Allogeneic Hematopoietic Stem Cell Transplantation in Thirty-Four Pediatric Cases of Mucopolysaccharidosis—A Ten-Year Report from the China Children Transplant Group

Highlights • We reported multicenter results of allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis throughout China • This study included not only mucopolysaccharidosis IH but also other types that were reported rarely • Overall survival, full donor chimerism, and normal enzyme levels were high and transplantation-related mortality was low • Hematopoietic stem cell transplantation was helpful to improve multisystem symptoms of mucopolysaccharidosis