Reduced-intensity allogeneic HCT for chronic granulomatous disease can lead to two-year survival rates exceeding 95%, according to the results of a prospective multi-center trial. Median age of the 56 patients enrolled in the study was 12.7 years (range 0-40), and 42 patients (75%) had high-risk features. Graft sources were HLA-matched related donors (n=21) and HLA-matched unrelated donors (n=35). At median follow-up of 21 months, the two-year probability of survival and event-free survival was 96% and 91%, respectively.
Güngör T, et al. Lancet
Reduced-Intensity HCT for Chronic Granulomatous Disease
Feb 2014