詹丽萍?阙丽萍?陈晗?吴正宙?刘典典?王开美?徐宏贵?方建培?黄科

【摘要】目的 了解自体脐血造血干细胞移植（ACBT）治疗儿童获得性再生障碍性贫血（AA）的疗效。方法 对接受ACBT治疗的3例获得性AA患儿的临床资料进行回顾性分析，其中男2例、女1例。以“再生障碍性贫血”和“自体脐血造血干细胞移植”（包括中英文）为关键词，对以下数据库的相关论文进行检索：PubMed、Web of Science、CNKI、万方数据知识服务平台，收集检索到的资料并进行分析。结果 3例患儿均成功获得造血重建，移植1年后骨髓均未见异常染色体克隆。病例1、2接受移植后继发血细胞减少，调整免疫抑制治疗方案后，血象均恢复正常，分别于移植后18个月、15个月停用免疫抑制剂。病例3接受移植后血象平稳，移植12个月顺利停用免疫抑制剂。3例患儿移植后540 d CD3+T淋巴細胞、CD3+CD4+T淋巴细胞、CD3+CD8+T淋巴细胞绝对计数均逐渐达到或者接近正常水平。检索到11例接受ACBT治疗的获得性AA患儿，其中8例于ACBT后使用了免疫抑制剂，10例病情完全缓解，1例首次ACBT后无使用免疫抑制剂，在接受了二次ACBT后死亡。结论 ACBT是治疗儿童获得性AA的安全有效方法。ACBT后适当应用免疫抑制剂有助于机体造血及免疫重建的稳定。

【关键词】儿童;再生障碍性贫血;自体脐血造血干细胞移植;免疫抑制;免疫重建

Successful autologous cord blood transplantation in 3 children with acquired aplastic anemia： case report and literature review Zhan Liping， Que Liping， Chen Han， Wu Zhengzhou， Liu Diandian， Wang Kaimei， Xu Honggui， Fang Jianpei， Huang Ke. Department of Pediatrics， Sun Yat-sen Memorial Hospital， Sun Yat-sen University， Guangzhou 510120， China Corresponding author， Huang Ke， E-mail： hke@mail. sysu. edu. cn

【Abstract】Objective To evaluate clinical efficacy of autologous cord blood transplantation （ACBT） in the treatment of pediatric acquired aplastic anemia （AA）. Methods Clinical data of three children with acquired AA， 2 male and 1 female， who received ACBT were retrospectively analyzed.Using the keywords of “aplastic anemia” and “autologous cord blood transplantation”， relevant literatures were searched from PubMed， Web of Science， CNKI and Wanfang databases， data were collected and analyzed. Results Successful hematopoietic reconstitution was performed in all three patients. No abnormal chromosomal clones were found after 1-year ACBT. Case 1 and case 2 suffered from cytopenia following ACBT. After adjusting the immunosuppressive therapy， hematopoiesis returned to normal. Both of them received post-transplantation immunosuppression therapy for 18 and 15 months. Case 3 achieved normal persistent hematopoiesis and received routine full-dose  administration of cyclosporine A for 6 months， followed by a 6-month taper. The absolute count of CD3+T cells， CD3+CD4+T cells and CD3+CD8+T cells reached or approached normal levels at 540 d after ACBT. Eleven children with acquired AA undergoing ACBT were retrieved. Eight of them were treated with immunosuppressants after ACBT， 10 cases achieved complete remission， and 1 child died after secondary ACBT without use of immunosuppressants after the initial ACBT. Conclusions ACBT is a safe and efficacious treatment for pediatric acquired AA. Appropriate post-transplantation immunosuppression contributes to stabilizing hematopoietic and immune reconstitution.

【Key words】Child;Aplastic anemia;Autologous cord blood transplantation;Immunosuppression;Immune reconstitution

获得性再生障碍性贫血（AA）是一种由多种病因引起的骨髓造血功能衰竭性疾病，治疗方法包括免疫抑制治疗（IST）及造血干细胞移植（HSCT）[1]。AA患者对IST的反应率为30% ～ 70%，但存在复发、克隆演变等问题[2]。据报道接受兔抗人胸腺细胞免疫球蛋白（r-ATG）治疗患者10年克隆演变发生率为5%，复发率高达15%[3]。……