Observational study of treatment outcome in early diffuse cutaneous systemic sclerosis

Journal of Rheumatology

Volumn 37, Issue 1, 2010, Pages 116-124

  Herrick, Ariane L ^a   Lunt, Mark ^a   Whidby, Nina ^a   Ennis, Holly ^a   Silman, Alan ^b   McHugh, Neil ^c   Denton, Christopher P ^d  

^a UNIVERSITY OF MANCHESTER   (United Kingdom)

^b Arthritis Research Campaign   (United Kingdom)

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

^d ROYAL FREE HOSPITAL   (United Kingdom)

Author keywords

Diffuse cutaneous systemic sclerosis; Observational study; Outcome; Treatment

Indexed keywords

AZATHIOPRINE; CYCLOPHOSPHAMIDE; IMMUNOSUPPRESSIVE AGENT; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; THYMOCYTE ANTIBODY;

ADULT; AGED; ARTICLE; COHORT ANALYSIS; DIFFUSE CUTANEOUS SYSTEMIC SCLEROSIS; DRUG DOSE INCREASE; FEMALE; FOLLOW UP; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; MAJOR CLINICAL STUDY; MALE; OBSERVATIONAL STUDY; OUTCOME ASSESSMENT; PRIORITY JOURNAL; SCORING SYSTEM; SKINFOLD; SYSTEMIC SCLEROSIS; TREATMENT OUTCOME;

ADULT; AGED; COHORT STUDIES; DISEASE PROGRESSION; FEMALE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MIDDLE AGED; QUESTIONNAIRES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SCLERODERMA, DIFFUSE; SEVERITY OF ILLNESS INDEX; SKIN; TREATMENT OUTCOME;

EID: 73649119523     PISSN: 0315162X     EISSN: None     Source Type: Journal    
DOI: 10.3899/jrheum.090668     Document Type: Article

References (28)

1. LeRoy EC, Black C, Fleischmajer R, Jablonska S, Kreig T, Medsger TA, et al. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol 1988;15:202-5.
2. Denton CP, Black CM. Scleroderma - clinical and pathological advances. Best Pract Res Clin Rheumatol 2004;18:271-90.
3. Tashkin DP, Elashoff R, Clements PJ, Goldin J, Roth MD, Furst DE, et al. Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med 2006;354:2655-66.
4. Hoyles RK, Ellis RW, Wellsbury J, Lees B, Newlands P, Roberts C, et al. A multicenter, prospective, randomized, double-blind, placebo-controlled trial of corticosteroids and intravenous cyclophosphamide followed by oral azathioprine for the treatment of pulmonary fibrosis in scleroderma. Arthritis Rheum 2006;54:3962-70.
5. Jimenez SA, Derk CT. Following the molecular pathways toward an understanding of the pathogenesis of systemic sclerosis. Ann Intern Med 2004;140:37-50.
6. Charles C, Clements P, Furst DE. Systemic sclerosis: hypothesis-driven treatment strategies. Lancet 2006;367:1683-91.
7. Denton CP, Black CM. Scleroderma and related disorders: therapeutic aspects. Balliere's Clin Rheumatol 2000;14:17-35.
8. Black CM, Silman AJ, Herrick AL, Denton CP, Wilson H, Newman J, et al. Interferon-alpha does not improve outcome at one year in patients with diffuse cutaneous scleroderma: results of a randomized double-blind placebo-controlled trial. Arthritis Rheum 1999;42:299-305.
9. Clements PJ, Furst DE, Wong WK, Mayes M, White B, Wigley F, et al. High-dose versus low-dose D-penicillamine in early diffuse systemic sclerosis: analysis of a two-year, double-blind, randomized, controlled clinical trial. Arthritis Rheum 1999;42:1194-203.
10. Denton CP, Merkel PA, Furst DE, Khanna D, Emery P, Hsu VM, et al. Recombinant human anti-transforming growth factor ß1 antibody therapy in systemic sclerosis: a multicenter, randomized, placebo-controlled phase I/II trial of CAT-192. Arthritis Rheum 2007;56:323-33.
11. Pope JE, Bellamy N, Seibold JR, Baron M, Ellman M, Carette S, et al. A randomized, controlled trial of methotrexate versus placebo in early diffuse scleroderma. Arthritis Rheum 2001;44:1351-8.
12. Vonk MC, Marjanovic Z, van den Hoogen FH, Zohar S, Schattenberg AV, Fibbe WE, et al. Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis. Ann Rheum Dis 2008;67:98-104.
13. Nash RA, McSweeney PA, Crofford LJ, Abidi M, Chen C, Godwin JD, et al. High-dose immunosuppressive therapy and autologous hematopoetic cell transplantation for severe systemic sclerosis: long-term follow-up of the US multicenter pilot study. Blood 2007;110:1388-96.
14. Stratton RJ, Wilson H, Black CM. Pilot study of anti-thymocyte globulin plus mycophenolate mofetil in recent-onset diffuse scleroderma. Rheumatol 2001;40:84-8.
15. Masi AT, Rodnan GP, Medsger TA, Altman RD, D'Angelo WA, Fries JF, et al. Preliminary criteria for the classification of systemic sclerosis (scleroderma). Arthritis Rheum 1980;23:581-90.
16. Steen VD, Medsger TA. The value of the health assessment questionnaire and special patient-generated scales to demonstrate change in systemic sclerosis over time. Arthritis Rheum 1997;40:1984-91.
17. Silman A, Akesson A, Newman J, Henriksson H, Sandquist G, Nihill M, et al. Assessment of functional ability in patients with scleroderma: a proposed new disability assessment instrument. J Rheumatol 1998;25:79-83.
18. Clements P, Lachenbruch P, Siebold J, White B, Weiner S, Martin R, et al. Inter and intraobserver variability of total skin thickness score (modified Rodnan TSS) in systemic sclerosis. J Rheumatol 1995;22:1281-5.
19. Sato T, Matsuyama Y. Marginal structural models as a tool for standardization. Epidemiology 2003;14:680-6.
20. Imbens GW. The role of the propensity score in estimating dose-response functions. Biometrica 2000:87:706-10.
21. Glynn RJ, Schneeweiss S, Sturmer T. Indications for propensity scores and review of their use in pharmacoepidemiology. Basic Clin Pharmacol Toxicol 2006;98:253-9.
22. Royston P. Multiple imputation of missing values: update of ice. Stata J 2005;5:527-36.
23. van Buuren S, Boshuizen HC, Knook DL. Multiple imputation of missing blood pressure covariates in survival analysis. Stat Med 1999;18:681-94.
24. Rubin DB. Multiple imputation for non-response in surveys. New York: J. Wiley and Sons; 1987.
25. Rawlins MD. The Harveian Oration of 2008: On the evidence for decisions about the use of therapeutic interventions. London: Royal College of Physicians; 2008.
26. Avouac J, Kowal-Bielecka O, Landewé RB, Chwiesko S, Miniati I, Czirják L, et al. European League Against Rheumatism (EULAR) Scleroderma Trial and Research group (EUSTAR) recommendations for the treatment of systemic sclerosis: Methods of elaboration and results of systematic literature research. Ann Rheum Dis 2009;68:629-34.
27. Liossis SNC, Bounas A, Andonopoulos AP. Mycophenolate mofetil as first-line treatment improves clinically early scleroderma lung disease. Rheumatology 2006;45:1005-8.
28. Nihtyanova SI, Brough GM, Black CM, Denton CP. Mycophenolate mofetil in diffuse cutaneous systemic sclerosis - a retrospective analysis. Rheumatology 2007;46:442-5.