Pierre Quartier

Université René Descartes - Paris 5, Lutetia Parisorum, Île-de-France, France

Are you Pierre Quartier?

Claim your profile

Publications (259)1243.11 Total impact

  • [Show abstract] [Hide abstract]
    ABSTRACT: Objective: Outcome of JDM is highly heterogeneous. Our objective was to determine clinical and muscle biopsy features associated with poor outcome and response to treatment. Methods: Clinical data and muscle biopsy were obtained from a monocentric cohort of 29 patients. Clinical subgroups were defined by latent class model analysis of initial and follow-up parameters. Myopathological features were analysed using validated scores. Capillary loss was determined on reconstructions of transversal sections and assessed in the different age groups to take into account variations of muscle capillarization during post-natal development. Regression models were used to identify initial predictors of therapeutic response. Results: Two distinct homogeneous subgroups of patients were identified according to clinical severity and pathological findings. The smallest group of patients (7/29) presented with severe JDM. Compared with the other group (22/29), patients had more severe muscle weakness at disease onset, low remission rate at 12 months, frequent subcutaneous limb oedema or gastrointestinal (GI) involvement and higher myopathological scores (capillary dropout, perifascicular necrosis/regeneration, fibres with internal myonuclei and fibrosis subscores). Relevance of capillary dropout to JDM severity was substantiated by age-based analysis, confirming its major role in JDM pathophysiology. Most of these manifestations could be related to vasculopathy (limb oedema, GI involvement, capillary dropout). Furthermore, Childhood Myositis Assessment Scale <34 with either GI involvement or muscle endomysial fibrosis at disease onset were the best predictors of poor response to treatment. Conclusion: Vasculopathy is prominent in severe JDM. Simple criteria can be used at initial evaluation to identify patients requiring a more intensive therapy.
    Rheumatology (Oxford, England) 10/2015; DOI:10.1093/rheumatology/kev359 · 4.48 Impact Factor

  • Neuromuscular Disorders 10/2015; 25:S247. DOI:10.1016/j.nmd.2015.06.227 · 2.64 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Objectives This three-year, international, multicentre, longitudinal, observational, cost-effectiveness study named RaDiCEA (RareDisease &Cost-EffectivenessAnalysis) will assess the economic evaluation (cost of illness - COI and cost-effectiveness analysis - CEA) of innovative therapies (i.e., anti IL-1 agents), quality of life (QoL) and effects of the prevention of otherwise irreversible central nervous system, eye, ear, kidney, and cartilage damages of different treatment strategies for cryopyrin-associated periodic syndromes (CAPS) of adults and children. Methods A virtual time-cohort approach and a Markov model simulating health states corresponding to different CAPS severity will be developed to assess the cost-effectiveness of two different treatment strategies: i.e., either anti IL-1 agents or other than anti IL-1. Due to the lack of a CAPS-specific severity index/damage score, a linear combination of existing indexes and damage scores will be used to rank patient's health status with respect to damages involving specific organs and systems. Coefficients of the resulting function will be assigned following both a top-down (Delphi) and an interim-ex post-bottom-up approach (principal component analysis) considering covariances of all the variables adopted to describe the disease evolution or response to therapies. The model uses relevant economic measures to quantify resource utilization for patients' care in the National Health Systems' perspectives and a broader societal perspective to take into account direct nonmedical costs and indirect costs, in addition to direct costs. QoL will be evaluated using EQ-5D questionnaires. To assess how the model reacts to changes in singular and multiple disease parameters, univariate and probabilistic sensitivity analyses will be performed. Expected results The RaDiCEA project will assess the long-term effectiveness of different potentially life-long treatment strategies and COI, while exploring the feasibility of a new CEA model to be generated from a rare disease (CAPS) observational study. The economic outcomes will be given as the number of years spent in each health state, the related yearly costs and QoL. Conclusions The importance and novelty of the model is twofold: i) in its application, adopting the cost-effectiveness approach for assessing the impact of CAPS therapies, and ii) in the methods, extending the analyses of the impact of CAPS therapies in reducing the speed of disease progression.
    Pediatric Rheumatology 09/2015; 13(Suppl 1):P185. DOI:10.1186/1546-0096-13-S1-P185 · 1.61 Impact Factor

  • Pediatric Rheumatology 09/2015; 13(Suppl 1):P32. DOI:10.1186/1546-0096-13-S1-P32 · 1.61 Impact Factor
  • Source

    Pediatric Rheumatology 09/2015; 13(Suppl 1):P186. DOI:10.1186/1546-0096-13-S1-P186 · 1.61 Impact Factor
  • Source

    ISSAID 2015; 09/2015
  • [Show abstract] [Hide abstract]
    ABSTRACT: To determine the type and frequency of musculoskeletal symptoms at onset and during follow-up of cryopyrin-associated periodic syndromes (CAPS). We retrospectively recorded the articular and muscular symptoms of patients with CAPS managed in French hospitals. Data were described as frequencies or median (range) and score groups were compared using the chi-square test, Fisher exact test, and Mann-Whitney test. 133 patients (33 children) were included, 20 with familial cold-induced auto-inflammatory syndrome, 88 with Muckle-Wells syndrome, 22 with chronic infantile neurologic cutaneous and articular syndrome, and 3 with unclassified CAPS. Median age was 35 (range 0-78) years at the time of the study, 1 (0-41) years at symptom onset, and 23 (0-58) years at diagnosis. The disease was sporadic in 17% of patients. Cutaneous symptoms predominated at onset (77%), followed by articular symptoms (30%). The p.Thr348Met and p.Arg260Trp NLRP3 mutations were significantly associated with presence and absence of articular symptoms at onset, respectively. During follow-up, 89% of patients had musculoskeletal symptoms; 88% had arthralgia and 58% arthritis, but only 9% had joint destruction. Tendinopathies occurred in 21.5% of patients, tender points in 16.5%, and myalgia in 33%. Only 3 patients had typical knee deformities. Radiographs were rarely performed. Except for bone deformities, osteoarticular symptoms occurred at similar frequencies in the different CAPS phenotypes. Joint manifestations were frequent in all CAPS phenotypes. Bone deformities were rare. Musculoskeletal manifestations varied within given families but tended to worsen over time. This article is protected by copyright. All rights reserved. © 2015, American College of Rheumatology.
    Arthritis and Rheumatology 08/2015; DOI:10.1002/art.39292
  • [Show abstract] [Hide abstract]
    ABSTRACT: Few studies have assessed Health-Related Quality of Life (HR-QoL) in adults following juvenile idiopathic arthritis, and none since the advent of biotherapies. The aim of our study is to assess the impact of juvenile idiopathic arthritis on quality of life in a large transitional cohort, evaluate which factors influence quality of life in juvenile idiopathic arthritis, and determine which questionnaire should be used in practice. All consecutive juvenile idiopathic arthritis patients followed during adulthood in a transitional care program were included. Demographical, clinical and biological data were collected. The following quality of life questionnaires were administered: SF36 and EuroQoL. Age- and sex-matched controls (without rheumatic disease) were included. One hundred and sixty-one juvenile idiopathic arthritis (120 women and 41 men) and 76 (51/25) controls were included. Out of 161, sixty-five (40%) were considered to be in remission. Juvenile idiopathic arthritis had a large impact on the physical scales of quality of life. Pain seemed to be the most important factor affecting quality of life in cases of juvenile idiopathic arthritis. No significant difference was found between sub-types of juvenile idiopathic arthritis. In this large transitional cohort of patients at the era of biotherapies, juvenile idiopathic arthritis has a larger effect on physical than mental scale of quality of life measures. Pain was the main factor influencing quality of life. Sub-types of juvenile idiopathic arthritis do not seem to influence quality of life. Copyright © 2015 Société française de rhumatologie. Published by Elsevier SAS. All rights reserved.
    Joint, bone, spine: revue du rhumatisme 07/2015; DOI:10.1016/j.jbspin.2015.03.005 · 2.90 Impact Factor
  • F. Uettwiller · M. Rodero · G. Sarrabay · Y. Marot · P. Quartier · Y. Crow ·
    [Show abstract] [Hide abstract]
    ABSTRACT: Background Autosomal recessive loss of function mutations in CECR1 encoding adenosine deaminase 2 (ADA2) were newly reported to cause early-onset stroke and inflammatory vasculoapthy with polyarteritis nodosa as pathological feature. Overexpression of interferon stimulated genes (ISGs) has been suggested as part of the physiopathology and neutrophil related genes signature has been described in some patients. Objectives To describe the clinical and genetic features of two siblings; to measure ADA2 activity, secretion of inflammatory cytokines, interferon signature and expression of neutrophil related genes. Methods Double stranded DNA sequencing (NM_00128225.1) was performed. ADA2 activity in serum was assessed using a commercial kit. Methods for the assessment of the expression of a panel of ISGs and neutrophil stimulated genes have been previously published and validated. Cytokine secretion was assessed by ELISA. Results We report the case of a 9 years old girl of non consanguineous parents presenting at the age of 3 years with fever, systemic inflammation, lesions of cutaneous vasculitis and inflammatory muscles lesions. Muscular biopsy fund focal necroziting angeitis of a minor interfascicular artery compatible with a periarteritis nodosa. The girl experienced neurological ischemic transient attack with mild lymphocytic pleocytosis in the cerebrospinal fluid and two strokes leaving here with spasticity and a diminution of her mental performances. MRI showed pedoncular and mesencephalic lesions. She was resistant to steroids, cyclosporine and azathioprine. Pulses of cyclophosphamid allowed a transient clinical remission but she presented once more an aggravation and we decided to move to antiTNF (etanercept) Her sister was followed for anemia since age of one year, with mild unexplained inflammatory syndrome. She had no cutaneous or muscular expression and no fever. At the age of 3, she presented with transient paralysis of the third cranial nerve. At the age of 5, she presented with transient vestibulitis. Both were considered as viral but cerebral MRI revealed a lacunar stroke. Double stranded DNA sequencing showed an biallelic mutation: p.Tyr453Cys, already published and known to be pathogen by prediction analysis and a deletion of exon 6 predicted to shift the reading frame. ADA2 activity showed a significant reduction compared to control. In the two cases, interferon related genes signature was significantly elevated. Neutrophil genes showed no increased expression. Inflammatory cytokines were not significantly oversecreted. Conclusions We describe here a new mutation with a surprising clinical picture of the youngest girl: start in a very young age with no cutaneous expression, no fever and two transient neurological events. Association of inflammatory syndrome even mild with neurological unexplained event must lead to test for ADA2 mutation. ADA2 activity is probably not linked to the phenotype, as already suggested. If some patients seem to have a neutrophil signature with remarkable response to antiTNF, some are not, perhaps consistent with different sub classes of ADA2 deficiency. New therapeutics targeting the interferon pathways could be a novel approach to try. Disclosure of Interest None declared
    Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):965.2-965. DOI:10.1136/annrheumdis-2015-eular.6226 · 10.38 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Background The long-term safety of anti-tumor necrosis factor (TNF) drugs is particularly important in pediatric patients (pts) who may require prolonged treatment of their inflammatory disease. Objectives To evaluate long-term rates of serious adverse events (AE) and anti-TNF AEs of special interest in adalimumab (ADA) clinical trials in pediatric pts with polyarticular or polyarticular course juvenile idiopathic arthritis (pJIA) or enthesitis-related arthritis (ERA). Methods Safety data from pts treated with ADA, either dosed 24 mg/m2 BSA every other week (eow) or 20 mg eow (<30 kg) to 40 mg eow (≥30 kg), in 4 clinical trials in pJIA and ERA were analyzed. Three studies in pJIA enrolled pts aged 2–17 years (yrs) treated with ADA for up to 8.5 yrs. One study enrolled pts with ERA aged 6–17 yrs who were treated with ADA for up to 52 wks in this analysis. AEs of special interest included malignancy, serious infections, tuberculosis (TB) and other opportunistic infections, and death. Events per 100 patient-years (PY) were calculated using AEs reported after first ADA dose through 70 days after last dose. Results ADA was administered to 274 pts, representing 769.0 PY of exposure. Infections, the most common AE, occurred in ≥10% of pts. Serious infection was the most frequently reported SAE (table). One case of latent TB was reported. No malignancies, opportunistic infections, or deaths were reported. 8.4% of pts (23/274) discontinued study due to AE (range, 5.1% in age <5 yr to 9.6% in ages 5 - <12 yrs). Other than uveitis, liver events, and injection site-related AEs, no differences in AE rates were observed between age groups.(table) Conclusions These data provide support for the long-term safety of ADA in pediatric pts aged 2–17 yrs with pJIA or ERA and demonstrate a safety profile consistent with ADA in adult pts and known information about the anti-TNF class. Acknowledgements AbbVie funded the studies (NCT00048542, NCT00690573, NCT00775437, and NCT01166282), contributed to their design and was involved in the collection, analysis, and interpretation of the data, and in the writing, review, and approval of the publication. Medical writing support was provided by Kathleen V. Kastenholz, PharmD, MS, of AbbVie. Disclosure of Interest N. Ruperto Grant/research support from: AbbVie, AstraZeneca, BMS, Janssen, “Francesco Angelini”, GlaxoSmithKline, Italfarmaco, Novartis, Pfizer, Roche, Sanofi Aventis, Schwarz Biosciences, Sobi, Xoma, and Wyeth for the PRINTO network paid to GASLINI Hospital, Consultant for: AbbVie, Amgen, Astellas, Alter, AstraZeneca, Biogen Idec, Boehringer, BMS, CD-Pharma, Celgene, Crescendo Bioscience, EMD Serono, Italfarmaco, Janssen, MedImmune, Medac, Novartis, Novo-Nordisk, Pfizer, Roche, Sanofi Aventis, Servier, Sinergie, Takeda, and Vertex, Speakers bureau: AbbVie, Amgen, Astellas, Alter, AstraZeneca, Biogen Idec, Boehringer, BMS, CD-Pharma, Celgene, Crescendo Bioscience, EMD Serono, Italfarmaco, Janssen, MedImmune, Medac, Novartis, Novo-Nordisk, Pfizer, Roche, Sanofi Aventis, Servier, Sinergie, Takeda, and Vertex, D. Lovell Consultant for: AbbVie, AstraZeneca, Boehringer Ingelheim, Celgene, Centocor, Bristol-Myers Squibb, Genentech, GlaxoSmithKline, Horizon Pharma, Janssen Biologics B.V., Novartis, Pfizer, Regeneron, Hoffman La-Roche, and UBC and served on data and safety monitoring boards for Forest Research, Speakers bureau: Genentech and Wyeth Pharmaceuticals, D. Kingsbury Grant/research support from: AbbVie, R. Burgos-Vargas Grant/research support from: AbbVie, Consultant for: AbbVie, BMS, Janssen, Pfizer, and Roche, Speakers bureau: AbbVie, BMS, Janssen, Pfizer, and Roche, T. Imagawa Grant/research support from: AbbVie/Eisai and Novartis, Consultant for: AbbVie/Eisai, Speakers bureau: AbbVie/Eisai, Chugai, and Mitsubishi Pharma, G. Horneff Grant/research support from: AbbVie, Pfizer, and Roche, Speakers bureau: AbbVie, Novartis, Pfizer, and Roche, P. Quartier Grant/research support from: AbbVie, Novartis, Pfizer, BMS, Chugai-Roche, Medimmune, Servier, and Swedish Orphan Biovitrum, Consultant for: AbbVie, Novartis, Pfizer, BMS, Chugai-Roche, Medimmune, Servier, and Swedish Orphan Biovitrum, S. Goodman Consultant for: Amgen, A. Reiff Consultant for: AbbVie and Amgen, Speakers bureau: AbbVie and Amgen, E. Giannini Consultant for: AbbVie, A. Cardoso Shareholder of: AbbVie, Employee of: AbbVie, J. Anderson Shareholder of: AbbVie, Employee of: AbbVie, N. Varothai Shareholder of: AbbVie, Employee of: AbbVie, J. Kalabic Shareholder of: AbbVie, Employee of: AbbVie, A. Martini Grant/research support from: AbbVie, AstraZeneca, BMS, Janssen, “Francesco Angelini”, GlaxoSmithKline, Italfarmaco, Novartis, Pfizer, Roche, Sanofi Aventis, Schwarz Biosciences, Xoma, and Wyeth for the PRINTO network paid to GASLINI Hospital, Consultant for: AbbVie, Amgen, Biogen Idec, Boehringer, BMS, Celgene, EMD Serono, Janssen, MedImmune, Medac, Novartis, Novo- Nordisk, Pfizer, Roche, Sanofi Aventis, Servier, Takeda, and Vertex, Speakers bureau: AbbVie, Amgen, Biogen Idec, Boehringer, BMS, Celgene, EMD Serono, Janssen, MedImmune, Medac, Novartis, Novo- Nordisk, Pfizer, Roche, Sanofi Aventis, Servier, Takeda, and Vertex
    Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):610.3-611. DOI:10.1136/annrheumdis-2015-eular.1273 · 10.38 Impact Factor

  • Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):613.2-613. DOI:10.1136/annrheumdis-2015-eular.4186 · 10.38 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Background Childhood-onset panniculitides are rare and classically include infectious, connective tissue, malignant, metabolic, and drugs, cold or trauma-induced panniculitis. Some specific types of panniculitis are only seen in neonates and very young infants, and include subcutaneous fat of the newborn, poststeroid panniculitis, sclerema neonatorum, and cold panniculitis. However, no specific discoverable etiology can be identified in many patients. Objectives To describe the spectrum of clinical and pathological manifestations of childhood-onset panniculitis of unknown cause Methods A retrospective single-center study of children presenting with panniculitis diagnosed by skin biopsy Results Eighteen patients were identified. The onset of symptoms occurred within the first two years of life in 15/18 patients (83%). Panniculitis was the revealing manifestation in 15/18 (83%) patients, and relapsed in 13/18 (72%) patients. Associated-non-cutaneous features occurred in 16/18 (89%) patients, especially autoinflammatory manifestations in 13/18 patients (72%), associated with immunodeficiency in 5 patients (low T or B cells count with hypogammaglobulinemia in 4 and 1 patients respectively). Autoinflammatory manifestations consisted in attacks of fever with an elevation of acute phase reactants associated with polyarthritis or polyarthralgia (2 patients), rash (2 patients) or aseptic adenitis (2 patients). Panniculitis was mostly lobular in 10 (55%) patients, septal in 3 (17%) patients or both in 5 patients (28%). Predominant infiltrate consisted of neutrophils, cytophagic histiocytes, lymphocytes, eosinophils in 8 (44%), 5 (28%), 3 (17%), and 1 (5.5%) patients respectively, and included granuloma in 1 patient. No relationship was found between clinical and pathological features. Genomic mutations were identified in 3 of the 4 patients who underwent genetic analysis: mutations in TRNT1, MVK (mevalonate kinase) and LCK genes. Conclusions This series underlines that auto-inflammatory diseases, possibly associated with inherited immunodeficiency, are a main cause of early-onset panniculitis. The identification of a monogenic cause in 3 patients and the early-onset presentation of the disease suggest that a subset of patients has a genetic predisposition to develop panniculitis, especially early-onset panniculitis. Disclosure of Interest None declared
    Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):1222.1-1222. DOI:10.1136/annrheumdis-2015-eular.4745 · 10.38 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Background The long-term efficacy of canakinumab and changes in the daily lives of patients (and their caregivers) have not been thoroughly evaluated since its first use in France in 2007 and its approval for CAPS in 2010. Objectives A multicentre retrospective, observational study set up to assess the “real life” use of canakinumab in all French CAPS patients ever treated since 2007, to describe their clinical course on a long-term, and to analyse changes in their (and their caregiver's) quality of lives. Methods We targeted the 70-80 patients ever treated for CAPS in France, at least once and even during a clinical trial. Investigators were known experts in the field of CAPS in France who accepted to take part in the study. Data were collected through questionnaires by phone interviews and medical chart reviews, at treatment initiation, 6 months, 12 months and at the last medical visit. They included: clinical data, canakinumab use in real life conditions, impact on patients' (and caregivers') quality of life, and care consumption. The significance limit was set at 5% for all of the statistical tests. Results 68 CAPS patients, >90% of the target number, were enrolled (23 children, 45 adults). Sixteen patients (24%) had FCAS, 43 (63%) had MWS and 9 (13%) had NOMID-CINCA. The median duration of treatment was 5 years (from July 2007 to July 2014). >95% of patients remained on treatment. Doses were not modified in nearly half cases (31/68). For 37 patients, dosage adjustments (more often increase) were required (102 in total), especially in younger patients and those with the most severe phenotypes. The global activity of the disease, skin disorders and most of the symptoms were significantly better after canakinumab treatment (p<0.001) at the different study timepoints. The quality of life score also showed a significant improvement as median was 8 before canakinumab versus 2 (p<0.0001). Canakinumab treatment allowed also improvement in patient's daily activities, mood, and social life. Patients reported less school absences (79% versus 36%), and less sick leaves (48% versus 6%) after the initiation of canakinumab. The effect was weaker in CINCA patients; due in part to the late initiation of anti -IL1β treatment. Caregivers (49) were mostly family members and 35% of them had CAPS. They dedicated a mean of 7 hours/week to the CAPS patients before treatment and 4 hours during the last year. They spent on average 11.1 days per year of their job before canakinumab treatment versus 3 days after. The trend was less pronounced for caregivers of NOMID-CINCA patients. Conclusions ENVOL study showed real-life results similar to those obtained during the phase III clinical trials with canakinumab, reinforcing its sustained activity. The maintenance of more than 95% of patients on therapy confirmed its major benefit to CAPS patients, which was demonstrated herein by the positive impact of canakinumab in patients (and their caregivers) social, emotional, educational and professional lives. Disclosure of Interest I. Kone-Paut Grant/research support from: SOBI, Novartis, Roche, Consultant for: SOBI, Novartis, Pfizer, Abbvie, Chugai, Speakers bureau: Novartis, SOBI, P. Quartier Grant/research support from: Abbvie, Novartis, Pfizer, Consultant for: Abbvie, Novartis, Servier, SOBI, Speakers bureau: Abbvie, Chugai Roche, Medimmune, Novartis, Sobi, O. Fain Consultant for: Shire et CSL Behring, G. Grateau Grant/research support from: Abbvie, Novartis, Pfizer, Consultant for: Abbvie, Novartis, Servier, SOBI, Speakers bureau: Abbvie, Chugai Roche, Medimmune, Novartis, Sobi, P. Pillet: None declared, V. Despert: None declared, K. STANKOVIC STOJANOVIC: None declared, S. quere Employee of: Novartis employee, L. willemins Employee of: Novartis employee, O. Reigneau Employee of: novartis employee, E. Hachulla Consultant for: Novartis, SOBI
    Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):1277.2-1278. DOI:10.1136/annrheumdis-2015-eular.2997 · 10.38 Impact Factor

  • Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):608.1-608. DOI:10.1136/annrheumdis-2015-eular.2050 · 10.38 Impact Factor

  • Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):92.3-93. DOI:10.1136/annrheumdis-2015-eular.1215 · 10.38 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Objective To evaluate the interleukin-6 receptor inhibitor tocilizumab for the treatment of patients with polyarticular-course juvenile idiopathic arthritis ( pcJIA). Methods This three-part, randomised, placebocontrolled, double-blind withdrawal study (NCT00988221) included patients who had active pcJIA for ≥6 months and inadequate responses to methotrexate. During part 1, patients received open-label tocilizumab every 4 weeks (8 or 10 mg/kg for body weight (BW) <30 kg; 8 mg/kg for BW ≥30 kg). At week 16, patients with ≥JIA-American College of Rheumatology (ACR) 30 improvement entered the 24- week, double-blind part 2 after randomisation 1:1 to placebo or tocilizumab (stratified by methotrexate and steroid background therapy) for evaluation of the primary end point: JIA flare, compared with week 16. Patients flaring or completing part 2 received open-label tocilizumab. Results In part 1, 188 patients received tocilizumab (<30 kg: 10 mg/kg (n=35) or 8 mg/kg (n=34); ≥30 kg: n=119). In part 2, 163 patients received tocilizumab (n=82) or placebo (n=81). JIA flare occurred in 48.1% of patients on placebo versus 25.6% continuing tocilizumab (difference in means adjusted for stratification: -0.21; 95% CI -0.35 to -0.08; p=0.0024). At the end of part 2, 64.6% and 45.1% of patients receiving tocilizumab had JIA-ACR70 and JIAACR90 responses, respectively. Rates/100 patient-years (PY) of adverse events (AEs) and serious AEs (SAEs) were 480 and 12.5, respectively; infections were the most common SAE (4.9/100 PY). Conclusions Tocilizumab treatment results in significant improvement, maintained over time, of pcJIA signs and symptoms and has a safety profile consistent with that for adults with rheumatoid arthritis.

  • Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):613.1-613. DOI:10.1136/annrheumdis-2015-eular.4945 · 10.38 Impact Factor

  • Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):91-91. DOI:10.1136/annrheumdis-2015-eular.4397 · 10.38 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: Background The Juvenile Arthritis Disease Activity Score (JADAS) is increasingly accepted for defining a treat-to-target strategy in patients (pts) with juvenile idiopathic arthritis (JIA)1 Objectives To use JADAS to evaluate clinical disease activity/control, with or without functional control, in pts with polyarticular or polyarticular-course JIA (pJIA), following the initiation of adalimumab (ADA) ± concurrent methotrexate (MTX) treatment. To assess the feasibility of JADAS definition of remission (REM) and minimal disease activity (MDA) as part of a treat-to-target strategy. Methods Data for this post hoc analysis originated from M10-444, an open-label study in pJIA pts 2 to <4 yrs old or ≥4 yrs weighing <15 kg with moderately to severely active pJIA, in the US and EU (in the EU pts had to previously fail, have an insufficient response, or intolerance to MTX). Pts received subcutaneous ADA (24 mg/m2 body surface area, up to 20 mg/dose), every other week (wk), for ≥24 wks, or until pts reached 4 yrs and weight ≥15 kg (in the EU, pts could continue for up to 1 additional yr). Disease activity was determined by JADAS based on C-reactive protein (CRP, normalization from 0-10), for 10 or 27 joints (JADAS10/27); functional impairment was determined by the Disability Index- Childhood Health Assessment questionnaire (DI-CHAQ). At wks 12 and 24, the proportion of pts achieving physician-assessed REM (JADAS≤2) or MDA (JADAS≤3.8), and the proportion of pts who achieved both MDA/REM and DI-CHAQ<0.5, was determined using observed cases. Results Out of 32 pts, 25 (78%) had received prior MTX and 27/32 (84%) received concomitant MTX during the study. At baseline, pts had a mean JADAS10 of 18.8, JADAS27 of 19.0, and DI-CHAQ of 1.2. After 12 wks on open-label ADA, improvements were observed in clinical and functional outcomes. At wks 12 and 24, the mean JADAS10 was 6.2 and 5.3; mean JADAS27 was 6.4 and 5.6; mean DI-CHAQ was 0.7 and 0.7, respectively. No pts were in REM/MDA at baseline; however after 12 and 24 wks, a sizeable proportion achieved disease control (table). After 12 and 24 wks of ADA treatment, the proportions of pts achieving both, disease control and DI-CHAQ<0.5 also increased from baseline. Conclusions Addition of open-label ADA treatment resulted in clinically important improvements in clinical and functional outcomes in pts with pJIA. JADAS 10 and -27 gave comparable results. JADAS REM and MDA are achievable targets with ADA treatment. References Acknowledgements AbbVie sponsored the study, participated in the design, data collection, analysis, and interpretation; and in the writing of the final version. Medical writing support was provided by Naina Barretto, PhD, of AbbVie Disclosure of Interest D. Kingsbury Grant/research support from: AbbVie, P. Quartier Grant/research support from: AbbVie, Novartis, Pfizer, BMS, Chugai-Roche, Medimmune, Servier, and Swedish Orphan Biovitrum, Consultant for: AbbVie, Novartis, Pfizer, BMS, Chugai-Roche, Medimmune, Servier, and Swedish Orphan Biovitrum, G. Horneff Grant/research support from: AbbVie, Pfizer, Roche, Speakers bureau: AbbVie, Novartis, Pfizer, and Roche, K. Minden Grant/research support from: Pfizer and Abbvie, Consultant for: Pfizer, Abbvie, Roche and Pharm-Allergan, M. Toth: None declared, N. Varothai Employee of: AbbVie, A. Cardoso Employee of: AbbVie, J. Kalabic Employee of: AbbVie
    Annals of the Rheumatic Diseases 06/2015; 74(Suppl 2):382.2-382. DOI:10.1136/annrheumdis-2015-eular.4171 · 10.38 Impact Factor
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Objectives To analyse the effect of biological agents (BAs) in terms of achieving inactive disease (ID) or clinical remission (CR) in patients with systemic juvenile idiopathic arthritis (SJIA), to describe effects of switching or discontinuing a BA and to assess the proportion of patients able to maintain ID or CR off steroids and after withdrawing BA therapy. Methods Retrospective study in a French paediatric rheumatology reference centre using the CEMARA (CEntre des MAladies RAres) register. Results Seventy-seven patients were included with a cumulative follow-up of 245.5 patient-years (median 1.1, range 0.5–8.0). On a first BA, ID was achieved in 37 patients, including 1 patient out of 12 patients on etanercept, 26 patients out of 51 on anakinra and 7 out of 10 on canakinumab. One patient on abatacept and two patients on tocilizumab also achieved ID. Switching of BA was common. The switch to a second (n=34), third (n=18) or fourth (n=4) BA resulted in ID in a further 13 patients, either on canakinumab (n=6) or tocilizumab (n=7). At last follow-up, 40 patients were in CR (27 patients off steroids, 5 patients having never received steroid treatment), either on (n=29) or off (n=11) BA. Conclusions In this series of patients with SJIA, interleukin-1 inhibitors were associated with a higher proportion of ID than tumour necrosis factor inhibitors when used as first BA. Switching allowed some patients to achieve ID when treated with canakinumab or tocilizumab. CR was eventually achieved in more than half of the patients.
    04/2015; 1(1):e000036-e000036. DOI:10.1136/rmdopen-2014-000036

Publication Stats

5k Citations
1,243.11 Total Impact Points


  • 2009-2015
    • Université René Descartes - Paris 5
      • Faculté de Médecine
      Lutetia Parisorum, Île-de-France, France
  • 2008-2015
    • Assistance Publique – Hôpitaux de Paris
      Lutetia Parisorum, Île-de-France, France
  • 2011-2014
    • IRCCS Istituto G. Gaslini
      Genova, Liguria, Italy
  • 2002-2014
    • Hôpital Universitaire Necker
      Lutetia Parisorum, Île-de-France, France
  • 2013
    • Institut Imagine
      Lutetia Parisorum, Île-de-France, France
    • Hacettepe University
      • Department of Pediatrics
      Ankara, Ankara, Turkey
  • 2012
    • Université Paris-Sorbonne - Paris IV
      Lutetia Parisorum, Île-de-France, France
  • 2010
    • Università degli Studi di Genova
      Genova, Liguria, Italy
    • CHU de Lyon - Hôpital Femme-Mère-Enfant
      Lyons, Rhône-Alpes, France
  • 2003-2005
    • Imperial College London
      • Department of Medicine
      Londinium, England, United Kingdom
    • University Medical Center Utrecht
      Utrecht, Utrecht, Netherlands
  • 1999
    • Karolinska Institutet
      Сольна, Stockholm, Sweden