Abstract: 【Abstract】 Objective The study was aimed to explore the efficacy and safety of haplotype allogeneic hematopoietic stem cell transplantation （allo-HSCT）in treatment of juvenile myelomonocytic leukemia（JMML）. Method From January 2011 to January 2014, six children with JMML receiving haploidentical allo-HSCT were included in the study, including 4 males and 2 females, aged from 4 to 12 years old, and the mean age was 5.6 years. The median time from diagnosis to transplantation was 6.5 months （2 to 25 months）.Donors received granulocyte colony-stimulating factor mobilization. Stem cell transplantation was collectedfrom both peripheral blood and bone marrow. CTX+FLU+ATG program was used as conditioning regimen, and combined immunosuppressive agents were used for graft-versus-host disease （GVHD） prophylaxis, including cyclosporine A, amethopterin, tacrolimus，etc. Toxic and side effects，incidence of GVHD and disease-free survival of these children after transplantation were observed. Result All patients reached hematopoietic reconstitution. The average time was 16.5 d and 18.2 d, respectively, with neutrophils ≥ 0.5×109/L and platelets ≥ 20×109/L. Implantation was confirmed by the evidence of 100% of donor hematopoiesis.With a median follow-up duration of 21.5 months （2 - 40 months）, three cases developed acute GVHD, two cases showed chronic GVHD. One case died of GVHD, one died of relapse, and the remaining 4 patients remained disease-free survival. The disease-free survival rate was 66.7%. Conclusion Haploidentical hematopoietic stem cell transplantation could be chosen as a treatment method for patients with JMML in the absence of matched donors.
2014, Vol. 2 
