Service de Médecine Interne, Hopital Saint Louis, Paris, France.
Autoimmunity. 2008 Dec;41(8):616-24. doi: 10.1080/08916930802197305.
Systemic sclerosis (SSc) is a generalised autoimmune disease, of yet unknown origin, with two major clinical subsets: the limited (lcSSc) and the diffuse cutaneous (dcSSc) forms, which can be distinguished by the extent of skin involvement, the autoantibody profile and the pattern of organ involvement. With an incidence of 1/10(5), SSc affects around 250,000 people in Europe and is responsible for significant morbidity with a 5-year mortality rate of at least 30% of all patients. In patients with rapidly progressive dcSSc, the 5-year mortality is estimated to be 40-50%. Hematopoietic stem cell transplantation (HSCT), mostly autologous but also allogeneic in some specific cases, has been employed worldwide since 1996 as a new therapeutic strategy in patients with a poor prognosis. In 2007, 150 HSCT procedures have been reported in the EBMT data base. We review herein both the short and the long-term reports from the various European and North American phase I-II studies, which have shown that autologous HSCT in selected patients with severe dcSSc results in sustained improvement of skin thickening and stabilisation of organ function up to seven years after transplantation. Based on these promising results, ongoing phase III trials have been designed in parallel, both in Europe (ASTIS) and in North America (SCOTT) aiming to analyse the respective benefits from autologous HSCT respectively without or with high dose irradiation. This review reports the current data concerning the effects of HSCT on survival, skin, and major organ function in patients with severe dcSSc.
系统性硬化症（SSc）是一种病因不明的全身性自身免疫性疾病，有两种主要的临床亚型：局限性（lcSSc）和弥漫性皮肤型（dcSSc），可通过皮肤受累的范围、自身抗体谱和器官受累的模式来区分。SSc 的发病率为 1/10(5)，在欧洲影响约 25 万人，发病率高，且具有很高的致残率，所有患者的 5 年死亡率至少为 30%。在快速进展性 dcSSc 患者中，估计 5 年死亡率为 40-50%。自 1996 年以来，造血干细胞移植（HSCT）作为一种新的治疗策略，已在全球范围内应用于预后不良的患者。2007 年，EBMT 数据库报告了 150 例 HSCT 手术。我们在此回顾了来自欧洲和北美的各种 I-II 期前瞻性研究的短期和长期报告，这些报告表明，在选择的严重 dcSSc 患者中进行自体 HSCT，可使皮肤增厚持续改善，并在移植后 7 年内稳定器官功能。基于这些有希望的结果，目前正在欧洲（ASTIS）和北美（SCOTT）进行 III 期试验，旨在分析自体 HSCT 分别在无或高剂量照射下对患者生存、皮肤和主要器官功能的各自获益。本综述报告了关于 HSCT 对严重 dcSSc 患者生存、皮肤和主要器官功能影响的最新数据。
