无血缘关系脐血移植治疗血液系统恶性疾病的临床研究

Unrelated cord blood transplantation for the treatment of hematological malignancies

目的 观察无血缘脐血移植 (UCBT)治疗儿童和成人血液系统恶性疾病的植入率、移植物抗宿主病 (GVHD)的发生和生存情况。方法 血液系统恶性疾病患者 17例 ,其中儿童 13例 ,成人 4例。 12例接受单份脐血 (CB) ,5例接受 2份CB。脐血与受者HLA 6个位点相合 6例 ,4～ 5个位点相合11例。 10例患者移植时处于疾病稳定期 ,7例处于复发或难治白血病阶段。预处理多数采用BU CY或CY TBI方案 ,同时应用抗胸腺细胞球蛋白。GVHD预防采用环孢菌素A(CsA)为主的方案。结果 14例存活超过 4 0d的患者进行了脐血植入率的评价。至移植后 6 0天 ,12例 (86 % )患者白细胞达到了植入标准 ,植活时间为 (2 1.0± 1.3)d ;10例 (71% )血小板达到植活水平 ,植活时间为 (39.0± 10 .3)d。 4例患者发生Ⅱ度急性GVHD ,2例发生慢性GVHD。 17例患者 11例存活 ,其中无事件生存 (EFS) 8例。10例处于疾病稳定期患者 ,2年总生存概率 (OS)为 90 % ,无事件生存率 (EFS)为 70 % ;7例处于复发或难治白血病阶段的患者 ,只有 2例存活而无复发。 4例成人患者 2例达到了持久植入 ,目前EFS分别为 18和 14个月。结论 初步结果表明 ,HLA相合及 1～ 2位点不合的UCBT是可行的 ,尤其是移植前处于疾病稳定状态的患者。对于 1份脐血有核细胞不足的成人患者

Objectives To investigate the engraftment, survival and graft versus host disease (GVHD) after transplantation of unrelated cord blood for the treatment of childhood and adult hematological malignancies. Methods Seventeen patients (13 children and 4 adults) with hematological malignancies were enrolled in this study. Twelve patients were transplanted with one unit and 5 with 2 units of cord blood. There were HLA matched in 6 and HLA mismatched at 1～2 loci in 11 patients. Ten patients were transplanted at stable status, 7 at advanced stage of leukemia. Conditioning regimens were BU/CY for 13 and CY/TBI for 3 patients. Most patients received additional ATG at a dose of 15～20 mg·kg -1 ·d -1 for 3 days. CsA, mycophenolate mofetil (MMF) and methylprednisolone were used for GVHD prophylaxis. Results Fourteen patients survived more than 40 days after transplantation were evaluated for engraftment. At day 60 after UCBT, 86% and 71% of the patients showed neutrophil and platelet engraftment, respectively. The time for an absolute neutrophil count≥0.5×10 9/L was (21.0±1.3) days and platelet≥20×10 9/L was (39.0±10.3) days. Four patients developed grade Ⅱ acute GVHD and 2 chronic GVHD. Of the 17 patients, 11 were still alive and 8 of them were in event free status. For the 10 patients transplanted at stable status, 2 year overall survival is 90%, and event free survival (EFS) 70%. However, for the 7 patients transplanted at advanced stage of leukemia, only 2 survived without relapse. Of the 4 adult patients, 2 had sustained engraftment and survived for 18 and 14 months, respectively. Conclusions HLA matched or 1～2 loci mismatched UCBT is a feasible procedure to cure a significant proportion of children or adults with leukemia, especially if performed in a favourable phase of disease. Two units of CBT can be used for adult patients if the cell number of one unit is not enough.