Conclusions
Acute GVHD grade III/IV developed in 9.3% of children undergoing HCT for NMD, and it was related to an adverse 3-year overall survival (52.9% [95% CI 34-83]). Although contributing to morbidity, grade I/II aGVHD or cGVHD did not impact overall survival. Recipients of mismatched unrelated BM or PB transplants, but not those of mismatched related donor transplants or mismatched cord blood transplants, were at a high risk for aGVHD grade III/IV. In order to reduce the incidence of aGVHD grade III/IV and improve survival, we recommend either avoiding mismatched unrelated donors or using novel methods for GVHD prophylaxis, ideally done on prospective clinical trials.