Haemopoietic stem-cell transplantation for systemic sclerosis – Authors' reply

There were no deaths in either treatment group, which is very unusual in severe systemic sclerosis and suggests either insufficient duration of follow-up or selection of patients with less severe disease. [...] meaningful improve ments in severe systemic sclerosis have been reported beyond 24 months...

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Published in:The Lancet (British edition) 2012, Vol.379 (9812), p.219-220
Main Authors: Burt, Richard K, Gheorghiade, Mahai, Shah, Sanjiv, Ruderman, Eric, Schroeder, James
Format: Article
Language:English
Subjects:
Autoimmune diseases
Disease
Drug dosages
Immune system
Internal Medicine
Rheumatology
Scleroderma
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Summary:There were no deaths in either treatment group, which is very unusual in severe systemic sclerosis and suggests either insufficient duration of follow-up or selection of patients with less severe disease. [...] meaningful improve ments in severe systemic sclerosis have been reported beyond 24 months after HSCT.2 As Burt and colleagues note, the multicentre ASTIS and SCOT trials are currently maturing to reach sufficient follow-up and numbers of patients to address these concerns. [...] Sullivan and colleagues' criticism of defining disease pro gression on the basis of only Rodnan skin score (mRSS) and forced vital capacity (FVC) endpoints overlooks the fact that the endpoints mRSS, FVC, high-resolution CT imaging of in volved lung, and 36-item Short Form Health Survey (SF-36) all changed equally in our study.1 Indeed, this endpoint critique would be applicable to previous studies in systemic sclerosis, including the Scleroderma Lung Research Study Group trial,2 which reported "modest benefit" of daily oral cyclophosphamide on the basis of a mean FVC that in reality deteriorated on cyclophosphamide treatment (but declined less rapidly when compared with placebo).
ISSN:0140-6736
1474-547X
DOI:10.1016/S0140-6736(12)60101-9