自体造血干细胞移植治疗导致心肌坏死的难治性抗磷脂综合征。

Autologous hematopoietic stem cell transplantation for refractory antiphospholipid syndrome causing myocardial necrosis.

作者信息

Hashimoto N, Iwasaki T, Sekiguchi M, Takatsuka H, Okamoto T, Hashimoto T, Sano H

机构信息

Division of Rheumatology and Clinical Immunology, Department of Internal Medicine, Hyogo College of Medicine, Nishinomiya, Hyogo, Japan.

出版信息

Bone Marrow Transplant. 2004 Apr;33(8):863-6. doi: 10.1038/sj.bmt.1704432.

DOI:10.1038/sj.bmt.1704432

PMID:14755313

Abstract

Autologous hematopoietic stem cell transplantation (HSCT) is currently being evaluated as a treatment for autoimmune diseases, including systemic lupus erythematosus (SLE), that are associated with a very severe prognosis. We describe a 27-year-old woman with SLE with a 10-year history of refractory antiphospholipid syndrome (APS). She developed progressive myocardial necrosis despite treatment with corticosteroids, cyclophosphamide (CYC), cyclosporine, and immunopheresis. After conditioning with CYC, fludarabine, and antithymocyte globulin, autologous HSCT using CD34(+) selection was performed. After transplantation, the clinical symptoms caused by APS remitted, and the serum anticardiolipin antibody level decreased. Remission has persisted for 21 months after transplantation. Although a longer follow-up is required for the assessment of efficacy, autologous HSCT may cure patients with refractory APS.

摘要

自体造血干细胞移植（HSCT）目前正在作为一种治疗自身免疫性疾病的方法进行评估，这些自身免疫性疾病包括预后非常严重的系统性红斑狼疮（SLE）。我们描述了一名27岁患有SLE且有10年难治性抗磷脂综合征（APS）病史的女性。尽管接受了皮质类固醇、环磷酰胺（CYC）、环孢素和免疫吸附治疗，她仍出现了进行性心肌坏死。在用CYC、氟达拉滨和抗胸腺细胞球蛋白进行预处理后，采用CD34(+)选择进行了自体HSCT。移植后，由APS引起的临床症状缓解，血清抗心磷脂抗体水平下降。移植后缓解已持续21个月。尽管需要更长时间的随访来评估疗效，但自体HSCT可能治愈难治性APS患者。