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Background Mass indoor gatherings were banned in early 2020 to prevent the spread of SARS-CoV-2. We aimed to assess, under controlled conditions, whether infection rates among attendees at a large, indoor gathering event would be similar to those in non-attendees, given implementation of a comprehensive prevention strategy including antigen-screening within 3 days, medical mask wearing, and optimised ventilation. Methods The non-inferiority, prospective, open-label, randomised, controlled SPRING trial was done on attendees at a live indoor concert held in the Accor Arena on May 29, 2021 in Paris, France. Participants, aged 18–45 years, recruited via a dedicated website, had no comorbidities, COVID-19 symptoms, or recent case contact, and had had a negative rapid antigen diagnostic test within 3 days before the concert. Participants were randomly allocated in a 2:1 ratio to the experimental group (attendees) or to the control group (non-attendees). The allocation sequence was computer-generated by means of permuted blocks of sizes three, six, or nine, with no stratification. The primary outcome measure was the number of patients who were SARS-CoV-2-positive by RT-PCR test on self-collected saliva 7 days post-gathering in the per-protocol population (non-inferiority margin

IntroductionLung cancer screening (LCS) using low-dose CT has been demonstrated to reduce lung cancer-related mortality in large randomised controlled trials. Moving from trials to practice requires answering practical questions about the level of expertise of CT readers, the need for double reading as in trials and the potential role of artificial intelligence (AI). In addition, most LCS studies have predominantly included male participants with women being under-represented, even though the benefit of screening is greater for them. Thus, this study aims to compare the performance of a single CT reading by general radiologists trained in LCS using AI as a second reader to that of a double reading by expert thoracic radiologists, in a campaign for low-dose CT screening in high-risk women.Methods and analysisThis observational cohort study will recruit 2400 asymptomatic women aged between 50 and 74 years, current or former smokers with at least a 20 pack-year smoking history, in 4 different French district areas. Assistance with smoking cessation will be offered to current smokers. An initial low-dose CT scan will be performed, with subsequent follow-ups at 1 year and 2 years. The primary objective is to compare CT scan readings by a single LCS-trained, AI-assisted radiologist to that of an expert double reading. The secondary objectives are: to evaluate the performance of AI as a stand-alone reader; the adherence to screening of female participants; the influence on smoking cessation; the psychological consequences of screening; the detection of chronic obstructive pulmonary disease (COPD), coronary artery disease and osteoporosis on low-dose CT scans and the costs incurred by screening.Ethics and disseminationEthics approval was obtained from the Comité de Protection des Personnes Sud-Est 1 (ethics approval number: 2021-A02265-36 with an amendment on 15 July 2022). Trial results will be disseminated at conferences, through relevant patient groups and published in peer-reviewed journals.Trial registration numberNCT05195385.

Contains fulltext : 289893.pdf (Publisher’s version ) (Open Access) Differences in disability perception between patients and care providers may impact outcomes. We aimed to explore differences in disability perception between patients and care providers in systemic sclerosis (SSc). We conducted a cross-sectional internet-based mirror survey. SSc patients participating in the online SPIN Cohort and care providers affiliated with 15 scientific societies were surveyed using the Cochin Scleroderma International Classification of Functioning, Disability and Health (ICF)-65 questionnaire, including 65 items (from 0 to 10), representing 9 domains of disability. Mean differences between patients and care providers were calculated. Care providers' characteristics associated with a mean difference >= 2 of 10 points were assessed in multivariate analysis. Answers were analyzed for 109 patients and 105 care providers. The mean age of patients was 55.9 (14.7) years and the disease duration was 10.1 (7.5) years. For all domains of the ICF-65, care providers' rates were higher than those of patients. The mean difference was 2.4 (1.0) of 10 points. Care providers' characteristics associated with this difference were organ-based specialty (OR = 7.0 [2.3-21.2]), younger age (OR = 2.7 [1.0-7.1]) and following patients with disease duration >= 5 years (OR = 3.0 [1.1-8.7]). We found systematic differences in disability perception between patients and care providers in SSc. 10 p.

ObjectiveDue to an annual progression rate of Barrett’s oesophagus (BO) with low-grade dysplasia (LGD) between 9% and 13% per year endoscopic ablation therapy is preferred to surveillance. Since this recommendation is based on only one randomised trial, we aimed at checking these results by another multicentre randomised trial with a similar design.DesignA prospective randomised study was performed in 14 centres comparing radiofrequency ablation (RFA) (maximum of 4 sessions) to annual endoscopic surveillance, including patients with a confirmed diagnosis of BO with LGD. Primary outcome was the prevalence of LGD at 3 years. Secondary outcomes were the prevalence of LGD at 1 year, the complete eradication of intestinal metaplasia (CE-IM) at 3 years, the rate of neoplastic progression at 3 years and the treatment-related morbidity.Results125 patients were initially included, of whom 82 with confirmed LGD (76 men, mean age 62.3 years) were finally randomised, 40 patients in the RFA and 42 in the surveillance group. At 3 years, CE-IM rates were 35% vs 0% in the RFA and surveillance groups, respectively (pConclusionRFA modestly reduced the prevalence of LGD as well as progression risk at 3 years. The risk-benefit balance of endoscopic ablation therapy should therefore be carefully weighted against surveillance in patients with BO with confirmed LGD.Trial registration numberNCT01360541.

People living with HIV (PLWH) are at risk of noninfectious comorbidities. It is important to individualize those at higher risk. In a single-center cohort of PLWH, we performed a cross-sectional analysis of comorbidities, diagnosed according to standard procedures. The primary endpoint was the prevalence of subclinical carotid/coronary atherosclerosis. Secondary endpoints were its association with selected inflammatory/immune activation biomarkers and with other comorbidities. Associations were examined by using Chi-square or Fisher's exact test for categorical variables and Student or Wilcoxon tests for quantitative variables, and a stepwise multivariate logistical model was performed for further exploration. Among 790 participants [median age: 49.8 years (interquartile range, IQR: 44.5-55.6), 77.1% males, median CD4: 536/mm

Background Patient-reported outcome measures (PROMs) aimed at assessing people with systemic sclerosis (SSc) have rarely involved the target population in the item- and domain-generation stage of the instrument construction. Objectives To develop a new PROM assessing activities and participation in people with SSc. Methods A provisional International Classification of Functioning, Disability and Health (ICF)-based 65-item questionnaire previously developed from interviews of people with SSc was sent by email to all patients followed in the internal medicine department of Cochin hospital (n = 184) and enrolled in the Scleroderma Patient-centered Intervention Network Cohort. Items were reduced according to their metric properties. Dimensional structure of the questionnaire was assessed by principal component analysis, convergent and divergent validities by Spearman's rank correlation coefficient, internal consistency by Cronbach's α, and reliability by a test-retest method using the intraclass correlation coefficient (ICC) and Bland-Altman analysis. Results Overall, 113 of 184 patients (61·4%) completed the provisional questionnaire. The item-reduction process resulted in a 17-item questionnaire, the Cochin 17-item Scleroderma Functional scale (CSF-17). Principal component analysis extracted two dimensions: 10 items related to mobility (CSF-17 section A) and seven items related to general tasks (CSF-17 section B). We observed convergent validity of the CSF-17 total score with global activity limitation, pain, depression and aesthetic burden, and divergent validity with anxiety. Cronbach's α was 0·94 for section A and 0·95 for section B. ICC (n = 25 patients) was 0·92 for the CSF-17 total score. Bland-Altman analysis did not reveal a systematic trend for the test-retest. Conclusions The CSF-17 is a new PROM assessing activities and participation specifically in people with SSc. Its content and construct validities are very high. What is already known about this topic? In the earliest stages of construction patient-reported outcomes (PROMs) for people with systemic sclerosis (SSc) rarely involve the target population. Instruments able to capture the specific needs of people with SSc in terms of activities and participation are lacking. What does this study add? The Cochin 17-item Scleroderma Functional Scale (CSF-17) is a new PROM assessing global activities and participation specifically in people with SSc. Patients' perspectives were prioritized at all stages of construction. What are the clinical implications of this work? The CSF-17 could be used in clinical practice and research to assess the efficacy of complex multidisciplinary interventions targeting activity limitations and participation restriction in people with SSc. Linked Comment: Clark and Denton. Br J Dermatol 2020; 183:610.

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159 Background: Abiraterone acetate (ABI) improves survival in mCRPC. In a previous observational study, worse PSA response and shorter progression-free survival (PFS) were associated with mean ABI plasma through concentration (ABI Cmin) < 8.5 ng/mL within the first three months (ms) of treatment (Carton E et al. Eur J Cancer 2017). The OPTIMABI study, a multicenter non-randomized study (NCT 03458247), aimed to investigate whether a dose escalation of ABI improve PFS in underexposed progressive mCRPC pts. Methods: In step 1, pts with docetaxel-naive progressive mCRPC received ABI at the standard dose of 1000 mg/d + Prednisone 10 mg/d. Mean ABI Cmin was calculated from three monthly dosages. Pts experiencing progression within the first 6 ms and for whom mean ABI Cmin was < 8.5 ng/mL were included in step 2 and received 1000 mg twice daily (2000 mg/d) of ABI. ABI Cmin was assessed every month. The primary endpoint was the non-progression rate at 12 weeks in step 2. Identification of risk factors of 6 ms-PFS was done using Cox regression analysis. Results: From 06/2018 to 09/2021, 81 of 93 pts (median age: 72 yrs) included in step 1 were evaluable for statistical analysis. The median ABI Cmin was 10.0 (IQR: 4.5-15. 0) ng/mL over the first three ms. Inter-individual variability was 59%. The 6 ms-PFS was 68% (IC 95%: 56% - 80%) with no statistical difference between pts with mean ABI Cmin ≥ or < 8.5 ng/mL (log-rank test, p= 0.24). In multivariate analysis, liver metastasis (Hazard ratio, HR = 5.95, CI95% = 1.22–28.92; p=0.027) and creatinine clearance < 60 mL/min (HR = 3.51, CI95% = 1.12–10.99; p=0.031) were independently associated with 6 ms-PFS. Among 21 pts (25.9%), with mean ABI Cmin < 8.5 ng/mL, 8 pts (38.1%) experienced tumor progression within the first 6 ms; four of them could be included in step 2. None of them fulfilled the primary endpoint despite a significant increase in plasma ABI Cmin (p

Background Spinal cord injury often results in erectile dysfunction and an ejaculation along with impaired semen parameters. Fertility is a major concern in spinal cord injury adult males and some fear that the delay post-spinal cord injury may negatively affect sperm quality. Objectives We aimed to (i) assess semen parameters over time in SCI patients according to age at spinal cord injury, time post-spinal cord injury, and the spinal cord injury level and completeness and (ii) measure markers in semen for inflammation and marker of oxidative stress to investigate their impact on sperm parameters. Materials and methods The study is a prospective, longitudinal, pilot study over 18 months. Thirty-five men with spinal cord injury from 18 to 60 years of age were enrolled. Their mean age was 29.4 ± 6.4 years. Semen retrieval was scheduled every 6 months, allowing analysis of four ejaculates, in association with measurement of granulocyte and seminal plasma elastase concentrations to assess markers in semen for inflammation and spermatozoa DNA fragmentation to assess oxidative stress. Results Based on reference limits, a normal total sperm number, decreased motility and vitality of the spermatozoa, and increased morphological abnormalities were found. Mean round cell and granulocyte concentrations were elevated in the semen. Markers in semen for inflammation and marker of oxidative stress were elevated in several semen samples, compared to reference limits. However, neither the presence of markers in semen for inflammation or oxidative stress, the completeness or the level of the spinal cord lesion, the age or the time post-spinal cord injury had a negative impact on the semen quality over time. Discussion There was no significant decline in semen quality in spinal cord injury patients over time within the limitations of this pilot study. Moreover, a chronic genital inflammatory status was not associated with impairment of semen quality. Conclusion The present findings are reassuring for men with spinal cord injury and could guide the management of their reproductive ability. According to these preliminary data, not all spinal cord injury patients who are able to ejaculate require systematic freezing of their spermatozoa.

Background There are conflicting data regarding the diagnostic performance of chest CT for COVID-19 pneumonia. Disease extent at CT has been reported to influence prognosis. Purpose To create a large publicly available data set and assess the diagnostic and prognostic value of CT in COVID-19 pneumonia. Materials and Methods This multicenter, observational, retrospective cohort study involved 20 French university hospitals. Eligible patients presented at the emergency departments of the hospitals involved between March 1 and April 30th, 2020, and underwent both thoracic CT and reverse transcription-polymerase chain reaction (RT-PCR) testing for suspected COVID-19 pneumonia. CT images were read blinded to initial reports, RT-PCR, demographic characteristics, clinical symptoms, and outcome. Readers classified CT scans as either positive or negative for COVID-19 based on criteria published by the French Society of Radiology. Multivariable logistic regression was used to develop a model predicting severe outcome (intubation or death) at 1-month follow-up in patients positive for both RT-PCR and CT, using clinical and radiologic features. Results Among 10 930 patients screened for eligibility, 10 735 (median age, 65 years; interquartile range, 51-77 years; 6147 men) were included and 6448 (60%) had a positive RT-PCR result. With RT-PCR as reference, the sensitivity and specificity of CT were 80.2% (95% CI: 79.3, 81.2) and 79.7% (95% CI: 78.5, 80.9), respectively, with strong agreement between junior and senior radiologists (Gwet AC1 coefficient, 0.79). Of all the variables analyzed, the extent of pneumonia at CT (odds ratio, 3.25; 95% CI: 2.71, 3.89) was the best predictor of severe outcome at 1 month. A score based solely on clinical variables predicted a severe outcome with an area under the curve of 0.64 (95% CI: 0.62, 0.66), improving to 0.69 (95% CI: 0.6, 0.71) when it also included the extent of pneumonia and coronary calcium score at CT. Conclusion Using predefined criteria, CT reading is not influenced by reader's experience and helps predict the outcome at 1 month. ClinicalTrials.gov identifier: NCT04355507 Published under a CC BY 4.0 license.

Objective To assess the feasibility, safety and precision of organ-based tracking (OBT)-fusion targeted focal microwave ablation (FMA), in patients with low to intermediate risk prostate cancer. Patients and method Ten patients with a visible index tumor of Gleason score ≤3+4, largest diameter Results Median [IQR] age was 64.5 [61–72] years and baseline PSA was 5 [4.3–8.1] ng/mL. Seven (70%) and 3 (30%) patients had a low and intermediate risk cancer, respectively. Median largest tumor axis was of 11 [9.0–15.0] mm. Median duration of procedure was of 82 [44–170] min. No patient reported any pain or rectal bleeding, and all 10 patients were discharged the next day. Seven days after ablation, total necrosis of the index tumor on MRI was obtained in eight (80% [95%CI 55%-100%]) patients. One patient was treated with radical prostatectomy. Re-biopsy at 6 months in the other 9 did not show evidence of cancer in 4 patients. IPSS, IIEF-5 and MSHQ-EjD-SF were not statistically different between baseline and 6 months follow up. Conclusions OBT-fusion targeted FMA was feasible, precise, and safe in patients with low to intermediate risk localized prostate cancer.

Synthetized by the liver and metabolized by the gut microbiota, BA are involved in metabolic liver diseases that are associated with cardiovascular disorders. Animal models of atheroma documented a powerful anti-atherosclerotic effect of bile acids (BA). This prospective study examined whether variations in circulating BA are predictive of coronary artery disease (CAD) in human. Consecutive patients undergoing coronary angiography were enrolled. Circulating and fecal BA were measured by high pressure liquid chromatography and tandem mass spectrometry. Of 406 screened patients, 80 were prospectively included and divided in two groups with (n = 45) and without (n = 35) CAD. The mean serum concentration of total BA was twice lower in patients with, versus without CAD (P = 0.005). Adjusted for gender and age, this decrease was an independent predictor of CAD. In a subgroup of 17 patients, statin therapy doubled the serum BA concentration. Decreased serum concentrations of BA were predictors of CAD in humans. A subgroup analysis showed a possible correction by statins. With respect to the anti-atherosclerotic effect of BA in animal models, and their role in human lipid metabolism, this study describe a new metabolic disturbance associated to CAD in human.

Background The patient acceptable symptom state (PASS) is a treatment-response criterion developed to determine the clinical relevance of a treatment effect. Its estimates for some patient-reported outcomes (PROs) in non-specific chronic low back pain (cLBP) are lacking and the stability of PRO estimates between independent cLBP populations is unknown. We hypothesized that these PRO estimates will be stable. Objectives To estimate and compare the PASS for PROs between 2 independent cLBP populations. Methods We conducted a secondary analysis of a randomized controlled trial (PREDID) and a cohort of outpatients with non-specific cLBP. Using an anchoring question, participants who self-rated their health as “excellent”, “very good” or “good” at 1 month were considered to have an acceptable symptom state. PASS estimates for 5 PROs were calculated by using the 75th percentile method. Estimates were compared between the 2 populations with bootstrap resampling. Results A total of 256 participants with non-specific cLBP were included: 135 patients with cLBP and active discopathy from the PREDID trial and 121 outpatients with cLBP without active discopathy followed up in an independent cohort. Overall, 137/256 (54%) participants had an acceptable symptom state at 1 month. PASS estimates were 47.5 (95% confidence interval [CI] 40.0 to 50.0)/100 for lumbar pain (0, no pain and 100, maximal pain), 30.5 (30.0 to 40.0)/100 for radicular pain, 39.3 (33.6 to 45.3)/100 for Quebec Back Pain Disability score (0, no disability and 100, maximal disability), 10.0 (9.2 to 10.0)/21 for the Hospital Anxiety Depression anxiety subscale (0, no anxiety, and 21, maximal anxiety) and 6.7 (6.0 to 8.0)/21 for the depression subscale (0, no depression, and 21, maximal depression). PASS estimates did not differ between the 2 populations. Conclusions Our study provides PASS estimates for 5 PROs commonly used in cLBP. Our estimates were stable between 2 independent populations of people with cLBP. The stability of our PASS estimates suggests that they are relevant for interpreting PRO values in clinical trials and practice. ClinicalTrials.gov no. (PREDID trial) NCT00804531 .

Importance Hypertensive events during cataract surgery may induce complications. Information and communication technologies applied to health are popular, but clinical evidence of its usefulness is rare and limited to treating anxiety without specific analysis. Recent publications have described the potential effects of personalized music choices as mobile-based intervention on pain but not on anxiety. Objective To demonstrate the effects of a web app-based music intervention on the incidence of hypertension in participants during cataract surgery performed under local anesthesia. Design, Setting, and Participants This randomized, single masked, controlled clinical trial was composed of 2 arms and took place at a single center at Cochin Hospital in Paris, France. A total of 313 participants were screened between February 2017 and July 2018. Among these, 311 participants undergoing a phacoemulsification procedure for elective surgery for their first eye cataract under local anesthesia were enrolled. Analysis was intention to treat and began in September 2018 and ended November 2018. Interventions Participants requiring cataract surgery were assigned randomly to either the experimental arm (web app-based music listening intervention delivered via headphones) or control arm (noise-canceling headphones without music) for 20 minutes before surgery. Main Outcomes and Measures The occurrence of at least 1 hypertensive event during surgery. Results A total of 310 participants were randomized in the study (155 to each arm) and 309 were analyzed (1 participant in the music arm had already undergone cataract surgery to the other eye). The mean (SD) age of the participants was 68.9 (10.8) years, and there were 176 female individuals (57%). On the primary end point, the incidence of hypertension was significantly lower in the music arm (21 [13.6%]) than in the control arm (82 [52.9%]), with a difference between the 2 arms of 39.3% (95% CI, 21.4%-48.9%; P

BACKGROUND: Diarrhea is a frequent complication of solid organ transplantation. Cryptosporidiosis is classically reported in patients with acquired immunodeficiency syndrome and emerged as a cause of persistent diarrhea in solid organ transplant patients. METHODS: Through the ANOFEL Cryptosporidium National Network and the French Transplantation Society, we collected all cryptosporidiosis cases identified in solid organ transplanted patients between 2006 and 2010 in France. RESULTS: We reported 47 solid organ transplant recipients (41 kidneys) with cryptosporidiosis, mostly men (68%), with a median age of 52 (6-70) years old. Five patients had additional immunodepression favoring cryptosporidiosis (CD40 ligand deficiency [n = 1], human immunodeficiency virus infection [n = 4]). Cryptosporidiosis occurred at a median time of 3.4 (0-19.8) years posttransplant. Exposure to environmental risk factors was found before infection onset in 18 patients. Time between first symptoms and diagnosis was 10 (2-110) days. Four patients had associated extraintestinal location (biliary tract [n = 3] and lung [n = 1]). Thirty-five patients received specific therapy against cryptosporidiosis ie nitozoxanide, 25 in monotherapy, and 10 in association with azithromycin, 13 in association with immunosuppression (IS) reduction. Four patients were cured with IS treatment tapering only. The others patients had neither IS reduction nor specific therapy against cryptosporidiosis. Cryptosporidiosis was complicated by renal failure in 15 patients. Symptoms resolved after a median of 10 days of treatment. Six patients relapsed and 3 died, 1 with evolutive infection. CONCLUSIONS: Cryptosporidiosis is a late posttransplant infection that disseminated to biliar duct or lung in 9% of patients. When limited to digestive tract, infection may resolve without IS reduction.

Introduction La maladie de Behcet (MB) est caracterisee par des ulcerations buccales et genitales, des lesions cutanees, une inflammation intraoculaire, et de nombreuses autres atteintes viscerales. Une association forte entre la MB et des polymorphismes du gene codant pour l’IL-23R/IL-12RB2 a ete montree, et l’implication des lymphocytes T helper (Th) 1 et Th17 a ete suggeree dans plusieurs etudes. L’ustekinumab, anticorps monoclonal anti-IL-12/IL-23, pourrait ainsi etre une option therapeutique interessante au cours de la MB. L’objectif de cette etude etait d’evaluer l’efficacite et la tolerance de l’ustekinumab au cours de la MB. Patients et methodes Nous avons realise une etude prospective de phase 2, ouverte, evaluant l’efficacite et la tolerance de l’ustekinumab chez les patients atteints de MB ( NCT02648581 ). Les criteres d’inclusion etaient : un diagnostic de MB selon les criteres de classification internationaux, et la presence d’ulceres buccaux ou genitaux recurrents definis comme ≥ 2 episodes dans les 3 derniers mois et resistant au moins a la colchicine a une dose ≥1 mg/j. Des injections sous-cutanees d’ustekinumab a la dose de 90 mg etaient realisees aux semaines (S) 0, 4 et 16, et des injections supplementaires etaient realisees a S28 et S40 chez les patients repondeurs a S24. Le critere de jugement principal etait la modification du nombre d’ulceres buccaux a S24 par rapport a la visite d’inclusion. Une reponse complete etait definie comme l’absence de toute ulceration buccale ou genitale, et une reponse partielle comme une diminution de ≥ 50 % du nombre d’ulceres buccaux. Le critere de jugement principal a ete evalue par un comite d’adjudication independant. Les criteres de jugements secondaires etaient l’evolution du nombre et de la douleur des aphtes, des scores d’activite de la MB (BSAS allant de 0 a 100, BDCAF allant de 0 a12) et des scores de qualite de vie (BDQol allant de 0 a 30, SF36, et RAPID3 allant de 0 a 30), et la tolerance du traitement. Un nombre de 15 patients etait necessaire afin de pouvoir demontrer une efficacite du traitement par ustekinumab chez > 50 % des patients. Resultats Quinze patients ont ete inclus et ont recu au moins une injection d’ustekinumab. L’âge moyen a l’inclusion etait de 35,5 ± 9,5 ans. A l’inclusion, le nombre moyen d’aphtes buccaux etait de 2,1 ± 1,3 et seuls 2 patients presentaient des aphtes genitaux. A S24, une reponse clinique etait observee chez 11 patients (73,3 % [60,0–95,7]), dont 9 (60 % [35,2–84,8]) reponses completes et 2 (13,3 % [0–30,5]) reponses partielles. Parmi les quatre patients en echec a S24, trois avaient interrompu l’ustekinumab pour une rechute ou une absence d’efficacite, avec un relais par infliximab dans 2 cas et cyclophosphamide dans un. Les causes d’echec etaient : la survenue d’abces aseptiques avec atteinte vasculaire arterielle grave necessitant une prise en charge chirurgicale urgente, une atteinte cutanee avec aphtose bipolaire et erytheme noueux, et enfin une orchite avec arthralgies et aphtose buccale. Sous traitement par ustekinumab, le nombre moyen d’ulceres buccaux diminuait de 2,1 ± 1,3 a 0,3 ± 0,5 a S24 (p = 0,0002). Il y avait egalement une diminution des scores d’activite et une amelioration des scores de qualite de vie a S24 : baisse du BSAS de 38 ± 13 a 14 ± 12 (p = 0,0006), du BDCAF de 3,7 ± 1,5 a 1,6 ± 1,5 (p = 0,001), du BD-QoL de 13,2 ± 7,4 a 8,2 ± 8,6 (p = 0,05) et du RAPID3 de 13,4 ± 5,6 a 4,7 ± 3,2 (p = 0,002). A S52, la reponse etait maintenue chez 10/11 patients (90,9 % [73,9-100]), dont 9 avec une reponse complete, avec un nombre moyen d’aphtes buccaux de 0,3 ± 0,8 (p = 0,0002 comparativement a la visite d’inclusion). Un seul patient a presente un effet indesirable grave, sous la forme d’un syndrome grippal justifiant une hospitalisation, avec une recuperation sans sequelle. Il n’y a eu aucun arret du traitement du fait d’un effet indesirable. Conclusion Cette etude montre une efficacite de l’ustekinumab pour le traitement de l’aphtose buccale resistante au cours de la MB, avec un excellent profil de tolerance.

Background Clinical relevance of commonly used patient-reported outcomes (PROs) is unclear in people with non-specific chronic low back pain (cLBP) (1). Objectives To estimate and compare patient acceptable symptom state (PASS) at 1 month post-intervention for 4 PROs between 2 populations of patients with non-specific cLBP and to determine which baseline variables contribute to having an acceptable symptom state at 1 month. Methods Overall, we included 256 patients: 135 patients with cLBP and active discopathy participated in a randomized controlled trial assessing the efficacy on pain at 1 month of a single glucocorticoid intradiscal injection compared to contrast alone (2), and 121 patients with cLBP and without active discopathy participated in a randomized controlled trial assessing the efficacy on pain at 4 months of 12 sessions of immersive virtual reality (VR) compared to usual care (3). Using an anchor-based method, PASS estimates for PROs were obtained using the 75th percentile method (4). Logistic regression was used to determine baseline variables contributing to achieving PASS at 1 month. Results At 1 month, 137/256 (53.52%) participants self-rated their health as acceptable. In the whole population, PASS (95% IC) were 47.50 (40.00 to 50.00) for the lumbar-pain VAS, 30.50 (30.00 to 40.00) for the radicular-pain VAS, 39.27 (33.60 to 45.26) for the QUEBEC score, 9.95 (9.16 to 10.00) for the HAD anxiety subscale and 6.70 (6.00 to 8.00) for the HAD depression subscale. The PASS estimates did not differ between the 2 populations of cLBP patients for any of the PRO. The only baseline variable contributing to having an acceptable symptom state at 1 month was symptom intensity. Conclusion PASS estimates at 1 month did not vary across 2 independent samples of people with cLBP and 2 distinct nociceptive sources of cLBP. The main contributor of the PASS was symptom intensity at baseline. Our findings can be useful in interpreting the clinical relevance of PROs values. References [1] Tubach F, Ravaud P, Baron G, Falissard B, Logeart I, Bellamy N, et al. Evaluation of clinically relevant states in patient reported outcomes in knee and hip osteoarthritis: the patient acceptable symptom state. Ann Rheum Dis. 2005;64(1):34-7. [2] Nguyen C, Boutron I, Baron G, Sanchez K, Palazzo C, Benchimol R, et al. Intradiscal Glucocorticoid Injection for Patients With Chronic Low Back Pain Associated With Active Discopathy: A Randomized Trial. Ann Intern Med. 2017;166(8):547-56. [3] Nguyen C, Boutron I, Perrodeau E, Sanchez K, Borderie D, Palazzo C et al. 3D immersive virtual reality in patients with chronic low back pain: a randomized controlled trial. PlosOne (under review). 2017. Disclosure of Interests None declared

Background: Few patient-reported outcomes measures (PROs) have been specifically designed to assess the functioning of patients with systemic sclerosis (SSc). In addition, the development of currently available instruments did not fully follow current guidelines (1) Objectives: To develop and validate a PRO assessing activity limitation and participation restriction of patients with SSc. Methods: A provisional International Classification of Functioning, Disability and Health (ICF)-based 65-item questionnaire previously developed from interviews of SSc patients was submitted online to French patients (n=184) of the Scleroderma Patient-centered Intervention Network e-cohort (2). Items were reduced according to their metric properties, dimensional structure of the questionnaire was assessed by principal component analysis, convergent and divergent validities using the Spearman correlation coefficient (ρ), internal consistency by the Cronbach α coefficient and reliability by a test-retest method using intraclass correlation coefficient (ICC) and Bland and Altman analysis. Results: Overall, 113/184 (61.4%) patients completed the provisional questionnaire. The item-reduction process resulted in a 17-item questionnaire, the Cochin 17-item Scleroderma Functional scale (CSF-17). Principal component analysis extracted 2 dimensions: 10 items related to mobility (CSF-17 section A) and 7 items related to general tasks (CSF-17 section B). We observed convergent validity with global activity limitation, pain, depression and aesthetic burden and divergent validity with anxiety. The Cronbach α coefficient was 0.94 for section A and 0.95 for section B. ICC (n=25 patients) was 0.92 for CSF-17 total score. Bland and Altman analysis did not reveal a systematic trend for the test-retest. Conclusion: The CSF-17 is a self-administered questionnaire assessing activity limitation and participation restriction of patients SSc with good content and construct validities References [1] Health USDo, Human Services FDA, Research, et al. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79. [2] Kwakkenbos L, Jewett LR, Baron M, Bartlett SJ, Furst D, Gottesman K, et al. The Scleroderma Patient-centered Intervention Network (SPIN) Cohort: protocol for a cohort multiple randomised controlled trial (cmRCT) design to support trials of psychosocial and rehabilitation interventions in a rare disease context. BMJ Open. 2013;3(8). Acknowledgement: Dr Camille Daste received a Master 2 grant from the French Society of Rheumatology Disclosure of Interests: None declared

BACKGROUND Chronic exchange transfusion is effective for primary and secondary prevention of stroke in children with sickle cell anemia (SCA). Erythrocytapheresis is recognized to be the most efficient approach; however, it is not widely implemented and is not suitable for all patients. The aim of our study was to compare automated exchange transfusion (AET) with our manual method of exchange transfusion and, in particular, to evaluate the efficacy, safety, and cost of our manual method. STUDY DESIGN AND METHODS Thirty-nine SCA children with stroke and/or abnormal findings on transcranial Doppler were included in the study. We retrospectively analyzed 1353 exchange sessions, including 333 sessions of AET and 1020 sessions of manual exchange transfusion (MET). RESULTS Both methods were well tolerated. The median decrease in hemoglobin (Hb)S per session was 21.5% with AET and 18.8% with our manual method (p

Introduction La sclerodermie systemique (SSc) a un impact important sur les activites et la participation des patients. De nombreux echelles d’evaluation sont utilises pour evaluer ces dimensions du point de vue des patients. Dans plusieurs pathologies rhumatologiques comme dans la SSc, la litterature met en evidence des situations de discordance entre soignant et patient dans l’evaluation de ces dimensions conduisant a de multiples consequences, notamment pour le suivi et l’adhesion aux traitements. L’objectif de notre etude etait d’evaluer les differences de perception pour les limitations d’activite et restrictions de participation entre les patients atteints de la SSc et les professionnels de sante. Patients et methodes Nous avons mene une etude transversale multicentrique en miroir. Dans une etude precedente, 109 patients avaient complete en ligne un questionnaire de 65 items, incluant 9 domaines, developpe a partir du point de vue des patients dans le cadre conceptuel de la Classification International du Fonctionnement, du Handicap et de la Sante (CIF). Le meme questionnaire a ete soumis en ligne a des professionnels de sante francais, recrutes par l’intermediaire de societes scientifiques et incluant medecins, dentistes, infirmiers, kinesitherapeutes et ergotherapeutes. La difference moyenne (ecart-type) des reponses entre patients et soignants a ete calculee pour chacun des 9 domaines du questionnaire. Un soignant etait considere comme concordant si la difference entre ses reponses et celles des patients etait ≤ 2 points pour la totalite du questionnaire. Les caracteristiques des soignants contribuant a cette difference ont ete evaluees en analyse multivariee. Resultats Au total, les patients atteints de SSc (n = 109) avaient 55,9 (14,7) ans, 41/109 (37,6 %) avaient une forme diffuse et la duree moyenne de la maladie etait de 10,1 (7,5) ans. Parmi les professionnels de sante (n = 105), 53/105 (50,5 %) etaient des femmes, 82/105 (78,1 %) etaient des medecins diplomes, 57/105 (54,3 %) etaient specialistes en medecine interne et 41/105 (39,0 %) etaient consideres comme concordants. Les soignants ont surevalue les limitations d’activite et restrictions de participation par rapport aux patients dans les 9 domaines du questionnaire. La difference la plus importante etait de 2,9 (1,5) points pour « l’entretien personnel » et la plus faible de 2,1 (1,0) points pour « l’acquisition et l’application des connaissances ». Les caracteristiques contribuant a cette difference etaient l’âge Discussion Notre etude a mis en evidence une surestimation systematique des limitations d’activite et restrictions de participation par les soignants. Cette difference de perception pourrait etre en partie expliquee par : 1/une representation cognitive de la SSc par les soignants en decalage avec le vecu reel des patients, et/ou 2/la grande resilience des patients sclerodermiques face a leur situation de handicap. Conclusion Les resultats de notre etude soulignent l’importance de prioriser le point de vue des patients dans l’evaluation du handicap au cours de la SSc. Enfin, ils suggerent egalement l’interet de sensibiliser les professionnels de sante aux differentes dimensions du handicap au cours de la SSc afin de mieux les prendre en compte dans le but d’ameliorer la prise en charge de ces patients.

Objectifs La tomographie par coherence optique (OCT) plein champ est une technique d’imagerie du tissu frais ex vivo. Notre objectif etait d’en evaluer les performances diagnostiques sur biopsies prostatiques pour la detection du cancer de la prostate. Methodes Cette etude diagnostique a ete proposee de janvier 2016 a janvier 2017 aux patients ayant une premiere serie de biopsies prostatiques dans notre centre. Une image OCT plein champ etait acquise pour chaque carotte. Toutes les carottes etaient analysees de maniere independante. Deux urologues et deux internes d’urologie ont analyse les images OCT plein champ en aveugle, pour determiner la presence de cancer. Un programme d’intelligence artificielle (IA) a ete entraine de maniere non supervisee sur les 506 premieres images, puis teste sur les 490 images restantes. La sensibilite (Se), specificite (Sp) et valeur predictives ont ete calculees pour chaque lecteur et pour l’IA. Resultats Quatre-vingt-huit patients ont ete inclus, et 996 carottes analysees. Pour la detection de cancer, les lecteurs obtenaient 0,37 Fig. 1 ). Conclusion Nos resultats suggerent que l’OCT plein champ a une VPN elevee pour eliminer la presence de cancer en temps reel sur carotte biopsique. Cette VPN augmente pour les cancers significatifs. Notre programme d’IA necessite un apprentissage sur un plus grand nombre d’image.

Background; Hypertensive events during cataract surgery may induce complications. We aimed to determine the effect of a musical intervention on anxiety-related hypertensive events during phacoemulsification performed under local anesthesia. Methods; Patients requiring cataract surgery were randomly assigned to either the experimental group (music delivered by earphones) or control group (earphones with no music) for 20 min before surgery. The primary outcome was the change in occurrence of at least one anxiety-related hypertensive event during surgery. Results; A total of 310 patients were randomized in the study (155 in the music group, 155 in the control group). The mean visual measure of anxiety was significantly (p = 0.005) lower in the music group (1.4 ± 2.0) than headphone group (3.1 ± 2.4). The incidence of hypertension related to anxiety was significantly lower (p Conclusions; A 20-minutes musical intervention before cataract surgery reduces patient's anxiety levels and anxiety-related hypertension during procedure and decrease the needs for sedative drugs.

We report an overall 24%(95% CI; 17–32%) cumulative incidence of pulmonary embolism in patients with COVID-19 pneumonia, 50% (30–70%) in ICU and 18% (12–27%) in other patients

Introduction Recent studies have suggested that intra-articular injection of botulinum toxin A (BTA) may have analgesic effects in degenerative joint diseases. We aim to assess the efficacy of intra-articular injection of BTA associated with splinting in patients with trapeziometacarpal osteoarthritis (TMC OA). Methods and analysis We will conduct a randomised double-blind controlled trial. Overall, 60 individuals with TMC OA fulfilling adapted 1990 American College of Rheumatology criteria for hand OA will be recruited in one tertiary care centre in France and randomised to receive splinting + a single ultrasound-guided injection in the TMC joint of 50 Allergan Units of BTA resuspended in 1 mL saline or splinting +1 mL saline. Randomisation will be centralised. The allocation ratio will be 1:1. The primary outcome will be the mean change from baseline in base-of-thumb pain on a self-administered 11-point Numeric Rating Scale in 10-point increments at 3 months after injection. Secondary outcomes will be the mean change in base-of-thumb pain at 1 and 6 months, mean change in hand-specific activity limitations assessed by the self-administered Cochin Hand Function Scale, proportion of responders assessed by the Osteoarthritis Research Society International -Outcome Measures in Rheumatology (OMERACT) criteria and consumption of analgesics and non-steroidal anti-inflammatory drugs assessed by a self-administered 4-class scale at 3 and 6 months. Cointerventions will be allowed in both groups and will be self-reported. Adverse events will be recorded at 3 and 6 months. Participants, care providers and statisticians will be blinded to the allocated treatment. Ethics and dissemination The RHIBOT trial has been authorised by the Agence Nationale de Securite du Medicament and approved by the Comite de Protection des Personnes de Tours Ouest-1. The findings of the study will be disseminated in peer-reviewed journals and at conferences. If the results are positive, intra-articular BTA could be an efficient and safe complementary therapeutic option for patients with TMC OA. Date and version identifier of the protocol 8 January 2018, V. 2.0. Trial registration number NCT03187626; Pre-results.

International audience; To the Editor:The Aicardi–Goutières syndrome is a genetic encephalopathy that is associated with childhood illness and death. The syndrome is hypothesized to be due to misidentification of self-derived nucleic acids as nonself and the subsequent induction of a type I interferon–mediated response that simulates an antiviral reaction.1 Endogenous retroelements, mobile genetic elements that can be transcribed to RNA and then to DNA by reverse transcription, constitute 40% of the human genome and represent a potential source of immunostimulatory nucleic acid in patients with this syndrome.2

Aims and objectives To assess the impact of a short hypnotic session on postoperative anxiety and pain in major orthopaedic surgery. Background Despite specific information given before a scheduled paediatric surgery, perioperative anxiety can become important. Design Randomised Clinical Study. Methods The study is an open single-centre randomised clinical study comparing a “control” group versus a “hypnosis” group receiving a short hypnosis pre-induction session as additional experimental analgesic procedure. The primary endpoint was the postoperative anxiety, blindly assessed using a visual analogue scale. Results The study involved 120 children (age 10–18 years). The results showed no difference between control group versus hypnosis group. Twenty-four hours after surgery (Day+1), the patient's anxiety score was not different between control and hypnosis groups (median [Q1–Q3]: 1 [0; 3] vs. 0 [0; 3], respectively, p = .17). Each group experienced a significant decrease in anxiety level between the day before surgery (Day−1) and the day after surgery (Day+1) (median ([Q1–Q3]) difference of the anxiety score: 2 [4; 0] and 2 [4; 0], respectively, p

Laparoscopic pyeloplasty in children remains controversial and is not included in most pediatric urology centers because of technical difficulties and lack of long-term results.To critically analyze our 10-yr experience with the retroperitoneal approach (RA), with a particular interest on the impact of the learning curve in a teaching center.Patients who underwent pyeloplasty between 1999 and 2010 at our institution were reviewed (n=390). The diagnosis of ureteropelvic junction obstruction was confirmed by ultrasound and technetium Tc 99m mercaptoacetyltriglycine-3 renal scan or magnetic resonance imaging; the same criteria were used to evaluate the outcome. The lateral RA was selected in children1 yr of age without abnormal migration or fusion of the kidney (n=104).Dismembered pyeloplasty and anastomosis were performed using running monofilament 5-0 or 6-0 absorbable suture. All were drained by double-J stent except 20 cases drained by external transanastomotic stent.We assessed intra- and postoperative morbidity and analyzed the teaching of technique and learning curve. Data are expressed as medians and interquartile range (25th, 75th percentiles) for quantitative variables.Median age was 6.2 yr (2.2-10.3). Thirty-three patients had crossing vessels. Median operative time was 185 min (160-235). Median hospital stay was 2 d (1-2). Redo pyeloplasty was needed in only two children (2%). Median follow-up was 2.1 yr (1.4-4.1). Operative time was3 h after 35 cases. After 30 cases performed by the same surgeon, standardization of the technique was feasible, which helped in the teaching process because 50% of the final 30 cases were done by trainees.Retroperitoneal dismembered laparoscopic pyeloplasty is a safe, reliable, and efficient procedure with an excellent outcome in selected children according to their indications and age, and the experience of the surgical team. Even if the transmission to trainees is successful, it is still a long learning process and remains a challenging task for a teaching center.

Mitochondrial fatty acid β-oxidation defects (FAODs) are a group of severe inherited metabolic diseases, most of which can be treated with favorable prognosis following diagnosis. A description of the broad range of phenotypes resulting from these defects remains incomplete, and for this study, we sought to investigate the semiology at diagnosis in a country without a newborn screening program for FAODs.Using a retrospective French multicentre study, we analyzed 187 children aged6 years at diagnosis with FAOD confirmed by enzymatic and/or molecular analyses. Clinical and biological parameters at diagnosis were assessed to screen liver, heart, neurological, and muscle symptoms. Information concerning the long-term prognosis was also collected.Predominant hepatic symptoms were observed in 89 % of patients regardless of the underlying defect. The most frequent symptoms observed were hepatomegaly (92 %), increased blood alanine aminotransferase (ALAT) level (82 %), and steatosis (88 %). Other frequent features included Reye syndrome (49 %), increased gamma-glutamyltranspeptidase (GGT) (37 %), and liver failure (27 %). Extrahepatic features were often associated in the foreground. Hypoglycemia (75 %), neurological (64 %), muscle (61 %), or cardiac features (55 %) [as either cardiomyopathy (47 %) or arrhythmias (31 %)] were frequently documented. Hemodynamic events (41 %) were represented by shock (31 %) or sudden death (35 %). Hyperammonemia (73 %) and hyperlactacidemia (57 %) were the two main biochemical features. Total, very-long-chain acyl-CoA dehydrogenase (VLCADD), long-chain 3-hydroxyacylCoA dehydrogenase (LCHADD), and medium-chain acyl-CoA dehydrogenase (MCADD) deficiency mortality rates were 48 %, 60 %, 63 %, and 20 % respectively.This study presents clinical features of a large cohort of patients with FAODs in a country without neonatal screening for FAODs. Our results highlight liver as the main organ involved at diagnosis regardless of age at diagnosis, classical phenotype (i.e., cardiac, hepatic, or muscular), or enzyme deficiency. Although steatosis may be observed in various inherited metabolic defects, it is a reliable indicator of FAOD and should prompt systematic screening when the diagnosis is suspected. The poor long-term prognoses reported are a strong argument for inclusion of FAODs in newborn screening programs.

Introduction La CRS est une des manifestations les plus graves au cours de la sclerodermie systemique (ScS). Malgre l’amelioration du pronostic depuis l’utilisation systematique des IEC, la mortalite reste de 36 % a 1 an et 19 % a 40 % des patients necessiteront une hemodialyse transitoire ou definitive. Au cours de la ScS, les antagonistes des recepteurs de l’endotheline 1 (ET-1) ont montre une efficacite dans le traitement de l’hypertension arterielle pulmonaire et la prevention secondaire des ulceres digitaux. L’objectif de l’etude est d’evaluer prospectivement l’efficacite d’un antagoniste mixte des recepteurs a l’ET-1, le bosentan au cours de la CRS en adjonction au traitement de reference comprenant un IEC ± autres anti-hypertenseurs. Objectif principal Amelioration de la survie renale et de la survie globale a 12 mois apres 6 mois de traitement par bosentan chez des patients venant de developper une CRS. La survie renale est definie par l’absence de dialyse et/ou doublement de la creatinine. Objectif secondaire Evaluation de la tolerance du bosentan. Patients et methodes Etude ouverte, prospective, multicentrique dans le cadre du programme hospitalier de recherche clinique national 2009. Les patients inclus repondaient aux criteres de ScS de l’ACR et/ou de Leroy et Medsger et presentaient une CRS definie par une HTA severe d’emblee ou d’aggravation progressive > 150/85 mmHg et une insuffisance renale rapidement progressive, sans autre explication que la ScS. Les patients ont ete inclus dans les 15 jours suivant le diagnostic de CRS et ont recu du bosentan a 62,5 mg ×2/j pendant 1 mois puis a 125 mg ×2/j pendant 5 mois. Leur suivi s’est poursuivi jusqu’a 12 mois. Le traitement par bosentan de cette etude a ete fourni par le laboratoire Actelion. Resultats Quarante-quatre patients ont ete evalues dont 28 non pas ete inclus (le plus souvent du fait d’un delai de plus de 15 jours apres debut de la crise renale). Parmi les 16 patients inclus, 10 (62,5 %) sont des femmes. Onze sont des formes diffuses (68,7 %) et 5 sont des formes limitees ou cutanees limitees avec un score de Rodnan moyen a 19 ± 11. L’âge moyen est de 65,3 ± 16 ans ; 14/16 patients ont des Ac specifiques (6 ont des anti-Scl70, 4 des anti-RNA polymerase III, 3 des anti-centromere et 1 des anti-PM/Scl). Onze patients (86,75 %) avaient recu des corticoides avant le debut de la CRS. Cinq ont une forme normotensive et 5 patients sont oligo-anuriques au diagnostic. La fonction renale avant la CRS etait en mediane de 75 mL/min/1,73 m2. Au diagnostic de CRS, la mediane de la creatinine etait de 288 μmol/L et la clairance de 16 mL/min/1,73 m2. Sept patients (43,75 %) ont ete dialyses au moins une fois entre M0 et M12 et 2/9 patients vivants sont encore dialyses a M12. La survie renale est obtenue dans 31 % des cas (5/16) a M6 et 18,75 % des cas (3/16) a M12. Neuf patients (56,25 %) sont vivants a M12. Un patient est decede d’anasarque avec defaillance cardiaque et renale, deux patients sont decedes de la progression de leur ScS. Les 4 autres deces ne sont pas lies a la ScS (demande d’arret de dialyse, cancer rectal metastatique, hypertension intracrânienne sur radionecrose d’un meningiome et ischemie aigue d’un membre inferieur sur atherome). La tolerance du traitement a ete bonne. Deux patients ont eu une cytolyse hepatique regressive a l’interruption du traitement, et une patiente a eu une anemie imputable au bosentan. Conclusion L’utilisation du bosentan au cours de la crise renale sclerodermique est bien toleree. Son benefice en termes d’amelioration du pronostic renal reste a confirmer sur une plus large cohorte de patients.

ObjectiveThere are few studies of outcome in male patients with undefined 46 XY disorder of sex development (DSD). We aimed to assess testicular function and clinical characteristics after puberty in men with idiopathic 46 XY DSD.DesignWe conducted a University Hospital-based observational follow-up study.MethodsNineteen patients with severe hypospadias associated with other signs of defective virilization, such as microphallus, cryptorchidism, and/or bifid scrotum, who were initially managed during childhood between 1988 and 1994, were evaluated at a median age of 17.6 (16.3; 17.8) years. Outcome measures included clinical findings and serum testosterone, FSH, LH, and inhibin B concentrations.ResultsTesticular function was normal in only five (26%) patients. Impaired testicular function was observed in 14 (74%) patients and was partial (n=6; 32%) or total (n=8; 42%), requiring testosterone treatment for the initial (n=2) or secondary (n=6) induction of puberty. Undescended testis (unilateral n=3, bilateral n=2) was found and surgically managed only in the 14 patients with testicular impairment. Testosterone treatment in early childhood greatly increased penis length in all patients, but persistent microphallus following surgical treatment was observed at the end of puberty in most patients, with no difference between patients with and without testicular dysfunction (penis length of 68 (60; 75) vs 65 (60; 65) mm; P=0.42). Half the patients presented an adult height more than 5 cm below their target height.ConclusionMen diagnosed with idiopathic 46 XY DSD during childhood are at high risk of testicular insufficiency and persistent micropenis, and this should be taken into account during the follow-up.

French hospital-based Biomedical Research System. (DOCX 14 kb)