Haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia.

Severe aplastic anemia (SAA) is a life-threatening disorder for which allogeneic hematopoietic stem cell transplantation (HSCT) is the current available curative treatment. HSCT from matched sibling donors (MSDs) is the preferred therapy for children with acquired SAA. For patients who lack MSDs, immunosuppressive therapy (IST) is widely accepted as a first-line treatment before considering HCT from an unrelated donor (URD). Given the recent progress in HSCT using URDs for childhood SAA, well-matched URDs became a realistic alternative for pediatric patients who have no suitable related donors and who are refractory to IST. However, it is quite challenging to treat patients with refractory SAA who lack suitable related or URDs. Even though haploidentical HSCT from genetically mismatched family members seemed to be an attractive procedure with the amazing benefit of readily available donors for most patients, early attempts were disappointing because of refractory graft-versus-host disease (GVHD) and excessively high transplant-related mortality. Recent advances with effective ex vivo depletion of T cells or unmanipulated in vivo regulation of T cells, better supportive care, and optimal conditioning regimens have significantly improved the outcome of haploidentical transplant. Besides considerable progress in the treatment of malignant diseases, recent emerging evidences for haploidentical HSCT in SAA has provided additional therapeutic options for patients with refractory diseases. Further improvements to decrease the rates of graft failure, GVHD, and infectious complications will facilitate the emergence of haploidentical HSCT as a front-line therapy for treating acquired SAA in children and adolescents who have no suitably matched donors.