T-cell replete haploidentical stem cell transplantation for children with relapsed or refractory acute leukemia.

Abstract

Human leukocyte antigen (HLA)-haploidentical stem cell transplantation (haplo-SCT) has been shown to be associated with a higher risk of early transplant-related mortality. This is because of complications in severe graft-versus-host disease arising due to allogeneic immune reactions and graft rejection. Therefore, traditionally, SCT from HLA-matched donors has been recommended. Contrastingly, allogeneic immune reactions induce a strong graft-versus-leukemia effect; thus, haplo-SCT is the treatment of choice for patients with relapsed／refractory leukemia. With recent advances in treatment methods, the survival rate in pediatric acute leukemia has improved. However, the prognosis of patients diagnosed with post-transplant relapsed／refractory leukemia or other relapsed diseases with poor prognostic factors continues to be dismal. We retrospectively analyzed 39 patients with relapsed／refractory acute leukemia who underwent T-cell-replete haplo-SCT between 2009 and 2016. The 3-year overall and disease-free survival rates were found to be 45.1±8.5% and 33.8±7.9%, respectively. Although these results are derived from case studies performed in a single institution, they will be important in evaluating the efficacy of prospective multi-center trials of T-cell-replete haplo-SCT.