Department of Internal Medicine, Unité Clinique de Médecine Interne, Maladies Auto-immunes et Pathologie Vasculaire, UF 04, Hôpital Saint-Louis, AP-HP Assistance Publique des Hôpitaux de Paris, INSERM UMRS 1160, Paris Denis Diderot University, Paris, France.
Department of Medicine, Division of Immunotherapy, Northwestern University, Chicago, IL, USA.
Bone Marrow Transplant. 2017 Nov;52(11):1495-1503. doi: 10.1038/bmt.2017.56. Epub 2017 May 22.
Systemic sclerosis (SSc) is a rare disabling autoimmune disease with a similar mortality to many cancers. Two randomized controlled trials of autologous hematopoietic stem cell transplantation (AHSCT) for SSc have shown significant improvement in organ function, quality of life and long-term survival compared to standard therapy. However, transplant-related mortality (TRM) ranged from 3-10% in patients undergoing HSCT. In SSc, the main cause of non-transplant and TRM is cardiac related. We therefore updated the previously published guidelines for cardiac evaluation, which should be performed in dedicated centers with expertize in HSCT for SSc. The current recommendations are based on pre-transplant cardiopulmonary evaluations combining pulmonary function tests, echocardiography, cardiac magnetic resonance imaging and invasive hemodynamic testing, initiated at Northwestern University (Chicago) and subsequently discussed and endorsed within the EBMT ADWP in 2016.
系统性硬化症（SSc）是一种罕见的致残自身免疫性疾病，其死亡率与许多癌症相当。两项自体造血干细胞移植（AHSCT）治疗 SSc 的随机对照试验显示，与标准治疗相比，器官功能、生活质量和长期生存率均有显著改善。然而，接受 HSCT 的患者的移植相关死亡率（TRM）在 3-10%之间。在 SSc 中，非移植和 TRM 的主要原因与心脏有关。因此，我们更新了之前发布的心脏评估指南，该指南应在具有 SSc 造血干细胞移植专业知识的专门中心进行。目前的建议基于移植前心肺评估，包括肺功能测试、超声心动图、心脏磁共振成像和有创血流动力学测试，这些评估由西北大学（芝加哥）发起，随后在 2016 年由 EBMT ADWP 进行讨论和认可。
