Will a reduced-toxicity conditioning regimen improve the results of stem cell transplantation in metabolic disease?

摘要

Escolar et al.1 have reported that early transplantation of asymptomatic newborns affected by Krabbe's disease favorably alters the course of the disease. Twenty-five newborns and infants underwent an unrelated cord blood transplantation after a standard myeloablative conditioning regimen with busulphan and cyclophoshamide and the survival was significantly better among asymptomatic newborns than symptomatic infants or untreated controls.