Alexander T, Henes J, Distler J H W, Schmalzing M, Blank N, Kötter I, Hiepe F
Medizinische Klinik m.S. Rheumatologie und Klinische Immunologie, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Charitéplatz 1, 10117, Berlin, Deutschland.
Deutsches Rheuma-Forschungszentrum (DRFZ) Berlin - ein Institut der Leibniz-Gemeinschaft, Berlin, Deutschland.
Z Rheumatol. 2020 Jun;79(5):429-436. doi: 10.1007/s00393-020-00789-0.
There have been three randomized controlled trials on autologous hematopoietic stem cell transplantation (AHSCT) in systemic sclerosis (SSc) that demonstrated significant superiority with respect to survival, improvement of cutaneous fibrosis, lung function and quality of life compared to standard treatment; however, these advantages must be carefully weighed against the transplantation-related risks. For this reason, an expert group from the stem cell therapy working party of the German Society for Rheumatology (DGRh) has now developed recommendations for the use of AHSCT in SSc. Based on the high-quality evidence, AHSCT is considered as the standard option for the treatment of selected SSc patients. Potential candidates for AHSCT are those with early, rapidly progressive, diffuse cutaneous SSc with visceral manifestations who have not yet developed severe damage to internal organs. A close cooperation between rheumatologists and transplantation centers is crucial for optimizing patient selection and treatment outcomes.
关于系统性硬化症（SSc）的自体造血干细胞移植（AHSCT）已有三项随机对照试验，结果表明与标准治疗相比，AHSCT在生存、皮肤纤维化改善、肺功能和生活质量方面具有显著优势；然而，这些优势必须与移植相关风险仔细权衡。因此，德国风湿病学会（DGRh）干细胞治疗工作组的一个专家组现已制定了SSc中使用AHSCT的建议。基于高质量证据，AHSCT被视为治疗选定SSc患者的标准选择。AHSCT的潜在候选者是那些早期、快速进展、有内脏表现的弥漫性皮肤SSc患者，且尚未对内脏器官造成严重损害。风湿病学家和移植中心之间的密切合作对于优化患者选择和治疗结果至关重要。
