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1. POS0826 PREDICTORS OF RESPONSE AND LONG-TERM EFFICACY AND SAFETY OF NINTEDANIB IN SYSTEMIC SCLEROSIS-INTERSTITIAL LUNG DISEASE: DATA FROM AN ITALIAN MULTICENTRE STUDY

Author

Campochiaro, C., primary, De Luca, G., additional, Lazzaroni, M. G., additional, Armentaro, G., additional, Ruaro, B., additional, Stanziola, A., additional, Benfaremo, D., additional, De Lorenzis, E., additional, Moccaldi, B., additional, Bonomi, F., additional, Bianchessi, L. M., additional, Iannone, F., additional, Cacciapaglia, F., additional, Spinella, A., additional, Guiducci, S., additional, Codullo, V., additional, Bosello, S. L., additional, Cuomo, G., additional, Zanatta, E., additional, Confalonieri, P., additional, Del Papa, N., additional, Airo’, P., additional, Moroncini, G., additional, Giuggioli, D., additional, Dagna, L., additional, and Matucci-Cerinic, M., additional

Published
2024
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3. The Updated Role of Ultrasound in Assessing Dermatological Manifestations in Systemic Sclerosis

Author

Ruaro B, Santiago T, Hughes M, Lepri G, Poillucci G, Baratella E, Salton F, and Confalonieri M

Subjects
systemic sclerosis, ultrasound, elastosonography, skin, skin thickness, digital ulcers, Diseases of the musculoskeletal system, RC925-935
Abstract

Barbara Ruaro,1 Tania Santiago,2,3 Michael Hughes,4 Gemma Lepri,5 Gabriele Poillucci,6 Elisa Baratella,6 Francesco Salton,1 Marco Confalonieri1 1Unit of Pulmonology, University Hospital of Trieste, Trieste, Italy; 2Department of Rheumatology, Centro Hospitalare Universitário de Coimbra, Coimbra, Portugal; 3Medicine Faculty, University of Coimbra, Coimbra, Portugal; 4Department of Rheumatology, Royal Hallamshire Hospital, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK; 5Department of Experimental and Clinical Medicine, Division of Rheumatology, University of Firenze, Florence, Italy; 6Department of Radiology, Department of Medicine, Surgery and Health Science, University of Trieste, Trieste, ItalyCorrespondence: Barbara RuaroUnit of Pulmonology, University Hospital of Trieste, Trieste, ItalyTel +39 3470502394Email barbara.ruaro@yahoo.itAbstract: Systemic sclerosis (SSc), an autoimmune connective tissue disease, characterized by skin fibrosis, increased dermal thickness and microvascular involvement. Fibroblasts and myofibroblasts deposit excessive amounts of collagenous and non-collagenous extracellular matrix components in the skin. This leads to microvascular abnormalities and Raynaud’s phenomenon, with painful digital ulcers (DU) at the fingertips adding to patient discomfort. The skin involvement and severity in SSc was evaluated by the Modified Rodnan skin score (mRSS). Although high-frequency ultrasound (HUS) has been widely researched in the study of skin thickness and DU in SSc, its adoption into clinical practice is not yet common. However, novel insights into the still relatively unknown disease pathogenesis in SSc and its evaluation may be provided by HUS, including early (pre-clinical) skin involvement. It may also be useful in both the evaluation and follow-up of DU. Indeed, it is a non-invasive, safe, inexpensive and reproducible method able to assess not only SSc patients’ cutaneous structural changes, but also their vascular system changes. Moreover, several recent studies have reported that elastosonography (ES) is of use when investigating skin involvement in systemic sclerosis. This review aims at providing information as to role HUS and ES play in research advancements and the clinical perspectives in the evaluation of skin thickness and DU in SSc patients.Keywords: systemic sclerosis, ultrasound, elastosonography, skin, skin thickness, digital ulcers

Published
2021

4. GRAd-COV2 vaccine provides potent and durable humoral and cellular immunity to SARS-CoV-2 in randomized placebo-controlled phase 2 trial

Author

Capone, S, Fusco, F, Milleri, S, Borre, S, Carbonara, S, Lo Caputo, S, Leone, S, Gori, G, Maggi, P, Cascio, A, Lichtner, M, Cauda, R, Dal Zoppo, S, Cossu, M, Gori, A, Roda, S, Confalonieri, P, Bonora, S, Missale, G, Codeluppi, M, Mezzaroma, I, Capici, S, Pontali, E, Libanore, M, Diani, A, Lanini, S, Battella, S, Contino, A, Piano Mortari, E, Genova, F, Parente, G, Dragonetti, R, Colloca, S, Visani, L, Iannacone, C, Carsetti, R, Folgori, A, Camerini, R, Ziviani, L, Malescio, F, Turrini, I, Lawlor, R, Romano, A, Nunziata, M, Armato, S, Mazzeo, N, Carleo, M, Dell'Isola, C, Pisapia, R, Pontarelli, A, Olivani, A, Grasselli, S, Laccabue, D, Leoni, M, Paolillo, F, Mancini, A, Ruaro, B, Confalonieri, M, Salton, F, Mancarella, G, Marocco, R, De Masi, M, Belvisi, V, Lamonica, S, Cingolani, A, Seguiti, C, Brambilla, P, Ferraresi, A, Lupi, M, Ludovisi, S, Renisi, G, Massafra, R, Pellicciotta, M, Armiento, L, Vimercati, S, Piacenza, M, Bonfanti, P, Columpsi, P, Cazzaniga, M, Rovelli, C, Ceresini, M, Previtali, L, Trentini, L, Alcantarini, C, Rugge, W, Biffi, S, Poletti, F, Rostagno, R, Moglia, R, De Negri, F, Fini, E, Cangialosi, A, Bruno, S, Rizzo, M, Niglio, M, Stritto, A, Matano, A, Petruzziello, A, Valsecchi, P, Pieri, T, Altamura, M, Calamo, A, Giannelli, A, Menolascina, S, Di Bari, S, Mauro, V, Aronica, R, Segala, D, Cultrera, R, Sighinolfi, L, Abbott, M, Gizzi, A, Marascia, F, Valenti, G, Feasi, M, Bobbio, N, Del Puente, F, Nicosia, A, Frasca, M, Mazzoleni, M, Garofalo, N, Ammendola, V, Grazioli, F, Napolitano, F, Vitelli, A, Marcellini, V, Capone S., Fusco F. M., Milleri S., Borre S., Carbonara S., Lo Caputo S., Leone S., Gori G., Maggi P., Cascio A., Lichtner M., Cauda R., Dal Zoppo S., Cossu M. V., Gori A., Roda S., Confalonieri P., Bonora S., Missale G., Codeluppi M., Mezzaroma I., Capici S., Pontali E., Libanore M., Diani A., Lanini S., Battella S., Contino A. M., Piano Mortari E., Genova F., Parente G., Dragonetti R., Colloca S., Visani L., Iannacone C., Carsetti R., Folgori A., Camerini R., Ziviani L., Malescio F., Turrini I., Lawlor R., Romano A., Nunziata M., Armato S., Mazzeo N., Carleo M. A., Dell'Isola C., Pisapia R., Pontarelli A., Olivani A., Grasselli S., Laccabue D., Leoni M. C., Paolillo F., Mancini A., Ruaro B., Confalonieri M., Salton F., Mancarella G., Marocco R., De Masi M., Belvisi V., Lamonica S., Cingolani A., Seguiti C., Brambilla P., Ferraresi A., Lupi M., Ludovisi S., Renisi G., Massafra R., Pellicciotta M., Armiento L., Vimercati S., Piacenza M., Bonfanti P., Columpsi P., Cazzaniga M. E., Rovelli C., Ceresini M., Previtali L., Trentini L., Alcantarini C., Rugge W., Biffi S., Poletti F., Rostagno R., Moglia R., De Negri F., Fini E., Cangialosi A., Bruno S. R., Rizzo M., Niglio M., Stritto A. D., Matano A., Petruzziello A., Valsecchi P., Pieri T., Altamura M., Calamo A., Giannelli A., Menolascina S., Di Bari S., Mauro V., Aronica R., Segala D., Cultrera R., Sighinolfi L., Abbott M., Gizzi A., Marascia F. G., Valenti G., Feasi M., Bobbio N., Del Puente F., Nicosia A., Frasca M., Mazzoleni M., Garofalo N., Ammendola V., Grazioli F., Napolitano F., Vitelli A., Marcellini V., Capone, S, Fusco, F, Milleri, S, Borre, S, Carbonara, S, Lo Caputo, S, Leone, S, Gori, G, Maggi, P, Cascio, A, Lichtner, M, Cauda, R, Dal Zoppo, S, Cossu, M, Gori, A, Roda, S, Confalonieri, P, Bonora, S, Missale, G, Codeluppi, M, Mezzaroma, I, Capici, S, Pontali, E, Libanore, M, Diani, A, Lanini, S, Battella, S, Contino, A, Piano Mortari, E, Genova, F, Parente, G, Dragonetti, R, Colloca, S, Visani, L, Iannacone, C, Carsetti, R, Folgori, A, Camerini, R, Ziviani, L, Malescio, F, Turrini, I, Lawlor, R, Romano, A, Nunziata, M, Armato, S, Mazzeo, N, Carleo, M, Dell'Isola, C, Pisapia, R, Pontarelli, A, Olivani, A, Grasselli, S, Laccabue, D, Leoni, M, Paolillo, F, Mancini, A, Ruaro, B, Confalonieri, M, Salton, F, Mancarella, G, Marocco, R, De Masi, M, Belvisi, V, Lamonica, S, Cingolani, A, Seguiti, C, Brambilla, P, Ferraresi, A, Lupi, M, Ludovisi, S, Renisi, G, Massafra, R, Pellicciotta, M, Armiento, L, Vimercati, S, Piacenza, M, Bonfanti, P, Columpsi, P, Cazzaniga, M, Rovelli, C, Ceresini, M, Previtali, L, Trentini, L, Alcantarini, C, Rugge, W, Biffi, S, Poletti, F, Rostagno, R, Moglia, R, De Negri, F, Fini, E, Cangialosi, A, Bruno, S, Rizzo, M, Niglio, M, Stritto, A, Matano, A, Petruzziello, A, Valsecchi, P, Pieri, T, Altamura, M, Calamo, A, Giannelli, A, Menolascina, S, Di Bari, S, Mauro, V, Aronica, R, Segala, D, Cultrera, R, Sighinolfi, L, Abbott, M, Gizzi, A, Marascia, F, Valenti, G, Feasi, M, Bobbio, N, Del Puente, F, Nicosia, A, Frasca, M, Mazzoleni, M, Garofalo, N, Ammendola, V, Grazioli, F, Napolitano, F, Vitelli, A, Marcellini, V, Capone S., Fusco F. M., Milleri S., Borre S., Carbonara S., Lo Caputo S., Leone S., Gori G., Maggi P., Cascio A., Lichtner M., Cauda R., Dal Zoppo S., Cossu M. V., Gori A., Roda S., Confalonieri P., Bonora S., Missale G., Codeluppi M., Mezzaroma I., Capici S., Pontali E., Libanore M., Diani A., Lanini S., Battella S., Contino A. M., Piano Mortari E., Genova F., Parente G., Dragonetti R., Colloca S., Visani L., Iannacone C., Carsetti R., Folgori A., Camerini R., Ziviani L., Malescio F., Turrini I., Lawlor R., Romano A., Nunziata M., Armato S., Mazzeo N., Carleo M. A., Dell'Isola C., Pisapia R., Pontarelli A., Olivani A., Grasselli S., Laccabue D., Leoni M. C., Paolillo F., Mancini A., Ruaro B., Confalonieri M., Salton F., Mancarella G., Marocco R., De Masi M., Belvisi V., Lamonica S., Cingolani A., Seguiti C., Brambilla P., Ferraresi A., Lupi M., Ludovisi S., Renisi G., Massafra R., Pellicciotta M., Armiento L., Vimercati S., Piacenza M., Bonfanti P., Columpsi P., Cazzaniga M. E., Rovelli C., Ceresini M., Previtali L., Trentini L., Alcantarini C., Rugge W., Biffi S., Poletti F., Rostagno R., Moglia R., De Negri F., Fini E., Cangialosi A., Bruno S. R., Rizzo M., Niglio M., Stritto A. D., Matano A., Petruzziello A., Valsecchi P., Pieri T., Altamura M., Calamo A., Giannelli A., Menolascina S., Di Bari S., Mauro V., Aronica R., Segala D., Cultrera R., Sighinolfi L., Abbott M., Gizzi A., Marascia F. G., Valenti G., Feasi M., Bobbio N., Del Puente F., Nicosia A., Frasca M., Mazzoleni M., Garofalo N., Ammendola V., Grazioli F., Napolitano F., Vitelli A., and Marcellini V.

Abstract

The ongoing severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic and heterologous immunization approaches implemented worldwide for booster doses call for diversified vaccine portfolios. GRAd-COV2 is a gorilla adenovirus-based COVID-19 vaccine candidate encoding prefusion-stabilized spike. The safety and immunogenicity of GRAd-COV2 is evaluated in a dose- and regimen-finding phase 2 trial (COVITAR study, ClinicalTrials.gov: NCT04791423) whereby 917 eligible participants are randomized to receive a single intramuscular GRAd-COV2 administration followed by placebo, or two vaccine injections, or two doses of placebo, spaced over 3 weeks. Here, we report that GRAd-COV2 is well tolerated and induces robust immune responses after a single immunization; a second administration increases binding and neutralizing antibody titers. Potent, variant of concern (VOC) cross-reactive spike-specific T cell response peaks after the first dose and is characterized by high frequencies of CD8s. T cells maintain immediate effector functions and high proliferative potential over time. Thus, GRAd vector is a valuable platform for genetic vaccine development, especially when robust CD8 response is needed.

Published
2023

5. Prolonged higher dose methylprednisolone vs. conventional dexamethasone in COVID-19 pneumonia: a randomised controlled trial (MEDEAS)

Author

Salton, F, Confalonieri, P, Centanni, S, Mondoni, M, Petrosillo, N, Bonfanti, P, Lapadula, G, Lacedonia, D, Voza, A, Carpene, N, Montico, M, Reccardini, N, Meduri, G, Ruaro, B, Confalonieri, M, Citton, G, Bozzi, C, Tavano, S, Pozzan, R, Andrisano, A, Jaber, M, Mari, M, Trotta, L, Mondini, L, Barbieri, M, Ruggero, L, Antonaglia, C, Soave, S, Torregiani, C, Bogatec, T, Baccelli, A, Nalesso, G, Re, B, Pavesi, S, Barbaro, M, Giuliani, A, Ravaglia, C, Poletti, V, Scala, R, Guidelli, L, Golfi, N, Vianello, A, Achille, A, Lucernoni, P, Gaccione, A, Romagnoli, M, Fraccaro, A, Malacchini, N, Malerba, M, Ragnoli, B, Zamparelli, A, Bocchino, M, Blasi, F, Spotti, M, Miele, C, Piedepalumbo, F, Barone, I, Baglioni, S, Dodaj, M, Franco, C, Andrani, F, Mangia, A, Mancini, A, Carrozzi, L, Rafanelli, A, Casto, E, Rogliani, P, Ora, J, Carpagnano, G, Di Lecce, V, Tamburrini, M, Papi, A, Contoli, M, Luzzati, R, Zatta, M, Di Bella, S, Caraffa, E, Francisci, D, Tosti, A, Pallotto, C, De Rosa, F, Pecori, A, Franceschini, M, Carlin, M, Orsini, V, Pollastri, E, Rugova, A, Sabbatini, F, Soria, A, Rossi, M, Santantonio, T, Meli, R, Sauro, S, Fedeli, C, Mangini, E, Biolo, G, Nunnari, A, Pietrangelo, A, Corradini, E, Bocchi, D, Boarini, C, Zucchetto, A, Lanini, S, Salton F., Confalonieri P., Centanni S., Mondoni M., Petrosillo N., Bonfanti P., Lapadula G., Lacedonia D., Voza A., Carpene N., Montico M., Reccardini N., Meduri G. U., Ruaro B., Confalonieri M., Citton G. M., Bozzi C., Tavano S., Pozzan R., Andrisano A. G., Jaber M., Mari M., Trotta L., Mondini L., Barbieri M., Ruggero L., Antonaglia C., Soave S., Torregiani C., Bogatec T., Baccelli A., Nalesso G., Re B., Pavesi S., Barbaro M. P. F., Giuliani A., Ravaglia C., Poletti V., Scala R., Guidelli L., Golfi N., Vianello A., Achille A., Lucernoni P., Gaccione A. T., Romagnoli M., Fraccaro A., Malacchini N., Malerba M., Ragnoli B., Zamparelli A. S., Bocchino M., Blasi F., Spotti M., Miele C., Piedepalumbo F., Barone I., Baglioni S., Dodaj M., Franco C., Andrani F., Mangia A., Mancini A., Carrozzi L., Rafanelli A., Casto E., Rogliani P., Ora J., Carpagnano G. E., Di Lecce V., Tamburrini M., Papi A., Contoli M., Luzzati R., Zatta M., Di Bella S., Caraffa E., Francisci D., Tosti A., Pallotto C., De Rosa F. G., Pecori A., Franceschini M., Carlin M., Orsini V., Pollastri E., Rugova A., Sabbatini F., Soria A., Rossi M., Santantonio T., Meli R., Sauro S., Fedeli C., Mangini E., Biolo G., Nunnari A., Pietrangelo A., Corradini E., Bocchi D., Boarini C., Zucchetto A., Lanini S., Salton, F, Confalonieri, P, Centanni, S, Mondoni, M, Petrosillo, N, Bonfanti, P, Lapadula, G, Lacedonia, D, Voza, A, Carpene, N, Montico, M, Reccardini, N, Meduri, G, Ruaro, B, Confalonieri, M, Citton, G, Bozzi, C, Tavano, S, Pozzan, R, Andrisano, A, Jaber, M, Mari, M, Trotta, L, Mondini, L, Barbieri, M, Ruggero, L, Antonaglia, C, Soave, S, Torregiani, C, Bogatec, T, Baccelli, A, Nalesso, G, Re, B, Pavesi, S, Barbaro, M, Giuliani, A, Ravaglia, C, Poletti, V, Scala, R, Guidelli, L, Golfi, N, Vianello, A, Achille, A, Lucernoni, P, Gaccione, A, Romagnoli, M, Fraccaro, A, Malacchini, N, Malerba, M, Ragnoli, B, Zamparelli, A, Bocchino, M, Blasi, F, Spotti, M, Miele, C, Piedepalumbo, F, Barone, I, Baglioni, S, Dodaj, M, Franco, C, Andrani, F, Mangia, A, Mancini, A, Carrozzi, L, Rafanelli, A, Casto, E, Rogliani, P, Ora, J, Carpagnano, G, Di Lecce, V, Tamburrini, M, Papi, A, Contoli, M, Luzzati, R, Zatta, M, Di Bella, S, Caraffa, E, Francisci, D, Tosti, A, Pallotto, C, De Rosa, F, Pecori, A, Franceschini, M, Carlin, M, Orsini, V, Pollastri, E, Rugova, A, Sabbatini, F, Soria, A, Rossi, M, Santantonio, T, Meli, R, Sauro, S, Fedeli, C, Mangini, E, Biolo, G, Nunnari, A, Pietrangelo, A, Corradini, E, Bocchi, D, Boarini, C, Zucchetto, A, Lanini, S, Salton F., Confalonieri P., Centanni S., Mondoni M., Petrosillo N., Bonfanti P., Lapadula G., Lacedonia D., Voza A., Carpene N., Montico M., Reccardini N., Meduri G. U., Ruaro B., Confalonieri M., Citton G. M., Bozzi C., Tavano S., Pozzan R., Andrisano A. G., Jaber M., Mari M., Trotta L., Mondini L., Barbieri M., Ruggero L., Antonaglia C., Soave S., Torregiani C., Bogatec T., Baccelli A., Nalesso G., Re B., Pavesi S., Barbaro M. P. F., Giuliani A., Ravaglia C., Poletti V., Scala R., Guidelli L., Golfi N., Vianello A., Achille A., Lucernoni P., Gaccione A. T., Romagnoli M., Fraccaro A., Malacchini N., Malerba M., Ragnoli B., Zamparelli A. S., Bocchino M., Blasi F., Spotti M., Miele C., Piedepalumbo F., Barone I., Baglioni S., Dodaj M., Franco C., Andrani F., Mangia A., Mancini A., Carrozzi L., Rafanelli A., Casto E., Rogliani P., Ora J., Carpagnano G. E., Di Lecce V., Tamburrini M., Papi A., Contoli M., Luzzati R., Zatta M., Di Bella S., Caraffa E., Francisci D., Tosti A., Pallotto C., De Rosa F. G., Pecori A., Franceschini M., Carlin M., Orsini V., Pollastri E., Rugova A., Sabbatini F., Soria A., Rossi M., Santantonio T., Meli R., Sauro S., Fedeli C., Mangini E., Biolo G., Nunnari A., Pietrangelo A., Corradini E., Bocchi D., Boarini C., Zucchetto A., and Lanini S.

Abstract

Background Dysregulated systemic inflammation is the primary driver of mortality in severe coronavirus disease 2019 (COVID-19) pneumonia. Current guidelines favour a 7-10-day course of any glucocorticoid equivalent to dexamethasone 6 mg daily. A comparative randomised controlled trial (RCT) with a higher dose and a longer duration of intervention was lacking. Methods We conducted a multicentre, open-label RCT to investigate methylprednisolone 80 mg as a continuous daily infusion for 8 days followed by slow tapering versus dexamethasone 6 mg once daily for up to 10 days in adult patients with COVID-19 pneumonia requiring oxygen or noninvasive respiratory support. The primary outcome was reduction in 28-day mortality. Secondary outcomes were mechanical ventilation-free days at 28 days, need for intensive care unit (ICU) referral, length of hospitalisation, need for tracheostomy, and changes in C-reactive protein (CRP) levels, arterial oxygen tension/inspiratory oxygen fraction (PaO2/FIO2) ratio and World Health Organization Clinical Progression Scale at days 3, 7 and 14. Results 677 randomised patients were included. Findings are reported as methylprednisolone (n=337) versus dexamethasone (n=340). By day 28, there were no significant differences in mortality (35 (10.4%) versus 41 (12.1%); p=0.49) nor in median mechanical ventilation-free days (median (interquartile range (IQR)) 23 (14) versus 24 (16) days; p=0.49). ICU referral was necessary in 41 (12.2%) versus 45 (13.2%) (p=0.68) and tracheostomy in 8 (2.4%) versus 9 (2.6%) (p=0.82). Survivors in the methylprednisolone group required a longer median (IQR) hospitalisation (15 (11) versus 14 (11) days; p=0.005) and experienced an improvement in CRP levels, but not in PaO2/FIO2 ratio, at days 7 and 14. There were no differences in disease progression at the prespecified time-points. Conclusion Prolonged, higher dose methylprednisolone did not reduce mortality at 28 days compared with conventional dexamethasone in COVID

Published
2023

8. Hematopoietic Stem Cell Transplantation in Late-onset X-linked Chronic Granulomatous Disease in a Female Carrier

Author

Trevisan M, Kang EM, Salton F, Ruaro B, Torregiani C, Confalonieri P, Naviglio S, Valencic E, Gabrielli M, Parta M, Kelly C, Notarangelo LD, Malech HL, Tommasini A, Confalonieri M., Trevisan, M, Kang, Em, Salton, F, Ruaro, B, Torregiani, C, Confalonieri, P, Naviglio, S, Valencic, E, Gabrielli, M, Parta, M, Kelly, C, Notarangelo, Ld, Malech, Hl, Tommasini, A, and Confalonieri, M.

Subjects
Interferon-gamma, Transplantation Conditioning, Immunology, Hematopoietic Stem Cell Transplantation, Humans, Immunology and Allergy, Female, Chronic Granulomatous Disease, Granulomatous Disease, Chronic, Article
Abstract

Chronic granulomatous disease (CGD) is a rare immunodeficiency disorder characterized by a defective NADPH oxidase complex leading to an impaired respiratory burst and defective killing of pathogens by phagocytes. The most common type of CGD is caused by hemizygous mutations of the CYBB gene on the X chromosome encoding the gp91phox subunit. It usually affects males, yet heterozygous females may rarely manifest clinical signs of the disease due to skewed X chromosome inactivation in leukocytes . Despite advances in gene therapy, hematopoietic stem cell transplantation (HSCT) remains the main definitive treatment. Older patients, however, are at greater risk of HSCT-related complications and mortality and may require a specific approach.

Published
2022

9. GRAd-COV2 vaccine provides potent and durable humoral and cellular immunity to SARS-CoV-2 in randomized placebo-controlled phase 2 trial

Author

Capone S., Fusco F. M., Milleri S., Borre S., Carbonara S., Lo Caputo S., Leone S., Gori G., Maggi P., Cascio A., Lichtner M., Cauda R., Dal Zoppo S., Cossu M. V., Gori A., Roda S., Confalonieri P., Bonora S., Missale G., Codeluppi M., Mezzaroma I., Capici S., Pontali E., Libanore M., Diani A., Lanini S., Battella S., Contino A. M., Piano Mortari E., Genova F., Parente G., Dragonetti R., Colloca S., Visani L., Iannacone C., Carsetti R., Folgori A., Camerini R., Ziviani L., Malescio F., Turrini I., Lawlor R., Romano A., Nunziata M., Armato S., Mazzeo N., Carleo M. A., Dell'Isola C., Pisapia R., Pontarelli A., Olivani A., Grasselli S., Laccabue D., Leoni M. C., Paolillo F., Mancini A., Ruaro B., Confalonieri M., Salton F., Mancarella G., Marocco R., De Masi M., Belvisi V., Lamonica S., Cingolani A., Seguiti C., Brambilla P., Ferraresi A., Lupi M., Ludovisi S., Renisi G., Massafra R., Pellicciotta M., Armiento L., Vimercati S., Piacenza M., Bonfanti P., Columpsi P., Cazzaniga M. E., Rovelli C., Ceresini M., Previtali L., Trentini L., Alcantarini C., Rugge W., Biffi S., Poletti F., Rostagno R., Moglia R., De Negri F., Fini E., Cangialosi A., Bruno S. R., Rizzo M., Niglio M., Stritto A. D., Matano A., Petruzziello A., Valsecchi P., Pieri T., Altamura M., Calamo A., Giannelli A., Menolascina S., Di Bari S., Mauro V., Aronica R., Segala D., Cultrera R., Sighinolfi L., Abbott M., Gizzi A., Marascia F. G., Valenti G., Feasi M., Bobbio N., Del Puente F., Nicosia A., Frasca M., Mazzoleni M., Garofalo N., Ammendola V., Grazioli F., Napolitano F., Vitelli A., Marcellini V., Capone, Stefania, Fusco, Francesco M, Milleri, Stefano, Borrè, Silvio, Carbonara, Sergio, Lo Caputo, Sergio, Leone, Sebastiano, Gori, Giovanni, Maggi, Paolo, Cascio, Antonio, Lichtner, Miriam, Cauda, Roberto, Dal Zoppo, Sarah, Cossu, Maria V, Gori, Andrea, Roda, Silvia, Confalonieri, Paola, Bonora, Stefano, Missale, Gabriele, Codeluppi, Mauro, Mezzaroma, Ivano, Capici, Serena, Pontali, Emanuele, Libanore, Marco, Diani, Augusta, Lanini, Simone, Battella, Simone, Contino, Alessandra M, Piano Mortari, Eva, Genova, Francesco, Parente, Gessica, Dragonetti, Rosella, Colloca, Stefano, Visani, Luigi, Iannacone, Claudio, Carsetti, Rita, Folgori, Antonella, Camerini, Roberto, Capone, S, Fusco, F, Milleri, S, Borre, S, Carbonara, S, Lo Caputo, S, Leone, S, Gori, G, Maggi, P, Cascio, A, Lichtner, M, Cauda, R, Dal Zoppo, S, Cossu, M, Gori, A, Roda, S, Confalonieri, P, Bonora, S, Missale, G, Codeluppi, M, Mezzaroma, I, Capici, S, Pontali, E, Libanore, M, Diani, A, Lanini, S, Battella, S, Contino, A, Piano Mortari, E, Genova, F, Parente, G, Dragonetti, R, Colloca, S, Visani, L, Iannacone, C, Carsetti, R, Folgori, A, Camerini, R, Ziviani, L, Malescio, F, Turrini, I, Lawlor, R, Romano, A, Nunziata, M, Armato, S, Mazzeo, N, Carleo, M, Dell'Isola, C, Pisapia, R, Pontarelli, A, Olivani, A, Grasselli, S, Laccabue, D, Leoni, M, Paolillo, F, Mancini, A, Ruaro, B, Confalonieri, M, Salton, F, Mancarella, G, Marocco, R, De Masi, M, Belvisi, V, Lamonica, S, Cingolani, A, Seguiti, C, Brambilla, P, Ferraresi, A, Lupi, M, Ludovisi, S, Renisi, G, Massafra, R, Pellicciotta, M, Armiento, L, Vimercati, S, Piacenza, M, Bonfanti, P, Columpsi, P, Cazzaniga, M, Rovelli, C, Ceresini, M, Previtali, L, Trentini, L, Alcantarini, C, Rugge, W, Biffi, S, Poletti, F, Rostagno, R, Moglia, R, De Negri, F, Fini, E, Cangialosi, A, Bruno, S, Rizzo, M, Niglio, M, Stritto, A, Matano, A, Petruzziello, A, Valsecchi, P, Pieri, T, Altamura, M, Calamo, A, Giannelli, A, Menolascina, S, Di Bari, S, Mauro, V, Aronica, R, Segala, D, Cultrera, R, Sighinolfi, L, Abbott, M, Gizzi, A, Marascia, F, Valenti, G, Feasi, M, Bobbio, N, Del Puente, F, Nicosia, A, Frasca, M, Mazzoleni, M, Garofalo, N, Ammendola, V, Grazioli, F, Napolitano, F, Vitelli, A, and Marcellini, V

Subjects
immunological memory, phase 2 clinical trial, safety, Sars-CoV-2 vaccine, COVID-19, CD8, T cell response, simian adenoviral vector, General Biochemistry, Genetics and Molecular Biology, CD4, neutralizing antibodie
Abstract

The ongoing severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic and heterologous immunization approaches implemented worldwide for booster doses call for diversified vaccine portfolios. GRAd-COV2 is a gorilla adenovirus-based COVID-19 vaccine candidate encoding prefusion-stabilized spike. The safety and immunogenicity of GRAd-COV2 is evaluated in a dose- and regimen-finding phase 2 trial (COVITAR study, ClinicalTrials.gov: NCT04791423) whereby 917 eligible participants are randomized to receive a single intramuscular GRAd-COV2 administration followed by placebo, or two vaccine injections, or two doses of placebo, spaced over 3weeks. Here, we report that GRAd-COV2 is well tolerated and induces robust immune responses after a single immunization; a second administration increases binding and neutralizing antibody titers. Potent, variant of concern (VOC) cross-reactive spike-specific Tcell response peaks after the first dose and is characterized by high frequencies of CD8s. Tcells maintain immediate effector functions and high proliferative potential over time. Thus, GRAd vector is a valuable platform for genetic vaccine development, especially when robust CD8 response is needed.

Published
2023

15. Real-life efficacy and safety of nintedanib in systemic sclerosis-interstitial lung disease: data from an Italian multicentre study

Author

Campochiaro, C., De Luca, G., Lazzaroni, M. -G., Armentaro, G., Spinella, A., Vigone, B., Ruaro, B., Stanziola, A., Benfaremo, D., De Lorenzis, E., Moccaldi, B., Bosello, Silvia Laura, Cuomo, G., Beretta, Carlo Luigi, Zanatta, E., Giuggioli, D., Del Papa, N., Airo, P., Confalonieri, Marco, Moroncini, G., Dagna, L., Matucci-Cerinic, M., Bosello S. L. (ORCID:0000-0002-4837-447X), Beretta L. (ORCID:0000-0001-9924-2066), Confalonieri M. (ORCID:0000-0002-3708-379X), Campochiaro, C., De Luca, G., Lazzaroni, M. -G., Armentaro, G., Spinella, A., Vigone, B., Ruaro, B., Stanziola, A., Benfaremo, D., De Lorenzis, E., Moccaldi, B., Bosello, Silvia Laura, Cuomo, G., Beretta, Carlo Luigi, Zanatta, E., Giuggioli, D., Del Papa, N., Airo, P., Confalonieri, Marco, Moroncini, G., Dagna, L., Matucci-Cerinic, M., Bosello S. L. (ORCID:0000-0002-4837-447X), Beretta L. (ORCID:0000-0001-9924-2066), and Confalonieri M. (ORCID:0000-0002-3708-379X)

Abstract

Introduction Nintedanib (NTD) has been shown to be effective in systemic sclerosis (SSc)-interstitial lung disease (ILD). Here we describe the efficacy and safety of NTD in a real-life setting. Methods Patients with SSc-ILD treated with NTD were retrospectively evaluated at 12 months prior to NTD introduction; at baseline and at 12 months after NTD introduction. The following parameters were recorded: SSc clinical features, NTD tolerability, pulmonary function tests and modified Rodnan skin score (mRSS). Results 90 patients with SSc-ILD (65% female, mean age 57.6±13.4 years, mean disease duration 8.8±7.6 years) were identified. The majority were positive for anti-topoisomerase I (75%) and 77 (85%) patients were on immunosuppressants. A significant decline in %predicted forced vital capacity (%pFVC) in the 12 months prior to NTD introduction was observed in 60%. At 12 months after NTD introduction, follow-up data were available for 40 (44%) patients and they showed a stabilisation in %pFVC (64±14 to 62±19, p=0.416). The percentage of patients with significant lung progression at 12 months was significantly lower compared with the previous 12 months (60% vs 17.5%, p=0.007). No significant mRSS change was observed. Gastrointestinal (GI) side effects were recorded in 35 (39%) patients. After a mean time of 3.6±3.1 months, NTD was maintained after dose adjustment in 23 (25%) patients. In nine (10%) patients, NTD was stopped after a median time of 4.5 (1-6) months. During the follow-up, four patients died. Conclusions In a real-life clinical scenario, NTD, in combination with immunosuppressants, may stabilise lung function. GI side effects are frequent and NTD dose adjustment may be necessary to retain the drug in patients with SSc-ILD.

Published
2023

21. Prolonged higher dose methylprednisolone vs. conventional dexamethasone in COVID-19 pneumonia: a randomised controlled trial (MEDEAS)

Author

Salton F., Confalonieri P., Centanni S., Mondoni M., Petrosillo N., Bonfanti P., Lapadula G., Lacedonia D., Voza A., Carpene N., Montico M., Reccardini N., Meduri G. U., Ruaro B., Confalonieri M., Citton G. M., Bozzi C., Tavano S., Pozzan R., Andrisano A. G., Jaber M., Mari M., Trotta L., Mondini L., Barbieri M., Ruggero L., Antonaglia C., Soave S., Torregiani C., Bogatec T., Baccelli A., Nalesso G., Re B., Pavesi S., Barbaro M. P. F., Giuliani A., Ravaglia C., Poletti V., Scala R., Guidelli L., Golfi N., Vianello A., Achille A., Lucernoni P., Gaccione A. T., Romagnoli M., Fraccaro A., Malacchini N., Malerba M., Ragnoli B., Zamparelli A. S., Bocchino M., Blasi F., Spotti M., Miele C., Piedepalumbo F., Barone I., Baglioni S., Dodaj M., Franco C., Andrani F., Mangia A., Mancini A., Carrozzi L., Rafanelli A., Casto E., Rogliani P., Ora J., Carpagnano G. E., Di Lecce V., Tamburrini M., Papi A., Contoli M., Luzzati R., Zatta M., Di Bella S., Caraffa E., Francisci D., Tosti A., Pallotto C., De Rosa F. G., Pecori A., Franceschini M., Carlin M., Orsini V., Pollastri E., Rugova A., Sabbatini F., Soria A., Rossi M., Santantonio T., Meli R., Sauro S., Fedeli C., Mangini E., Biolo G., Nunnari A., Pietrangelo A., Corradini E., Bocchi D., Boarini C., Zucchetto A., Lanini S., Salton, F, Confalonieri, P, Centanni, S, Mondoni, M, Petrosillo, N, Bonfanti, P, Lapadula, G, Lacedonia, D, Voza, A, Carpene, N, Montico, M, Reccardini, N, Meduri, G, Ruaro, B, Confalonieri, M, Citton, G, Bozzi, C, Tavano, S, Pozzan, R, Andrisano, A, Jaber, M, Mari, M, Trotta, L, Mondini, L, Barbieri, M, Ruggero, L, Antonaglia, C, Soave, S, Torregiani, C, Bogatec, T, Baccelli, A, Nalesso, G, Re, B, Pavesi, S, Barbaro, M, Giuliani, A, Ravaglia, C, Poletti, V, Scala, R, Guidelli, L, Golfi, N, Vianello, A, Achille, A, Lucernoni, P, Gaccione, A, Romagnoli, M, Fraccaro, A, Malacchini, N, Malerba, M, Ragnoli, B, Zamparelli, A, Bocchino, M, Blasi, F, Spotti, M, Miele, C, Piedepalumbo, F, Barone, I, Baglioni, S, Dodaj, M, Franco, C, Andrani, F, Mangia, A, Mancini, A, Carrozzi, L, Rafanelli, A, Casto, E, Rogliani, P, Ora, J, Carpagnano, G, Di Lecce, V, Tamburrini, M, Papi, A, Contoli, M, Luzzati, R, Zatta, M, Di Bella, S, Caraffa, E, Francisci, D, Tosti, A, Pallotto, C, De Rosa, F, Pecori, A, Franceschini, M, Carlin, M, Orsini, V, Pollastri, E, Rugova, A, Sabbatini, F, Soria, A, Rossi, M, Santantonio, T, Meli, R, Sauro, S, Fedeli, C, Mangini, E, Biolo, G, Nunnari, A, Pietrangelo, A, Corradini, E, Bocchi, D, Boarini, C, Zucchetto, A, Lanini, S, Salton, Francesco, Confalonieri, Paola, Centanni, Stefano, Mondoni, Michele, Petrosillo, Nicola, Bonfanti, Paolo, Lapadula, Giuseppe, Lacedonia, Donato, Voza, Antonio, Carpenè, Nicoletta, Montico, Marcella, Reccardini, Nicolò, Meduri, Gianfranco Umberto, Ruaro, Barbara, MEDEAS Collaborative, Group, and Confalonieri, Marco

Subjects
Pulmonary and Respiratory Medicine, glucocorticoids, Settore MED/10 - Malattie dell'Apparato Respiratorio, pneumonia, COVID-19, glucocorticoid, dexamethasone, ARDS, acute respiratory distress syndrome, methylprednisolone
Abstract

BackgroundDysregulated systemic inflammation is the primary driver of mortality in severe coronavirus disease 2019 (COVID-19) pneumonia. Current guidelines favour a 7–10-day course of any glucocorticoid equivalent to dexamethasone 6 mg daily. A comparative randomised controlled trial (RCT) with a higher dose and a longer duration of intervention was lacking.MethodsWe conducted a multicentre, open-label RCT to investigate methylprednisolone 80 mg as a continuous daily infusion for 8 days followed by slow taperingversusdexamethasone 6 mg once daily for up to 10 days in adult patients with COVID-19 pneumonia requiring oxygen or noninvasive respiratory support. The primary outcome was reduction in 28-day mortality. Secondary outcomes were mechanical ventilation-free days at 28 days, need for intensive care unit (ICU) referral, length of hospitalisation, need for tracheostomy, and changes in C-reactive protein (CRP) levels, arterial oxygen tension/inspiratory oxygen fraction (PaO2/FIO2) ratio and World Health Organization Clinical Progression Scale at days 3, 7 and 14.Results677 randomised patients were included. Findings are reported as methylprednisolone (n=337)versusdexamethasone (n=340). By day 28, there were no significant differences in mortality (35 (10.4%)versus41 (12.1%); p=0.49) nor in median mechanical ventilation-free days (median (interquartile range (IQR)) 23 (14)versus24 (16) days; p=0.49). ICU referral was necessary in 41 (12.2%)versus45 (13.2%) (p=0.68) and tracheostomy in 8 (2.4%)versus9 (2.6%) (p=0.82). Survivors in the methylprednisolone group required a longer median (IQR) hospitalisation (15 (11)versus14 (11) days; p=0.005) and experienced an improvement in CRP levels, but not inPaO2/FIO2ratio, at days 7 and 14. There were no differences in disease progression at the prespecified time-points.ConclusionProlonged, higher dose methylprednisolone did not reduce mortality at 28 days compared with conventional dexamethasone in COVID-19 pneumonia.

Published
2022

22. The role of chest CT in deciphering interstitial lung involvement: systemic sclerosis versus COVID-19

Author

Orlandi, M, Landini, N, Sambataro, G, Nardi, C, Tofani, L, Bruni, C, Bellando-Randone, S, Blagojevic, J, Melchiorre, D, Hughes, M, Denton, C, Luppi, F, Ruaro, B, Della Casa, F, Rossi, F, De Luca, G, Campochiaro, C, Spinicci, M, Zammarchi, L, Tomassetti, S, Caminati, A, Cavigli, E, Albanesi, M, Melchiorre, F, Palmucci, S, Vegni, V, Guiducci, S, Moggi-Pignone, A, Allanore, Y, Bartoloni, A, Confalonieri, M, Dagna, L, De Cobelli, F, De Paulis, A, Harari, S, Khanna, D, Kuwana, M, Taliani, G, Lavorini, F, Miele, V, Morana, G, Pesci, A, Vancheri, C, Colagrande, S, Matucci-Cerinic, M, Orlandi, Martina, Landini, Nicholas, Sambataro, Gianluca, Nardi, Cosimo, Tofani, Lorenzo, Bruni, Cosimo, Bellando-Randone, Silvia, Blagojevic, Jelena, Melchiorre, Daniela, Hughes, Michael, Denton, Christopher P, Luppi, Fabrizio, Ruaro, Barbara, Della Casa, Francesca, Rossi, Francesca W, De Luca, Giacomo, Campochiaro, Corrado, Spinicci, Michele, Zammarchi, Lorenzo, Tomassetti, Sara, Caminati, Antonella, Cavigli, Edoardo, Albanesi, Marco, Melchiorre, Fabio, Palmucci, Stefano, Vegni, Virginia, Guiducci, Serena, Moggi-Pignone, Alberto, Allanore, Yannick, Bartoloni, Alessandro, Confalonieri, Marco, Dagna, Lorenzo, De Cobelli, Francesco, De Paulis, Amato, Harari, Sergio, Khanna, Dinesh, Kuwana, Masataka, Taliani, Gloria, Lavorini, Federico, Miele, Vittorio, Morana, Giovanni, Pesci, Alberto, Vancheri, Carlo, Colagrande, Stefano, Matucci-Cerinic, Marco, Orlandi, M, Landini, N, Sambataro, G, Nardi, C, Tofani, L, Bruni, C, Bellando-Randone, S, Blagojevic, J, Melchiorre, D, Hughes, M, Denton, C, Luppi, F, Ruaro, B, Della Casa, F, Rossi, F, De Luca, G, Campochiaro, C, Spinicci, M, Zammarchi, L, Tomassetti, S, Caminati, A, Cavigli, E, Albanesi, M, Melchiorre, F, Palmucci, S, Vegni, V, Guiducci, S, Moggi-Pignone, A, Allanore, Y, Bartoloni, A, Confalonieri, M, Dagna, L, De Cobelli, F, De Paulis, A, Harari, S, Khanna, D, Kuwana, M, Taliani, G, Lavorini, F, Miele, V, Morana, G, Pesci, A, Vancheri, C, Colagrande, S, Matucci-Cerinic, M, Orlandi, Martina, Landini, Nicholas, Sambataro, Gianluca, Nardi, Cosimo, Tofani, Lorenzo, Bruni, Cosimo, Bellando-Randone, Silvia, Blagojevic, Jelena, Melchiorre, Daniela, Hughes, Michael, Denton, Christopher P, Luppi, Fabrizio, Ruaro, Barbara, Della Casa, Francesca, Rossi, Francesca W, De Luca, Giacomo, Campochiaro, Corrado, Spinicci, Michele, Zammarchi, Lorenzo, Tomassetti, Sara, Caminati, Antonella, Cavigli, Edoardo, Albanesi, Marco, Melchiorre, Fabio, Palmucci, Stefano, Vegni, Virginia, Guiducci, Serena, Moggi-Pignone, Alberto, Allanore, Yannick, Bartoloni, Alessandro, Confalonieri, Marco, Dagna, Lorenzo, De Cobelli, Francesco, De Paulis, Amato, Harari, Sergio, Khanna, Dinesh, Kuwana, Masataka, Taliani, Gloria, Lavorini, Federico, Miele, Vittorio, Morana, Giovanni, Pesci, Alberto, Vancheri, Carlo, Colagrande, Stefano, and Matucci-Cerinic, Marco

Abstract

Objective: The aim of this study was to identify the main CT features that may help in distinguishing a progression of interstitial lung disease (ILD) secondary to SSc from COVID-19 pneumonia. Methods: This multicentric study included 22 international readers grouped into a radiologist group (RADs) and a non-radiologist group (nRADs). A total of 99 patients, 52 with COVID-19 and 47 with SSc-ILD, were included in the study. Results: Fibrosis inside focal ground-glass opacities (GGOs) in the upper lobes; fibrosis in the lower lobe GGOs; reticulations in lower lobes (especially if bilateral and symmetrical or associated with signs of fibrosis) were the CT features most frequently associated with SSc-ILD. The CT features most frequently associated with COVID- 19 pneumonia were: consolidation (CONS) in the lower lobes, CONS with peripheral (both central/peripheral or patchy distributions), anterior and posterior CONS and rounded-shaped GGOs in the lower lobes. After multivariate analysis, the presence of CONs in the lower lobes (P < 0.0001) and signs of fibrosis in GGOs in the lower lobes (P < 0.0001) remained independently associated with COVID-19 pneumonia and SSc-ILD, respectively. A predictive score was created that was positively associated with COVID-19 diagnosis (96.1% sensitivity and 83.3% specificity). Conclusion: CT diagnosis differentiating between COVID-19 pneumonia and SSc-ILD is possible through a combination of the proposed score and radiologic expertise. The presence of consolidation in the lower lobes may suggest COVID-19 pneumonia, while the presence of fibrosis inside GGOs may indicate SSc-ILD.

Published
2022

24. POS0890 NINTEDANIB REAL-LIFE EFFICACY AND SAFETY IN SYSTEMIC SCLEROSIS (SSc)-INTERTISTIAL LUNG DISEASE (ILD): AN ITALIAN MULTICENTRE PRELIMINARY STUDY.

Author

Campochiaro, C., primary, De Luca, G., additional, Lazzaroni, M. G., additional, Armentaro, G., additional, Spinella, A., additional, Vigone, B., additional, Ruaro, B., additional, Stanziola, A., additional, Benfaremo, D., additional, De Lorenzis, E., additional, Benvenuti, F., additional, Bosello, S. L., additional, Moroncini, G., additional, Cuomo, G., additional, Confalonieri, M., additional, Beretta, L., additional, Zanatta, E., additional, Giuggioli, D., additional, Del Papa, N., additional, Airò, P., additional, Dagna, L., additional, and Matucci-Cerinic, M., additional

Published
2022
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28. POS1228 THE ROLE OF CHEST CT IN UNDERSTANDING INTERSTITIAL LUNG DISEASE (ILD): SYSTEMIC SCLEROSIS (SSc). VERSUS COVID-19

Author

Orlandi, M., primary, Landini, N., additional, Sambataro, G., additional, Nardi, C., additional, Bruni, C., additional, Bellando-Randone, S., additional, Denton, C., additional, Luppi, F., additional, Ruaro, B., additional, Tomassetti, S., additional, Cavigli, E., additional, Melchiorre, F., additional, Palmucci, S., additional, Guiducci, S., additional, Moggi Pignone, A., additional, Allanore, Y., additional, Bartoloni, A., additional, Confalonieri, M., additional, Cortese, G., additional, Dagna, L., additional, De Cobelli, F., additional, De Paulis, A., additional, Harari, S., additional, Khanna, D., additional, Kuwana, M., additional, Miele, V., additional, Taliani, G., additional, Hughes, M., additional, Vanchieri, C., additional, Colagrande, S., additional, and Matucci-Cerinic, M., additional

Published
2021
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29. Systemic sclerosis: State of the art on clinical practice guidelines

Author

Smith, V, Scire, C, Talarico, R, Airo, P, Alexander, T, Allanore, Y, Bruni, C, Codullo, V, Dalm, V, De Vries-Bouwstra, J, Della Rossa, A, Distler, O, Galetti, I, Launay, D, Lepri, G, Mathian, A, Mouthon, L, Ruaro, B, Sulli, A, Tincani, A, Vandecasteele, E, Vanhaecke, A, Vanthuyne, M, Van Den Hoogen, F, Van Vollenhoven, R, Voskuyl, A, Zanatta, E, Bombardieri, S, Burmester, G, Eurico, F, Frank, C, Hachulla, E, Houssiau, F, Mueller-Ladner, U, Schneider, M, Van Laar, J, Vieira, A, Cutolo, M, Mosca, M, Matucci-Cerinic, M, Smith V., Scire C. A., Talarico R., Airo P., Alexander T., Allanore Y., Bruni C., Codullo V., Dalm V., De Vries-Bouwstra J., Della Rossa A., Distler O., Galetti I., Launay D., Lepri G., Mathian A., Mouthon L., Ruaro B., Sulli A., Tincani A., Vandecasteele E., Vanhaecke A., Vanthuyne M., Van Den Hoogen F., Van Vollenhoven R., Voskuyl A. E., Zanatta E., Bombardieri S., Burmester G., Eurico F. J., Frank C., Hachulla E., Houssiau F., Mueller-Ladner U., Schneider M., Van Laar J. M., Vieira A., Cutolo M., Mosca M., Matucci-Cerinic M., Smith, V, Scire, C, Talarico, R, Airo, P, Alexander, T, Allanore, Y, Bruni, C, Codullo, V, Dalm, V, De Vries-Bouwstra, J, Della Rossa, A, Distler, O, Galetti, I, Launay, D, Lepri, G, Mathian, A, Mouthon, L, Ruaro, B, Sulli, A, Tincani, A, Vandecasteele, E, Vanhaecke, A, Vanthuyne, M, Van Den Hoogen, F, Van Vollenhoven, R, Voskuyl, A, Zanatta, E, Bombardieri, S, Burmester, G, Eurico, F, Frank, C, Hachulla, E, Houssiau, F, Mueller-Ladner, U, Schneider, M, Van Laar, J, Vieira, A, Cutolo, M, Mosca, M, Matucci-Cerinic, M, Smith V., Scire C. A., Talarico R., Airo P., Alexander T., Allanore Y., Bruni C., Codullo V., Dalm V., De Vries-Bouwstra J., Della Rossa A., Distler O., Galetti I., Launay D., Lepri G., Mathian A., Mouthon L., Ruaro B., Sulli A., Tincani A., Vandecasteele E., Vanhaecke A., Vanthuyne M., Van Den Hoogen F., Van Vollenhoven R., Voskuyl A. E., Zanatta E., Bombardieri S., Burmester G., Eurico F. J., Frank C., Hachulla E., Houssiau F., Mueller-Ladner U., Schneider M., Van Laar J. M., Vieira A., Cutolo M., Mosca M., and Matucci-Cerinic M.

Abstract

Systemic sclerosis (SSc) is an orphan disease characterised by autoimmunity, fibrosis of the skin and internal organs, and vasculopathy. SSc may be associated with high morbidity and mortality. In this narrative review we summarise the results of a systematic literature research, which was performed as part of the European Reference Network on Rare and Complex Connective Tissue and Musculoskeletal Diseases project, aimed at evaluating existing clinical practice guidelines or recommendations. Only in the domains 'Vascular & Ulcers' (ie, non-pharmacological approach to digital ulcer), 'PAH' (ie, screening and treatment), 'Treatment' and 'Juveniles' (ie, evaluation of juveniles with Raynaud's phenomenon) evidence-based and consensus-based guidelines could be included. Hence there is a preponderance of unmet needs in SSc referring to the diagnosis and (non-)pharmacological treatment of several SSc-specific complications. Patients with SSc experience significant uncertainty concerning SSc-related taxonomy, management (both pharmacological and nonpharmacological) and education. Day-to-day impact of the disease (loss of self-esteem, fatigue, sexual dysfunction, and occupational, nutritional and relational problems) is underestimated and needs evaluation.

Published
2018

30. Racial differences in systemic sclerosis disease presentation: A European Scleroderma Trials and Research group study

Author

Jaeger, Veronika K, Tikly, Mohammed, Dong, Xu, Siegert, Elise, Hachulla, Eric, Airò, Paolo, Valentini, Gabriele, Matucci Cerinic, Marco, Distler, Oliver, Cozzi, Franco, Carreira, Patricia, Allanore, Yannick, Müller-Ladner, Ulf, Ananieva, Lidia P, Balbir-Gurman, Alexandra, Distler, Jörg H W, Czirják, Laszlo, Mengtao, Li, Henes, Jörg, Jimenez, Sergio A, Smith, Vanessa, Damjanov, Nemanja, Denton, Christopher P, Delgaldo, Francesco, Saketkoo, Lesley Ann, Walker, Ulrich, A, Randone, Sb, Bannert, B, Iannone, F, Maurer, B, Jordan, S, Dobrota, R, Becker, M, Mihai, C, Becvarare, R, Tomčík, M, Bielecka, Ok, Gindzienska-Sieskiewicz, E, Karaszewska, K, Cutolo, M, Pizzorni, C, Paolino, S, Sulli, A, Ruaro, B, Alessandri, E, Riccardi, A, Giacco, V, Messitini, V, Irace, R, Kedor, C, Casteleyn, V, Hilger, J, Hoeppner, J, Rednic, S, Szabo, I, Petcu, A, Avouac, J, Camelia, F, Desbas, C, Vlachoyiannopoulos, P, Montecucco, C, Caporali, R, Cavagna, L, Stork, J, Inanc, M, Joven, Be, Novak, S, Anic, F, Varju, C, Minier, T, Chizzolini, C, Allai, D, Kucharz, Ej, Kotulska, A, Kopec-Medrek, M, Widuchowska, M, Dolnicar, As, Coleiro, B, Gabrielli, A, Manfredi, L, Benfaremo, D, Ferrarini, A, Bancel, Df, Hij, A, Lansiaux, P, Lazzaroni, Mg, Hesselstrand, R, Wuttge, D, Andréasson, R, Martinovic, D, Bozic, I, Radic, M, Braun-Moscovici, Y, Monaco, Al, Furini, F, Hunzelmann, N, Moinzadeh, P, Pellerito, R, Caimmi, C, Bertoldo, E, Morovic-Vergles, J, Culo, Im, Pecher, Ac, Santamaria, Vo, Heitmann, S, Codagnone, M, Pflugfelder, J, Krasowska, D, Michalska-Jakubus, M, Seidel, M, Hasler, P, Kretschmar, S, Kohm, M, Bajocchi, G, Salvador, Mj, Silva, Japd, Stamenkovic, B, Stankovic, A, Selmi, Cf, Santis, M, Ceribelli, A, Garzanova, L, Koneva, O, Starovoytova, M, Herrick, A, Puppo, F, Negrini, S, Murdaca, G, Engelhart, M, Szücs, G, Szamosi, S, de la Puente, C, Grande, Cs, Villanueva, Mjg, Midtvedt, Sø, Hoffmann-Vold, Am, Launay, D, Sobanski, V, Riccieri, V, Vasile, M, Ionescu, Rm, Opris, D, Sha, A, Woods, A, Gheorghiu, Am, Bojinca, M, Sunderkötter, C, Ehrchen, J, Ingegnoli, F, Mouthon, L, Dunogue, B, Chaigne, B, Legendre, P, Cantatore, Fp, Corrado, A, Ullman, S, Iversen, L, von Mühlen CA, Pozzi, Mr, Eyerich, K, Lauffer, F, Wiland, P, Szmyrka-Kaczmarek, M, Sokolik, R, Morgiel, E, Madej, M, Vanthuyne, M, Frédéric, H, Alegre-Sancho, Jj, Aringer, M, Herrmann, K, Günther, C, Westhovens, R, Langhe, E, Lenaerts, J, Anic, B, Baresic, M, Mayer, M, Üprus, M, Otsa, K, Yavuz, S, Granel, B, Radominski, Sc, De, C, Müller, S, Azevedo, Vf, Mendoza, F, Busquets, J, Popa, S, Agachi, S, Zenone, T, Pileckyte, M, Stebbings, S, Mathieu, A, Vacca, A, Sampaio-Barros, Pd, Stamp, L, Solanki, K, Silva, C, Schollum, J, Barns-Graham, H, Veale, D, O'Rourke, M, Loyo, E, Tineo, C, Paulino, G, Mohamed, Waaa, Rosato, E, Gigante, A, Oksel, F, Yargucu, F, Tanaseanu, Cm, Popescu, M, Dumitrascu, A, Tiglea, I, Foti, R, Visalli, E, Benenati, A, Amato, G, Ancuta, C, Villiger, P, Adler, S, Fröhlich, J, Kayser, C, Eduardo, Al, Fathi, N, Alii, S, Ahmed, M, Hasaneen, S, Hakeem, Ee, de la PG, Lefebvre, P, Martin, Jjg, Sibilia, J, Chatelus, E, Gottenberg, Je, Chifflot, H, Litinsky, I, Galdo, Fd, Abignano, G, Eng, S, Seskute, G, Butrimiene, I, Rugiene, R, Karpec, D, Pascal, M, Kerzberg, E, Bianchi, W, Bianchi, Bv, Bianchi, Dv, Barcellos, Y, Castellví, I, Millan, M, Limonta, M, Rimar, D, Rosner, I, Slobodin, G, Couto, M, Spertini, F, Ribi, C, Buss, G, Marcoccia, A, Bondanini, F, Ciani, A, Kahl, S, Hsu, Vm, Martin, T, Poindron, V, Meghit, K, Moiseev, S, Novikov, P, Chung, L, Kolstad, K, Stark, M, Schmeiser, T, Thiele, A, Majewski, D, Zdrojewski, Z, Zaneta, S, Wierzba, K, Martínez-Barrio, J, López-Longo, Fj, Bernardino, V, Moraes-Fontes, Mf, Rodrigues, Ac, Riemekasten, G, Sommerlatte, S, Jendreck, S, Arnold, S, Levy, Y, Rezus, E, Cardoneanu, A, Burlui, Am, Pamuk, On, Puttini, Ps, Talotta, R, Bongiovanni, S, Poormoghim, H, Andalib, E, Almasi, S, Kötter, I, Krusche, M, Cuomo, G, Danzo, F, Masini, F, Gaches, F, Michaud, M, Cartos, F, Belloli, L, Casu, C, Sfikakis, P, Tektonidou, M, Furst, D, Feldman, Gr, Ramazan, Am, Nurmambet, E, Miroto, A, Suta, C, Andronache, I, Huizinga, Twj, de Vries-Bouwstra, J., Chizzolini, Carlo, Jaeger, Veronika K, Tikly, Mohammed, Xu, Dong, Siegert, Elise, Hachulla, Eric, Airò, Paolo, Valentini, Gabriele, Matucci Cerinic, Marco, Distler, Oliver, Cozzi, Franco, Carreira, Patricia, Allanore, Yannick, Müller-Ladner, Ulf, Ananieva, Lidia P, Balbir-Gurman, Alexandra, Distler, Jörg H W, Czirják, Laszlo, Li, Mengtao, Henes, Jörg, Jimenez, Sergio A, Smith, Vanessa, Damjanov, Nemanja, Denton, Christopher P, Delgaldo, Francesco, Saketkoo, Lesley Ann, Walker, Ulrich A, University of Zurich, Cerinic, Marco Matucci, Walker Ulrich, A, Randone, Silvia Bellando, Bannert, Bettina, Iannone, Florenzoaa, Maurer, Brittaab, Jordan, Suzanaab, Dobrota, Rucsandraab, Becker, Mikeab, Mihai, Carinaa, Becvarare, Radima, Tomcik, Michala, Bielecka, Otylia Kowala, Gindzienska-Sieskiewicz, Ewaa, Karaszewska, Katarzynaa, Cutolo, Maurizioa, Pizzorni, Carmena, Paolino, Sabrinaae, Sulli, Albertoa, Ruaro, Barbara, Alessandri, Elisa, Riccardi, Antonella, Giacco, Veronica, Messitini, Valentina, Irace, Rosaria, Kedor, Claudia, Casteleyn, Vincent, Hilger, Julia, Hoeppner, Jakob, Rednic, Simona, Szabo, Iulia, Petcu, Ana, Avouac, Jérome, Camelia, Frantz, Desbas, Carole, Vlachoyiannopoulos, Panayioti, Montecucco, Carlo Maurizio, Caporali, Roberto, Cavagna, Lorenzo, Stork, Jiri, Inanc, Murat, Joven, Beatriz E., Novak, Srdan, Anic, Felina, Varju, Cecilia, Minier, Tunde, Allai, Daniela, Kucharz, Eugene J., Kotulska, Anna, Kopec-Medrek, Magdalena, Widuchowska, Malgorzata, Dolnicar, Alenka Sipek, Coleiro, Bernard, Gabrielli, Armando, Manfredi, Lucia, Benfaremo, Devi, Ferrarini, Alessia, Bancel, Dominique Farge, Hij, Adrian, Lazzaroni, Maria Grazia, Hesselstrand, Roger, Wuttge, Dirk, Andréasson, Kristofer, Martinovic, Duska, Bozic, Ivona, Radic, Mislav, Braun-Moscovici, Yolanda, Monaco, Andrea Lo, Furini, Federica, Hunzelmann, Nicola, Moinzadeh, Pia, Pellerito, Raffaele, Caimmi, Cristian, Bertoldo, Eugenia, Morovic-Vergles, Jadranka, Culo, Ivana Melanie, Pecher, Ann-Christian, Santamaria, Vera Ortiz, Heitmann, Stefan, Codagnone, Medeleine, Pflugfelder, Johanne, Krasowska, Dorota, Michalska-Jakubus, Malgorzata, Seidel, Matthia, Hasler, Paul, Kretschmar, Samuel, Kohm, Michaela, Bajocchi, Gianluigi, Salvador, Maria João, Da Silva, JoséAntonio Pereira, Stamenkovic, Bojana, Stankovic, Aleksandra, Selmi, Carlo Francesco, De Santis, Maria, Ceribelli, Angela, Garzanova, Ludmila, Koneva, Olga, Starovoytova, Maya, Herrick, Ariane, Puppo, Francesco, Negrini, Simone, Murdaca, Giuseppe, Engelhart, Merete, Szücs, Gabriela, Szamosi, Szilvia, De La Puente, Carlo, Grande, Cristina Sobrino, Villanueva, Maria Jesus Garcia, Midtve, Øyvindbw, Hoffmann-Vold, Anna-Mariabw, Launay, Davidbx, Sobanski, Vincentbx, Riccieri, Valeriaby, Vasile, Massimilianoby, Stefantoni, Katia, Ionescu, Ruxandra Maria, Opris, Daniela, Sha, Ami, Woods, Adrianne, Gheorghiu, Ana Maria, Bojinca, Mihai, Sunderkötter, Cord, Ehrchen, Jan, Ingegnoli, Francesca, Mouthon, Luc, Dunogue, Bertrand, Chaigne, Benjamin, Legendre, Paul, Cantatore, Francesco Paolo, Corrado, Ada, Ullman, Susanne, Iversen, Line, Von Mühlen, Carlos Alberto, Pozzi, Maria Rosa, Eyerich, Kilian, Lauffer, Felix, Wiland, Piotr, Szmyrka-Kaczmarek, Magdalena, Sokolik, Renata, Morgiel, Ewa, Madej, Marta, Vanthuyne, Marie, Frédéric, Houssiau, Alegre-Sancho, Juan Jose, Aringer, Martin, Herrmann, Kristine, Günther, Claudia, Westhovens, Rene, De Langhe, Ellen, Lenaerts, Jan, Anic, Branimir, Baresic, Marko, Mayer, Miroslav, Üprus, Maria, Otsa, Kati, Yavuz, Sule, Granel, Brigitte, Radominski, Sebastião Cezar, De Souza Müller, Carolina, Feijóazevedo, Valderílio, Mendoza, Fabian, Busquets, Joanna, Popa, Sergei, Agachi, Svetlana, Zenone, Thierry, Pileckyte, Margarita, Stebbings, Simon, Jordan, Sarah, Mathieu, Alessandro, Vacca, Alessandra, Sampaio-Barros, Percival D., Stamp, Lisa, Solanki, Kamal, Silva, Cherumi, Schollum, Joanne, Barns-Graham, Helen, Veale, Dougla, O'Rourke, Marie, Loyo, Esthela, Tineo, Carmen, Paulino, Glenny, Mohamed, Walid Ahmed Abdel Atty, Rosato, Edoardo, Gigante, Antonietta, Oksel, Fahrettin, Yargucu, Figen, Tanaseanu, Cristina-Mihaela, Popescu, Monica, Dumitrascu, Alina, Tiglea, Isabela, Foti, Rosario, Visalli, Elisa, Benenati, Alessia, Amato, Giorgio, Ancuta, Codrina, Villiger, Peter, Adler, Sabine, Fröhlich, Johanne, Kayser, Cristiane, Eduardo, Andrade Lui, Fathi, Nihal, Alii, Safa, Ahmed, Marrow, Hasaneen, Samar, El Hakeem, Eman, De La Peña Lefebvre, Paloma García, Martin, Jorge Juan Gonzalez, Sibilia, Jean, Chatelus, Emmanuel, Gottenberg, Jacques Eric, Chifflot, Hélène, Litinsky, Ira, Del Galdo, Francesco, Abignano, Giuseppina, Eng, Sookho, Seskute, Goda, Butrimiene, Irena, Rugiene, Rita, Karpec, Diana, Pascal, Melanie, Kerzberg, Eduardo, Bianchi, Washington, Bianchi, Breno Valdetaro, Bianchi, Dante Valdetaro, Barcellos, Yeda, Castellví, Ivan, Millan, Milena, Limonta, Massimiliano, Rimar, Doron, Rosner, Itzhak, Slobodin, Gleb, Couto, Maura, Spertini, Françoi, Ribi, Camillo, Buss, Guillaume, Marcoccia, Antonella, Bondanini, Francesco, Ciani, Aldo, Kahl, Sarah, Hsu, Vivien M., Martin, Thierry, Poindron, Vincent, Meghit, Kilifa, Moiseev, Sergey, Novikov, Pavel, Chung, Lori, Kolstad, Kathleen, Stark, Marianna, Schmeiser, Tim, Thiele, Astrid, Majewski, Dominik, Zdrojewski, Zbigniew, Zaneta, Smolenska, Wierzba, Karol, Martínez-Barrio, Julia, López-Longo, Francisco Javier, Bernardino, Vera, Moraes-Fontes, Maria Francisca, Rodrigues, Ana Catarina, Riemekasten, Gabriela, Sommerlatte, Sabine, Jendreck, Sebastian, Arnold, Sabrina, Levy, Yair, Rezus, Elena, Cardoneanu, Anca, Burlui, Alexandra Maria, Pamuk, Omer Nuri, Puttini, Piercarlo Sarzi, Talotta, Rossella, Bongiovanni, Sara, Poormoghim, Hadi, Andalib, Elham, Almasi, Simin, Kötter, Ina, Krusche, Matrin, Cuomo, Giovanna, Danzo, Fiammetta, Masini, Francesco, Gaches, Franci, Michaud, Martin, Cartos, Florian, Belloli, Laura, Casu, Cinzia, Sfikakis, Petro, Tektonidou, Maria, Furst, Daniel, Feldman, Gary R., Ramazan, Ana-Maria, Nurmambet, Emel, Miroto, Amalia, Suta, Cristina, Andronache, Iulia, Huizinga, Tom W. J., De Vries-Bouwstra, Jeska, and Walker, Ulrich A.

Subjects
Male, Vital capacity, Organ manifestations, systemic sclerosis, Type I, race difference, Systemic scleroderma, Gastroenterology, Scleroderma, immunology, 0302 clinical medicine, Diffusing capacity, middle aged, pulmonary hypertension, Medicine, Pharmacology (medical), 030212 general & internal medicine, organ manifestations, races, skin and connective tissue diseases, Lung, race, pathophysiology, African Continental Ancestry Group, ddc:616, integumentary system, disease course, Hazard ratio, Races, 10051 Rheumatology Clinic and Institute of Physical Medicine, Pulmonary, Middle Aged, Blacks, cohort analysis, Autoantibodie, 3. Good health, Asians, female, priority journal, DNA Topoisomerases, Type I, Black, centromere, Cohort, Hypertension, organ manifestation, Systemic sclerosis, Female, systemic sclerosi, Human, Adult, Asian Continental Ancestry Group, medicine.medical_specialty, Hypertension, Pulmonary, European Continental Ancestry Group, Black People, 610 Medicine & health, complication, Caucasian, White People, Article, lung, 03 medical and health sciences, Black person, Rheumatology, Asian People, forced vital capacity, Internal medicine, geographic distribution, Humans, controlled study, human, DNA topoisomerase, Aged, Autoantibodies, 030203 arthritis & rheumatology, Scleroderma, Systemic, Asian, business.industry, Whites, Systemic, Odds ratio, medicine.disease, Pulmonary hypertension, major clinical study, mortality, clinical feature, business, DNA Topoisomerases, autoantibody
Abstract

Objectives Racial factors play a significant role in SSc. We evaluated differences in SSc presentations between white patients (WP), Asian patients (AP) and black patients (BP) and analysed the effects of geographical locations. Methods SSc characteristics of patients from the EUSTAR cohort were cross-sectionally compared across racial groups using survival and multiple logistic regression analyses. Results The study included 9162 WP, 341 AP and 181 BP. AP developed the first non-RP feature faster than WP but slower than BP. AP were less frequently anti-centromere (ACA; odds ratio (OR) = 0.4, P < 0.001) and more frequently anti-topoisomerase-I autoantibodies (ATA) positive (OR = 1.2, P = 0.068), while BP were less likely to be ACA and ATA positive than were WP [OR(ACA) = 0.3, P < 0.001; OR(ATA) = 0.5, P = 0.020]. AP had less often (OR = 0.7, P = 0.06) and BP more often (OR = 2.7, P < 0.001) diffuse skin involvement than had WP. AP and BP were more likely to have pulmonary hypertension [OR(AP) = 2.6, P < 0.001; OR(BP) = 2.7, P = 0.03 vs WP] and a reduced forced vital capacity [OR(AP) = 2.5, P < 0.001; OR(BP) = 2.4, P < 0.004] than were WP. AP more often had an impaired diffusing capacity of the lung than had BP and WP [OR(AP vs BP) = 1.9, P = 0.038; OR(AP vs WP) = 2.4, P < 0.001]. After RP onset, AP and BP had a higher hazard to die than had WP [hazard ratio (HR) (AP) = 1.6, P = 0.011; HR(BP) = 2.1, P < 0.001]. Conclusion Compared with WP, and mostly independent of geographical location, AP have a faster and earlier disease onset with high prevalences of ATA, pulmonary hypertension and forced vital capacity impairment and higher mortality. BP had the fastest disease onset, a high prevalence of diffuse skin involvement and nominally the highest mortality.

Published
2020

33. Systemic sclerosis: State of the art on clinical practice guidelines

Author

Smith, V. (Vanessa), Scirè, C.A. (Carlo Alberto), Talarico, R. (Rosaria), Airo, P. (Paolo), Alexander, T. (Tobias), Allanore, Y. (Yannick), Bruni, C. (Cosimo), Codullo, V. (Veronica), Dalm, V.A.S.H. (Virgil), Vries-Bouwstra, J.K. (Jeska) de, Della Rossa, A. (Alessandra), Distler, O. (Oliver), Galetti, I. (Ilaria), Launay, D. (David), Lepri, G. (Gemma), Mathian, A. (Alexis), Mouthon, L. (Luc), Ruaro, B. (Barbara), Sulli, A. (Alberto), Tincani, A. (Angela), Vandecasteele, E. (Els), Vanhaecke, A. (Amber), Vanthuyne, M. (Marie), Hoogen, F.H.J. van den, Van Vollenhoven, R.F. (Ronald F.), Voskuyl, R.A. (Robert), Zanatta, E. (Elisabetta), Bombardieri, S. (Stefano), Burmester, G.R. (Gerd), Eurico, F.J. (Fonseca João), Frank, C. (Charissa), Hachulla, E. (Eric), Houssiau, F. (Frederic), Mueller-Ladner, U. (Ulf), Schneider, M. (Matthias), Van Laar, J.M. (Jacob M), Vieira, A. (Ana), Cutolo, M. (Maurizio), Mosca, M. (Marta), Matucci-Cerinic, M. (Marco), Smith, V. (Vanessa), Scirè, C.A. (Carlo Alberto), Talarico, R. (Rosaria), Airo, P. (Paolo), Alexander, T. (Tobias), Allanore, Y. (Yannick), Bruni, C. (Cosimo), Codullo, V. (Veronica), Dalm, V.A.S.H. (Virgil), Vries-Bouwstra, J.K. (Jeska) de, Della Rossa, A. (Alessandra), Distler, O. (Oliver), Galetti, I. (Ilaria), Launay, D. (David), Lepri, G. (Gemma), Mathian, A. (Alexis), Mouthon, L. (Luc), Ruaro, B. (Barbara), Sulli, A. (Alberto), Tincani, A. (Angela), Vandecasteele, E. (Els), Vanhaecke, A. (Amber), Vanthuyne, M. (Marie), Hoogen, F.H.J. van den, Van Vollenhoven, R.F. (Ronald F.), Voskuyl, R.A. (Robert), Zanatta, E. (Elisabetta), Bombardieri, S. (Stefano), Burmester, G.R. (Gerd), Eurico, F.J. (Fonseca João), Frank, C. (Charissa), Hachulla, E. (Eric), Houssiau, F. (Frederic), Mueller-Ladner, U. (Ulf), Schneider, M. (Matthias), Van Laar, J.M. (Jacob M), Vieira, A. (Ana), Cutolo, M. (Maurizio), Mosca, M. (Marta), and Matucci-Cerinic, M. (Marco)

Abstract

Systemic sclerosis (SSc) is an orphan disease characterised by autoimmunity, fibrosis of the skin and internal organs, and vasculopathy. SSc may be associated with high morbidity and mortality. In this narrative review we summarise the results of a systematic literature research, which was performed as part of the European Reference Network on Rare and Complex Connective Tissue and Musculoskeletal Diseases project, aimed at evaluating existing clinical practice guidelines or recommendations. Only in the domains 'Vascular & Ulcers' (ie, non-pharmacological approach to digital ulcer), 'PAH' (ie, screening and treatment), 'Treatment' and 'Juveniles' (ie, evaluation of juveniles with Raynaud's phenomenon) evidence-based and consensus-based guidelines could be included. Hence there is a preponderance of unmet needs in SSc referring to the diagnosis and (non-)pharmacological treatment of several SSc-specific complications. Patients with SSc experience significant uncertainty concerning SSc-related taxonomy, management (both pharmacological and nonpharmacological) and education. Day-to-day impact of the disease (loss of self-esteem, fatigue, sexual dysfunction, and occupational, nutritional and relational problems) is underestimated and needs evaluation.

Published
2018
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43. FRI0423 Effects of selexipag in cultured scleroderma skin fibroblasts

Author

Cutolo, M., primary, Ruaro, B., additional, Montagna, P., additional, Brizzolara, R., additional, Trombetta, A.C., additional, Scabini, S., additional, Tavilla, P.P., additional, Parodi, A., additional, Corallo, C., additional, Giordano, N., additional, Paolino, S., additional, Pizzorni, C., additional, Sulli, A., additional, Smith, V., additional, and Soldano, S., additional

Published
2018
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