A multi-center retrospective study of 240 infants with severe combined immunodeficiency (SCID) has demonstrated that five-year transplant survival is 74% overall, but can reach 88-100% in select patient groups. Donors included matched sibling (MSD), T-cell depleted (TCD) mismatched related (MMRD), and unmodified other related/unrelated donors (OD). Multivariate analysis of survival revealed superior outcomes for MSD when compared to TCD MMRD or unmodified OD grafts. Regardless of donor type, however, transplant recipients had “excellent outcomes” according to the researchers of 88-100% if they were <3.5 months old, or were of any age and free of infection at time of transplant. The researchers concluded that transplant results in excellent survival when applied to infants with SCID identified early and before onset of infections, and that this underscores the critical importance of universal newborn screening for SCID.
Pai S-Y, et al. Biol Blood Marrow Transplant (Full abstract only available to BBMT subscribers)