摘要
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 clinical efficacy and complications of allo geneic hematopoietic stem cell transplant (allo HSCT) for children with hematologic diseases Methods Nine patients with β thalassemia major (β thal),1 patient with chronic idiopathic hamolytic anemia (CIHA) and 4 patients with acute leukemia accepted umbilical blood transplant (UCBT) Five cases of β thal and 1 case of chronic myelogenous leukemia(CML) accepted allo peripheral blood stem cell transplant (allo PBSCT) In UCBT treated cases (11 male and 3 female cases at the median age of 5 5 years), 8 cases (7 cases of β Thal and 1 case of CIHA )accepted UCBT from siblings and another 6 cases (2 cases of β Thal and 4 cases of acute leukemia ) accepted unrelated donor UCBT (UD UCBT) In allo PBSCT treated cases, 3 were male and 3 were female Their median age was 6 years and 2 months In 8 cases who accepted UCBT from siblings, 5 cases were HLA identical (6/6), One was 1 HLA antigen mismatched (5/6) and 2 cases were 3 HLA antigen mismatched (3/6) In 6 cases who accepted UD UCBT, 4 cases were HLA identical, one was 5/6 and the other one received 2 units of UCB from 6/6 and 5/6 donors Of the 6 patients who accepted allo PBSCT, 5 donors were HLA 6/6 siblings and one 5/6 parent The conditioning regimen including busalphan (BU), cyclophosphamide (CY) and ATG was given to the 18 patients Melphlan (melph) was added to 10 cases accepting UCBT and 2 allo PBSCT, and Fludarabine (Fluda) was added to five cases accepting UD UCBT and 3 allo PBSCT Thiotepa(TT) was added to 3 cases of β thal, TBI(7 5Gy)+Fluda+Melph+ATG to 1 case of AML M 2a , and Cy+Melph+Fluda+IDA(30 mg/m 2) to 1 case of ALL All patients received cyclosporine alone or combined with methotrexate, FK506 and prednisone for graft versus host desease (GVHD) prophylactic therapy G CSF started from day+1 until engraftment UCB NC infused was 7 5×10 7/kg (range, 3 4 to 9 4×10 7/kg), CFU GM 1 16×10 5/kg (range, 0 24 to 23 00×10 5/kg),CD34+ cells 2 98×10 5/kg (range, 0 6 to 11 7×10 5/kg) In the PBSCT treated cases, NC was 9 39×10 8/kg (range, 2 5 to 14 4×10 8/kg) Results Ten were engrafted in 13 evaluated cases accepting UCBT, but one rejected 2 months after UCBT Two cases of β thal and one case of AML(M3a) had autologous reconstitution The median time to reach an absolute neutrophil count (ANC)≥0 5×10 9/L was 17 4 days(range, 10 22 days), platelet≥20×10 9/L was 35 days (range, 19 106 days), independent infusion of red blood cells was 34 days in β thal and CIHA Eight patients developed acute GVHD (80%), 2 of whom were above grade III Extensive chronic GVHD was observed in 2 patients One case of β thal died from hepatic veno occlusive disease (HVOD), 2 patients with AML were engrafted and had complete remission(CR) But one of them relapsed 53 days after UCBT and one died from CMV interstitial pneumonia at 2 months of post UCBT Total survival and disease free survival (DFS) of β thal was 90%(9/10) and 60%(6/10), respectively The median survival time was 21 months (2 42months) All patients treated with allo PBSCT were engrafted The time to reach ANC ≥0 5×10 9/L was 11 5 days (range, 10 13 days), PLT≥20×10 9/L was 33 days (range,11 92 days), and independent infusion of RBC was 35 3 days There were 5 patients with aGVHD, 2 of whom had aGVHD of above grade III and 3 had extensive cGVHD One died from HVOD Median survival time was 10 months (3 26 months) Conclusion UCBT and allo PBSC are of value in treating hematological diseases in children In particular, the β thal was firstly treated with UD UCBT in this study
出处
《中华儿科杂志》
CAS
CSCD
北大核心
2001年第10期583-587,共5页
Chinese Journal of Pediatrics
基金
美国中华医学基金 (MB)资助项目 ( 96 6 30 )
关键词
移植
同种
造血干细胞移植
血液病
儿童
Transplantation, homologous
Hematopoietic stem cell transplantation
Hematologic diseases
