Similar survival following HLA-identical sibling transplantation for standard indication in children with haematologic malignancies:: A single center comparison of mobilized peripheral blood stem cell with bone marrow transplantation

Background: Peripheral blood stem cell (PBSC) grafts are increasingly used for autologous and allogeneic haematopoietic stem cell transplantation (alloHSCT) with the aim to hasten neutrophil and platelet engraftment and thereby to reduce transplant-related complications due to infections, bleeding and graft failure. Based on the paucity of data on PBSC transplantation in children we performed a retrospective single-center analysis comparing the outcome of children receiving mobilized PBSC from human leukocyte antigen (HLA)-identical sibling donors to bone marrow (BM) transplant recipients. Patients and Methods: Between 1996 and 2004, 16 children with haematologic malignancies and standard indication for alloHSCF underwent PBSC transplantation from HLA-identical sibling donors. The outcome of these children was compared to a historic control group of 19 bone marrow (BM) transplant recipients. Time to neutrophil engraftment, incidence of acute and chronic graft-versus-host disease (GvHD), relapse rate, transplant-related mortality, event-free and overall survival were analyzed. Results: Neutrophil engraftment was achieved significantly faster after PBSC compared to BM transplantation with a median time to neutrophil engraftment of 11 (range: 8-21) and 19 (16-44) days for the PBSC and BM cohort, respectively (p < 0.001). Two of 19 (11 %) BM recipients did not achieve primary neutrophil engraftment and both patients died due to infectious complications. The rate of clinically significant acute GvHD >= grade II was higher in the PBSC compared to the BM group (75 vs. 39%; p=0.045). Incidences of chronic GvHD (PBSC vs. BM: 60 vs. 44%), death of disease (13 vs. 21%) and death of complication (13 vs. 16%) were comparable between both groups (p = ns). With a median follow up of 4.7 years (PBSC) and 10.2 years (BM) overall survival (PBSC vs. BM: 68.6 +/- 13.5 vs. 63.2 +/- 11.1%; p = 0.65) and event-free survival (67.0 +/- 12.1 vs. 63.2 +/- 11.1%; p = 0.80) is without demonstrable difference in both groups. Conclusions: Transplantation of PBSC compared to BM is associated with faster neutrophil engraftment and a higher rate of >= grade II acute GvHD. As overall survival and event-free survival is similar when using PBSC and BM, PBSC is an alternative stem cell source for HLA-identical sibling transplantation. Further prospective analyses with higher number of patients stratified according to well established risk factors are required to define the precise role of both stem cell sources for children with haematologic malignancies.