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目的 评估异基因造血干细胞移植治疗儿童血液病的疗效及合并症。方法 脐血移植(UCBT)治疗 9例重症 β地中海贫血 (β thal)、1例慢性特发性溶血性贫血 (CIHA)、3例急性髓性白血病(AML M3b、M2a、M5)及 1例急性淋巴细胞白血病 (ALL)合并中枢神经系统白血病 ;异基因外周血干细胞移植 (allo PBSCT)治疗 5例 β thal及 1例慢性粒细胞性白血病慢性期 (CML CP)患儿。同胞UCBT 8例中 5例HLA相合 (6 /6 ) ,5 /6为 1例 ,3/6为 2例。 6例非血缘相关脐血移植 (UD UCBT)中 4例为 6 /6 ,1例 5 /6 ,双份脐血混合者为 5 /6和 6 /6。allo PBSCT中 5例均为血缘相关 ,HLA相合同胞及 1例 5 /6父亲供者。输入脐血 (UCB)有核细胞数 (NC)为 7 5 (3 4～ 19 4)× 10 7/kg ,外周血NC为 9 39(2 5～14 4)× 10 8/kg。结果 血缘相关脐血移植 (RD UCBT) 8例中植入 6例 ,其中排斥 1例 ,死于肝静脉闭塞病 (HVOD) 1例 ,恢复地中海贫血状态 2例 ;UD UCBT中 2例 β thal及 2例AML均植入 ,1例AML M5复发 ,AML M3b自体恢复造血并完全缓解 ,1例AML M2a死于巨细胞病毒间质性肺炎 ,1例ALL未植入 ,死于败血症。急性移植物抗宿主病 (aGVHD) 8例 (80 % ) ,III度以上 2例 ,广泛慢性移植物抗宿主病(cGVHD 2 )例。β thal总生存率为 90 % ,无病存?
Objective To evaluate the efficacy and complications of allogeneic hematopoietic stem cell transplantation in children with hematological diseases. Methods UCBT was performed in 9 patients with β thalidomide, 1 with chronic idiopathic hemolytic anemia (CIHA), 3 with acute myeloid leukemia (AML M3b, M2a, M5) and 1 with acute Lymphocytic leukemia (ALL) combined with central nervous system leukemia; allo PBSCT for 5 cases of β thal and 1 case of chronic myeloid leukemia chronic phase (CML CP) children. Five of the 8 UCBT siblings had HLA matched (6/6), 5/6 as 1 and 3/6 as 2. Of the 6 unrelated umbilical cord blood transplants (UD UCBT), 4 were 6/6, 1 of 5/6, and 5/12 and 6/6 of double cord blood mixed. Allo PBSCT in 5 cases were blood related, HLA matched siblings and 1 case of 5/6 father donor. The number of nucleated cells (UC) in UCB was 75 (34 ~ 19 4) × 10 7 / kg and the peripheral blood NC was 9 39 (25 ~ 14 4) × 10 8 / kg. Results Among the 8 cases of RD UCBT, 6 cases were implanted, including 1 case of rejection, 1 case of hepatic veno-occlusive disease (HVOD) and 2 cases of thalassemia reversion. 2 cases of β thal and 2 cases of U UCBT 2 cases of AML were implanted, 1 case of AML M5 recurrence, AML M3b autologous hematopoietic recovery and complete remission, 1 case of AML M2a died of cytomegalovirus interstitial pneumonia, 1 case of ALL not implanted, died of sepsis. Eight cases (80%) of acute graft-versus-host disease (aGVHD), two cases of grade III or higher, and a group of patients with extensive chronic graft-versus-host disease (cGVHD 2). β thal total survival rate of 90%, disease-free survival?