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HLA 单倍体相合骨髓移植联合移植后环磷酰胺可扩大镰状细胞病患者的供者池。

HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease.

机构信息

Hematologic Malignancies and Bone Marrow Transplantation Program, Department of Oncology, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins and Johns Hopkins University School of Medicine, Baltimore, MD 21231-1000, USA.

出版信息

Blood. 2012 Nov 22;120(22):4285-91. doi: 10.1182/blood-2012-07-438408. Epub 2012 Sep 6.

Abstract

Allogeneic marrow transplantation can cure sickle cell disease; however, HLA-matched donors are difficult to find, and the toxicities of myeloablative conditioning are prohibitive for most adults with this disease. We developed a nonmyeloablative bone marrow transplantation platform using related, including HLA-haploidentical, donors for patients with sickle cell disease. The regimen consisted of antithymocyte globulin, fludarabine, cyclophosphamide, and total body irradiation, and graft-versus-host disease prophylaxis with posttransplantation high-dose cyclophosphamide, mycophenolate mofetil, and tacrolimus or sirolimus. After screening 19 patients, we transplanted 17, 14 from HLA-haploidentical and 3 from HLA-matched related donors. Eleven patients engrafted durably. With a median follow-up of 711 days (minimal follow up 224 days), 10 patients are asymptomatic, and 6 patients are off immunosupression. Only 1 patient developed skin-only acute graft-versus-host disease that resolved without any therapy; no mortality was seen. Nonmyeloablative conditioning with posttransplantation high-dose cyclophosphamide expands the donor pool, making marrow transplantation feasible for most patients with sickle cell disease, and is associated with a low risk of complications, even with haploidentical related donors. Graft failure, 43% in haploidentical pairs, remains a major obstacle but may be acceptable in a fraction of patients if the majority can be cured without serious toxicities.

摘要

同种异体骨髓移植可治愈镰状细胞病;然而,HLA 匹配的供体难以找到,而且大多数患有这种疾病的成年人都无法承受清髓性预处理的毒性。我们为镰状细胞病患者开发了一种非清髓性骨髓移植平台,使用相关的供体,包括 HLA 单倍体不全相合供体。该方案包括抗胸腺细胞球蛋白、氟达拉滨、环磷酰胺和全身照射,以及用移植后高剂量环磷酰胺、霉酚酸酯和他克莫司或西罗莫司进行移植物抗宿主病预防。在筛选了 19 名患者后,我们移植了 17 名患者,其中 14 名来自 HLA 单倍体不全相合供体,3 名来自 HLA 匹配的相关供体。11 名患者持久植入。中位随访 711 天(最小随访 224 天),10 名患者无症状,6 名患者停用免疫抑制剂。只有 1 名患者发生仅有皮肤的急性移植物抗宿主病,未经任何治疗即缓解;未观察到死亡率。移植后高剂量环磷酰胺的非清髓性预处理扩大了供体库,使骨髓移植对大多数镰状细胞病患者变得可行,且并发症风险低,即使是与单倍体不全相合相关供体。植入失败(在单倍体不全相合对中占 43%)仍然是一个主要障碍,但如果大多数患者可以在没有严重毒性的情况下治愈,那么对于一部分患者来说,这可能是可以接受的。

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