Donor availability still limits the applicability of HSCT to thalassemia major patients and this review provides an overview of alternative HSCT approaches in thalassemia major. Outcomes after matched unrelated HSCT in thalassemia major has been reported now approach those obtained in the matched family donor setting, provided that the donor selection is performed using high-resolution molecular typing for HLA class I and II loci and according to strict criteria of donor/recipient compatibility. Besides, novel strategies of graft manipulation, associated with the infusion of engineered donor lymphocytes, are emerging as a promising strategy in addressing the limitation of delayed immune reconstitution, rendering this approach a viable alternative. The significant advances achieved in transplantation techniques, both in the matched unrelated and in the haploidentical setting, have been reported to significantly broaden the applicability of HSCT to patients with thalassemia major.

This summary is prepared by Sule Unal, M.D.