Mycophenolate Mofetil Versus Placebo for Systemic Sclerosis–Related Interstitial Lung Disease: An Analysis of Scleroderma Lung Studies I and II

Arthritis and Rheumatology

Volumn 69, Issue 7, 2017, Pages 1451-1460

  Volkmann, Elizabeth R ^a   Tashkin, Donald P ^a   Li, Ning ^a   Roth, Michael D ^a   Khanna, Dinesh ^b   Hoffmann Vold, Anna Maria ^c   Kim, Grace ^a   Goldin, Jonathan ^a   Clements, Philip J ^a   Furst, Daniel E ^a   Elashoff, Robert M ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^c OSLO UNIVERSITY HOSPITAL   (Norway)

Author keywords

[No Author keywords available]

Indexed keywords

CARBON MONOXIDE; CYCLOPHOSPHAMIDE; MYCOPHENOLATE MOFETIL; IMMUNOSUPPRESSIVE AGENT; MYCOPHENOLIC ACID;

ADULT; AGED; ANEMIA; ARTICLE; CONTROLLED STUDY; DISEASE DURATION; DISEASE SEVERITY; DRUG DOSE TITRATION; DRUG EFFICACY; DRUG SAFETY; DRUG TREATMENT FAILURE; DYSPNEA; FEMALE; FOLLOW UP; FORCED VITAL CAPACITY; HEALTH ASSESSMENT QUESTIONNAIRE; HEMATURIA; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INTENTION TO TREAT ANALYSIS; INTERSTITIAL LUNG DISEASE; LEUKOPENIA; LUNG DIFFUSION CAPACITY; MAJOR CLINICAL STUDY; MALE; MINIMAL CLINICALLY IMPORTANT DIFFERENCE; MODIFIED RODNAN SKIN THICKNESS SCORE; NEUTROPENIA; PATIENT SAFETY; PNEUMONIA; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SCORING SYSTEM; SECONDARY ANALYSIS; SHORT FORM 36; SIDE EFFECT; SKIN DISEASE ASSESSMENT; SYSTEMIC SCLEROSIS; THERAPY EFFECT; THROMBOCYTOPENIA; COMPARATIVE STUDY; COMPLICATION; LUNG DISEASES, INTERSTITIAL; MIDDLE AGED; PATHOPHYSIOLOGY; RANDOMIZED CONTROLLED TRIAL (TOPIC); SCLERODERMA, SYSTEMIC; SKIN; TREATMENT OUTCOME; VERY ELDERLY; VITAL CAPACITY; YOUNG ADULT;

ADULT; AGED; AGED, 80 AND OVER; CYCLOPHOSPHAMIDE; DYSPNEA; FEMALE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; LUNG DISEASES, INTERSTITIAL; MALE; MIDDLE AGED; MYCOPHENOLIC ACID; PULMONARY DIFFUSING CAPACITY; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SCLERODERMA, SYSTEMIC; SKIN; TREATMENT OUTCOME; VITAL CAPACITY; YOUNG ADULT;

EID: 85021382806     PISSN: 23265191     EISSN: 23265205     Source Type: Journal    
DOI: 10.1002/art.40114     Document Type: Article

References (25)

1. Elhai M, Meune C, Avouac J, Kahan A, Allanore Y. Trends in mortality in patients with systemic sclerosis over 40 years: a systematic review and meta-analysis of cohort studies. Rheumatology (Oxford) 2012;51:1017–26.
2. Tyndall AJ, Bannert B, Vonk M, Airo P, Cozzi F, Carreira PE, et al. Causes and risk factors for death in systemic sclerosis: a study from the EULAR Scleroderma Trials and Research (EUSTAR) database. Ann Rheum Dis 2010;69:1809–15.
3. Tashkin DP, Elashoff R, Clements PJ, Goldin J, Roth M, 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, Goh NS, 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. Volkmann ER, Tashkin DP. Treatment of systemic sclerosis-related interstitial lung disease: a review of existing and emerging therapies. Ann Am Thorac Soc 2016;13:2045–56.
6. Nihtyanova SI, Brough GM, Black CM, Denton CP. Mycophenolate mofetil in diffuse cutaneous systemic sclerosis: a retrospective analysis. Rheumatology (Oxford) 2007;46:442–5.
7. Derk CT, Grace E, Shenin M, Naik M, Schulz S, Xiong W. A prospective open-label study of mycophenolate mofetil for the treatment of diffuse systemic sclerosis. Rheumatology (Oxford) 2009;48:1595–9.
8. Gerbino AJ, Goss CH, Molitor JA. Effect of mycophenolate mofetil on pulmonary function in scleroderma-associated interstitial lung disease. Chest 2008;133:455–60.
9. Simeón-Aznar CP, Fonollosa-Plá V, Tolosa-Vilella C, Selva-O'Callaghan A, Solans-Laque R, Vilardell-Tarres M. Effect of mycophenolate sodium in scleroderma-related interstitial lung disease. Clin Rheumatol 2011;30:1393.
10. Swigris JJ, Olson AL, Fischer A, Lynch DA, Cosgrove GP, Frankel SK, et al. Mycophenolate mofetil is safe, well tolerated and preserves lung function in patients with connective tissue disease-related interstitial lung disease. Chest 2006;130:30–6.
11. Zamora AC, Wolters PJ, Collard HR, Connolly MK, Elicker BM, Webb WR, et al. Use of mycophenolate mofetil to treat scleroderma-associated interstitial lung disease. Respir Med 2008;102:150–55.
12. Fischer A, Brown KK, Du Bois RM, Frankel SK, Cosgrove GP, Fernandez-Perez ER, et al. Mycophenolate mofetil improves lung function in connective tissue disease-associated interstitial lung disease. J Rheumatol 2013;40:640–6.
13. Tashkin DP, Roth MD, Clements PJ, Furst DE, Khanna D, Kleerup EC, et al. Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomised controlled, double-blind, parallel group trial. Lancet Respir Med 2016;4:708–19.
14. Wells AU. Interstitial lung disease in systemic sclerosis. Press Med 2014;43:e329–e343.
15. Tashkin DP, Elashoff R, Clements PJ, Roth MD, Furst DE, Silver RM, et al. Effects of 1-year treatment with cyclophosphamide on outcomes at 2 years in scleroderma lung disease. Am J Respir Crit Care Med 2007;176:1026–34.
16. Mahler DA, Weinberg DH, Wells CK, Feinstein AR. The measurement of dyspnea: contents, interobserver agreement and physiologic correlates of two new clinical indexes. Chest 1984;85:751–8.
17. Mahler DA, Ward J, Fierro-Carrion G, Waterman LA, Lentine TF, Mejia-Alfaro R, et al. Development of self-administered versions of modified baseline and transition dyspnea indexes in COPD. COPD 2004;1:1–8.
18. Clements PJ, Lachenbruch PA, Seibold JR, Zee B, Steen VD, Brennan P, et al. Skin thickness score in systemic sclerosis (SSc): an assessment of inter-observer variability in three independent studies. J Rheumatol 1993;20:1892–6.
19. Goldin J, Elashoff R, Kim HJ, Yan X, Lynch D, Strollo D, et al. Treatment of scleroderma-interstitial lung disease with cyclophosphamide is associated with less progressive fibrosis on serial thoracic high-resolution CT scan than placebo: findings from the scleroderma lung study. Chest 2009;136:1333–40.
20. Kim HJ, Li G, Gjertson D, Elashoff R, Shah SK, Ochs R, et al. Classification of parenchymal abnormality in scleroderma lung using a novel approach to denoise images collected via a multicenter study. Acad Radiol 2008;15:1004–16.
21. Li N, Elashoff RM, Li G, Tseng CH. Joint analysis of bivariate longitudinal ordinal outcomes and competing risks survival times with nonparametric distributions for random effects. Stat Med 2012;31:1707–21.
22. Elashoff RM, Li G, Li N. A joint model for longitudinal measurements and survival data in the presence of multiple failure types. Biometrics 2008;64:762–71.
23. Khanna D, Tseng CH, Furst DE, Clements PJ, Elashoff R, Roth M, et al. Minimally important differences in the Mahler's Transition Dyspnoea Index in a large randomized controlled trial: results from the Scleroderma Lung Study. Rheumatology (Oxford) 2009;48:1537–40.
24. Steen VD, Lanz JK Jr, Conte C, Owens GR, Medsger TA Jr. Therapy for severe interstitial lung disease in systemic sclerosis. a retrospective study. Arthritis Rheum 1994;37:1290–6.
25. Clements PJ, Medsger TA Jr, Feghali CA. Cutaneous involvement in systemic sclerosis. In: Clements PJ, Furst DE, editors. Systemic sclerosis. 2nd ed. New York: Lippincott Williams and Wilkins; 2004. p. 129–50.