• Hiroyuki Nakamura, Toshio Odani, Shinsuke Yasuda, Atsushi Noguchi, Yuichiro Fujieda, Masaru Kato, Kenji Oku, Toshiyuki Bohgaki, Junichi Sugita, Tomoyuki Endo, Takanori Teshima, and Tatsuya Atsumi.
• a Department of Rheumatology, Endocrinology and Nephrology, Faculty of Medicine and Graduate School of Medicine , Hokkaido University , Sapporo , Japan.
• Mod Rheumatol. 2018 Sep 1; 28 (5): 879-884.

ObjectivesThe objective of this study is to elucidate the efficacy and safety of autologous haematopoietic stem cell transplantation (HSCT) for Japanese patients with systemic sclerosis (SSc).MethodsA phase II clinical trial included SSc patients diagnosed within the last three years having at least one of the following clinical features: diffuse skin sclerosis with modified Rodman total thickness skin score (mRSS) ≥ 15, refractory digital ulcer or interstitial lung disease (ILD). HSCT were performed after conditioning using cyclophosphamide.ResultsFourteen patients were enrolled and underwent HSCT. Median follow-up period was 137 months. Overall survival or event-free survival rate was 93% or 40% at 10 years, respectively. Eight patients (57%) achieved more than a 50% decrease in mRSS from baseline within six months after HSCT. Six patients (43%) required additional immunosuppressive treatments due to progression of diffuse skin sclerosis and/or ILD during follow-up period. Adverse events related to HSCT occurred in six patients (43%). Severe cardiomyopathy occurred in two patients, and one of them had a fatal course.ConclusionHSCT is a feasible treatment bringing favourable results to more than half of our patients with SSc. Careful selection of the patients is essential for whom benefited from HSCT, considering the risk-benefit balance of the treatment.

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