HLA-haploidentical donor transplantation in severe combined immunodeficiency

Abstract

Curative treatment of Severe Combined Immunodeficiency (SCID) by Hematopoietic Cell Transplantation (HCT) remains a challenge, in particular in infants presenting with serious, poorly controllable complications. In the absence of a matched family donor, HLA-haploidentical transplantation from parental donors represents a uniformly and readily available treatment option, offering a high chance to be successful. Concerning outcomes of HCT in SCID, other important parameters beside survival need to be taken into consideration, in particular the stability and robustness of the graft and its function, as well as potential late complications, related either to the disease or to the treatment.