Objective: To evaluate the efficacy and safety of haploidentical (from family member donors) hematopoietic stem cell transplantation (HSCT) for children.
Patients and methods: Fifty-eight children under fourteen years old with hematological malignancies underwent haploidentical HSCT. Outcomes were analyzed.
Results: Of Fifty-eight patient/donor pairs, seven (12.1%) were mismatched in two HLA loci, twenty (34.5%) in three loci, and thirty-one (53.4%) in four loci. Follow-ups were performed for a median of 915 (227-1898) days after transplantation. All patients achieved stable engraftments. The cumulative incidence of acute graft-versus-host disease (GVHD) of grade 2-4 was 54.8%7.6%, and that of grade 3-4 was 11.4%4.8%. The cumulative incidence of chronic GVHD was 45.6%7.8% for total and 19.6%6.5% for extensive. Fourty patients survived with a 3-year probability of leukemia-free survival (LFS) 44.7%13.9%. Eighteen patients died, five from infection, eight from relapse of leukemia, two from heart failure, two from GVHD, and one from lymphoproliferative disorders.
Conclusion: The results encourage extending haploidentical HSCT without T-cell depletion treatments to children with an indication for transplantation.