幼年粒单细胞白血病造血干细胞移植疗效初探

目的探讨造血干细胞移植治疗幼年粒单细胞白血病(JMML)的疗效。方法 5例JMML患儿接受无关供者脐血造血干细胞移植治疗。预处理均采用Bu/Cy+Mel+ATG方案:马利兰(0.8～1.0)mg/kg,每6小时1次共用16次(-8d～-5d);环磷酰胺60mg/(kg·d)用2d(-4～-3d);马法兰140mg/m2用1次(-2d):抗胸腺细胞球蛋白2.5mg/(kg·d)连用4d(-4～-1d)。GVHD预防采用CsA+MP±MMF。结果 5例成功植入,3例复发,1例复发后死于卡氏肺囊虫肺炎,1例死于CMV感染相关间质性肺炎,1例长期无病存活。结论 5例JMML移植初步治疗结果不满意,移植失败主要原因为白血病复发。为减少复发,今后需进一步改进移植方法 ,包括选择其他供体、改进GVHD预防方案。

Preliminary study of hematopoietic stem cell transplantation in children with juvenile myelomonocytic leukemia

Objective To explore the effect of hematopoietic stem cell transplantation in children with juvenile myelomonocytic leukemia(JMML). Methods We performed unrelated donor cord blood transplantations in 5 children,aged 3.3 to 7.0 yr,with juvenile myelomonocytic leukemia. Conditioning regimen was Bu/Cy+Mel+ATG:busulfan,0.8~1.0 mg/kg,q6h,i.v. for 4 days(-8 d~-5 d); cyclophosphamide,60 mg/kg once daily i.v. for 2 days(-4 d~-3 d); melphalan,140 mg/m2 i.v. for one day(-2 d); antithymocyte globulin,2.5 mg/kg once daily i.v. for 4 days(-4 d~-1 d). GVHD prophylaxis consisted of cyclosporine A (CsA),methylprednisolone(MP) and mycophenolate mofetil (MMF),2 patients without MMF. Results All 5 patients achieved neutrophil engraftment. 3 patients relapsed after transplantation. Of the 3 relapsed patients,one patient died of carini pneumocystis pneumonitis. One patient died of CMV related interstitial pneumonitis. Only one case long-term surviving without disease. Conclusion The preliminary treatment outcomes of the 5 JMML children were not satisfactory,leukemia recurrence was the major cause of treatment failure after transplantation. For reducing relapse,we should improve present transplant methods,including alternative donor selection,modifying GVHD prophylaxis protocol.