单倍型异基因造血干细胞移植治疗儿童难治性或复发白血病30例

Haplotype allogeneic hematopoietic stem cell transplantation for refractory and relapsed childhood leukemia

目的探讨单倍型异基因造血干细胞移植（allo-HSCT）治疗儿童难治性或复发白血病的疗效和安全性。方法2007年6月至2011年6月间难治性或复发白血病患儿30例接受亲缘单倍型相合a110-HSCT。患儿的原发病为急性髓系白血病（AML）14例，急性淋巴细胞白血病（ALL）16例。30例中，首次复发11例，2次或以上复发16例，原发难治性3例。获取供者骨髓＋外周血干细胞用于移植。预处理药物采用阿糖胞苷、白消安（Bu）、氟达拉滨及全身照射等。联合使用环孢素A（CsA）＋短程甲氨蝶呤（MTX）＋吗替麦考酚酯（MMF）＋抗人胸腺细胞球蛋白（ATG）等预防移植物抗宿生病（GVHD）。结果30例均获造血重建，中性粒细胞≥0．5×10^9／L及血小板≥20×10^9／L的平均时间分别为18．5d及24．2d，均为完全供者植人。患儿随访时间中位数为22．5个月，随访期间共12例发生急性GVHD，6例发生慢性GVHD。因GVHD死亡4例，感染死亡3例，复发死亡6例，其余17例患儿仍无病存活，2年无病存活率为55％。结论单倍型a110-HSCT治疗儿童难治性或复发白血病安全、可行。

Objective To explore the effect and feasibility of haplotype allogeneic hematopoietic stem cell transplantation （alIo-HSCT） used in the childhood patients with refractory and relapsed leukemia. Methods Thirty children with refractory and relapsed leukemia received haplotype alloHSCT from June 2007 to June 2011 in Beijing Military General Hospital, including 14 cases of acute myeloid leukemia （AML） and 16 cases of acute lymphoblastic leukemia （ALL）. Of the 30 cases, there were 11 cases of initial recurrence, 16 cases of second or more relapse, and 3 cases of primary refractory leukemia. The bone marrow and peripheral blood of donors were used for transplantation. All children were subjected to pretreatment consisting of cytarabine, busulfan, fludarabine and total body irradiation （TBI）, etc. Graft-versus-host disease （GVHD） was prevented by combining variety of immunosuppressants including Cyclosporin A （CsA）, Methotrexate （MTX）, Mycophenolate mofetil （MMF） and anti-thymocyte immunoglobulin （ATG）, etc. The regimen-associated side effect incidence of GVHD and disease-free survival probabilities were observed after HSCT. Results The results showed that all of the 30 children acquired hematopoietic reconstitution, and the median time of granulocytes exceeding 0. 5 x 10^9/L and platelets exceeding 20 x 10^9/L which were transplanted 100% by donors was 18. 5 days and 24. 2 clays respectively. The mean follow-up period was 22. 5 months （3 -48 months）. Twelve children had experience of acute GVHD, and 6 children had experience of chronic GVHD. Four children died of GVHD, 3 died of infection and 6 died of relapse, and the rest children were alive in free situation. The 2-year disease-free survival rate was 55%. Conclusion Haplotype allo -HSCT was an safe and feasible therapy for the childhood patients with refractory and relapsed leukemia.