非血缘脐血移植治疗儿童高危急性淋巴细胞白血病的疗效

Therapeutic effect of unrelated umbilical cord blood transplantation in treating children with high risk acute lymphoblastic leukemia

目的探讨单份非血缘脐血移植（UCBT）治疗儿童高危急性淋巴细胞白血病（ALL）的疗效。 方法回顾性分析2008年9月至2014年12月安徽医科大学附属省立医院采用UCBT治疗高危ALL儿童66例的临床资料。患儿均采用强化清髓性预处理方案，分别为全身照射/环磷酰胺/阿糖胞苷（TBI/Cy/Ara-C）13例，白消安/环磷酰胺/氟达拉滨（BU/Cy/Flu）47例，BU/Cy/Ara-C 6例。环孢素联合短程霉酚酸酯预防移植物抗宿主病（GVHD）。 结果所有患儿输入复温后脐血总有核细胞数为4.70（2.07～13.74）×107/kg，CD34＋细胞中位数为2.40（0.73～9.89）×105/kg。移植后42 d中性粒细胞累积植入率为97.0%（64例），移植后120 d血小板累积植入率为87.6%（57例）；中性粒细胞和血小板中位植入时间分别为移植后16（12～37） d和39（15～148） d。Ⅱ～Ⅳ度急性GVHD、Ⅲ～Ⅳ度急性GVHD和慢性GVHD发生率分别为27.3%（17例）、17.2%（10例）和10.3%（6例），可评估的360 d移植相关病死率为21.2%（14例），3年累积复发率为14.8%（9例），3年无病生存率和总体生存率分别为65.1%（45例）和67.6%（46例）。 结论UCBT是治疗儿童高危ALL的一种安全有效的方法。

Objective To analyze the clinical outcomes of single unrelated umbilical cord blood transplanta- tion（UCBT） in treating children with high risk acute lymphoblastic leukemia（ALL）. Methods Between September 2008 and December 2014, a total of 66 consecutive pediatric patients with ALL received single UCBT treatment in De- partment of Hematology, Anhui Provincial Hospital Affiliated to Anhui Medical University. Myeloablative conditioning regimen was applied. The conditioning regimen consisted of total body irradiation （TBI）/ Cyclophosphamide （Cy）/ Cytarabine （Ara -C）in 13 patients,Busulfan（ BU）/Cy/Fludarabine（Flu） in 47 patients and BU/Cy/Ara- C in 6 patients. All patients received a combination of Cyclosporine and Mycophenolate mofetil for the prophylaxis of graft - ver- sus - host disease （GVHD）. Results The median dose of infused total nucleated cells and CD34 ＋ cells were 4.70 （2.07 - 13.74 ） ×107/kg and 2.40 （0.73 -9. 89 ）× 105/kg. The cumulative incidence of neutrophil cells engraftment at day ＋42 and platelet recovery at day ＋ 120 was 97.0% （64 cases） at a median of 16（ 12 -37） days after trans- plantation and 87.6% （ 57 cases ） with a median time of recovery of 39 （ 15 - 148 ） days, respectively. Incidence of grades Ⅱ-Ⅳ acute GVHD, grades Ⅲ - Ⅳacute GVHD and chronic GVHD were 27.3% （17 cases）, 17.2% （ 10 cases） ,and 10.3% （6 cases） ,respectively. The transplant - related mortality at 360 day was 21.2% （ 14 cases）. The 3 - year cumulative incidence of relapse was 14.8% （9 cases）. The 3 - year - disease - free survival and overall survival rates were 65.1% （45 cases） and 67.6% （46 cases）, respectively. Conclusions For pediatric patients with high risk ALL,UCBT is a safe and effective treatment option.