Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Children with Advanced Acute Leukemia with a Fludarabine, Busulfan and Melphalan Based Conditioning.

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (2015)


Post-Transplantation Cyclophosphamide (PTCY) has made haploidentical transplantation a global reality in adults, but the literature is largely silent on the feasibility of this approach in children. We conducted a prospective study on 20 patients (median age 12 years; range 2-20) with advanced acute leukemia to evaluate the feasibility of PTCY based haploidentical peripheral blood stem cell (PBSC) transplantation in children. The conditioning regimen comprised of Fludarabine, intravenous Busulfan and Melphalan (FLU-BU-Mel). PTCY on day +3 and +4 was followed by mycophenolate mofetil for 14-21 days and cyclosporine for 60 days. Thirteen patients (65%) had refractory or relapsed myeloid leukemia and the rest had high risk lymphoblastic leukemia. Prompt engraftment was noted at a median of 14 days with full donor chimerism on Day + 28. Cumulative incidences of acute and chronic graft-versus-host disease were 35% and 5% respectively. Non-relapse mortality at one year was 20%. The incidence of disease progression was 25.7%. The actuarial overall survival at 2 years was 64.3% (95% CI53.4 -75.2%). Our data suggests FLU-BU-MEL based conditioning followed by PTCY based Haploidentical PBSC transplantation with reduced duration of immunosuppression is feasible in patients of pediatric age group with advanced leukemia.