Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network

摘要: Abstract The leukodystrophies are a heterogeneous group of inherited diseases characterized by progressive demyelination the central nervous system leading to devastating neurologic symptoms and premature death. Hematopoietic stem cell transplantation (HSCT) has been successfully used treat certain leukodystrophies, including adrenoleukodystrophy, globoid leukodystrophy (Krabbe disease), metachromatic leukodystrophy, over past 30 years. To date, these complex patients have primarily transplanted at limited number pediatric centers. As cases identified through pregnancy newborn screening is increasing, additional centers will be required children. Hunter's Hope created Leukodystrophy Care Network in part create standardize high-quality clinical practice guidelines guide care affected patients. In this report for with undergoing treatment HSCT presented. initial transplant evaluation, determination patient eligibility, donor selection, conditioning, supportive care, post-transplant follow-up discussed. Throughout need early detection role partnership between families multidisciplinary providers emphasized.