Saccardi R, Tyndall A, Coghlan G, Denton C, Edan G, Emdin M, Farge D, Fassas A, Finke J, Furst D, Lassus M, Mancardi G, Miniati I, Mini E, Pagliai F, Passweg J, Pignone A, van Laar J M, Bocelli-Tyndall C, Matucci-Cerinic M
Haematology Unit, University of Florence, Italy.
Bone Marrow Transplant. 2004 Nov;34(10):877-81. doi: 10.1038/sj.bmt.1704656.
Autologous haematopoietic stem cell transplantation is now a feasible and effective treatment for selected patients with severe autoimmune diseases. Worldwide, over 650 patients have been transplanted in the context of phase I and II clinical trials. The results are encouraging enough to begin randomised phase III trials. However, as predicted, significant transplant-related morbidity and mortality have been observed. This is primarily due to complications related to either the stage of the disease at transplant or due to infections. The number of deaths related to cardiac toxicity is low. However, caution is required when cyclophosphamide or anthracyclines such as mitoxantrone are used in patients with a possible underlying heart damage, for example, systemic sclerosis patients. In November 2002, a meeting was held in Florence, bringing together a number of experts in various fields, including rheumatology, cardiology, neurology, pharmacology and transplantation medicine. The object of the meeting was to analyse existing data, both published or available, in the European Group for Blood and Marrow Transplantation autoimmune disease database, and to propose a safe approach to such patients. A full cardiological assessment before and during the transplant emerged as the major recommendation.
自体造血干细胞移植目前是治疗部分重症自身免疫性疾病患者的一种可行且有效的方法。在全球范围内，已有超过650名患者在I期和II期临床试验中接受了移植。这些结果足以令人鼓舞，从而启动随机III期试验。然而，正如所预测的那样，已观察到显著的与移植相关的发病率和死亡率。这主要是由于与移植时疾病阶段相关的并发症或感染所致。与心脏毒性相关的死亡人数较少。然而，对于可能存在潜在心脏损害的患者，如系统性硬化症患者，在使用环磷酰胺或米托蒽醌等蒽环类药物时需要谨慎。2002年11月，在佛罗伦萨召开了一次会议，汇聚了包括风湿病学、心脏病学、神经病学、药理学和移植医学等多个领域的专家。会议的目的是分析欧洲血液和骨髓移植组自身免疫性疾病数据库中已发表或可得的现有数据，并为这类患者提出一种安全的治疗方法。移植前和移植期间进行全面的心脏评估成为主要建议。
