异基因造血干细胞移植治疗骨髓增生异常综合征8例疗效分析

Therapeutic efficacy evaluation of allogeneic hematopoietic stem cell transplantation in 8 patients with myelodysplastic syndrome

关键词:  骨髓增生异常综合征  异基因造血干细胞移植  移植物抗宿主病  地西他滨

Key words:  myelodysplastic syndrome  allogeneic hematopoietic stem cell transplantation  GVHD  decitabine

摘要:

目的　观察异基因造血干细胞移植（allo-HSCT）治疗骨髓增生异常综合征（MDS）的疗效及安全性。方法　对8例MDS患者进行异基因造血干细胞移植，其中同胞全相合供者4例，亲缘间单倍型供者4例。预处理方案为改良BU/CY 4例，地西他滨桥接BU/CY 2例，FLU-IDA/FLU-BU 1例，FLU-BU/CY 1例，同胞全相合移植采集供者外周血造血干细胞，移植物抗宿主病（GVHD）预防给予环孢素A（CSA）加短程甲氨蝶呤（MTX）；血缘单倍型移植采集骨髓和外周血造血干细胞，GVHD预防采用CSA、短程MTX及吗替麦考酚酯（MMF），预处理期间加用抗胸腺细胞球蛋白（ATG）。随访时间1～13个月。结果　8例患者全部完成造血重建，顺利出仓。粒系植入时间为11.2 d，巨系植入时间为15.8 d，经STR-PCR检测均为完全供者型。Ⅰ～Ⅱ度急性移植物抗宿主病（aGVHD）3例，因自行停用抗排异药物导致Ⅳ度aGVHD并于移植后4个月死亡1例。慢性移植物抗宿主病（cGVHD）2例；移植后复发1例，死亡1例，存活6 例。结论　allo-HSCT治疗MDS安全有效，对中高危MDS可能是唯一有效的治疗方法，并宜尽早进行。

Abstract:

Objective　To evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) therapy in patients with myelodysplastic syndrome (MDS).Methods　The clinical characteristics and curative effect of 8 MDS patients who received allo-HSCT were retrospectively analyzed. Of them, 4 patients received HLA-identical-related donor hematopoietic transplantation (HIR-HSCT), while another 4 MDS patients received HLA-haploidentical-related donor hematopoietic transplantation (Haplo-HSCT). Regarding to the preparative regimen, 4 MDS patients underwent modified BU/CY, 2 with decitabine bridging modified BU/CY, 1 with FLU-IDA/FLU-BU, 1 with FLU-BU/CY. Patients with HIR-HSCT received peripheral blood stem cells only. Cyclosporine A and short term methotrexate (MTX) were used to prevent from graft versus-host disease (GVHD). Patients with Haplo-HSCT received stem cells from both bone marrow and peripheral blood. Mycophenolate mofetil (MMF) was further included in the indicated GVHD preventing regimen. During conditioning, Antithymocyte Globuin (ATG) was additionally used. The follow-up period was 13 months after HSCT.Results　All patients were successfully engrafted. Engraftment of granulocytes and megakaryocytes was 11.2 days and 15.8 days, respectively. Results of STR-PCR showed complete chimerism. The incidence of grade Ⅰ-Ⅱ acute graft-versus-host disease (aGVHD) was 3/8, and that of grade Ⅳ was 1/8 due to self-withdrawal of GVHD drugs in 4 months after HSCT. The cumulative incidence of chronic GVHD (cGVHD) was 2/8. The respective incidence of relapse and death was 1/8, while the survival rate was 6/8.Conclusion　Allo-HSCT is an effective and safe therapy for MDS, and especially it might be the only effective way for MDS patients at intermediate-high risk level.