Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia

Summary Haploidentical haematopoietic stem cell transplantation (haplo-HSCT) used to be a third-line treatment option for childhood severe aplastic anaemia (SAA). We conducted this retrospective study of 36 children (38 transplants) who received haplo-HSCT from human leucocyte antigen (HLA)-mismatched related donors between July 2002 and November 2013 at five HSCT centres in China, including 17 cases that were 5/6 HLA matched (Group 1) and 21 that were 4/6 or 3/6 HLA matched (Group 2). Although patients in Group 2 had a higher incidence of grade II-IV acute graft-versus-host disease (57·9% vs. 5·9%, P = 0·001), they had similar rates of graft failure (5·3% vs. 5·9%, P = 0·742) and overall survival (80·8% vs. 93·8%, P = 0·234) as Group 1. Unmanipulated haplo-HSCT is an effective treatment for SAA children with satisfactory outcome of this cohort, especially in the 5/6 HLA-matched group. For patients in critical situations, such as unresponsive to immunosuppressive therapy, refractory infection and failing first HSCT, to bring forward the timing of haplo-HSCT is a feasible salvage strategy with better and faster donor accessibility.