A small cohort of children with severe aplastic anemia demonstrated tolerance to haploidentical hematopoietic stem cell transplantation, according to results of a recent investigation.
Twelve eligible children and adolescents received in vitro T-cell–depleted peripheral blood stem cells.
There were 15 transplantations with in vitro CD3-depleted grafts performed between July 2009 and July 2012.
Eleven of the 12 patients experienced neutrophil engraftment. The researchers observed this result at a median of 10 days (range, 9-13 days) after the procedure.
Researchers reported one primary engraftment failure and two graft rejections. Each of these three patients received a second haploidentical hematopoietic stem cell transplantation (HSCT). The result was a 100% final engraftment rate.
Investigators assessed acute graft-versus-host disease (GVHD) in nine patients, excluding those who experienced early graft failure. Two patients experienced grade 2 or higher GVHD, and one patient experienced grade 3 GVHD.
At a median follow up of 14.3 months (range, 4.1-40.7 months), all 12 patients were alive and transfusion-independent.
“Hematopoietic stem cell transplantation from haploidentical family donors with in vitro CD3 T-cell depletion is a reasonable therapeutic option for children and adolescents with acquired [severe aplastic anemia],” the researchers wrote.
Future studies will be undertaken with a uniform protocol to potentially solve problems linked to this type of transplantation, they added.