Treatment outcome in early diffuse cutaneous systemic sclerosis: The European Scleroderma Observational Study (ESOS)

Annals of the Rheumatic Diseases

Volumn 76, Issue 7, 2017, Pages 1207-1218

  Herrick, Ariane L ^a,b   Pan, Xiaoyan ^a   Peytrignet, Sébastien ^a   Lunt, Mark ^a   Hesselstrand, Roger ^d   Mouthon, Luc ^e   Silman, Alan ^f   Brown, Edith ^a   Czirják, László ^g   Distler, Jörg H W ^h   Distler, Oliver ⁱ   Fligelstone, Kim ^j   Gregory, William J ^k   Ochiel, Rachel ^j   Vonk, Madelon ^l   Ancuta, Codrina ^m   Ong, Voon H ⁿ   Farge, Dominique ^o   Hudson, Marie ^p   Matucci Cerinic, Marco ^q   Balbir Gurman, Alexandra ^r   Midtvedt, Øyvind ^s   Jordan, Alison C ^t   Jobanputra, Paresh ^t   Stevens, Wendy ^u   Moinzadeh, Pia ^v   Hall, Frances C ^w   Agard, Christian ^x   Anderson, Marina E ^y   Diot, Elisabeth ^z   Madhok, Rajan ^aa   Akil, Mohammed ^ab   Buch, Maya H ^ac   Chung, Lorinda ^ad   Damjanov, Nemanja ^ae   Gunawardena, Harsha ^af   Lanyon, Peter ^ag   Ahmad, Yasmeen ^ah   Chakravarty, Kuntal ^ai   Jacobsen, Søren ^aj   MacGregor, Alexander J ^ak   McHugh, Neil ^al   Müller Ladner, Ulf ^am   Riemekasten, Gabriela ^an   Becker, Michael ^ao   Roddy, Janet ^ap   Carreira, Patricia E ^aq   Fauchais, Anne Laure ^ar   Hachulla, Eric ^as   Hamilton, Jennifer ^at   Inanç, Murat ^au   McLaren, John S ^av   Van Laar, Jacob M ^aw   Pathare, Sanjay ^ax   Proudman, Susannah ^ay   Rudin, Anna ^az   Sahhar, Joanne ^ba   Coppere, Brigitte ^bb   Serratrice, Christine ^bc   Sheeran, Tom ^bd   Veale, Douglas J ^be   Grange, Claire ^bf   Trad, Georges Selim ^c   Denton, Christopher P ⁿ   more..

^a UNIVERSITY OF MANCHESTER   (United Kingdom)

^b MANCHESTER UNIVERSITY NHS FOUNDATION TRUST   (United Kingdom)

^c HÔPITAL AMBROISE PARÉ   (France)

^d LUND UNIVERSITY   (Sweden)

^e Institut Cochin   (France)

^f UNIVERSITY OF OXFORD   (United Kingdom)

^g UNIVERSITY OF PÉCS   (Hungary)

^h UNIVERSITY OF ERLANGEN NUREMBERG   (Germany)

ⁱ UNIVERSITY OF ZURICH   (Switzerland)

^j ROYAL FREE LONDON NHS FOUNDATION TRUST   (United Kingdom)

^k SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

^l RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^m GRIGORE T POPA UNIVERSITY OF MEDICINE AND PHARMACY   (Romania)

ⁿ UNIVERSITY COLLEGE LONDON   (United Kingdom)

^o SAINT LOUIS HOSPITAL   (France)

^p MCGILL UNIVERSITY   (Canada)

^q UNIVERSITY OF FLORENCE   (Italy)

^r TECHNION ISRAEL INSTITUTE OF TECHNOLOGY   (Israel)

^s OSLO UNIVERSITY HOSPITAL   (Norway)

^t QUEEN ELIZABETH HOSPITAL   (United Kingdom)

^u ST VINCENT S HOSPITAL   (Australia)

^v UNIVERSITY OF COLOGNE   (Germany)

^w CAMBRIDGE UNIVERSITY HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^x UNIVERSITÉ DE NANTES   (France)

^y UNIVERSITY OF LIVERPOOL   (United Kingdom)

^z CHU BRETONNEAU   (France)

^aa GLASGOW ROYAL INFIRMARY   (United Kingdom)

^ab SHEFFIELD TEACHING HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^ac UNIVERSITY OF LEEDS   (United Kingdom)

^ad STANFORD UNIVERSITY   (United States)

^ae UNIVERSITY OF BELGRADE   (Serbia)

^af NORTH BRISTOL NHS TRUST   (United Kingdom)

^ag NOTTINGHAM UNIVERSITY HOSPITALS NHS TRUST   (United Kingdom)

^ah Peter Maddison Rheumatology Centre   (United Kingdom)

^ai QUEEN S HOSPITAL   (United Kingdom)

^aj UNIVERSITY OF COPENHAGEN   (Denmark)

^ak UNIVERSITY OF EAST ANGLIA   (United Kingdom)

^al ROYAL NATIONAL HOSPITAL FOR RHEUMATIC DISEASES   (United Kingdom)

^am JUSTUS LIEBIG UNIVERSITY   (Germany)

^an UNIVERSITY OF LÜBECK   (Germany)

^ao CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

^ap ROYAL PERTH HOSPITAL   (Australia)

^aq HOSPITAL UNIVERSITARIO 12 DE OCTUBRE   (Spain)

^ar LIMOGES UNIVERSITY HOSPITAL   (France)

^as UNIV LILLE   (France)

^at GATESHEAD HEALTH NHS FOUNDATION TRUST   (United Kingdom)

^au ISTANBUL UNIVERSITY   (Turkey)

^av NHS FIFE   (United Kingdom)

^aw UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^ax JAMES COOK UNIVERSITY HOSPITAL   (United Kingdom)

^ay UNIVERSITY OF ADELAIDE   (Australia)

^az UNIVERSITY OF GOTHENBURG   (Sweden)

^ba MONASH UNIVERSITY   (Australia)

^bb EDOUARD HERRIOT HOSPITAL   (France)

^bc HÔPITAL SAINT JOSEPH   (France)

^bd ROYAL WOLVERHAMPTON HOSPITALS NHS TRUST   (United Kingdom)

^be ST VINCENT S UNIVERSITY HOSPITAL   (Ireland)

^bf CENTRE HOSPITALIER LYON SUD   (France)

more..

Author keywords

Cyclophosphamide; Methotrexate; Systemic Sclerosis; Treatment

Indexed keywords

AZATHIOPRINE; CYCLOPHOSPHAMIDE; METHOTREXATE; MYCOPHENOLATE MOFETIL; ANTINUCLEAR ANTIBODY; AUTOANTIBODY; CENTROMERE ANTIBODY; DNA DIRECTED RNA POLYMERASE III; IMMUNOSUPPRESSIVE AGENT; MYCOPHENOLIC ACID; NUCLEAR PROTEIN; SCL 70 ANTIGEN, HUMAN;

ADULT; ARTICLE; CONTROLLED STUDY; DIFFUSE CUTANEOUS SYSTEMIC SCLEROSIS; DISEASE DURATION; DRUG EFFICACY; DRUG WITHDRAWAL; FEMALE; FOLLOW UP; HUMAN; MAJOR CLINICAL STUDY; MALE; OBSERVATIONAL STUDY; PATIENT ASSESSMENT; PRIORITY JOURNAL; SURVIVAL; SYSTEMIC SCLEROSIS; TREATMENT OUTCOME; UNSPECIFIED SIDE EFFECT; COHORT ANALYSIS; EARLY INTERVENTION; EUROPE; IMMUNOLOGY; MIDDLE AGED; PROSPECTIVE STUDY; SCLERODERMA, DIFFUSE; SEVERITY OF ILLNESS INDEX; SURVIVAL RATE;

ADULT; ANTIBODIES, ANTINUCLEAR; AUTOANTIBODIES; COHORT STUDIES; CYCLOPHOSPHAMIDE; EARLY MEDICAL INTERVENTION; EUROPE; FEMALE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MALE; METHOTREXATE; MIDDLE AGED; MYCOPHENOLIC ACID; NUCLEAR PROTEINS; PROSPECTIVE STUDIES; RNA POLYMERASE III; SCLERODERMA, DIFFUSE; SEVERITY OF ILLNESS INDEX; SURVIVAL RATE; TREATMENT OUTCOME;

EID: 85020703821     PISSN: 00034967     EISSN: 14682060     Source Type: Journal    
DOI: 10.1136/annrheumdis-2016-210503     Document Type: Article

References (29)

1. Nikpour M, Stevens WM, Herrick AL, et al. Epidemiology of systemic sclerosis. Best Practice Res Clin Rheumatol 2010;24:857-69.
2. Rubio-Rivas M, Royo C, Simeon CP, et al. Mortality and survival in systemic sclerosis : systematic review and meta-analysis. Sem Arthritis Rheum 2014;44:208-19.
3. Nihtyanova SI, Schreiber BE, Ong VH, et al. Prediction of pulmonary complications and long-term survival in systemic sclerosis. Arthritis Rheum 2014;66:1625-35.
4. Merkel PA, Silliman NP, Clements PJ, et al. Patterns and predictors of change in outcome measures in clinical trials in scleroderma: An individual patient meta-analysis of 629 subjects with diffuse cutaneous systemic sclerosis. Arthritis Rheum 2012;64:3420-9.
5. Silverman SL. From randomized controlled trials to observational studies. Am J Med 2009;122:114-20.
6. Halpern SD, Karlawish JT, Berlin JA. The continuing unethical conduct of underpowered clinical trials. JAMA 2002;288:358-62.
7. Pope JE, Bellamy N, Seibold JR, et al. A randomized, controlled trial of methotrexate versus placebo in early diffuse scleroderma. Arthritis Rheum 2001;44:1351-8.
8. Denton CP, Merkel PA, Furst DE, et al. Recombinant human anti-transforming growth factor β1 antibody therapy in systemic sclerosis: a multicentre, randomized, placebo-controlled Phase I/II trial of CAT-192. Arthritis Rheum 2007;56: 323-3.
9. Kowal-Bielecka O, Landewé R, Avouac J, et al. EULAR recommendations for the treatment of systemic sclerosis: a report from the EULAR Scleroderma Trials and Research Group (EUSTAR). Ann Rheum Dis 2009;68:620-8.
10. Rawlins MD. The Harveian Oration of 2008: On the evidence for decisions about the use of therapeutic interventions. London: Royal College of Physicians, 2008.
11. Gagne J, Thompson L, O'Keefe K, et al. Innovative research methods for studying treatments for rare diseases: methodological review. BMJ 2014;349:g6802.
12. Le Roy EC, Black C, Fleischmajer R, et al. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol 1988;15:202-5.
13. Clements P, Lachenbruch P, Siebold J, et al. Inter- and intraobserver variability of total skin thickness score (modified Rodnan TSS) in systemic sclerosis. J Rheumatol 1995;22:1281-5.
14. Clements PJ, Hurwitz EL, Wong WK, et al. Skin thickness score as a predictor and correlate of outcome in systemic sclerosis. Arthritis Rheum 2000;43:2445-54.
15. Steen VD, Medsger TA. The value of the health assessment questionnaire and special patient-generated scales to demonstrate change in systemic sclerosis patients over time. Arthritis Rheum 1997;40:1984-91.
16. Webster K, Cella D, Yost K. The Functional Assessment of Chronic Illness Therapy (FACIT) Measurement System: properties, applications, and interpretation. Health Qual Life Outcomes 2003;1:79.
17. Harel D, Thombs BD, Hudson M, et al. Measuring fatigue in SSc: a comparison of the Short Form-36 Vitality subscale and Functional Assessment of Chronic Illness Therapy-Fatigue scale. Rheumatol 2012;51:2177-85.
18. Rannou F, Poiraudeau S, Berezne A, et al. Assessing disability and quality of life in systemic sclerosis: construct validities of the Cochin Hand Function Scale, Health Assessment Questionnaire (HAQ), Systemic Sclerosis HAQ, and Medical Outcomes Study 36-Item Short Form Health Survey. Arthritis Rheum 2007;57:94-102.
19. Brookhart MA, Schneeweiss S, Rothman KJ, et al. Variable selection for propensity score models. Amer J Epidemiol 2006;163:1149-56.
20. Austin PC, Grootendorst P, Normand S-LT, et al. Conditioning on the propensity score can result in biased estimation of common measures of treatment effect: a Monte Carlo study. Stat Med 2007;26:754-68.
21. Sato T, Matsuyama Y. Marginal structural models as a tool for standardization. Epidemiology 2003;14:680-6.
22. Imbens G. The role of the propensity score in estimating dose-response functions. Biometrika 2000;87:706-10.
23. Fewell Z, Hernan MA, Wolfe F, et al. Controlling for time-dependant confounding using marginal structural models. Stata J 2004;4:402-20.
24. Cole SR, Hernán MA. Constructing inverse probability weights for marginal structural models. Amer J Epidemiol 2008;168:656-64.
25. Tashkin DP, Elashoff R, Clements PJ, et al. Cyclophosphamide versus placebo in scleroderma lung disease. N Eng J Med 2006;354:2655-66.
26. Tashkin DP, Roth MD, Clements PJ, et al. Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomsed controlled, double-blind, parallel group trial. Lancet Respir Med 2016;4:708-19.
27. Van Laar JM, Farge D, Sont JK, et al. Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis. JAMA 2014;311:2490-8.
28. Herrick A, Lunt M, Whidby N, et al. Observational study of treatment outcome in early diffuse cutaneous systemic sclerosis. J Rheumatol 2010;37; 116-24.
29. Khanna D, Denton CP, Jahreis A, et al. Safety and efficacy of subcutaneous tocilizumab in adults with systemic sclerosis (faSScinate): a phase 2, randomised controlled trial. Lancet 2016;387:2630-40.