Allogeneic stem cell transplantation for children with acquired severe aplastic anaemia: a retrospective study by the Viva- Asia Blood and Marrow Transplantation Group.

We retrospectively analysed the outcomes of 127 children with acquired severe aplastic anaemia ( SAA) who had received haematopoietic stem cell transplantation ( HSCT) between 2000 and 2011 in one of the 10 Asia Pacific institutions. Fifty-three were matched sibling donor ( MSD) and 74 were alternative donor ( AD), including 22 matched unrelated donor, 32 mismatched unrelated donor and 20 mismatched related donor. With a median follow up 45·5 months (13-139) and when compared to the MSD group, AD recipients had more grade II- IV acute graft-versus-host disease (a GVHD; 14·3% vs. 32·8%, P = 0·029), but similar grade III- IV a GVHD (10·2% vs. 12·5%, P = 0·774), graft failure ( GF) (15·1% vs. 15·5%, P = 0·658) and 5-year overall survival (90·6% vs. 83·7%, P = 0·251). As a source of stem cell, peripheral blood stem cells ( PBSC) resulted in less GF (18% vs. 9·1% P = 0·013), similar grade II-IV a GVHD (28·1% vs. 17·4%, P = 0·258), chronic GVHD (25·8% vs. 29·3%, P = 0·822) and similar outcomes (89·7% vs. 82·4%, P =0 ·665) when compared to bone marrow ( BM). In univariate analysis, GF ( P < 0·001) and grade II- IV a GVHD ( P = 0·009) were predictors of poor survival. In multivariate analysis, only GF was associated with poor survival ( P = 0·012). The outcome of AD and PBSC HSCT were comparable to that of MSD and BM HSCT in the Asia Pacific region. [ABSTRACT FROM AUTHOR]