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1. Cardiac assessment and inflammatory markers in children with paediatric inflammatory multisystem syndrome temporally associated with SARS-CoV2 (PIMS-TS) treated with methylprednisolone versus intravenous immunoglobulins: 6-month follow-up outcomes of the randomised controlled Swissped RECOVERY trialResearch in context

Author

Maya C. Andre, Carlos Sanchez, Sabrina Bressieux-Degueldre, Marie-Helene Perez, Daniela Wütz, Geraldine Blanchard-Rohner, Serge Grazioli, Nina Schöbi, Johannes Trück, Tatjana Welzel, Andrew Atkinson, Luregn J. Schlapbach, Julia Bielicki, Henrik Koehler, Spyridoula Gysi, Indra Janz, Andreas Bieri, Birgit Donner, Jürg Hammer, Ulrich Heininger, Clemens von Kalckreuth, Malte Kohns, Nicole Mettauer, Alexandra Meyer, Diana Reppucci, Chloé Schlaeppi, Daniel Trachsel, Nina Vaezipour, Andreas Woerner, Andreas Zutter, Federica Vanoni, Lisa Kottanattu, Calogero Mazzara, Alessia Severi Conti, Christoph Aebi, Philipp Agyeman, Andrea Duppenthaler, Martin Glöckler, Sabine Pallivathukal, Thomas Riedel, Petra Zimmermann, Hong-Phuc Cudré-Cung, Mladen Pavlovic, Alice Bordessoule, Anne-Laure Martin, Angelo Polito, Noemie Wagner, Marie Rohr, Arnaud L'Huillier, Vivianne Amiet, Thomas Ferry, David Longchamp, Julia Natterer, Rebecca Oppenheim, Michael Hofer, Michael Buettcher, Katharina Wechselberger, Alex Donas, Sara Germann, Michaela Lütolf Erni, Daniela Kaiser, Katharina Schwendener Scholl, Hans Peter Kuen, Katja Hrup, Janine Stritt, Douggl G.N. Bailey, Tanja Wachinger, Ingrid Beck, André Birkenmaier, Bjarte Rogdo, Philip Lorenz, Ivo Iglowstein, Konstanze Zöhrer, Martin Flade, Seraina Prader, Jana Pachlopnik Schmid, Michelle Seiler, Patrick Meyer Sauteur, Barbara Brotschi, Kathrin Weber, Elizabeth Whittaker, and Saul N. Faust

Subjects
Children, Immunoglobulins, PIMS-TS, Methylprednisolone, RCT, Coronary artery aneurysm, Medicine (General), R5-920
Abstract

Summary: Background: Previous findings from the Swissped RECOVERY trial showed that patients with Pediatric Inflammatory Multisystem Syndrome–Temporally Associated with SARS-CoV-2 (PIMS-TS) who were randomly assigned to intravenous immunoglobulins or methylprednisolone have a comparable length of hospital stay. Here, we report the 6-month follow-up outcomes of cardiac pathologies and normalisation of clinical or laboratory signs of inflammation from this study population. Methods: This pre-planned follow-up of patients with PIMS-TS included the Swissped RECOVERY Trial reports on the 6-month outcomes of the cohort after randomisation, with a focus on cardiac, haematological, and biochemical findings. The trial was an investigator-initiated randomised multicentre open-label two-arm trial in children and adolescents hospitalised with PIMS-TS at ten hospitals in Switzerland. Cardiological assessments and laboratory analyses were prospectively collected in the intention-to-treat analysis on pre-defined intervals after hospital discharge. Differences between randomised arms were investigated using Chi-square test for categorical and Wilcoxon test for continuous variables. The trial is registered with the Swiss National Clinical Trials Portal (SNCTP000004720) and ClinicalTrials.gov (NCT04826588). Findings: Between May 21, 2021 and April 15, 2022, 75 patients with a median age of 9.1 years (IQR 6.2–12.2) were included in the intention-to-treat population (37 in the methylprednisolone group and 38 in the intravenous immunoglobulin group). During follow-up, the incidence of abnormal left ventricular systolic function, coronary artery aneurysms (CAA), and other signs of inflammation were comparable in both groups. However, we detected cardiac abnormalities with low incidence and a mild degree grade of pathology. CAAs were observed in 2/38 children (5.3%) in the IVIG group and 1/37 children (2.7%) in the methylprednisolone group at 6-month follow-up (difference proportion 0.75; 95% confidence interval (CI) −0.05 to 1.0; p = 0.39). Interpretation: Methylprednisolone alone may be an acceptable first-line treatment as left ventricular systolic dysfunction and clinical/laboratory evidence for inflammation quickly resolved in all children. However, our findings need further confirmation through larger studies as our sample size is likely to be of insufficient power to address rare clinically relevant adverse outcomes. Funding: NOMIS, Vontobel, and Gaydoul Foundation.

Published
2024
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2. Individual and environmental determinants associated with longer times to access pediatric rheumatology centers for patients with juvenile idiopathic arthritis, a JIR cohort study

Author

Aurélie Chausset, Céline Lambert, Alexandre Belot, Etienne Merlin, Elvira Cannizzaro, Isabelle Kone-Paut, Claire Ballot, Valérie Devauchelle, Sylvaine Poignant, Raffaella Carlomagno, Anne Lohse, Catherine Barbier, Véronique Despert, Aurélia Carbasse, Laetitia Sparsa, Eva Adank, Federica Vanoni, Héloise Reumaux, Pascal Pillet, Daniela Kaiser, Michael Hofer, Caroline Freychet, and Anne-Marie Schott

Subjects
Juvenile idiopathic arthritis, Access to care, Time to referral, Socio-economic factors, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background Despite guidelines, poor access to appropriate care for juvenile idiopathic arthritis (JIA) patients remains a global issue. Prompt referral to a pediatric rheumatology (PR) center and effective care is known to be critical for changing the natural history of the disease and improving long-term prognosis. This project assesses socio-economic factors of delayed referral to a pediatric rheumatologist (PRst) for JIA patients in France and Switzerland within the Juvenile Inflammatory Rheumatism (JIR) Cohort. Methods All patients diagnosed with JIA, presenting at one center of the JIRcohort in France or Switzerland with additional data on referral pathway were included. Patient characteristics at first visit to the PR center, dates of visits to healthcare providers during referral, and parent characteristics were extracted from the JIRcohort database. Results Two hundred fifty children were included. The overall median time to first PR assessment was 2.4 months [1.3; 6.9] and ranged widely across the JIA subtypes, from 1.4 months [0.6; 3.8] for children with systemic juvenile idiopathic arthritis (sJIA) to 5.3 months [2.0; 19.1] for children with enthesitis-related arthritis (ERA). A diagnosis of ERA and an appointment with an orthopedist during the referral pathway were significantly associated with a longer time before the first PR visit (hazard ratio HR 0.50 [95% CI: 0.29; 0.84]) and HR 0.68 [95% CI: 0.49; 0.93], respectively) in multivariable analysis. Having a mother with a post-graduate educational attainment level was tendentially associated with a shorter time before the first PR visit, (HR 1.32 [95% CI: 0.99; 1.78]). Conclusions Time to first PRst visit was most often short compared to other studies and close to the British recommendations. However, this time remained too long for many patients. We observed no social inequities in access to a PRst, but we show the need to improve effective pathway and access to a PR center for JIA patients.

Published
2023
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3. Cardiac involvement in children with paediatric multisystem inflammatory syndrome temporally associated with SARS-CoV-2 (PIMS-TS): data from a prospective nationwide surveillance study

Author

Anita Uka, Sabrina Bressieux-Degueldre, Michael Buettcher, Lisa Kottanattu, Margerita Plebani, Anita Niederer-Loher, Nina Schöbi, Michael Hofer, Julie Tomasini, Johannes Trück, Reto Villiger, Noémie Wagner, Daniela Wuetz, Nicole Ritz, and Petra Zimmermann

Subjects
Medicine
Abstract

BACKGROUND: Paediatric inflammatory multisystem syndrome temporally associated with SARS-CoV-2 (PIMS-TS) may occur 4 to 8 weeks after SARS-CoV-2 infection. The acute presentation of PIMS-TS has been well described, but data on longer-term outcomes, particularly cardiac, is scarce. METHODS: This prospective nationwide surveillance study included children and adolescents less than 18 years of age who were hospitalised with PIMS-TS in Switzerland between March 2020 and March 2022. Data was collected from all 29 paediatric hospitals through the Swiss Paediatric Surveillance Unit (SPSU) during hospitalisation and approximately six weeks after discharge. The data was analysed after categorising the participants into three groups based on their admission status to the intensive care unit (ICU) (non-ICU, ICU-moderate) and the requirement for invasive ventilatory and/or inotropic support (ICU-severe). RESULTS: Overall, 204 children were included of whom 194 (95.1%) had follow-up data recorded. Median age was 9.0 years (interquartile range [IQR] 6.0–11.5) and 142 (69.6%) were male. In total, 105/204 (51.5%) required ICU admission, of whom 55/105 (52.4%) received inotropic support and 14/105 (13.3%) mechanical ventilation (ICU-severe group). Echocardiography was performed in 201/204 (98.5%) children; 132 (64.7%) had a cardiac abnormality including left ventricular systolic dysfunction (73 [36.3%]), a coronary artery abnormality (45 [22.4%]), pericardial effusion (50 [24.9%]) and mitral valve regurgitation (60 [29.9%]). Left ventricular systolic dysfunction was present at admission in 62/201 (30.8%) children and appeared during hospitalisation in 11 (5.5%) children. A coronary artery abnormality was detected at admission in 29/201 (14.2%) children and developed during hospitalisation or at follow-up in 13 (6.5%) and 3 (1.5%) children, respectively. None of the children had left ventricular systolic dysfunction at follow-up, but a coronary abnormality and pericardial effusion were found in 12 (6.6%) and 3 (1.7%) children, respectively. School absenteeism at the time of follow-up was more frequent in children who had been admitted to the ICU (2.5% in the non-ICU group compared to 10.4% and 17.6% in the ICU-moderate and ICU-severe group, respectively) (p = 0.011). CONCLUSION: Cardiac complications in children presenting with PIMS-TS are common and may worsen during the hospitalisation. Irrespective of initial severity, resolution of left ventricular systolic dysfunction is observed, often occurring rapidly during the hospitalisation. Most of the coronary artery abnormalities regress; however, some are still present at follow-up, emphasising the need for prolonged cardiac evaluation after PIMS-TS.

Published
2023
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4. Criteria Associated with Treatment Decisions in Juvenile Idiopathic Arthritis with a Focus on Ultrasonography: Results from the JIRECHO Cohort

Author

Sara Baydoun, Sandrine Jousse-Joulin, Alain Saraux, Perrine Dusser-Benesty, Charlotte Borocco, Caroline Galeotti, Annette Von Scheven, Michael Hofer, Brigitte Bader-Meunier, Florence Aeschlimann, Sylvain Breton, Laetitia Sparsa, Aurélia Carbasse, Gaël Mouterde, Linda Rossi-Semerano, and Valérie Devauchelle-Pensec

Subjects
Juvenile idiopathic arthritis, Ultrasonography, Synovitis, Therapeutic decision, Treatment, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background The treatment of children with juvenile idiopathic arthritis (JIA) to prevent disability is a major challenge in paediatric rheumatology. The presence of synovitis, which is difficult to detect in children, is associated with structural damage. Musculoskeletal ultrasonography (MSUS) can be used in patients with JIA to reveal subclinical synovitis. Objective The primary aim was to determine whether the use of MSUS was associated with therapeutic modification in patients with JIA. The secondary aim was to identify other factors associated with therapeutic decisions. Methods We conducted an observational study based on the JIRECHO multi-centre cohort, which was developed to provide a systematic MSUS follow-up for patients with JIA. Follow-up occurred every 6 months and included clinical and MSUS examinations. We included children who underwent MSUS of the elbows, wrists, second metacarpophalangeal joints, knees and ankles, which was performed by expert sonographers. Clinical and biological data, disease activity scores and information on therapeutics were collected. Results A total of 185 visits concerning 112 patients were recorded. Three groups were defined according to the therapeutic decision: escalation (22%, n = 40), de-escalation (14%, n = 26) or stable (64%, n = 119). In the “therapeutic escalation” group: the presence of ultrasonographic synovitis in B-mode and the presence of grade 2 or 3 synovitis in B-mode were not significantly more frequent than in the “stable therapeutic or de-escalation” group (80% versus 65%, p = 0.06; 33% versus 19%, p = 0.06), and the patient’s and physician’s visual analogue scale (VAS) scores, the clinical JADAS and the C-reactive protein level were significantly higher, but only physician’s VAS score remained in the model of logistic regression. In the “therapeutic de-escalation” group: there was no difference in the presence of US synovitis compared with the “stable therapeutic or escalation” group (62% versus 69%, p = 0.48). Conclusion Even though US synovitis tended to be more frequent in patients with therapeutic escalation, the study did not show that the presence of synovitis in MSUS was statistically associated with therapeutic modifications in patients with JIA. Treatment remained stable despite the presence of US synovitis.

Published
2022
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5. Vaccination completeness in children with rheumatic diseases: A longitudinal, observational multicenter cohort study in Switzerland

Author

Tatjana Welzel, Jasmin Kuemmerle-Deschner, Constantin Sluka, Raffaella Carlomagno, Elvira Cannizzaro Schneider, Daniela Kaiser, Michael Hofer, Veronique Hentgen, and Andreas Woerner

Subjects
vaccination adherence, infection risk, vaccination recommendations, immunosuppression, vaccination, Pediatrics, RJ1-570
Abstract

IntroductionChildren with pediatric inflammatory rheumatic diseases (PRD) have an increased infection risk. Vaccinations are effective to avoid vaccine-preventable diseases. This study aimed to assess the vaccination completeness in Swiss PRD patients stratified by immunosuppressive treatment (IST).Materials and methodsThis multicenter observational cohort study of PRD patients was performed in Basel, Geneva, Lucerne, Lausanne, and Zurich in PRD patients aged < 18 years included in the Juvenile Inflammatory Rheumatism Cohort. Completeness was assessed for i) the overall vaccination status (Swiss national immunization program (NIP) and specific additional PRD-recommended vaccinations), ii) for all and each vaccination of the NIP at PRD diagnosis and reference date (RefD) and iii) all and each specific additional PRD-recommended vaccination at RefD. Completeness was assessed over the disease course and stratified by IST.ResultsOf 616 eligible patients, 234 children were analyzed. Of these, 147 (63%) were girls. Median age at PRD diagnosis was 6.5 years (IQR 2.9–10.3) and 10.9 years at RefD (6.9–14.3). The median follow-up since PRD diagnosis was 3 years (1.1–5.5). 120/234 children received IST. At RefD, overall vaccination completeness was 3.8% (9/234 children), completeness for the NIP vaccinations was 70.1% (164/234 children; IST 65%, no IST: 75.4%) and for all specific additional PRD-recommended vaccinations was 3.8% (9/234 children; IST 2.5%; no IST 5.3%). Vaccination completeness against pneumococcal disease, hepatitis B virus, and human papilloma virus (HPV) was 50.4, 20, 37.9%, respectively. In 25/35 children with negative varicella zoster virus history vaccination status was complete (IST: 94.4%, no IST: 47%). Annual non-live influenza vaccination was complete in 24.2% of children during IST; adherence decreased over the disease course.DiscussionThis study identified a low overall vaccination completeness in children with PRD. Particularly, the completeness of specific additional PRD-recommended vaccinations was low. If not performed early after PRD diagnosis, vaccination status remained frequently incomplete. Close collaboration between pediatrician and rheumatologist to improve vaccination completeness is essential. Exchange of vaccination records, standardized assessment of specific PRD-recommended vaccinations and those of the NIP, and annual reminder for influenza vaccination are crucial to improve vaccination completeness in this vulnerable pediatric population.

Published
2022
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6. Pharmacovigilance in juvenile idiopathic arthritis patients treated with biologic or synthetic drugs: combined data of more than 15,000 patients from Pharmachild and national registries

Author

Joost Swart, Gabriella Giancane, Gerd Horneff, Bo Magnusson, Michael Hofer, Еkaterina Alexeeva, Violeta Panaviene, Brigitte Bader-Meunier, Jordi Anton, Susan Nielsen, Fabrizio De Benedetti, Sylvia Kamphuis, Valda Staņēviča, Maria Tracahana, Laura Marinela Ailioaie, Elena Tsitsami, Ariane Klein, Kirsten Minden, Ivan Foeldvari, Johannes Peter Haas, Jens Klotsche, Anna Carin Horne, Alessandro Consolaro, Francesca Bovis, Francesca Bagnasco, Angela Pistorio, Alberto Martini, Nico Wulffraat, Nicolino Ruperto, and for the Paediatric Rheumatology International Trials Organisation (PRINTO), BiKeR and the board of the Swedish Registry

Subjects
Juvenile idiopathic arthritis, Registry, Safety, Adverse events, Methotrexate, Biologics, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background The availability of methotrexate and the introduction of multiple biological agents have revolutionized the treatment of juvenile idiopathic arthritis (JIA). Several international and national drug registries have been implemented to accurately monitor the long-term safety/efficacy of these agents. This report aims to present the combined data coming from Pharmachild/PRINTO registry and the national registries from Germany (BiKeR) and Sweden. Methods Descriptive statistics was used for demographic, clinical data, drug exposure, adverse events (AEs) and events of special interest (ESIs). For the Swedish register, AE data were not available. Results Data from a total of 15,284 patients were reported: 8274 (54%) from the Pharmachild registry and 3990 (26%) and 3020 (20%) from the German and the Swedish registries, respectively. Pharmachild children showed a younger age (median of 5.4 versus 7.6 years) at JIA onset and shorter disease duration at last available visit (5.3 versus 6.1–6.8) when compared with the other registries. The most frequent JIA category was the rheumatoid factor–negative polyarthritis (range of 24.6–29.9%). Methotrexate (61–84%) and etanercept (24%–61.8%) were the most frequently used synthetic and biologic disease-modifying anti-rheumatic drugs (DMARDs), respectively. There was a wide variability in glucocorticoid use (16.7–42.1%). Serious AEs were present in 572 (6.9%) patients in Pharmachild versus 297 (7.4%) in BiKeR. Infection and infestations were the most frequent AEs (29.4–30.1%) followed by gastrointestinal disorders (11.5–19.6%). The most frequent ESIs were infections (75.3–89%). Conclusions This article is the first attempt to present a very large sample of data on JIA patients from different national and international registries and represents the first proposal for data merging as the most powerful tool for future analysis of safety and effectiveness of immunosuppressive therapies in JIA. Registry registration The Pharmachild registry is registered at ClinicalTrials.gov (NCT01399281) and at the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) (http://www.encepp.eu/encepp/viewResource.htm?id=19362). The BiKeR registry is registered at ENCePP (http://www.encepp.eu/encepp/viewResource.htm?id=20591).

Published
2018
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7. Best Practice Recommendations for the Diagnosis and Management of Children With Pediatric Inflammatory Multisystem Syndrome Temporally Associated With SARS-CoV-2 (PIMS-TS; Multisystem Inflammatory Syndrome in Children, MIS-C) in Switzerland

Author

Luregn J. Schlapbach, Maya C. Andre, Serge Grazioli, Nina Schöbi, Nicole Ritz, Christoph Aebi, Philipp Agyeman, Manuela Albisetti, Douggl G. N. Bailey, Christoph Berger, Géraldine Blanchard-Rohner, Sabrina Bressieux-Degueldre, Michael Hofer, Arnaud G. L'Huillier, Mark Marston, Patrick M. Meyer Sauteur, Jana Pachlopnik Schmid, Marie-Helene Perez, Bjarte Rogdo, Johannes Trück, Andreas Woerner, Daniela Wütz, Petra Zimmermann, Michael Levin, Elizabeth Whittaker, Peter C. Rimensberger, and the PIMS-TS working group of the Interest Group for Pediatric Neonatal Intensive Care (IGPNI) of the Swiss Society of Intensive Care and the Pediatric Infectious Diseases Group Switzerland (PIGS)

Subjects
child, COVID-19, Kawasaki disease, multisystem inflammatory syndrome, MIS-C, pediatric inflammatory multisystem syndrome, Pediatrics, RJ1-570
Abstract

Background: Following the spread of the coronavirus disease 2019 (COVID-19) pandemic a new disease entity emerged, defined as Pediatric Inflammatory Multisystem Syndrome temporally associated with COVID-19 (PIMS-TS), or Multisystem Inflammatory Syndrome in Children (MIS-C). In the absence of trials, evidence for treatment remains scarce.Purpose: To develop best practice recommendations for the diagnosis and treatment of children with PIMS-TS in Switzerland. It is acknowledged that the field is changing rapidly, and regular revisions in the coming months are pre-planned as evidence is increasing.Methods: Consensus guidelines for best practice were established by a multidisciplinary group of Swiss pediatric clinicians with expertise in intensive care, immunology/rheumatology, infectious diseases, hematology, and cardiology. Subsequent to literature review, four working groups established draft recommendations which were subsequently adapted in a modified Delphi process. Recommendations had to reach >80% agreement for acceptance.Results: The group achieved agreement on 26 recommendations, which specify diagnostic approaches and interventions across anti-inflammatory, anti-infectious, and support therapies, and follow-up for children with suspected PIMS-TS. A management algorithm was derived to guide treatment depending on the phenotype of presentation, categorized into PIMS-TS with (a) shock, (b) Kawasaki-disease like, and (c) undifferentiated inflammatory presentation.Conclusion: Available literature on PIMS-TS is limited to retrospective or prospective observational studies. Informed by these cohort studies and indirect evidence from other inflammatory conditions in children and adults, as well as guidelines from international health authorities, the Swiss PIMS-TS recommendations represent best practice guidelines based on currently available knowledge to standardize treatment of children with suspected PIMS-TS. Given the absence of high-grade evidence, regular updates of the recommendations will be warranted, and participation of patients in trials should be encouraged.

Published
2021
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8. Case Report: Pansclerotic Morphea-Clinical Features, Differential Diagnoses and Modern Treatment Concepts

Author

Sarah Ventéjou, Agnes Schwieger-Briel, Rebecca Nicolai, Stephanie Christen-Zaech, Caroline Schnider, Michael Hofer, Sofia Bogiatzi, Daniel Hohl, Fabrizio De Benedetti, and Marie-Anne Morren

Subjects
pansclerotic morphea, stiff skin, scleroderma, tocilizumab, IL-6, case report, Immunologic diseases. Allergy, RC581-607
Abstract

Pansclerotic morphea (PSM) is a rare skin disease characterized by progressive stiffening of the skin with or without the typical superficial skin changes usually seen in morphea (localized scleroderma). Standard therapy, consisting of a combination of systemic glucocorticoids and methotrexate or mycophenolate mofetil, does rarely stop disease progression, which may lead to severe cutaneous sclerosis and secondary contractures. Little is known about the efficacy of newer biologicals such as abatacept, a fusion protein antibody against CTLA-4, or tocilizumab, a fully humanized IL-6R antibody, in the treatment of this pathology. We present the case of an 8 years old girl with an unusual, progressive stiffening of the skin, which was eventually diagnosed as pansclerotic morphea. A treatment with systemic glucocorticoids and methotrexate combined with tocilizumab led to a good clinical response within 2 months after initiation. In this paper, we discuss differential diagnoses to be considered and this new promising treatment option based on a case review of the literature.

Published
2021
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9. Case Report: Case Series of Children With Multisystem Inflammatory Syndrome Following SARS-CoV-2 Infection in Switzerland

Author

Athina Fouriki, Yves Fougère, Caroline De Camaret, Géraldine Blanchard Rohner, Serge Grazioli, Noémie Wagner, Christa Relly, Jana Pachlopnik Schmid, Johannes Trück, Lisa Kottanatu, Estefania Perez, Marie-Helene Perez, Damien Schaffner, Sandra Andrea Asner, and Michael Hofer

Subjects
SARS-CoV-2, child, MIS-C, IL-1ra, Anakinra, Pediatrics, RJ1-570
Abstract

Since the beginning of the severe SARS-CoV-2 pandemic, an increasing number of countries reported cases of a systemic hyperinflammatory condition defined as multi-system inflammatory syndrome in children (MIS-C). The clinical features of MIS-C can be an overlap of Kawasaki Disease (KD), Toxic Shock Syndrome (TSS), Macrophage Activation Syndrome (MAS), or have often an acute abdominal presentation. Intravenous immunoglobulin (IVIG) is recommended as first line therapy in KD. Recent evidence suggests intravenous immunoglobulins (IVIG) resistance in some cases of SARS-CoV-2 related MIS-C, thereby questioning the benefit of immunomodulators such as IL-1 or IL-6 blocking agents. We report on a cohort of 6 Swiss children with SARS-CoV2 related MIS-C presenting with clinical features compatible with Incomplete KD and Toxic Shock Syndrome associated to a cytokine storm. Serum cytokine profile investigations showed increased IL1RA levels (8 to 22-fold) in 5 of the 6 patients (one patient had not been tested), whereas, IL-6 serum levels were increased only in the 3 patients of the 6 who were tested. With exception of one patient who had only benefited by Anakinra, all patients received at least one dose of IVIG. One patient has only received Anakinra with favorable evolution, and three patients had also a steroid treatment. In addition to all this anti-inflammatory medication two patients have also received one dose of anti-IL6. In conclusion, our case series reports on clinical and laboratory findings of most of Swiss cases with MIS-C and suggests the use of Anakinra as an alternative to steroids in these children, most of whom presented with high IL-1RA levels.

Published
2021
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10. Long-Term Follow-Up and Optimization of Interleukin-1 Inhibitors in the Management of Monogenic Autoinflammatory Diseases: Real-Life Data from the JIR Cohort

Author

Véronique Hentgen, Isabelle Koné-Paut, Alexandre Belot, Caroline Galeotti, Gilles Grateau, Aurelia Carbasse, Anne Pagnier, Pascal Pillet, Marc Delord, Michael Hofer, and Sophie Georgin-Lavialle

Subjects
anakinra, canakinumab, cryopyrin-associated periodic syndrome, Tumor Necrosis factor (TNF)-receptor-associated periodic syndrome, mevalonate kinase deficiency, posology, Therapeutics. Pharmacology, RM1-950
Abstract

Objectives: The major role of interleukin (IL)-1 in the pathogenesis of hereditary recurrent fever syndromes favored the employment of targeted therapies modulating IL-1 signaling. However the best use of IL1 inhibitors in terms of dosage is difficult to define at present.Methods: In order to better understand the use of IL1 inhibitors in a real-life setting, our study assessed the dosage regimens of French patients with one of the four main hereditary recurrent fever syndromes (Familial Mediterranean Fever (FMF), TNF receptor associated periodic syndrome (TRAPS), cryopyrin associated periodic fever (CAPS) and mevalonate kinase deficiency). The patients were retrieved retrospectively from the JIR cohort, an international platform gathering data of patients with pediatric inflammatory diseases.Results: Forty five patients of the JIR cohort with a hereditary recurrent fever syndrome had received at least once an IL1 inhibitor (anakinra or canakinumab). Of these, 43% received a lower dosage than the one suggested in the product recommendations, regardless of the type of the IL1 inhibitor. Especially patients with FMF and TRAPS seemed to need lower treatment regimens; in our cohort none of the FMF or TRAPS patients received an intensified dose of IL-inhibitor. On-demand treatment with a short half-life IL-1 inhibitor has also been used successfully for some patients with one of these two conditions The standard dose was given to 42% of the patients; whereas an intensified dose of IL-1 inhibitors was given to 15% of the patients (44% of CAPS patients and 17% of mevalonate kinase deficiency patients). In our cohort each individual patient’s need for treatment seemed highly variable, ranging from on demand treatment regimens to intensified dosage maintenance therapies depending on the activity and the severity of the underlying disease.Conclusion: IL-1 inhibitors are a good treatment option for patients with a hereditary recurrent fever syndrome, but the individual need of the dosage of IL-1 inhibitors to control the disease effectively seems highly variable. Severity, activity but also the type of the underlying disease, belong to the parameters underpinning the treat-to-target strategy implemented in an everyday life practice.

Published
2021
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11. Resting TcPO2 levels decrease during liner wear in persons with a transtibial amputation.

Author

Martin C Berli, Michèle Jundt-Ecker, Margrit R Meier, Michael Hofer, Madlaina Schöni, Tobias Götschi, Ilker Uçkay, Thomas Böni, and Felix W A Waibel

Subjects
Medicine, Science
Abstract

BackgroundIn our clinic, a substantial number of patients present with transtibial residual limb pain of no specific somatic origin. Silicone liner induced tissue compression may reduce blood flow, possibly causing residual limb pain. Thus, as a first step we investigated if the liner itself has an effect on transcutaneous oxygen pressure (TcPO2).MethodsPersons with unilateral transtibial amputation and residual limb pain of unknown origin were included. Medical history, including residual limb pain, was recorded, and the SF-36 administered. Resting TcPO2 levels were measured in the supine position and without a liner at 0, 10, 20 and 30 minutes using two sensors: one placed in the Transverse plane over the tip of the Tibia End (= TTE), the other placed in the Sagittal plane, distally over the Peroneal Compartment (= SPC). Measurements were repeated with specially prepared liners avoiding additional pressure due to sensor placement. Statistical analyses were performed using SPSS.ResultsTwenty persons (9 women, 11 men) with a mean age of 68.65 years (range 47-86 years) participated. The transtibial amputation occurred on average 43 months prior to study entry (range 3-119 months). With liner wear, both sensors measured TcPO2 levels that were significantly lower than those measured without a liner (TTE: p < 0.001; SPC: p = 0.002) after 10, 20 and 30 minutes. No significant differences were found between TcPO2 levels over time between the sensors. There were no significant associations between TcPO2 levels and pain, smoking status, age, duration of daily liner use, mobility level, and revision history.ConclusionResting TcPO2 levels decreased significantly while wearing a liner alone, without a prosthetic socket. Further studies are required to investigate the effect of liner wear on exercise TcPO2 levels.

Published
2020
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12. An international delphi survey for the definition of the variables for the development of new classification criteria for periodic fever aphtous stomatitis pharingitis cervical adenitis (PFAPA)

Author

Federica Vanoni, Silvia Federici, Jordi Antón, Karyl S. Barron, Paul Brogan, Fabrizio De Benedetti, Fatma Dedeoglu, Erkan Demirkaya, Veronique Hentgen, Tilmann Kallinich, Ronald Laxer, Ricardo Russo, Natasa Toplak, Yosef Uziel, Alberto Martini, Nicolino Ruperto, Marco Gattorno, Michael Hofer, and for Eurofever and the Paediatric Rheumatology International Trials Organisation (PRINTO)

Subjects
Autoinflammatory diseases, Monogenic periodic fever, PFAPA, Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Abstract

Abstract Background Diagnosis of Periodic fever, aphthous stomatitis, pharyngitis and cervical adenitis (PFAPA) is currently based on a set of criteria proposed in 1999 modified from Marshall’s criteria. Nevertheless no validated evidence based set of classification criteria for PFAPA has been established so far. The aim of this study was to identify candidate classification criteria PFAPA syndrome using international consensus formation through a Delphi questionnaire survey. Methods A first open-ended questionnaire was sent to adult and pediatric clinicians/researchers, asking to identify the variables thought most likely to be helpful and relevant for the diagnosis of PFAPA. In a second survey, respondents were asked to select, from the list of variables coming from the first survey, the 10 features that they felt were most important, and to rank them in descending order from most important to least important. Results The response rate to the first and second Delphi was respectively 109/124 (88%) and 141/162 (87%). The number of participants that completed the first and second Delphi was 69/124 (56%) and 110/162 (68%). From the first Delphi we obtained a list of 92 variables, of which 62 were selected in the second Delphi. Variables reaching the top five position of the rank were regular periodicity, aphthous stomatitis, response to corticosteroids, cervical adenitis, and well-being between flares. Conclusion Our process led to identification of features that were felt to be the most important as candidate classification criteria for PFAPA by a large sample of international rheumatologists. The performance of these items will be tested further in the next phase of the study, through analysis of real patient data.

Published
2018
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13. Chronic Nonbacterial Osteomyelitis in Children

Author

Aikaterini Koryllou, Manel Mejbri, Katerina Theodoropoulou, Michael Hofer, and Raffaella Carlomagno

Subjects
chronic nonbacterial osteomyelitis (CNO), chronic recurrent multifocal osteomyelitis (CRMO), chronic nonbacterial osteitis, SAPHO, DIRA, PAPA syndrome, Pediatrics, RJ1-570
Abstract

Chronic nonbacterial osteomyelitis (CNO) is an auto-inflammatory bone disorder with a wide spectrum of clinical manifestations, from unifocal to multifocal lesions. When it manifests with multifocal lesions, it is also referred to as chronic recurrent multifocal osteomyelitis (CRMO). CNO/CRMO can affect all age groups, with the pediatric population being the most common. Patients may present with systemic inflammation, but there is no pathognomonic laboratory finding. Magnetic resonance imaging (MRI) is the gold standard radiological tool for diagnosis. In the absence of validated diagnostic criteria, CNO/CRMO remains an exclusion diagnosis. Bone biopsy does not show a specific disease pattern, but it may be necessary in unifocal or atypical cases to differentiate it from malignancy or infection. First-line treatments are non-steroidal anti-inflammatory drugs (NSAIDs), while bisphosphonates or TNF-α blockers can be used in refractory cases. The disease course is unpredictable, and uncontrolled lesions can complicate with bone fractures and deformations, underlying the importance of long-term follow-up in these patients.

Published
2021
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14. Proceedings of the 23rd Paediatric Rheumatology European Society Congress: part one

Author

F. De Benedetti, J. Anton, M. Gattorno, H. Lachmann, I. Kone-Paut, S. Ozen, J. Frenkel, A. Simon, A. Zeft, E. Ben-Chetrit, H. M. Hoffman, Y. Joubert, K. Lheritier, A. Speziale, J. Guido, Roberta Caorsi, Federica Penco, Alice Grossi, Antonella Insalaco, Maria Alessio, Giovanni Conti, Federico Marchetti, Alberto Tommasini, Silvana Martino, Romina Gallizzi, Annalisa Salis, Francesca Schena, Francesco Caroli, Alberto Martini, Gianluca Damonte, Isabella Ceccherini, Marco Gattorno, Marie-Louise Frémond, Carolina Uggenti, Lien Van Eyck, Isabelle Melki, Darragh Duffy, Vincent Bondet, Yoann Rose, Bénédicte Neven, Yanick Crow, Mathieu P. Rodero, Yvonne Kusche, Johannes Roth, Katarzyna Barczyk-Kahlert, Giovanna Ferrara, Annalisa Chiocchetti, Silvio Polizzi, Josef Vuch, Diego Vozzi, Anna Mondino, Erica Valencic, Serena Pastore, Andrea Taddio, Flavio Faletra, Umberto Dianzani, Ugo Ramenghi, Qing Zhou, Xiaomin Yu, Erkan Demirkaya, Natalie Deuitch, Deborah Stone, Wanxia Tsai, Amanda Ombrello, Tina Romeo, Elaine F. Remmers, JaeJin Chae, Massimo Gadina, Steven Welch, Seza Ozen, Rezan Topaloglu, Mario Abinun, Daniel L. Kastner, Ivona Aksentijevich, Donatella Vairo, Rosalba Monica Ferraro, Giulia Zani, Jessica Galli, Micaela De Simone, Marco Cattalini, Elisa Fazzi, Silvia Giliani, Ebun Omoyinmi, Ariane Standing, Dorota Rowczenio, Annette Keylock, Sonia Melo Gomes, Fiona Price-Kuehne, Sira Nanthapisal, Claire Murphy, Thomas Cullup, Lucy Jenkins, Kimberly Gilmour, Despina Eleftheriou, Helen Lachmann, Philip Hawkins, Nigel Klein, Paul Brogan, Anita Dhanrajani, Mercedes Chan, Stephanie Pau, Janet Ellsworth, Jaime Guzman, Florence A. Aeschlimann, Marinka Twilt, Simon W. Eng, Shehla Sheikh, Ronald M. Laxer, Diane Hebert, Damien Noone, Christian Pagnoux, Susanne M. Benseler, Rae S. Yeung, Christoph Kessel, Katrin Lippitz, Toni Weinhage, Claas Hinze, Helmut Wittkowski, Dirk Holzinger, Niklas Grün, Dirk Föll, Pieter Van Dijkhuizen, Federica Del Chierico, Clara Malattia, Alessandra Russo, Denise Pires Marafon, Nienke M. ter Haar, Silvia Magni-Manzoni, Sebastiaan J. Vastert, Bruno Dallapiccola, Berent Prakken, Fabrizio De Benedetti, Lorenza Putignani, Berna Eren Fidanci, Kenan Barut, Serap Arıcı, Dogan Simsek, Mustafa Cakan, Ezgi D. Batu, Sezgin Şahin, Ayşenur Kısaarslan, Ebru Yilmaz, Özge Basaran, Ferhat Demir, Kubra Ozturk, Zübeyde Gunduz, Betül Sozeri, Balahan Makay, Nuray Ayaz, Onder Yavascan, Ozlem Aydog, Yelda Bilginer, Zelal Ekinci, Dilek Yıldız, Faysal Gök, Muferret Erguven, Erbil Unsal, Ozgur Kasapcopur, For the FMF Arthritis Vasculitis and Orphan Disease Research in Paediatric Rheumatology (FAVOR), Hafize E. Sönmez, Betül Sözeri, Yonatan Butbul, Seza Özen, Claudia Bracaglia, Giusi Prencipe, Manuela Pardeo, Geneviève Lapeyre, Emiliano Marasco, Walter Ferlin, Robert Nelson, Cristina de Min, N. Ruperto, H. I. Brunner, P. Quartier, T. Constantin, E. Alexeeva, K. Marzan, N. Wulffraat, R. Schneider, S. Padeh, V. Chasnyk, C. Wouters, J. B. Kuemmerle-Deschner, T. Kallinich, B. Lauwerys, E. Haddad, E. Nasonov, M. Trachana, O. Vougiouka, K. Leon, E. Vritzali, A. Martini, D. Lovell, PRINTO/PRCSG, Stefano Volpi, Claudia Pastorino, Francesca Kalli, Alessia Omenetti, Sabrina Chiesa, Arinna Bertoni, Paolo Picco, Gilberto Filaci, Elisabetta Traggiai, Marie-Louise Fremond, Naoki Kitabayashi, Olivero Sacco, Isabelle Meyts, Marie-Anne Morren, Carine Wouters, Eric Legius, Isabelle Callebaut, Christine Bodemer, Frederic Rieux-Laucat, Mathieu Rodero, Nadia Jeremiah, Alexandre Belot, Eric Jeziorski, Didier Bessis, Guilhem Cros, Gillian I. Rice, Bruno Charbit, Anne Hulin, Nihel Khoudour, Consuelo Modesto Caballero, Monique Fabre, Laureline Berteloot, Muriel Le Bourgeois, Philippe Reix, Thierry Walzer, Despina Moshous, Stéphane Blanche, Alain Fischer, Brigitte Bader-Meunier, Frédéric Rieux-Laucat, K. Annink, N. ter Haar, S. Al-Mayouf, G. Amaryan, K. Barron, S. Benseler, P. Brogan, L. Cantarini, M. Cattalini, A. Cochino, F. Dedeoglu, A. De Jesus, O. Dellacasa, E. Demirkaya, P. Dolezalova, K. Durrant, G. Fabio, R. Gallizzi, R. Goldbach-Mansky, E. Hachulla, V. Hentgen, T. Herlin, M. Hofer, H. Hoffman, A. Insalaco, A. Jansson, I. Koné-Paut, A. Kozlova, J. Kuemmerle-Deschner, R. Laxer, S. Nielsen, I. Nikishina, A. Ombrello, E. Papadopoulou-Alataki, A. Ravelli, D. Rigante, R. Russo, Y. Uziel, Nienke ter Haar, Jerold Jeyaratnam, Anna Simon, Matteo Doglio, Jordi Anton, Consuelo Modesto, Pierre Quartier, Esther Hoppenreijs, Luca Cantarini, Loredana Lepore, Inmaculada Calvo Penades, Christina Boros, Rita Consolini, Donato Rigante, Ricardo Russo, Jana Pachlopnik Schmid, Thirusha Lane, Nicolino Ruperto, Joost Frenkel, Chiara Passarelli, Elisa Pisaneschi, Virginia Messia, Antonio Novelli, Fabrizio Debenedetti, P. A. Brogan, X. Wei, Martina Finetti, Francesca Orlando, Elisabetta Cortis, Angela Miniaci, Nicola Ruperto, Charlotte Eijkelboom, Pavla Dolezalova, Isabelle Koné-Paut, Marija Jelusic-Drazic, Liliana Bezrodnik, Mari Carmen Pinedo, Valda Stanevicha, Marielle van Gijn, Silvia Federici, Hermann Girschick, Gerd Ganser, Susan Nielsen, Troels Herlin, Sulaiman Mohammed Al-Mayouf, Michael Hofer, Jasmin Kuemmerle-Deschner, Susanne Schalm, Annette Jansson, on behalf of PRINTO and Eurofever registry, Marta Marchi, Chiara Marini, Angelo Ravelli, Alberto Garaventa, Sonia Carta, Enrica Balza, Patrizia Castellani, Caterina Pellecchia, Silvia Borghini, Maria Libera Trotta, Anna Rubartelli, Andrew Henrey, Thomas Loughin, Roberta Berard, Natalie Shiff, Roman Jurencak, Susanne Benseler, Lori Tucker, on behalf of ReACCh-Out Investigators, Charalampia Papadopoulou, Ying Hong, Petra Krol, Yiannis Ioannou, Clarissa Pilkington, Hema Chaplin, Stephania Simou, Marietta Charakida, Lucy Wedderburn, Lynn R. Spiegel, Sara Ahola Kohut, Jennifer Stinson, Paula Forgeron, Miriam Kaufman, Nadia Luca, Khush Amaria, Mary Bell, J Swart, F. Boris, E. Castagnola, A. Groll, G. Giancane, G. Horneff, H. I. Huppertz, T. Wolfs, E. Alekseeva, V. Panaviene, F. Uettwiller, V. Stanevicha, L. M. Ailioaie, E. Tsitami, S. Kamphuis, G. Susic, F. Sztajnbok, B. Flato, A. Pistorio, Stephanie J. W. Shoop, Suzanne M. M. Verstappen, Janet E. McDonagh, Wendy Thomson, Kimme L. Hyrich, CAPS, Maarit Tarkiainen, Pirjo Tynjala, Pekka Lahdenne, Janne Martikainen, Acute-JIA Study Group, Meredyth Wilkinson, Christopher Piper, Georg Otto, Claire T. Deakin, Stefanie Dowle, Stefania Simou, Daniel Kelberman, Claudia Mauri, Elizabeth Jury, David Isenberg, Lucy R. Wedderburn, Kiran Nistala, I. Foeldvari, D. J. Lovell, G. Simonini, M. Bereswill, J. Kalabic, Kiem Oen, Brian M. Feldman, Brenden Dufault, Jennifer Lee, Karen Watanabe Duffy, Ciaran Duffy, ReACCh-Out Investigators, N. Tzaribachev, G. Vega-Cornejo, I. Louw, A. Berman, I. Calvo, R. Cuttica, F. Avila-Zapata, R. Cimaz, E. Solau-Gervais, R. Joos, G. Espada, X. Li, M. Nys, R. Wong, S. Banerjee, For Pediatric Rheumatology International Trials Organization (PRINTO)/Pediatric Rheumatology Collaborative Study Group (PRCSG), Rebecca Nicolai, Margherita Verardo, Adele D’Amico, Luisa Bracci-Laudiero, Gian Marco Moneta, Gillian Rice, Anne-Laure Mathieu, Sulliman O. Omarjee, Tracy A. Briggs, James O’Sullivan, Simon Williams, Rolando Cimaz, Eve Smith, Michael W. Beresford, Yanick J. Crow, GENIAL Investigators, UK JSLE Study Group, Madeleine Rooney, Nick Bishop, joyce davidson, Clarissa pilkington, Michael Beresford, Jacqui Clinch, Rangaraj Satyapal, Helen Foster, Janet Gardner Medwin, Janet McDonagh, Sue Wyatt, On Behalf of the British Society for Paediatric and Adolescent Rheumatology, Valentina Litta Modignani, Francesco Baldo, Stefano Lanni, Alessandro Consolaro, Giovanni Filocamo, Helen J. Lachmann, on behalf of Eurofever Registry, Gianmarco Moneta, Camilla Celani, Bilade Cherqaoui, Linda Rossi-Semerano, Perrine Dusser, Véronique Hentgen, Claire Grimwood, Linda Rossi, Isabelle Kone Paut, Veronique Hentgen, Denise Lasigliè, Denise Ferrera, Giulia Amico, Marco Di Duca, Laura Obici, Roberto Ravazzolo, Ryuta Nishikomori, Juan Arostegui, Andrea Petretto, Chiara Lavarello, Elvira Inglese, Federica Vanoni, Michaël Hofer, on behalf of EUROFEVER PROJECT, P. N. Hawkins, T. van der Poll, U. A. Walker, H. H. Tilson, Pascal N. Tyrrell, Raphaela Goldbach-Mansky, Norbert Blank, Hal M. Hoffman, Elisabeth Weissbarth-Riedel, Boris Huegle, Tilmann Kallinich, Ahmet Gul, Marlen Oswald, Fatma Dedeoglu, Aki Hanaya, Takako Miyamae, Manabu Kawamoto, Yumi Tani, Takuma Hara, Yasushi Kawaguchi, Satoru Nagata, Hisashi Yamanaka, Almira Ćosićkić, Fahrija Skokić, Belkisa Čolić, Sanimir Suljendić, Anna Kozlova, Irina Mersiyanova, Mariya Panina, Lily Hachtryan, Vasiliy Burlakov, Elena Raikina, Alexey Maschan, Anna Shcherbina, Banu Acar, Meryem Albayrak, Betul Sozeri, Sezgin Sahin, Amra Adrovic, Nese Inan, Serhan Sevgi, Caroline M. Andreasen, Anne Grethe Jurik, Mia B. Glerup, Christian Høst, Birgitte T. Mahler, Ellen-Margrethe Hauge, Cecilia Lazea, Laura Damian, Calin Lazar, Rodica Manasia, Chloe M. Stephenson, Vimal Prajapati, Paivi M. Miettunen, Dilek Yılmaz, Yavuz Tokgöz, Yasin Bulut, Harun Çakmak, Ferah Sönmez, Elif Comak, Gülşah Kaya Aksoy, Mustafa Koyun, Sema Akman, Yunus Arıkan, Ender Terzioğlu, Osman Nidai Özdeş, İbrahim Keser, Hüseyin Koçak, Ayşen Bingöl, Aygen Yılmaz, Reha Artan, X. Xu, Fatemeh F. Mehregan, Vahid Ziaee, Mohammad H. Moradinejad, Francesco La Torre, Clotilde Alizzi, Pio D’Adamo, G. Junge, J. Gregson, Hasmik Sargsyan, Hulya Zengin, Berna E. Fidanci, Cagla Kaymakamgil, Dilek Konukbay, Dilek Yildiz, Faysal Gok, Iris Stoler, Judith Freytag, Banu Orak, Christine Seib, Lars Esmann, Eva Seipelt, Faekah Gohar, Dirk Foell, Ismail Dursun, Sebahat Tulpar, Sibel Yel, Demet Kartal, Murat Borlu, Funda Bastug, Hakan Poyrazoglu, Zubeyde Gunduz, Kader Kose, Mehmet E. Yuksel, Abdullah Calıskan, Ahmet B. Cekgeloglu, Ruhan Dusunsel, Katerina Bouchalova, Jana Franova, Marcel Schuller, Marie Macku, Katerina Theodoropoulou, Raffaella Carlomagno, Annette von Scheven-Gête, Claudia Poloni, Laura O. Damian, Dan Cosma, Amanda Radulescu, Dan Vasilescu, Liliana Rogojan, Simona Rednic, Mihaela Lupse, Lien De Somer, Pierre Moens, Rocio Galindo Zavala, Laura Martín Pedraz, Esmeralda Núñez Cuadros, Gisela Díaz-Cordovés Rego, Antonio L. Urda Cardona, Ilaria Dal Forno, Sara Pieropan, Ombretta Viapiana, Davide Gatti, Gloria Dallagiacoma, Paola Caramaschi, Domenico Biasi, Daniel Windschall, Ralf Trauzeddel, Hartwig Lehmann, Rainer Berendes, Maria Haller, Manuela Krumrey-Langkammerer, Antje Nimtz-Talaska, Philipp Schoof, Ralf Felix Trauzeddel, Christine Nirschl, Estefania Quesada-Masachs, Carla Aguilar Blancafort, Sara Marsal Barril, Francisca Aguiar, Rita Fonseca, Duarte Alves, Ana Vieira, Alberto Vieira, Jorge A. Dias, Iva Brito, Gordana Susic, Vera Milic, Goran Radunovic, Ivan Boricic, Pauline Marteau, Catherine Adamsbaum, Michel De Bandt, Irène Lemelle, Chantal Deslandre, Tu Anh Tran, Anne Lohse, Elisabeth Solau-Gervais, Pascal Pillet, Julien Wipff, Cécile Gaujoux-Viala, Sylvain Breton, Valérie Devauchelle-Pensec, Sandra Gran, Olesja Fehler, Stefanie Zenker, Michael Schäfers, Thomas Vogl, Severine Guillaume Czitrom, EH Pieter Van Dijkhuizen, Silvia Magni Manzoni, Francesca Magnaguagno, Laura Tanturri de Horatio, Nienke M. Ter Haar, Annemieke S. Littooij, Vitor A. Teixeira, Raquel Campanilho-Marques, Ana F. Mourão, Filipa O. Ramos, Manuela Costa, Wafa A. Madan, Orla G. Killeen, Adriana Rodriguez Vidal, Diana Sueiro Delgado, Maria Isabel Gonzalez Fernandez, Berta Lopez Montesinos, Aleksey Kozhevnikov, Nina Pozdeeva, Mikhail Konev, Evgeniy Melchenko, Vladimir Kenis, Gennadiy Novik, Aysenur Pac Kısaarslan, Butsabong Lerkvaleekul, Suphaneewan Jaovisidha, Witaya Sungkarat, Niyata Chitrapazt, Praman Fuangfa, Thumanoon Ruangchaijatuporn, Soamarat Vilaiyuk, Dan Ø. Pradsgaard, Arne Hørlyck, Anne H. Spannow, Carsten W. Heuck, Talia Diaz, Fernando Garcia, Lorenia De La Cruz, Nadina Rubio, Joanna Świdrowska-Jaros, Elzbieta Smolewska, Mirta Lamot, Lovro Lamot, Mandica Vidovic, Edi Paleka Bosak, Ivana Rados, Miroslav Harjacek, Nikolay Tzaribachev, Polymnia Louka, Romiesa Hagoug, Chiara Trentin, Olga Kubassova, Mark Hinton, Mikael Boesen, Olena A. Oshlianska, Illya A. Chaikovsky, G. Mjasnikov, A. Kazmirchyk, Umberto Garagiola, Irene Borzani, Paolo Cressoni, Fabrizia Corona, Eszter Dzsida, Giampietro Farronato, Antonella Petaccia, Alenka Gagro, Agneza Marija Pasini, Goran Roic, Ozren Vrdoljak, Lucija Lujic, Matija Zutelija-Fattorini, Monika M. Esser, Deepthi R. Abraham, Craig Kinnear, Glenda Durrheim, Mike Urban, Eileen Hoal, Victoria B. Nikolayenko, Kubilay Şahin, Yasar Karaaslan, Adele Civino, Giovanni Alighieri, Sergio Davì, Roberto Rondelli, Andrea Magnolato, Francesca Ricci, Alma Olivieri, Valeria Gerloni, Bianca Lattanzi, Francesca Soscia, Alessandro De Fanti, Stefania Citiso, Lorenzo Quartulli, Maria Cristina Maggio, Manuela Marsili, Maria Antonietta Pelagatti, Valentino Conter, Franca Fagioli, Andrea Pession, Marco Garrone, Mariangela Rinaldi, Jaime De Inocencio, Stella Garay, Daniel J. Lovell, Berit Flato, EPOCA Study Group, Angela Aquilani, Simona Cascioli, Ivan Caiello, Denise Pires-Marafón, Rita Carsetti, Emily Robinson, Salvatore Albani, Wilco de Jager, Sytze de Roock, Trang Duong, Justine Ellis, Kimme Hyrich, Laetitia Jervis, Daniel Lovell, Lucy Marshall, Elizabeth D. Mellins, Kirsten Minden, Jane Munro, Peter A. Nigrovic, Jason Palman, Sunil Sampath, Laura E. Schanberg, Susan D. Thompson, Richard Vesely, Chris Wallace, Chris Williams, Qiong Wu, Nico Wulffraat, Rae S. M. Yeung, M. B. Seyger, D. Arikan, J. K. Anderson, A. Lazar, D. A. Williams, C. Wang, R. Tarzynski-Potempa, J. S. Hymans, Gabriele Simonini, Erika Scoccimarro, Irene Pontikaki, Teresa Giani, Alessandro Ventura, Pier Luigi Meroni, Gaetana Minnone, Marzia Soligo, Luigi Manni, Luisa Bracci Laudiero, Noortje Groot, I. Grein, N. M. Wulffraat, R. Schepp, G. Berbers, C. C. Barbosa Sandoval de Souza, V. Paes Leme Ferriani, G. Pileggi, S. de Roock, Ingrid H. R. Grein, Silvia Scala, Elisa Patrone, Casper Schoemaker, on behalf of Dutch JIA patient organization, Wendy Costello, on behalf of ENCA, Suzanne Parsons, Jean-David Cohen, Damien Bentayou, Marc-Antoine Bernard Brunel, Sonia Trope, Jens Klotsche, Miriam Listing, Martina Niewerth, Gerd Horneff, Angelika Thon, Hans-Iko Huppertz, Kirsten Mönkemöller, Ivan Foeldvari, ICON study group, Achille Marino, Stefano Stagi, Niccolò Carli, Federico Bertini, Adriana S. Díaz-Maldonado, Sally Pino, Pilar Guarnizo, Alfonso Ragnar Torres-Jimenez, Berenice Sanchez-Jara, Eunice Solis-Vallejo, Adriana Ivonne Cespedes-Cruz, Maritza Zeferino-Cruz, Julia Veronica Ramirez-Miramontes, Ankur Kumar, Anju Gupta, Deepti Suri, Amit Rawat, Nandita Kakkar, Surjit Singh, Özge A. Gücenmez, Erbil Ünsal, Bo Magnusson, Karina Mördrup, Anna Vermé, Christina Peterson, Board of the Swedish Pediatric Rheumatology Registry, Caroline Freychet, Jean Louis Stephan, Cathryn E. Harkness, Leanne Foster, Emma Henry, Pauline Taggart, Coskun F. Ozkececi, Esra Kurt, Gokalp Basbozkurt, Daiva Gorczyca, Jacek Postępski, Aleksandra Czajkowska, Bogumiła Szponar, Mariola Paściak, Anna Gruenpeter, Iwona Lachór-Motyka, Daria Augustyniak, Edyta Olesińska, Emediong S. Asuka, Tatyana Golovko, Samuel U. Aliejim, Emilio Inarejos Clemente, Estibaliz Iglesias Jimenez, Joan Calzada Hernandez, Sergi Borlan Fernandez, Clara Gimenez Roca, David Moreno Romo, Natalia Rodriguez Nieva, Juan Manuel Mosquera Angarita, Jordi Anton Lopez, Esmeralda Nuñez-Cuadros, Gisela Diaz-Cordovés, Rocío Galindo-Zavala, Antonio Urda-Cardona, Antonio Fernández-Nebro, Daniel Álvarez de la Sierra, Marina Garcia Prat, Mónica Martínez Gallo, Ricardo Pujol Borrell, Ana M. Marín Sánchez, Etienne Merlin, Sylvie Fraitag, Jean-Louis Stephan, Federico Annoni, Giancarla Di Landro, Sofia Torreggiani, Marta Torcoletti, Georgina Tiller, Jo Buckle, Angela Cox, Peter Gowdie, Roger C. Allen, Jonathan D. Akikusa, Hayde G. Hernández-Huirache, Edel R. Rodea-Montero, William Fahy, Christelle Sordet, Karin B. Berggren, Johanna T. Kembe, Joyce Bos, Wineke Armbrust, Marco van Brussel, Jeanette Cappon, Pieter Dijkstra, Jan Geertzen, Elizabeth Legger, Marion van Rossum, Pieter Sauer, Otto Lelieveld, Levent Buluc, Gur Akansel, Bahar Muezzinoglu, Ljubov Rychkova, Tatyana Knyazeva, Anna Pogodina, Tatyana Belova, Tamara Mandzyak, Ekaterina Kulesh, Alessandro Cafarotti, Cosimo Giannini, Roberta Salvatore, Giuseppe Lapergola, Caterina Di Battista, Maria Loredana Marcovecchio, Raffaella Basilico, Piernicola Pelliccia, Francesco Chiarelli, Luciana Breda, Beverley Almeida, Sarah Tansley, Harsha Gunawardena, Neil McHugh, Juvenile Dermatomyositis Research Group (JDRG), Jessie Aouizerate, Marie De Antonio, Christine Barnerias, Guillaume Bassez, Isabelle Desguerre, Romain Gherardi, Jean-Luc Charuel, François-Jérôme Authier, Cyril Gitiaux, C. H. Spencer, Rabheh Abdul Aziz, Chack-Yung Yu, Brent Adler, Sharon Bout-Tabaku, Katherine Lintner, Melissa Moore-Clingenpeel, Liza McCann, Nicola Ambrose, Mario Cortina-Borja, Juvenile Dermatomyositis Cohort and Biomarker Study (JCDBS), Prasad T. Oommen, Fabian Speth, Johannes-Peter Haas, Working Group “Juvenile Dermatomyositis” of the German Society for Paediatric and Adolescent Rheumatology (GKJR), Claudio Lavarello, Gabriella Giancane, Angela Pistorio, Lisa Rider, Rohit Aggarwal, Sheila K. Oliveira, Ruben Cuttica, Michel Fischbach, Gary Sterba, Karine Brochard, Frank Dressler, Patrizia Barone, Ruben Burgos-Vargas, Elizabeth Candell Chalom, Marine Desjonqueres, Graciela Espada, Anders Fasth, Stella Maris Garay, Rose-Marie Herbigneaux, Claire Hoyoux, Chantal Job Deslandre, Frederick W. Miller, Jiri Vencovsky, Erdal Sag, Gulsev Kale, Haluk Topaloglu, Beril Talim, Francesco Zulian, Tadej Avcin, Roberto Marini, Anne Pagnier, Michel Rodiere, Christine Soler, Rebecca Ten Cate, Yosef Uziel, Jelena Vojinovic, Ana V. Villarreal, Nydia Acevedo, Yuridiana Ramirez, Enrique Faugier, Rocio Maldonado, Bita Arabshahi, John H. Lee, Ian Leibowitz, Lawrence O. Okong’o, Jo Wilmshurst, Monika Esser, Christiaan Scott, Ezgi Deniz Batu, Nagehan Emiroglu, Hafize Emine Sonmez, Gokcen Dilsa Tugcu, Zehra Serap Arici, Ebru Yalcin, Deniz Dogru, Ugur Ozcelik, Mithat Haliloglu, Nural Kiper, Masato Yashiro, Mutsuko Yamada, Toshihiko Yabuuchi, Tomonobu Kikkawa, Nobuyuki Nosaka, Yosuke Fujii, Yukie Saito, Hirokazu Tsukahara, Sulaiman M. Al-Mayouf, Nora AlMutiari, Mohammed Muzaffer, Rawiah shehata, Adel Al-Wahadneh, Reem Abdwani, Safia Al-Abrawi, Mohammed Abu-shukair, Zeyad El-Habahbeh, Abdullah Alsonbul, Aleksandra Szabat, Monika Chęć, Violetta Opoka-Winiarska, Biman Saikia, Ranjana W. Minz, Christine Arango, Clara Malagon, Maria D. P. Gomez, Angela C. Mosquera, Ricardo Yepez, Tatiana Gonzalez, Camilo Vargas, GRIP study group, Marta Balzarin, Biagio Castaldi, Elena Reffo, Francesca Sperotto, Giorgia Martini, Alessandra Meneghel, Ornella Milanesi, Ozgur Kasapçopur, Maria Teresa Terreri, Ekaterina Alexeeva, Maria Katsicas, Mikhail Kostik, Thomas Lehman, W.-Alberto Sifuentes-Giraldo, Vanessa Smith, Flavio Sztajnbok, Tadey Avcin, Maria Jose Santos, Dana Nemcova, Cristina Battagliotti, Liora Harel, Mahesh Janarthanan, Kathryn Torok, Nicola Helmus, Eileen Baildem, Michael Blakley, Kim Fligelstone, Antonia Kienast, Clare Pain, Amanda Saracino, Gabriele Simoni, Lisa Weibel, Maria K. Osminina, Nathalia A. Geppe, Olga V. Niconorova, Olesya V. Karashtina, Oksana V. Abbyasova, Olga V. Shpitonkova, Sinem Durmus, Hafize Uzun, Angela Mauro, Eleonora Fanti, Fabio Voller, Franca Rusconi, Fernando Garcia-Rodriguez, Ana V. Villarreal-Treviño, Angel J. Flores-Pineda, Paola B. Lara-Herrea, Diego R. Salinas-Encinas, Talia Diaz-Prieto, Maria R. Maldonado-Velazquez, Sarbelio Moreno-Espinosa, Enrique Faugier-Fuentes, Mirella Crapanzano, Ilaria Parissenti, Man S. Parihar, Pandiarajan Vignesh, ManojKumar Rohit, Kavitha Gopalan, Savita V. Attri, Alan Salama, David Jayne, Mark Little, Yulia Kostina, Galina Lyskina, Olga Shpitonkova, Alena Torbyak, Olga Shirinsky, Maria Francesca Gicchino, Maria Cristina Smaldone, Mario Diplomatico, Alma Nunzia Olivieri, C H. Spencer, Richard McClead, Hiren Patel, Chung-Yung Yu, Dita Cebecauerová, Tomáš Dallos, Edita Kabíčková, Martin Kynčl, Daniela Chroustová, Jozef Hoza, Dana Němcová, Vladimír Tesař, Pavla Doležalová, Tuncay Hazirolan, Fatih Ozaltin, Fabiola Almeida, Isabela H. Faria de Paula, Maíra M. Sampaio, Fernando N. Arita, Andressa G. Alves, Maria Carolina Santos, Eunice M. Okuda, Silvana B. Sacchetti, Fernanda Falcini, Marini Francesca, Gemma Lepri, Marco Matucci-Cerinic, Maria Luisa Brandi, Hakan Kisaoglu, Sema Misir, Selim Demir, Yuksel Aliyazicioglu, Mukaddes Kalyoncu, Carlos Eduardo Ramalho, Fabiola D. Almeida, Joan Calzada-Hernández, Rosa Bou, Estíbaliz Iglesias, Judith Sánchez-Manubens, Fredy Hermógenes Prada Martínez, Clara Giménez Roca, Sergi Borlan Fernández, Marek Bohm, Kamran Mahmood, Valentina Leone, Mark Wood, Ken-Ichi Yamaguchi, Satoshi Fujikawa, Working Group of Behçet’s Disease, Pediatric Rheumatology Association of Japan (PRAJ), Kyu Yeun Kim, Do Young Kim, Dong Soo Kim, Maka Ioseliani, Ivane Chkhaidze, Maia Lekishvili, Nana Tskhakaia, Shorena Tvalabeishvili, Aleksandre Kajrishvili, Maiko Takakura, Masaki Shimizu, Natsumi Inoue, Mao Mizuta, Akihiro Yachie, Giovanni Corsello, Maryam Piram, Carla Maldini, Sandra Biscardi, Nathalie Desuremain, Catherine Orzechowski, Emilie Georget, Delphine Regnard, Isabelle Kone-Paut, Alfred Mahr, Mihaela Sparchez, Zeno Sparchez, Nydia Acevedo Silva, Ana V. Villarreal Treviño, Yuridiana Ramirez Loyola, Talia Diaz Prieto, Enrique Faugier Fuentes, Maria D. R. Maldonado Velazquez, Pilar Perez, Sagar Bhattad, Ranjana Minz, Jitendra Shandilya, Pediatric Allergy and Immunology Unit, PGIMER, Chandigarh, Ana Villarreal, Yuridiana Ramírez, Zeynep Birsin Özçakar, Suat Fitoz, Fatos Yalcinkaya, Annacarin Horne, Francesca Minoia, Francesca Bovis, Sergio Davi, Priyankar Pal, Kimo Stein, Sandra Enciso, Michael Jeng, Despoina Maritsi, Randy C. Cron, Anne Thorwarth, Sae Lim von Stuckrad, Angela Rösen-Wolff, Hella Luksch, Patrick Hundsdoerfer, Peter Krawitz, Nuray Aktay Ayaz, Doğan Simsek, Şebnem Sara Kılıc, Emine Sonmez, Aysenur Pac Kisaarslan, Ozge Altug Gucenmez, Z. Serap Arıcı, Fatih Kelesoglu, Zelal Ekinci Ekinci, Maria Miranda-Garcia, Carolin Pretzer, Michael Frosch, F. Gohar, Angela McArdle, Niamh Callan, Belinda Hernandez, Miha Lavric, Oliver FitzGerald, Stephen R. Pennington, Joachim Peitz, Joern Kekow, Ariane Klein, Anna C. Schulz, Frank Weller-Heinemann, Anton Hospach, J-Peter Haas, BIKER collaborative group, Karen Put, Jessica Vandenhaute, Anneleen Avau, Annemarie van Nieuwenhuijze, Ellen Brisse, Tim Dierckx, Omer Rutgeerts, Josselyn E. Garcia-Perez, Jaan Toelen, Mark Waer, Georges Leclercq, An Goris, Johan Van Weyenbergh, Adrian Liston, Patrick Matthys, Carine H. Wouters, Yasuo Nakagishi, Michael J. Ombrello, Victoria Arthur, Anne Hinks, Patricia Woo, International Childhood Arthritis Genetics (INCHARGE) Consortium, Barbara Stanimirovic, Biljana Djurdjevic-Banjac, Olivera Ljuboja, Boris Hugle, MArgarita Onoufriou, Olga Vougiouka, Kenza Bouayed, Sanae El Hani, Imane Hafid, Nabiha Mikou, Nunu Shelia, Mari Laan, Jaanika Ilisson, and Chris Pruunsild

Subjects
Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Published
2017
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15. Proceedings of the 23rd Paediatric Rheumatology European Society Congress: part two

Author

Olga Lomakina, Ekaterina Alekseeva, Sania Valieva, Tatiana Bzarova, Irina Nikishina, Elena Zholobova, Svetlana Rodionovskaya, Maria Kaleda, Yasuo Nakagishi, Masaki Shimizu, Mao Mizuta, Akihiro Yachie, Yuko Sugita, Nami Okamoto, Kousuke Shabana, Takuji Murata, Hiroshi Tamai, Eve M. Smith, Peng Yin, Andrea L. Jorgensen, Michael W. Beresford, on behalf of On behalf of the UK JSLE Cohort Study, Antonio Eleuteri, Beatrice Goilav, Laura Lewandowski, Angel Phuti, Dawn Wahezi, Tamar Rubinstein, Caroline Jones, Paul Newland, Stephen Marks, Rachel Corkhill, Diana Ekdawy, Clarissa Pilkington, Kjell Tullus, Chaim Putterman, Chris Scott, Antony C. Fisher, Andrea Jorgensen, Ezgi Deniz Batu, Can Kosukcu, Ekim Taskiran, Sema Akman, Kubra Ozturk, Betul Sozeri, Erbil Unsal, Zelal Ekinci, Yelda Bilginer, Mehmet Alikasifoglu, Seza Ozen, Hanna Lythgoe, Hermine I. Brunner, Gaurav Gulati, Jordan T. Jones, Mekibib Altaye, Jamie Eaton, Mark Difrancesco, Joo Guan Yeo, Jingyao Leong, Loshinidevi D/O Thana Bathi, Thaschawee Arkachaisri, Salvatore Albani, Nagla Abdelrahman, Michael W Beresford, Valentina Leone, UK JSLE study group supported by the National Institute of Health Research Clinical Research Network, Noortje Groot, D. Shaikhani, I. E. M. Bultink, M. Bijl, R. J. E. M. Dolhain, Y. K. O. Teng, E. Zirkzee, K. de Leeuw, R. Fritsch-Stork, S. S. M. Kamphuis, Rachael D. Wright, Reem Abdawani, Laila Al Shaqshi, Ibrahim Al Zakwani, Natali W. Gormezano, David Kern, Oriany L. Pereira, Gladys C. C. Esteves, Adriana M. Sallum, Nadia E. Aikawa, Rosa M. Pereira, Clovis A. Silva, Eloisa Bonfa, Jessica Beckmann, Nora Bartholomä, Nils Venhoff, Philipp Henneke, Ulrich Salzer, Ales Janda, Alina Lucica Boteanu, Sandra Garrote Corral, Alberto Sifuentes Giraldo, Mariluz Gámir Gámir, Antonio Zea Mendoza, Amra Adrovic, Reyhan Dedeoglu, Sezgin Sahin, Kenan Barut, Aida Koka, Funda Oztunc, Ozgur Kasapcopur, Ana Luisa Rodriguez-Lozano, Francisco Rivas-Larrauri, Silvestre García de la Puente, Andressa G. F. Alves, Maria F. D. A. Giacomin, Juliana Farhat, Alfésio L. F. Braga, Adriana M. E. Sallum, Lúcia M. D. A. Campos, Luiz A. A. Pereira, Ana J. D. F. C. Lichtenfels, Clóvis A. Silva, Sylvia C. L. Farhat, Banu Acar, Z. Birsin Ozcakar, Nilgün Çakar, Nermin Uncu, Gökçe Gür, Semanur Özdel, Fatoş Yalçınkaya, Christiaan Scott, Nicky Brice, Peter Nourse, Christine Arango, Angela C. Mosquera, Clara Malagon, Ana P. Sakamoto, Marco F. C. D. Silva, Ananadreia S. Lopes, Gleice C. S. Russo, Adriana E. M. Sallum, Katia Kozu, Eloisa Bonfá, Claudia Saad-Magalhães, Rosa M. R. Pereira, Claudio A. Len, Maria T. Terreri, Deepti Suri, Siyaram Didel, Amit Rawat, Surjit Singh, Despoina Maritsi, MArgarita Onoufriou, Olga Vougiouka, Maria Tsolia, Edi Paleka Bosak, Mandica Vidović, Mirta Lamot, Lovro Lamot, Miroslav Harjaček, Erika Van Nieuwenhove, Adrian Liston, Carine Wouters, Fatemeh Tahghighi, Vahid Ziaee, Seid-Reza Raeeskarami, Francisca Aguiar, Sandra Pereira, Mariana Rodrigues, Cláudia Moura, Gustavo Rocha, Hercília Guimarães, Iva Brito, Rita Fonseca, Gerd Horneff, Ariane Klein, Kirsten Minden, Hans-Iko Huppertz, Frank Weller-Heinemann, Jasmin Kuemmerle-Deschner, J-Peter Haas, Anton Hospach, BIKER collaborative group, Ricardo Menendez-Castro, Boris Huegle, Johannes-Peter Haas, Joost Swart, Gabriella Giancane, Francesca Bovis, Elio Castagnola, Andreas Groll, Daniel J. Lovell, Tom Wolfs, Michael Hofer, Violeta Panaviene, Susan Nielsen, Jordi Anton, Florence Uettwiller, Valda Stanevicha, Maria Trachana, Denise Pires Marafon, Constantin Ailioaie, Elena Tsitsami, Sylvia Kamphuis, Troels Herlin, Pavla Doležalová, Gordana Susic, Berit Flatø, Flavio Sztajnbok, Angela Pistorio, Alberto Martini, Nico Wulffraat, Nicolino Ruperto, Marco Gattorno, Antonio Brucato, Martina Finetti, George Lazaros, Silvia Maestroni, Mara Carraro, Davide Cumetti, Alessandra Carobbio, Monia Lorini, Alessandro Rimini, Renzo Marcolongo, Anna Valenti, Gian Luca Erre, Riccardo Belli, Fiorenzo Gaita, Maria Pia Sormani, Massimo Imazio, Mario Abinun, Nicola Smith, Tim Rapley, Flora McErlane, Lianne Kearsley-Fleet, Kimme L. Hyrich, Helen Foster, Nikolay Tzaribachev, Andrew Zeft, Rolando Cimaz, John Bohnsack, Thomas Griffin, Ruy Carrasco, Jason Dare, Ivan Foeldvari, Richard Vehe, Teresa Simon, Hermine Brunner, S. Verazza, S. Davì, A. Consolaro, A. Insalaco, V. Gerloni, R. Cimaz, F. Zulian, S. Pastore, F. Corona, G. Conti, P. Barone, M. Cattalini, E. Cortis, L. Breda, A. N. Olivieri, A. Civino, R. Podda, D. Rigante, F. La Torre, G. D’Angelo, M. Jorini, R. Gallizzi, M. C. Maggio, R. Consolini, A. De Fanti, M. G. Alpigiani, A. Martini, A. Ravelli, on behalf of Italian Pediatric Rheumatology Study Group, Aysenur Pac Kısaarslan, Zubeyde Gunduz, Ruhan Dusunsel, Ismail Dursun, Hakan Poyrazoglu, Ekaterina Kuchinskaya, Farida Abduragimova, Mikhail Kostik, Erik Sundberg, Soley Omarsdottir, Lena Klevenvall, Helena Erlandsson-Harris, Gokalp Basbozkurt, Ozge Erdemli, Dogan Simsek, Fatih Yazici, Yildirim Karsioglu, Aysen Tezcaner, Dilek Keskin, Huseyin Ozkan, Cengizhan Acikel, Erkan Demirkaya, Ilonka Orbán, Krisztina Sevcic, Valentin Brodszky, Emese Kiss, Ismaiel A. Tekko, Madeleine Rooney, James McElnay, Cliff Taggart, Helen McCarthy, Ryan F. Donnelly, Drug Delivery Group, Mary Slatter, Zohreh Nademi, Mark Friswell, Sharmila Jandial, Terence Flood, Sophie Hambleton, Andrew Gennery, Andrew Cant, Phoi-Ngoc Duong, Isabelle Koné-Paut, Giovanni Filocamo, María Luz Gamir, Helga Sanner, Laura Carenini, Mesut Topdemir, Yildirim Karslioglu, Faysal Gok, Nadezhda Tsurikova, Elena Ligostaeva, Navdha R. Ramchurn, O. Kostareva, I. Nikishina, S. Arsenyeva, S. Rodionovskaya, M. Kaleda, D. Alexeev, Ismail Dursun Dursun, Sara Murias, Estefania Barral, Rosa Alcobendas, Eugenia Enriquez, Agustin Remesal, Jaime de Inocencio, Tania M. Castro, Simone A. Lotufo, Tatjana Freye, Raffaella Carlomagno, Thomas Zumbrunn, Jan Bonhoeffer, Elvira Cannizzaro Schneider, Daniela Kaiser, Michaël Hofer, Véronique Hentgen, Andreas Woerner, Juvenile Inflammatory Rheumatism (JIR) Cohort, Tobias Schwarz, Jens Klotsche, Martina Niewerth, Gerd Ganser, ICON study group, Jerold Jeyaratnam, Nienke ter Haar, Donato Rigante, Fatma Dedeoglu, Ezgi Baris, Sebastiaan Vastert, Joost Frenkel, Jonathan S. Hausmann, Kathleen G. Lomax, Ari Shapiro, Karen L. Durrant, P. A. Brogan, M. Hofer, J. B. Kuemmerle-Deschner, B. Lauwerys, A. Speziale, K. Leon, X. Wei, R. M. Laxer, Sara Signa, Marta Rusmini, Elena Campione, Sabrina Chiesa, Alice Grossi, Alessia Omenetti, Roberta Caorsi, Gianmaria Viglizzo, Isabella Ceccherini, Silvia Federici, Helen Lachmann, Nicola Ruperto, on behalf of PRINTO and Eurofever Registry, Federica Vanoni, on behalf of PRINTO and Eurofever Project, Sonia Melo Gomes, Ebun Omoyinmi, Juan I. Arostegui, Eva Gonzalez-Roca, Despina Eleftheriou, Nigel Klein, Paul Brogan, Stefano Volpi, Elettra Santori, Paolo Picco, Claudia Pastorino, Gillian Rice, Alessandra Tesser, Yanick Crow, Fabio Candotti, Ada B. Sinoplu, Gozde Yucel, Gizem Pamuk, Laura O. Damian, Cecilia Lazea, Mihaela Sparchez, Paulina Vele, Laura Muntean, Adriana Albu, Simona Rednic, Calin Lazar, Leonardo O. Mendonça, Alessandra Pontillo, Jorge Kalil, Fabio M. Castro, Myrthes T. Barros, Manuela Pardeo, Virginia Messia, Fabrizio De Benedetti, Antonella Insalaco, Giorgia Malighetti, Chiara Gorio, Francesca Ricci, Ilaria Parissenti, Paola Montesano, Barbara Bonafini, Veronica Medeghini, Marco Cattalini, Lucio Giordano, Giulia Zani, Rosalba Ferraro, Donatella Vairo, Silvia Giliani, Maria Cristina Maggio, Girolamo Luppino, Giovanni Corsello, Maria Isabel Gonzalez Fernandez, Berta Lopez Montesinos, Adriana Rodriguez Vidal, Juan I. Arostegui Gorospe, Inmaculada Calvo Penades, Nadia K. Rafiq, Karen Wynne, Khalid Hussain, Paul A. Brogan, Elizabeth Ang, Nicholas Ng, Ayla Kacar, Ozge Altug Gucenmez, Balahan Makay, Sevket Erbil Unsal, Yasin Sahin, Tufan Kutlu, Fugen Cullu-Cokugras, Hasret Ayyildiz-Civan, Tulay Erkan, Sana Al Zuhbi, Eiman Abdalla, Ricardo A. Russo, María M. Katsicas, Francesca Minoia, Angelo Ravelli, Sagar Bhattad, Anju Gupta, Vignesh Pandiarajan, Ritambhra Nada, Kaara Tiewsoh, Philip Hawkins, Dorota Rowczenio, Sarka Fingerhutova, Jana Franova, Leona Prochazkova, Eva Hlavackova, Pavla Dolezalova, Havva Evrengül, Selçuk Yüksel, Mustafa Doğan, Dolunay Gürses, Harun Evrengül, Silvia De Pauli, Serena Pastore, Anna Monica Bianco, Giovanni Maria Severini, Andrea Taddio, Alberto Tommasini, Svetlana O. Salugina, Evgeny Fedorov, Elena Kamenets, Ekaterina Zaharova, Tatiana Sleptsova, Ekaterina Alexeeva, Kirill Savostyanov, Alexander Pushkov, Tatyana Bzarova, Saniya Valieva, Rina Denisova, Kseniya Isayeva, Evgeniya Chistyakova, Margarita Soloshenko, Elena Kaschenko, Utako Kaneko, Chihaya Imai, Akihiko Saitoh, Vitor A. Teixeira, Filipa O. Ramos, Manuela Costa, Yonatan Butbul Aviel, Shafe Fahoum, Riva Brik, Zeynep Birsin Özçakar, Banu Acar Celikel, Fatos Yalcinkaya, Benedetta Schiappapietra, Sergio Davi’, Federica Mongini, Luisa Giannone, Cecilia Bava, Maria Giannina Alpigiani, Alessandro Consolaro, Dragana S. Lazarevic, Jelena Vojinovic, Jelena Basic, Valentina Muratore, Valentina Marzetti, Neus Quilis, Belen Serrano Benavente, Alessandra Alongi, Adele Civino, Lorenzo Quartulli, Giedre Januskeviciute, Pieter van Dijkhuizen, N. Groot, W. van Dijk, A. Kardolus, Raul Gutiérrez Suárez, Ellen B. Nordal, Veronika G. Rypdal, Lillemor Berntson, Maria Ekelund, Kristiina Aalto, Suvi Peltoniemi, Marek Zak, Mia Glerup, Ellen D. Arnstad, Anders Fasth, Marite Rygg, the Nordic Study Group of Pediatric Rheumatology (NoSPeR), Ana Catarina Duarte, Sandra Sousa, Lídia Teixeira, Ana Cordeiro, Mª José Santos, Ana Filipa Mourão, Maria José Santos, Mónica Eusébio, Ana Lopes, Filipa Oliveira-Ramos, Manuel Salgado, Paula Estanqueiro, José Melo-Gomes, Fernando Martins, José Costa, Carolina Furtado, Ricardo Figueira, Jaime C. Branco, João E. Fonseca, Helena Canhão, Ana F. Mourão, Maria Jose Santos, Andrea Coda, Samuel Cassidy, Kerry West, Gordon Hendry, Debra Grech, Julie Jones, Fiona Hawke, Davinder Singh Grewal, Charlene Foley, Orla Killeen, Emma MacDermott, Douglas Veale, Ursula Fearon, Dilek Konukbay, Ela Tarakci, Nilay Arman, Sezgin Şahin, Jane Munro, Esi Morgan, Meredith Riebschleger, Jennifer Horonjeff, Vibeke Strand, Clifton Bingham, Ma. Theresa M. Collante, Margarita Ganeva, Stefan Stefanov, Albena Telcharova, Dimitrina Mihaylova, Radoslava Saraeva, Reni Tzveova, Radka Kaneva, Adelina Tsakova, Katya Temelkova, GRANT Medical University, Sofia 68/, Maria Mercedes C. Picarelli, Luiz C. Danzmann, Florencia Barbé-Tuana, Lucas K. Grun, Marcus H. Jones, Marijan Frković, Karla Ištuk, Ika Birkić, Saša Sršen, Marija Jelušić, Alan Easton, Rachael Quarmby, Raju Khubchandani, Mercedes Chan, Radoslav Srp, Katerina Kobrova, Dana Nemcova, Jozef Hoza, Michal Uher, Melania Saifridova, Lenka Linkova, Sirirat Charuvanij, Isree Leelayuwattanakul, Thita Pacharapakornpong, Sakda A.-O. Vallipakorn, Butsabong Lerkvaleekul, Soamarat Vilaiyuk, Stefano Lanni, Sergio Davì, Randy Q. Cron, Chiara Passarelli, Elisa Pisaneschi, Antonio Novelli, Claudia Bracaglia, Ivan Caiello, Kathy de Graaf, Florence Guilhot, Walter Ferlin, Grant Schulert, Alexi A. Grom, Robert Nelson, Cristina de Min, Dirk Holzinger, Christoph Kessel, Ndate Fall, Alexei Grom, Wilco de Jager, Raffaele Strippoli, Anna Horne, Stephan Ehl, Sandra Ammann, Kai Lehmberg, Karin Beutel, Dirk Foell, AnnaCarin Horne, Laura Pagani, Graciela Espada, Yi-jin Gao, Susan Shenoi, Sheila Weitzman, Giusi Prencipe, Antonia Pascarella, Walter G. Ferlin, Laurence Chatel, Philippe Jacqmin, Kathy De Graaf, Maria Ballabio, Zoë Johnson, Geneviève Lapeyre, Fabrizio de Benedetti, de Min Cristina, Hiroyuki Wakiguchi, Shunji Hasegawa, Reiji Hirano, Fumiko Okazaki, Tamaki Nakamura, Hidenobu Kaneyasu, Shouichi Ohga, Kazuko Yamazaki, Tomo Nozawa, Taichi Kanetaka, Shuichi Ito, Shumpei Yokota, Kirsty McLellan, Ishbel MacGregor, Neil Martin, Joyce Davidson, Sandra Hansmann, Andreas Eikelberg, Iris Haug, Sabrina Schuller, Susanne M. Benseler, Single Hub and Access point for paediatric Rheumatology in Europe (SHARE), Liliia S. Nazarova, Kseniia V. Danilko, Viktor A. Malievsky, Tatiana V. Viktorova, Angela Mauro, Angela Barnicoat, Jane Hurst, Nathalie Canham, Sandrine Lacassagne, Anastasia Wiener, Boris Hügle, Bernd Denecke, Ivan Costa-Filho, Johannes Peter Haas, Klaus Tenbrock, David Popp, Arjan Boltjes, Frank Rühle, Stefanie Herresthal, Femke van Wijk, Joachim Schultze, Monika Stoll, Luisa Klotz, Thomas Vogl, Johannes Roth, Estefania Quesada-Masachs, Daniel Álvarez de la Sierra, Marina Garcia Prat, Ana M. Marín Sánchez, Ricardo Pujol Borrell, Sara Marsal Barril, Mónica Martínez Gallo, Consuelo Modesto Caballero, Iryna Chyzheuskaya, Lyudmyla M. Byelyaeva, Rostislav M. Filonovich, Helena K. Khrustaleva, Larisa I. Zajtseva, Tamara M. Yuraga, Thomas Giner, Lukas Hackl, Julia Albrecht, Reinhard Würzner, Juergen Brunner, Marta Minute, Fulvio Parentin, Agostino Nocerino, Mette Nørgaard, Mikel Alberdi-Saugstrup, Marek S. Zak, Susan M. Nielsen, Ellen Nordal, Klaus G. Müller, Nordic Study Group of Pediatric Rheumatology (NoSPeR), Mojca Zajc Avramovič, Vita Dolžan, Nataša Toplak, Tadej Avčin, N. Ruperto, D. J. Lovell, C. Wallace, M. Toth, I. Foeldvari, J. Bohnsack, D. Milojevic, C. Rabinovich, D. Kingsbury, K. Marzan, P. Quartier, K. Minden, E. Chalom, G. Horneff, R. M. Kuester, J. Dare, M. Heinrich, H. Kupper, J. Kalabic, H. I. Brunner, on behalf of PRINTO and PRCSG, Ruben Burgos-Vargas, Tamas Constantin, Joke Dehoorne, Valda Stanevica, Katarzyna Kobusinska, Zbigniew Zuber, Richard Mouy, Ingrida Rumba-Rozenfelde, Chantal Job-Deslandre, Ronald Pederson, Jack Bukowski, Tina Hinnershitz, Bonnie Vlahos, Paula Keskitalo, Salla Kangas, Paula Vähäsalo, Raul A. Chavez Valencia, David Martino, Anne-Louise Ponsonby, Rachel Chiaroni-Clarke, Braydon Meyer, Roger C. Allen, Jonathan D. Akikusa, Jeffrey M. Craig, Richard Saffrey, Justine A. Ellis, Carol Wallace, Yosef Uziel, Gary Sterba, Rayfel Schneider, Ricardo Russo, Athimalaipet V. Ramanan, Jana Pachlopnik Schmid, Kim E Nichols, Paivi Miettunen, Toshiyuki Kitoh, Norman T. Ilowite, Jan-Inge Henter, Alexei A Grom, Edward M. Behrens, Tadej Avcin, Maurizio Aricò, Sriharsha Grevich, Peggy Lee, Sarah Ringold, Brian Leroux, Hannah Leahey, Megan Yuasa, Jessica Foster, Jeremy Sokolove, Lauren Lahey, William Robinson, Joshua Newson, Anne Stevens, Stephanie J. W. Shoop, Suzanne M. M. Verstappen, Wendy Thomson, Janet E. McDonagh, CAPS, Timothy Beukelman, Yuki Kimura, Marc Natter, Norm Ilowite, Kelly Mieszkalski, Grendel Burrell, Brian Best, Helen Bristow, Shannon Carr, Anne Dennos, Rachel Kaufmann, Laura Schanberg, for the CARRA Registry Investigators, Gabriele Simonini, Francesca Lancini, Margaux Gerbaux, Phu-Quoc Lê, Laurence Goffin, Valérie Badot, Céline La, Laure Caspers, François Willermain, Alina Ferster, Maria Ceci, Francesco Licciardi, Marco Turco, Francesca Santarelli, Davide Montin, Claudia Toppino, Clotilde Alizzi, Bruno Papia, Beatrice Vergara, Umberto Corpora, Luca Messina, Maria Tsinti, Vasiliko Dermentzoglou, Panagiotis Tziavas, Marija Perica, Lana Tambić Bukovac, Mustafa Çakan, Nuray Aktay Ayaz, Gonca Keskindemirci, Michael Lang, Catherine Laing, Susanne Benseler, Tommy Gerschman, Nadia Luca, Heinrike Schmeling, Anastasia Dropol, Jaymi Taiani, Nicole Johnson, Brian Rusted, Panagiota Nalbanti, Polyxeni Pratsidou, Grigoris Pardalos, Vasiliki Tzimouli, Anna Taparkou, Maria Stavrakidou, Fotios Papachristou, Florence Kanakoudi-Tsakalidou, Peter Bale, Emily Robinson, Jason Palman, Elizabeth Ralph, Kimberly Gilmour, Clare Heard, Lucy R. Wedderburn, Yara Barrense-Dias, Antonarakis Gregory, Dhouib Amira, Scolozzi Paolo, Hanquinet Sylviane, Hofer Michaël, Nataliya Panko, Salah Shokry, Liudmila Rakovska, Sally Pino, Adriana Diaz-Maldonado, Pilar Guarnizo, Sofia Torreggiani, Paolo Cressoni, Umberto Garagiola, Giancarla Di Landro, Giampietro Farronato, Fabrizia Corona, Samantha Bell, Parveen Bhatti, Lee Nelson, Beth A. Mueller, T. A. Simon, A. Baheti, N. Ray, Z. Guo, Anasuya Hazra, Thomas Stock, Ronnie Wang, Charles Mebus, Christine Alvey, Manisha Lamba, Sriram Krishnaswami, Umberto Conte, Min Wang, Daniel Kingsbury, Elena Koskova, Elzbieta Smolewska, Richard K. Vehe, Daniel Lovell, Tomohiro Kubota, Junko Yasumura, Toshitaka Kizawa, Masato Yashiro, Tsuyoshi Yamatou, Yuichi Yamasaki, Syuji Takei, Yoshifumi Kawano, Ulrika Järpemo Nykvist, Bo Magnusson, Rikard Wicksell, Karin Palmblad, Gunnar L. Olsson, Mohammadreza Modaressi, Mohammad-Hassan Moradinejad, Valentina Seraya, Alisa Vitebskaya, Veronica Moshe, Gil Amarilyo, Liora Harel, Phillip J Hashkes, Amir Mendelson, Noa Rabinowicz, Yonit Reis, Zane Dāvidsone, Arina Lazareva, Ruta Šantere, Dace Bērziņa, Valda Staņēviča, Giulia Camilla Varnier, Susan Maillard, Cristina Ferrari, Silvia Zaffarano, Juvenile Dermatomyositis Research Group and European Federation of Immunological Societies, Judith Wienke, Felicitas Bellutti Enders, Lucas L. van den Hoogen, Jorre S. Mertens, Timothy R. Radstake, Henny G. Hotten, Ruth Fritsch, Lucy Wedderburn, Kiran Nistala, Berent Prakken, Annet van Royen-Kerkhof, Mohammad Alhemairi, Mohammed Muzaffer, Pieter Van Dijkhuizen, Claire T. Deakin, Stefania Simou, Maria De Iorio, Qiong Wu, Tania Amin, Lee Dossetter, Juvenile Dermatomyositis Research Group (JDRG), Raquel Campanilho-Marques, Claire Deakin, Clarissa A. Pilkington, on behalf of Juvenile Dermatomyositis Research Group (JDRG), Silvia Rosina, Sirisucha Soponkanaporn, on behalf of the UK Juvenile Dermatomyositis Research Group (JDRG), Zehra S. Arıcı, Gökçen D. Tuğcu, Ezgi D. Batu, Hafize E. Sönmez, Deniz Doğru-Ersöz, Beril Talim, Nural Kiper, Seza Özen, Alexander Solyom, Ezgi Batu, John Mitchell, Ariana Kariminejad, Fatemeh Hadipour, Zahra Hadipour, Marta Torcoletti, Carlo Agostoni, Maja Di Rocco, Pranoot Tanpaiboon, Andrea Superti-Furga, Luisa Bonafé, Nur Arslan, Norberto Guelbert, Karoline Ehlert, Giedre Grigelioniene, Ratna Puri, Edward Schuchman, Pilar Gomez, Tatiana Gonzalez, Ricardo Yepez, Camilo Vargas, GRIP study group, Falcini Fernanda, Gemma Lepri, Alessandra Ferrari, Marco Matucci-Cerinic, Antonella Meini, Gian Marco Moneta, Emiliano Marasco, Rebecca Nicolai, Luisa Bracci-Laudiero, Olga Kopchak, Alexander Mushkin, Alexey Maletin, Catalina Mosquera, Rita A. Amorim, Juliana Molina, Gustavo Moreira, Flávia H. Santos, Melissa Fraga, Livia Keppeke, Vanessa M. Silva, Camila Hirotsu, Sergio Tufik, Maria Teresa Terreri, Vinícius L. Braga, Maria Beatriz Fonseca, Vania Schinzel, Maria Teresa R. Terreri, Liliana Jorge, Liana Guerra, Edson Amaro Junior, Maria Cristina Castiglione, Alessandra Tricarico, Emily Boulter, Andre Schultz, Kevin Murray, Fernanda Falcini, Stefano Stagi, Eleonora Bellucci, Ingrid H. R. Grein, Gecilmara Pileggi, Natália B. F. Pinto, Aline L. de Oliveira, Lyudmila Belyaeva, Rostislav Filonovich, Helena Khrustaleva, Larisa Zajtseva, Jaanika Ilisson, Chris Pruunsild, Olivier Gilliaux, Francis Corazza, Christophe Lelubre, on behalf of PANLAR Pediatric Rheumatology Study Group, Zoilo Morel, Claudia Saad-Magalhães C, Luis Lira, Mabel Ladino, Ruth Eraso, Ivonne Arroyo, Clovis Silva, Carlos Rose, and PANLAR Pediatric Rheumatology Study Group

Subjects
Pediatrics, RJ1-570, Diseases of the musculoskeletal system, RC925-935
Published
2017
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16. Systemic-onset juvenile idiopathic arthritis: the changing life of a rare disease

Author

Rolando Cimaz, Annette Von Scheven, and Michael Hofer

Subjects
pediatric rheumatology, Medicine
Abstract

Systemic-onset juvenile idiopathic arthritis (SoJIA), sometimes called Still’s disease, is a systemic inflammatory disease classified within the spectrum of juvenile idiopathic arthritis (JIA). It is an orphan disease with often a chronic course and a major impact on the affected children and their families. This disorder is unique in terms of clinical manifestations, prognosis and response to conventional immunosuppressants. The objectives of this review are to describe SoJIA and emphasise the recent advances in the pathogenesis and treatment, which have transformed the care and the prognosis of this potentially life-threatening paediatric condition.

Published
2012
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17. Methylprednisolone versus intravenous immunoglobulins in children with paediatric inflammatory multisystem syndrome temporally associated with SARS-CoV-2 (PIMS-TS): an open-label, multicentre, randomised trial

Author

Tatjana Welzel, Andrew Atkinson, Nina Schöbi, Maya C Andre, Douggl G N Bailey, Geraldine Blanchard-Rohner, Michael Buettcher, Serge Grazioli, Henrik Koehler, Marie-Helene Perez, Johannes Trück, Federica Vanoni, Petra Zimmermann, Carlos Sanchez, Julia A Bielicki, Luregn J Schlapbach, Andreas Bieri, Spyridoula Gysi, Indra Janz, Birgit Donner, Jürg Hammer, Ulrich Heininger, Malte Kohns Vasconcelos, Nicole Mettauer, Alexandra Meyer, Diana Reppucci, Chloé Schlaeppi, Daniel Trachsel, Nina Vaezipour, Clemens von Kalckreuth, Andreas Woerner, Andreas Zutter, Lisa Kottanattu, Calogero Mazzara, Alessia Severi Conti, Christoph Aebi, Philipp K A Agyeman, Andrea Duppenthaler, Martin Glöckler, Sabine Pallivathukal, Thomas Riedel, Hong-Phuc Cudré-Cung, Mladen Pavlovic, Alice Bordessoule, Arnaud G L'Huillier, Anne-Laure Martin, Angelo Polito, Marie Rohr, Maria Isabel Rodriguez, Noemie Wagner, Vivianne Chanez, Sabrina Bressieux-Degueldre, Thomas Ferry, Michael Hofer, David Longchamp, Julia Natterer, Rebecca Oppenheim, Alex Donas, Sara Germann, Katja Hrup, Daniela Kaiser, Hans Peter Kuen, Michaela Lütolf Erni, Katharina Schwendener Scholl, Janine Stritt, Katharina Wechselberger, Ingrid Beck, André Birkenmaier, Martin Flade, Bjarte Rogdo, Ivo Iglowstein, Philip Lorenz, Tanja Wachinger, Konstanze Zöhrer, Barbara Brotschi, Patrick M Meyer Sauteur, Jana Pachlopnik Schmid, Seraina Prader, Michelle Seiler, Kathrin Weber, Daniela Wütz, Elizabeth Whittaker, and Saul N Faust

Subjects
Pediatrics, Perinatology and Child Health, Developmental and Educational Psychology
Published
2023

21. Breaking down the fences among registries on Autoinflammatory diseases: the E-Merge project

Author

Yulia Vyzhga, Veronique Hentgen, Roberta Caorsi, Helmut Wittkowski, Michael Hofer, Nicolino Ruperto, Elke Lainka, Katerina Theodoropoulou, Dirk Foell, Eugenia Mosci, and Marco Gattorno

Abstract

Background. Between the various number of different AIDs, the absolute majority remains to be rare, with a single representative among big populations. Project, endorsed by PRES and supported by the EMERGE fellowship program, performed in line with the Metadata registry for the ERN RITA (MeRITA), had an objective to perform data harmonization attempt of the most relevant research questions regarding clinical features, diagnostic strategy, and optimal management of the autoinflammatory diseases. Results. Analysis of 3 large European registries: Eurofever, JIR-cohort, and AID-Net with total coverage of 7825 patients from 278 participating centers all over the world was performed in the context of the epidemiological and clinical data merging. Information summarized in the registries covers not only pediatric patients, but as well adults, with newly diagnosed AID. General aspects of the existing epidemiological data were discussed in the context of patient distribution around the world, potential diagnostic delay, access to genetic testing, and availability of the treatment. Conclusions. Overall work results indicate great potential for upcoming collaborative work using existing data in cohorts, which will definitely benefit the quality of medical care performed for patients with autoinflammatory diseases.

Published
2023

22. Évènements indésirables infectieux chez des enfants atteints d’arthrite juvénile idiopathique et traités en vie réelle par biothérapies : données issues de la JIR cohorte

Author

Cécile Dumaine, Véronique Hentgen, Sara Bekkar, Michael Hofer, Andreas Woerner, Aurélia Carbasse, Natalia Cabrera, Salma Malik, Pascal Pillet, Silke Schroeder, Alexandre Belot, Kenza Bouayed, Annette von Scheven, and Carine Wouters

Subjects
030203 arthritis & rheumatology, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, 030212 general & internal medicine
Abstract

Resume Objectifs Notre etude avait pour principal objectif d’evaluer les evenements indesirables infectieux survenus chez des enfants atteints d’arthrite juvenile idiopathique (AJI) et traites par biotherapies. Methodes Les patients ont ete selectionnes dans le module retrospectif de la cohorte JIR, donnees collectees entre janvier 2001 et aout 2015. Tous les effets indesirables infectieux (EII) ont ete enregistres. Pour chaque EII, la date, la severite, le recours a l’hospitalisation, le type de pathogene et l’organe concerne ont ete notes. L’incidence des EII a ete exprimee en nombre d’evenements pour 100 patients/annees (100p-a) et l’OR a ete calcule. Resultats Six cent soixante-dix-sept patients atteints d’AJI ont ete inclus dans l’etude. Au total, 3075,4 personnes/annees d’exposition ont ete analysees. Cent quatre-vingt-quatre evenements indesirables infectieux ont ete decrits (6,0 evenements/100 p-a) : Tocilizumab (TCZ) 15,5/100 p-a, Canakinumab (CAN) 9,6/100 p-a, Abatacept (ABA) 7,4/100 p-a, Golimumab (GOL) 6,9/100 p-a, Anakinra (ANA) 6,7/100 p-a, Infliximab 6,3/100 p-a, Etanercept 4,8/100 p-a et Adalimumab 3,7/100 p-a. Le risque de developper une infection s’est avere significativement plus eleve avec les antagonistes de l’IL-6 ou de l’IL-1 comparativement aux anti-TNF. Dans 40,8 % des cas, les evenements indesirables infectieux (EII) interessaient les voies respiratoires superieures ou le systeme ORL (oreilles, nez, gorge, larynx). Douze evenements indesirables ont ete qualifies de grave ou tres graves (0,4/100p-a). Il n’a ete rapporte aucun cas de tuberculose ou de deces. Conclusion Les complications infectieuses secondaires aux traitements biologiques, chez les enfants atteints d’AJI, sont rares et dans la plupart des cas d’intensite legere a moderee, et touchent preferentiellement les voies respiratoires superieures ou la sphere ORL.

Published
2021

23. Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised Phase 3 CLUSTER trial

Author

Jeremy Levy, Tamás Constantin, Joost Frenkel, Brigitte Bader-Meunier, Anna Shcherbina, Elise L. Dekker, Marco Gattorno, Anna Simon, Inmaculada Calvo, Michael Hofer, Jerold Jeyaratnam, Alberto Martini, Fabrizio De Benedetti, and Bas Vastert

Subjects
medicine.medical_specialty, Antibodies, Monoclonal, Humanized, Double-Blind Method, Rheumatology, Internal medicine, Hyper IgD syndrome, Mevalonate kinase deficiency, auto-inflammatory diseases, canakinumab, interleukin-1, medicine, Humans, Pharmacology (medical), Serum amyloid A, Adverse effect, Serum Amyloid A Protein, Mevalonate kinase deficiency, biology, business.industry, Cumulative dose, Hyper-IgD syndrome, C-reactive protein, Antibodies, Monoclonal, medicine.disease, Clinical trial, Canakinumab, Treatment Outcome, biology.protein, Mevalonate Kinase Deficiency, business, Inflammatory diseases Radboud Institute for Molecular Life Sciences [Radboudumc 5], medicine.drug
Abstract

Objectives To evaluate the long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency during the open label extension (weeks 41–113) of the randomized controlled CLUSTER trial. Methods During a 72-week period, patients received open-label canakinumab 150 or 300 mg, every 4 or 8 weeks. The disease activity was evaluated every 8 weeks using physician global assessment and counting the number of flares. Concentrations of CRP and serum amyloid A protein were measured. The safety was studied by determination and classification of observed adverse events. The safety and efficacy were analysed separately in three subgroups of patients receiving a cumulative dose of less than Results Of the 74 patients who started the CLUSTER study, 66 entered Epoch 4 and 65 completed it. During the 72-week period, 42 (64%) patients experienced no flares, while 13 (20%) had one flare, as compared with a median of 12 flares per year reported at baseline. Low physician global assessment scores were seen at the end of the study for all groups with >90% reporting minimal disease activity or none at all. Median CRP concentrations were consistently equal or lower than 10 mg/l, while median serum amyloid A concentrations remained only slightly above the normal range of 10 mg/l. The study showed no new or unexpected adverse events. Conclusion Canakinumab proved effective to control disease activity and prevent flares in mevalonate kinase deficiency during the 72-week study period. No new safety concerns were reported. Trial registration NCT02059291. https://clinicaltrials.gov.

Published
2021

25. Comparaison des critères de classification pédiatrique et adulte dans les arthrites juvéniles idiopathiques à la transition des soins pédiatriques aux soins adultes

Author

Michael Hofer, Agnès Duquesne, Alexandre Belot, Marie Couret, Adrien Rougelot, Bérengère Aubry-Rozier, Jean-Paul Larbre, Amandine Beaumel, Fabienne Coury, Natalia Cabrera, and Anne-Cécile Debrach

Subjects
Rheumatology
Abstract

Resume Objectifs Determiner les caracteristiques des patients avec une arthrite juvenile idiopathique (AJI) vus a la transition dans le but de comparer les criteres de classification pediatrique et adulte. Methodes Les patients avec une AJI definie par la classification ILAR vus en consultation de transition entre 2010 et 2017 ont ete inclus dans une etude retrospective bicentrique (Lyon, Lausanne). Les criteres de classification des AJI ont ete compares aux criteres ACR/EULAR de 2010 de la polyarthrite rhumatoide (PR), aux criteres de Yamaguchi pour la maladie de Still de l’adulte (MSA), aux criteres ASAS de spondyloarthrite et CASPAR du rhumatisme psoriasique. Resultats Cent trente patients ont ete inclus : 13,9 % avec une AJI systemique, 22,3 % avec une AJI polyarticulaire, 22,3 % avec une AJI oligoarticulaire, 34,6 % avec une AJI associee aux enthesites (ERA) et 6,9 % avec une AJI psoriasique. Parmi eux, 13,1 % ont souffert d’uveite. Les atteintes structurales etaient representees par 14,5 % d’erosions et de carpite, principalement observees dans les AJI psoriasiques, polyarticulaires ou systemiques. Dans le groupe ERA, 37,5 % des patients presentaient une sacro-iliite radiologique. En comparant les AJI aux rhumatismes adultes, nous avons trouve que : 66,6 % des patients avec une AJI systemique remplissaient les criteres de MSA, 87,5 % et 9,5 % des AJI polyarticulaires, respectivement avec et sans facteur rhumatoide, remplissaient les criteres de PR et 34,5 % des AJI oligoarticulaires remplissaient les criteres de spondyloarthrite. Enfin, 77,7 % des patients avec une ERA remplissaient les criteres de spondyloarthrite et 100 % des patients avec une AJI psoriasique remplissaient les criteres de rhumatisme psoriasique. Conclusion Les AJI oligoarticulaires et polyarticulaires sans facteur rhumatoide semblent etre des entites pediatriques, tandis que les autres types d’AJI remplissent les criteres de classification des rhumatismes adultes correspondants.

Published
2021

31. A Structural View on the Stereospecificity of Plant Borneol‐Type Dehydrogenases

Author

Carl P. O. Helmer, Elia Calderini, Robert Kourist, Loreto P. Parra, Volker Sieber, Andrea M Chánique, Mónica P. Pantín, Bernhard Loll, Michael Hofer, Nicole Dimos, and Ivana Drienovská

Subjects
Borneol, Full Paper, Terpenoids, Organic Chemistry, Enantioselective synthesis, Dehydrogenase, Enantioselectivity, Full Papers, Catalysis, ddc, Kinetic resolution, Inorganic Chemistry, Salvia rosmarinus, chemistry.chemical_compound, Camphor, Stereospecificity, chemistry, Docking (molecular), Organic chemistry, Physical and Theoretical Chemistry, Enantiomer, 500 Naturwissenschaften und Mathematik::540 Chemie::540 Chemie und zugeordnete Wissenschaften, Short-chain dehydrogenase-reductase (SDR)
Abstract

The development of sustainable processes for the valorization of byproducts and other waste streams remains an ongoing challenge in the field of catalysis. Racemic borneol, isoborneol and camphor are currently produced from α‐pinene, a side product from the production of cellulose. The pure enantiomers of these monoterpenoids have numerous applications in cosmetics and act as reagents for asymmetric synthesis, making an enzymatic route for their separation into optically pure enantiomers a desirable goal. Known short‐chain borneol‐type dehydrogenases (BDHs) from plants and bacteria lack the required specificity, stability or activity for industrial utilization. Prompted by reports on the presence of pure (−)‐borneol and (−)‐camphor in essential oils from rosemary, we set out to investigate dehydrogenases from the genus Salvia and discovered a dehydrogenase with high specificity (E>120) and high specific activity (>0.02 U mg−1) for borneol and isoborneol. Compared to other specific dehydrogenases, the one reported here shows remarkably higher stability, which was exploited to obtain the first three‐dimensional structure of an enantiospecific borneol‐type short‐chain dehydrogenase. This, together with docking studies, led to the identification of a hydrophobic pocket in the enzyme that plays a crucial role in the stereo discrimination of bornane‐type monoterpenoids. The kinetic resolution of borneol and isoborneol can be easily integrated into the existing synthetic route from α‐pinene to camphor thereby allowing the facile synthesis of optically pure monoterpenols from an abundant renewable source., Highly Specific Borneol‐Type Dehydrogenase: A borneol‐ type dehydrogenase with high specificity for borneol and isoborneol was characterized from the genome of Salvia rosmarinus. The crystallographic structure was determined, showing an hydrophobic pocket that plays a crucial role in the stereo discrimination of bornane‐type monoterpenoids.

Published
2021

34. Immune deficiency, autoimmune disease and intellectual disability: A pleiotropic disorder caused by biallelic variants in the <scp> TPP2 </scp> gene

Author

Fabio Candotti, Claudia Poloni, Christophe Bonvin, Belinda Campos-Xavier, Jean-Louis Blouin, Athina Fouriki, Mathieu Quinodoz, Carlo Rivolta, Stéphanie Christen-Zaech, Orbicia Riccio, Armand Bottani, Michael Hofer, Andrea Superti-Furga, Camille Kumps, Maja Beck-Popovic, Virginie G. Peter, Mattia Rizzi, Raffaele Renella, Felicitas Bellutti Enders, Frossard Valérie, Federica Angelini, Isis Atallah, and Sheila Unger

Subjects
Adult, Male, 0301 basic medicine, Genetic counseling, Human leukocyte antigen, 030105 genetics & heredity, Aminopeptidases, Autoimmune Diseases, Frameshift mutation, Young Adult, 03 medical and health sciences, Immune system, Amino acid homeostasis, Intellectual disability, Genetics, medicine, Humans, Child, Dipeptidyl-Peptidases and Tripeptidyl-Peptidases, Frameshift Mutation, Genetics (clinical), Immunodeficiency, Autoimmune disease, business.industry, Serine Endopeptidases, Immunologic Deficiency Syndromes, Exons, medicine.disease, 030104 developmental biology, Child, Preschool, Immunology, Female, business
Abstract

Four individuals from two families presented with a multisystemic condition of suspected genetic origin that was diagnosed only after genome analysis. The main phenotypic features were immune system dysregulation (severe immunodeficiency with autoimmunity) and intellectual disability. The four individuals were found to be homozygous for a 4.4 Kb deletion removing exons 20 to 23 (NM_003291.4) of the TPP2 gene, predicting a frameshift with premature termination of the protein. The deletion was located on a shared chromosome 13 haplotype indicating a Swiss founder mutation. Tripeptidyl peptidase 2 (TPP2) is a protease involved in HLA/antigen complex processing and amino acid homeostasis. Biallelic variants in TPP2 have been described in ten individuals with variable features including immune deficiency, autoimmune cytopenias, and intellectual disability or chronic sterile brain inflammation mimicking multiple sclerosis. Our observations further delineate this severe condition not yet included in the OMIM catalog. Timely recognition of TPP2 deficiency is crucial since (1) immune surveillance is needed and hematopoietic stem cell transplantation may be necessary, and (2) for provision of genetic counseling. Additionally, enzyme replacement therapy, as already established for TPP1 deficiency, might be an option in the future. This article is protected by copyright. All rights reserved.

Published
2021

37. Suboptimal vaccination coverage of recommended vaccines among French children with recurrent autoinflammatory fever syndromes: a study from the Juvenile Inflammatory Rheumatism cohort

Author

Virginie Rollet-Cohen, Marie-Aliette Dommergues, Glory Dingulu, Justine Mirete, Michael Hofer, François Hofer, Véronique Hentgen, and Andreas Woerner

Subjects
Pediatrics, medicine.medical_specialty, Vaccination Coverage, Adolescent, Fever, Familial Mediterranean fever, Rubella, Measles, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, Lymphadenitis, Humans, Medicine, 030212 general & internal medicine, Child, 030203 arthritis & rheumatology, Vaccines, business.industry, Diphtheria, Infant, Pharyngitis, Syndrome, General Medicine, medicine.disease, Poliomyelitis, Vaccination, Child, Preschool, Cohort, Stomatitis, Aphthous, Rheumatic Fever, medicine.symptom, business
Abstract

To determine vaccination coverage among a French cohort of children with recurrent autoinflammatory fever syndromes (RFS).All RFS children aged 2 to 19 years from the Juvenile Inflammatory Rheumatism cohort and followed at the French Reference Center for Autoinflammatory Diseases, Versailles Hospital, were included in our observational study. Immunisation status at ages 2, 7 and 15 years and at the last outpatient visit was evaluated according to the standard French vaccine schedule and recommended supplementary vaccines for patients with immunosuppressive therapy.Of 200 patients, 90 (45%) had periodic fever, aphthous stomatitis, pharyngitis and adenitis syndrome; 52 (26%) had familial Mediterranean fever and 50 (25%) had undefined recurrent fever. Complete immunisation as per the standard schedule was obtained by 32% of patients at 2 years, 28% at 7 years, 6% at 15 years and 44% at the last outpatient visit. Similar or higher coverage was obtained by the last outpatient visit for most vaccines, compared to immunisation coverage at 2 years: pneumococcus (91% vs 88%), diphtheria tetanus poliomyelitis (82% vs 86%), hepatitis B (79% vs 69%) and measles, mumps, rubella (91% vs 50%). No patients with immunosuppressive therapy (n = 14) were up to date for all supplementary immunisations recommended for them.Vaccination coverage for RFS children is suboptimal, especially for infants who present with recurrent febrile episodes. The initial vaccination delay is partially corrected through specialist follow-up in later years. Coverage according to the supplementary vaccine recommendations for immunosuppressed patients is poor. Key Points • Vaccination coverage for RFS children is suboptimal, especially at 2 years of age which is likely due to the prevalence of early recurrent febrile symptoms. • The initial vaccination delay is partially recovered during later follow-up at an expert rheumatology center. • Specific recommendations are particularly difficult to apply to patients on immunosuppressive therapy.

Published
2021

39. Why and How Should We Treat Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Syndrome?

Author

Michael Hofer

Subjects
Male, Pediatrics, medicine.medical_specialty, PFAPA syndrome, Fever, Spontaneous remission, Disease, Asymptomatic, 03 medical and health sciences, 0302 clinical medicine, Pharmacotherapy, Quality of life, Lymphadenitis, medicine, Humans, Pharmacology (medical), 030203 arthritis & rheumatology, Anakinra, business.industry, Pharyngitis, Syndrome, Child, Preschool, Pediatrics, Perinatology and Child Health, Quality of Life, Female, Stomatitis, Aphthous, medicine.symptom, business, 030217 neurology & neurosurgery, medicine.drug
Abstract

Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome is characterized by recurrent fever flares accompanied by symptoms and signs forming the disease acronym and alternating with asymptomatic periods. Despite the disease having a generally favorable outcome, with spontaneous remission after a few years, it does have a major impact on the quality of life of the child and his or her relatives. Beside symptomatic medications during fever flares, the most used treatment consists of a single dose of corticosteroids at flare onset to interrupt the attack; fever resolves usually within a few hours, but often with a shorter interval between the attacks. For these patients, colchicine has been shown to decrease the frequency of the flares. Other medications were also reported in case series of patients with PFAPA syndrome. These include the interleukin-1 blocker anakinra to treat flares, cimetidine (which showed no convincing effect), and other drugs with anecdotal use. The pediatrician faces a difficult question: should they wait for spontaneous disease resolution and only treat the flares medically, or should they propose tonsillectomy that may induce remission? Due to the lack of strong evidence, the answer will vary on an individual basis, depending on the impact on the patient's quality of life. The choice of the best therapeutic strategy will be based on the response to the flare-based therapy and on disease severity (quality of life, school performances); long-term data on the remission rate under the different therapeutic strategies are currently missing. More randomized controlled studies are needed to help the treating physician to choose the best therapy, as well as real-life long-term data to evaluate the long-term outcome of children with PFAPA syndrome.

Published
2020

40. Interleukin-1 Blockade in Systemic Juvenile Idiopathic Arthritis

Author

Katerina Theodoropoulou, Michael Hofer, Manel Mejbri, and Rolando Cimaz

Subjects
medicine.medical_specialty, Arthritis, Bioinformatics, medicine.disease_cause, Autoimmunity, 03 medical and health sciences, 0302 clinical medicine, Pharmacotherapy, Synovitis, Internal medicine, Humans, Medicine, Pharmacology (medical), Child, 030203 arthritis & rheumatology, business.industry, medicine.disease, Arthritis, Juvenile, Rheumatology, Blockade, Clinical research, Macrophage activation syndrome, Pediatrics, Perinatology and Child Health, business, 030217 neurology & neurosurgery, Interleukin-1
Abstract

Systemic juvenile idiopathic arthritis (sJIA) is a severe disorder now linked more to autoinflammation than to autoimmunity. Clinical and laboratory evidence support the pathogenetic role of interleukin-1 (IL-1), and blockade of this cytokine has proved to be very effective in the treatment of sJIA. There are now several agents that block IL-1 available on the market. This article reviews the efficacy and safety of these drugs for the treatment of sJIA on the basis of published data, and offers the current view on treating sJIA according to its different phenotypes. There are no head to head trials among the different IL-1 inhibitors, and although efficacy has been demonstrated for all of them, it is still unknown which one would be more appropriate for which particular situation. The presence of synovitis in addition to active systemic features might be relevant for these choices. In addition, complications such as macrophage activation syndrome can be important since, on one hand, it has been associated with biologic therapy administration and, on another, there have been some reports of this complication being treated with anti-IL-1. Current recommendations by the American College of Rheumatology are now outdated, and new ones are being prepared. In the meantime, basic and clinical research is advancing in order to identify new treatment targets and to evaluate the different protocols currently in use.

Published
2020

41. Sécurité des agents biologiques dans les maladies rhumatismales pédiatriques : étude rétrospective multicentrique en situation réelle dans la base de données JIRcohorte

Author

Véronique Hentgen, Elvira Cannizzaro, Daniela Kaiser, N. Cabrera, Aurélie Chausset, Jean-Christophe Lega, Anne Maes, Salma Malik, Agnès Duquesne, Isabelle Koné-Paut, Michael Hofer, Gerald Berthet, Anuela Kondi, Samuel Roethlisberger, Andreas Woerner, Cyril Jeanneret, Florence Aeschlimann, Behrouz Kassai, Laetitia Higel, Alexandre Belot, and Carine Wouters

Subjects
030203 arthritis & rheumatology, 03 medical and health sciences, 0302 clinical medicine, Rheumatology, 030212 general & internal medicine
Abstract

Resume Objectif Analyser et decrire l’incidence des effets indesirables des agents biologiques chez des enfants atteints de maladies inflammatoires, dans une cohorte suivie en situation reelle. Methodes Cette etude observationnelle retrospective multicentrique internationale a evalue, par la methode de Kaplan–Meier, la survenue d’effets indesirables chez des enfants recevant un traitement biologique et suivis dans le reseau JIRcohorte de rhumatologie pediatrique (JIR, Juvenile Inflammatory Rheumatism). Un modele de Cox a ete elabore pour identifier des facteurs predictifs independants d’effets indesirables. Resultats Au total, 813 patients totalisant 3439 patients-annee (p-a) d’agents biologiques ont ete inclus. Le principal diagnostic etait l’arthrite juvenile idiopathique (AJI) (84 %). Au total, 222 patients (27,3 %) ont presente 419 effets indesirables, soit un taux d’incidence de 12,2 pour 100 p-a, IC 95 % [11,0 ; 13,4]. L’incidence globale des effets indesirables graves etait de 3,9 pour 100 p-a, IC 95 % [3,2 ; 4,6]. Le tocilizumab et l’infliximab ont ete associes de maniere significative a des effets indesirables et le canakinumab a des effets indesirables graves. L’analyse univariee et multivariee des effets indesirables et des effets indesirables graves a montre qu’ils etaient plus frequents chez les patients prenant des agents biologiques en association avec des immunosuppresseurs (autres que le methotrexate). Conclusion Cette etude suggere que la securite des agents biologiques est globalement acceptable chez les enfants atteints de maladies rhumatismales inflammatoires traites par des agents biologiques. Cependant, la prescription d’immunosuppresseurs associee cree un risque notable d’effets indesirables.

Published
2020

42. Design and Application Considerations of Packaging of Dc-Dc Converter Micromodules

Author

Michael Brooks, Michael Hofer, Martin Haug, Dragan Dinulovic, and Mahmoud Shousha

Subjects
Data acquisition, Computer science, Derating, business.industry, EMI, Power electronics, Thermal, Electrical engineering, Electronics, business, Vertical integration, Dc dc converter
Abstract

This paper presents different design considerations of two different packaging approaches for micromodules, namely vertically integrated ferrite substrate based [1] and conventional overmolded side-by-side integration [2]. Production steps, EMI, and thermal considerations are covered in this paper. Two dc-dc converter micromodules with the same specifications are built using both packaging approaches and tested. The vertically integrated micromodule has dimensions of 3.2 X 2.5 X 1.6mm3 while the side-by-side integrated micromodule achieves 2.5 X 2.5 X 1.2mm3 dimensions. Both modules achieve peak efficiency of 87% for 5Vin-to-1.8Vout conversion. Experimental evaluation and market requirements suggest the suitability of vertically integrated micromodule for applications, which require very low noise level (radiated emissions) and excellent thermal derating performance such as data acquisition and measurements. On the other hand, side-by-side integrated micromodules are suitable for applications that require low profile height and have aggressive cost requirements such as consumer electronics, i.e. tablets, smart phones, and laptops.

Published
2021

43. Infektionen unter der Behandlung mit Biologika: Erfahrung der Juvenile Inflammatory Rheumatism cohort (JIR-Kohorte)

Author

François Hofer, Alexandre Belot, Michael Hofer, and Véronique Hentgen

Subjects
General Medicine
Abstract

ZUSAMMENFASSUNGDie Juvenile Inflammatory Rheumatism (JIR) Kohorte ist eine internationale, multizentrische Beobachtungsstudie, deren Daten webbasiert erfasst werden. Sie ist modular aufgebaut und kann als elektronische Patientenakte sowie für wissenschaftliche Untersuchungen genutzt werden. Primär wurde die JIR-Kohorte auf den Weg gebracht, um mehr Informationen zur Sicherheit und Wirksamkeit von krankheitsmodifizierenden antirheumatischen Medikamenten (DMARDs) bei Kindern und Jugendlichen mit entzündlichen rheumatischen Erkrankungen zu gewinnen. Die erste Analyse zur Sicherheit von biologischen DMARDs bei 813 Patienten mit entzündlich rheumatischen Erkrankungen wies keine neuen Sicherheitssignale nach. Die weitere Überwachung von Patienten mit langfristiger Arzneimittelexposition bereits ab dem Kindes- und Jugendalter ist notwendig, um zuverlässige Daten über die Langzeitsicherheit und -wirksamkeit von DMARDs bei juvenilen rheumatischen Erkrankungen zu erhalten. Darüber hinaus wird die JIR-Kohorte für weitere Forschungsfragen eingesetzt, z. B. zur Untersuchung der Verträglichkeit von Impfungen bei immunsupprimierten Kindern oder zur Charakterisierung spezieller Krankheitsmanifestationen, wie einer Uveitis.

Published
2019

44. Safety of biological agents in paediatric rheumatic diseases: A real-life multicenter retrospective study using the JIRcohorte database

Author

Véronique Hentgen, Daniela Kaiser, Elvira Cannizzaro, Michael Hofer, Anne Maes, Salma Malik, Gerald Berthet, Carine Wouters, Behrouz Kassai, Anuela Kondi, Andreas Woerner, Agnès Duquesne, Aurélie Chausset, Isabelle Koné-Paut, Natalia Cabrera, Samuel Roethlisberger, Cyril Jeanneret, Florence Aeschlimann, Jean-Christophe Lega, Alexandre Belot, Laetitia Higel, University of Zurich, and Cabrera, Natalia

Subjects
Male, Internationality, Databases, Factual, 2745 Rheumatology, Kaplan-Meier Estimate, Etanercept, Arthritis, Rheumatoid, Cohort Studies, paediatric rheumatology, Biological Factors, chemistry.chemical_compound, Range of Motion, Articular, Child, Pain Measurement, Incidence (epidemiology), Prognosis, Treatment Outcome, Antirheumatic Agents, Child, Preschool, Cohort, Female, JIRcohorte, medicine.drug, Cohort study, medicine.medical_specialty, Adolescent, 610 Medicine & health, Antibodies, Monoclonal, Humanized, Risk Assessment, Abatacept, Biological agents, Tocilizumab, Rheumatology, Internal medicine, medicine, Humans, Adverse effect, Proportional Hazards Models, Retrospective Studies, business.industry, Retrospective cohort study, Arthritis, Juvenile, Infliximab, adverse events, Canakinumab, chemistry, 10036 Medical Clinic, serious adverse events, Multivariate Analysis, juvenile idiopathic arthritis, business
Abstract

OBJECTIVE: To analyse and report the incidence of side effects of biological agents in paediatric patients with inflammatory diseases using of real-life follow-up cohort. METHODS: In this international, observational, retrospective, multicentre study of children treated by biological agents and followed in the Juvenile Inflammatory Rheumatism (JIR) cohort (JIRcohorte) network, a Kaplan-Meier method was used to estimate the occurrence of adverse events. A Cox model was constructed to identify independent predictors of adverse events. RESULTS: Overall 813 patients totalling 3439 patients-year (PY) of biological agents were included. The main diagnosis was juvenile idiopathic arthritis (84%). A total of 222 patients (27.3%) had 419 adverse events, representing an incidence rate of 12.2 per 100 PY 95% CI [11.0; 13.4]. The overall incidence rate of serious adverse events was 3.9 per 100 PY 95% CI [3.2; 4.6]. Tocilizumab and infliximab were significantly associated with adverse events and canakinumab with serious adverse events. Univariate and multivariable analysis of adverse events and serious adverse events indicated that patients under biological agents with concomitant immunosuppressive drugs (excluding methotrexate) suffered from more of these events. CONCLUSION: This study suggests an overall an acceptable safety of biologic agents in children with inflammatory rheumatic diseases treated with biological agents. However, the concomitant prescription of immunosuppressive drugs with biological agents represents a substantial risk of adverse events. ispartof: JOINT BONE SPINE vol:86 issue:3 pages:343-350 ispartof: location:France status: published

Published
2019

45. L’échographie ostéo-articulaire est-elle associée au changement thérapeutique chez les patients atteints d’arthrite juvénile idiopathique : résultats de la cohorte JIRECHO

Author

I Kone-Paut, Aurélia Carbasse, Michael Hofer, Sylvain Breton, Linda Rossi-Semerano, Brigitte Bader-Meunier, S. Jousse Joulin, V. Devauchelle Pensec, A. Von Scheven-Gète, S. Mahmoud, Gaël Mouterde, A. Saraux, J.D. Cohen, and L. Sparsa

Subjects
Rheumatology
Abstract

Introduction L’arthrite juvenile idiopathique (AJI) est un groupe heterogene de maladies inflammatoires. Les traitements constituent un enjeu majeur afin de prevenir le handicap. La presence de synovites, parfois difficiles a detecter chez l’enfant, est associee au risque de destruction articulaire et l’echographie articulaire permet de detecter des synovites infra-cliniques. Notre objectif principal etait de determiner si l’echographie articulaire etait associee a une modification therapeutique chez les patients atteints d’AJI. Les objectifs secondaires etaient d’identifier les autres facteurs associes au changement therapeutique. Patients et methodes Nous avons mene une etude observationnelle basee sur la cohorte multicentrique JIRECHO, issue de la JIR Cohorte, incluant des patients atteints d’AJI. Les patients avaient un suivi clinique et echographique semestriel standardise. L’echographie articulaire etait realisee par des echographistes prealablement entraines et reproductibles selon les recommandations OMERACT [1] . La synovite echographique etait definie par la presence d’un epanchement et/ou d’une hypertrophie synoviale en mode B (≥ grade 1) associee ou non a une vascularisation doppler (≥ grade 1). Les donnees cliniques, d’activite globale (EVA, JADAS), biologiques (CRP, VS) et therapeutiques etaient recueillies. Resultats Nous avons inclus 112 patients. Le nombre total de visites medicales etait de 185. Trois groupes ont ete definis selon le traitement : augmente (22 %), diminue (14 %) et stable (64 %). Dans un premier temps, nous avons compare les patients chez qui le traitement a ete augmente avec les autres patients. La presence d’au moins une synovite echographique en mode B n’etait pas statistiquement plus eleve dans le groupe « augmentation de traitement » quoique numeriquement plus elevee (80 % vs 65 %, p = 0,06). Il n’y avait pas de difference significative pour la presence d’au moins une synovite en mode doppler (30 % vs 23 %, p = 0,4). Les EVA patients et medecins etaient en revanche significativement plus elevees lorsque le traitement etait augmente [3,3 vs 1,7, p Fig. 1 ). Conclusion Notre etude a montre que la presence d’au moins une synovite echographique en mode B et/ou D n’etait pas statistiquement associee au changement de traitement dans l’AJI, meme si la balance Se/Sp est proche de celle de l’avis du medecin et du score d’activite clinique.

Published
2021

46. Élaboration d’une check-list pour la transition vers les soins adultes en rhumatologie : une étude de type Delphi

Author

Thanh-Thao Dang, Anne Meynard, Johannes Mattar, Béatrice Fonjallaz, Christina Akre, Michael Hofer, Jean-Paul Larbre, Susanne Schalm, Agnès Duquesne, Marie Couret, Alexandre Belot, Sophie Georgin-Lavialle, and Joan-Carles Suris

Subjects
03 medical and health sciences, 0302 clinical medicine, Rheumatology, 030225 pediatrics, 030212 general & internal medicine
Abstract

Resume Objectifs Elaborer une check-list pour la transition vers les soins adultes destinee aux professionnels de la sante et concue pour faciliter le processus de transition des enfants atteints d’affections rhumatismales chroniques et de leur famille. Methodes Une enquete de type Delphi en quatre etapes a ete realisee aupres d’un groupe international d’experts : (1) Un groupe de travail compose de 6 specialistes a ete en charge de rediger une premiere ebauche du projet ; (2) Un sondage en ligne aupres d’un groupe international d’experts a permis d’en evaluer le contenu ; (3) Une conference de consensus de 2 jours a reuni plusieurs experts charges de discuter des items discordants ; (4) Un sondage en ligne a ete mene aupres d’un panel d’experts internationaux et portait sur les items prealablement reformules. Resultats La premiere version de cette check-list comprenait 38 items repartis sur 3 phases de transition et 5 tranches d’âge. Trente-trois experts internationaux ont ete en charge d’evaluer la check-list et sont parvenus a un consensus ≥ 80 % pour 26 items et ≤ 80 % pour 12. A l’occasion d’une conference de consensus reunissant 12 experts, ces 12 items ont ete discutes et redefinis. Vingt-cinq experts internationaux ont participe au sondage en ligne et sont parvenus a un consensus d’au moins 80 % pour tous les items a l’exception d’un. La check-list a ete finalisee. Conclusions Cette etude de type Delphi a permis de definir les themes a inclure et l’âge approprie auquel il convenait de les aborder avec les patients atteints d’une maladie rhumatismale chronique et leur famille au cours du processus de transition. Cette check-list pour la transition a obtenu un solide consensus international et interdisciplinaire tout en abordant la transition dans son aspect le plus large. Elle devrait etre communiquee a l’ensemble des professionnels de la sante en charge d’adolescents âges de 12 ans et plus au moment de la transition. Elle peut etre transposee a la plupart des maladies chroniques. Des recommandations pour la poursuite des recherches sont donnees.

Published
2018

47. Simple Plug-In Synthetic Step for the Synthesis of (-)-Camphor from Renewable Starting Materials

Author

Robert Kourist, Kamela Myrtollari, Michael Hofer, Ivana Drienovská, Michaela Pressnig, Elia Calderini, Volker Sieber, and Helmut Schwab

Subjects
010402 general chemistry, 01 natural sciences, Biochemistry, Kinetic resolution, Borneol, chemistry.chemical_compound, Camphor, Hydrolysis, esterases, Organic chemistry, Moiety, Rhodococcus, kinetic resolution, Molecular Biology, Bicyclic Monoterpenes, biology, Molecular Structure, Full Paper, 010405 organic chemistry, Organic Chemistry, Serine Endopeptidases, Enantioselective synthesis, asymmetric catalysis, borneol, Stereoisomerism, Rhodococcus rhodochrous, Fenchol, Full Papers, biology.organism_classification, 0104 chemical sciences, ddc, Cephalosporins, chemistry, Molecular Medicine, Burkholderia gladioli
Abstract

Racemic camphor and isoborneol are readily available as industrial side products, whereas (1R)‐camphor is available from natural sources. Optically pure (1S)‐camphor, however, is much more difficult to obtain. The synthesis of racemic camphor from α‐pinene proceeds via an intermediary racemic isobornyl ester, which is then hydrolyzed and oxidized to give camphor. We reasoned that enantioselective hydrolysis of isobornyl esters would give facile access to optically pure isoborneol and camphor isomers, respectively. While screening of a set of commercial lipases and esterases in the kinetic resolution of racemic monoterpenols did not lead to the identification of any enantioselective enzymes, the cephalosporin Esterase B from Burkholderia gladioli (EstB) and Esterase C (EstC) from Rhodococcus rhodochrous showed outstanding enantioselectivity (E>100) towards the butyryl esters of isoborneol, borneol and fenchol. The enantioselectivity was higher with increasing chain length of the acyl moiety of the substrate. The kinetic resolution of isobornyl butyrate can be easily integrated into the production of camphor from α‐pinene and thus allows the facile synthesis of optically pure monoterpenols from a renewable side‐product., Enzymes are a valuable tool for upcycling side‐stream from renewable sources. We have found that EstB from Burkholderia gladioli could achieve high enantiopurity in upcycling α‐pinene, a side‐product from turpentine production, whereas common lipases and esterases could not. This reaction can be easily integrated with the existing chemical synthetic route for the production of the racemic camphor with minimal adjustments as the route goes through an intermediate isobornyl acetate.

Published
2021

48. Epidemiology of Childhood Uveitis in a Tertiary Care Center: A 20-Year Study

Author

Sakina Ezziat, Daniele C. Rossi, Yan Guex-Crosier, Aikaterini Koryllou, Florence Hoogewoud, Catherine Guex-Crosier, Michael Hofer, and Judith Cohen

Subjects
Adult, Systemic disease, medicine.medical_specialty, Pediatrics, Adolescent, Disease, Tertiary Care Centers, Uveitis, Epidemiology, medicine, Humans, Child, Aged, Retrospective Studies, business.industry, Panuveitis, Infant, Newborn, Infant, Retrospective cohort study, Uveitis, Posterior, medicine.disease, Uveitis, Anterior, Ophthalmology, Child, Preschool, Etiology, Intermediate uveitis, business
Abstract

To investigate demographics and causes of pediatric uveitis in a Swiss tertiary reference center over a 20-year period.Retrospective cohort study on patients with uveitis aged less than 16 years seen at Jules-Gonin Eye Hospital between 1 January 2000 and 31 December 2019.Out of 2846 patients with uveitis seen in the Jules-Gonin Eye Hospital Ocular Immune-Infectiology Department, 317 (11.1%) were under 16 years of age and were included in this study. Median age at onset of the uveitis was 8.9 years (range 0 - 16). Anterior uveitis was the most frequent presentation (45.1%) followed by posterior uveitis in 26.2%, intermediate uveitis in 23.3%, and panuveitis in 5.4%. The inflammation was most frequently bilateral and non-granulomatous. A systemic inflammatory disease was found in 34% of the cases and an infectious cause in 24%.The repartition of the location of the uveitis was similar to previous reports from Western countries. Uveitis in juvenile idiopathic arthritis is the most frequent etiology related to a systemic disease in children. An infectious cause was found in 24% of our patients, which is a greater proportion than in adult cohorts.Die Studie untersucht die Demografie und Ätiologie der Kindheitsuveitis über eine 20-Jahres-Periode in einem Universitätsspital.Diese Studie hat Kinder unter 16 Jahren eingeschlossen, die zwischen dem 1. Januar 2000 und dem 31. Dezember 2019 im Jules Gonin Augenspital auf eine Uveitis untersucht worden sind. Die Daten wurden retrospektiv analysiert.2846 Patienten mit Uveitis wurden untersucht und 317 (11,1%) davon waren Kinder. Das mediane Alter des Krankheitsbeginns war 8,9 Jahre (Range 0 – 16). Die anatomische Lokalisation war am häufigsten anterior (45,1%). Dann folgten die posteriore (26,2%) und die intermediäre (23,3%) Uveitis. Die Panuveitis war mit 5,4% am seltensten vorhanden. Die Entzündung war am häufigsten bilateral und nicht granulomatös. Eine systemische Immunerkrankung wurde in 34% der Fälle entdeckt und 24% der Kinder hatten eine infektiöse Ursache der Uveitis.Die Aufteilung der anatomischen Lokalisation war vergleichbar zu epidemiologischen Studien der 20 früheren Jahre. Die juvenile idiopathische Arthritis ist immer noch die häufigste Ursache für eine Uveitis in der Kindheit. Eine infektiöse Ursache wurde in fast einem Viertel der Fälle gefunden, was verhältnismäßig häufiger ist als bei Erwachsenen.

Published
2021

49. Nonlinear Optics Measurements in IOTA

Author

Alexander Valishev, Aleksandr Romanov, Nikita Kuklev, Rogelio Tomás García, Giulio Stancari, Michael Hofer, and Sebastian Szustkowski

Subjects
Iota, Physics, Optics, business.industry, Physics::Accelerator Physics, Nonlinear optics, business, Accelerators and Storage Rings
Abstract

Nonlinear integrable optics is a recently proposed accelerator lattice design approach which allows to generate an amplitude dependent tune shift which is needed in high brightness accelerators to mitigate fast coherent instabilities. Whereas usually octupoles are used to achieve this task, this concept allows doing so without exciting any resonances, in turn preventing any particle loss. The concept is based around a special magnet design, together with specific constraints on the optics of the accelerator. To study such a system, the Integrable Optics Test Accelerator (IOTA) was recently constructed and commissioned at Fermilab. For the assessment of the performance of this concept, good knowledge of the optics and the (non-)linear dynamics without the special magnet is of key importance. As such, measurements were conducted in the IOTA ring, using the captured turn-by-turn data by the beam position monitors after excitation to infer quantities such as amplitude detuning and resonance driving terms. In this note, first results of these measurements are presented.

Published
2021

50. [Pediatric COVID-19 - Pathophysiology, immune responses, genetic predispositions, hyperinflammatory syndrome]

Author

Géraldine, Blanchard-Rohner, Jana, Pachlopnik Schmid, Fabio, Candotti, and Michael, Hofer

Subjects
SARS-CoV-2, Immunity, COVID-19, Humans, Genetic Predisposition to Disease, Child, Systemic Inflammatory Response Syndrome
Abstract

Children appeared to be initially spared by the SARS-CoV-2 pandemic, however, in spring 2020, a new clinical entity was described related to the SARS-CoV-2 infection and named multisystem inflammatory syndrome in children (MIS-C) or pediatric inflammatory multisystem syndrome (PIMS). The gravity of this inflammatory syndrome, the time interval between infection and MIS-C, the response to the various immunomodulatory treatments are all suggestive of an immunologic reaction rather than a virus-mediatred phenomenon. The pathophysiological mechanisms and possible risk factors for MIS-C have not been elucidated. In this article, we summarize what is known to date about the immune response to SARS-CoV-2 in children and about the immune response to SARS-CoV-2 in children and about the MIS-C.Alors que les enfants semblaient épargnés par la pandémie due au SARS-CoV-2, une nouvelle entité clinique a été décrite au printemps 2020 en lien avec cette infection et appelée syndrome inflammatoire multisystémique de l’enfant (MIS-C ou PIMS en anglais). La gravité du syndrome inflammatoire, le délai entre l’infection et le MIS-C, et la réponse aux différents traitements immunomodulateurs sont tous suggestifs d’une réaction immunologique plutôt que d’un phénomène uniquement médié par le virus. Les mécanismes physiopathologiques et les facteurs de risque possibles du MIS-C n’ont pas encore été élucidés. Dans cet article, nous résumons ce qui est connu à ce jour sur la réponse immune au SARS-CoV-2 chez l’enfant et sur le MIS-C.

Published
2021

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