The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update.
Haematopoietic cell transplantation (HCT) can benefit some selected subsets of patients with lysosomal diseases. Results had been impressive in children with MPS I-H, but poor in other disorders. Careful, multidisciplinary decision-making regarding whether to recommend HCT and how to provide optimal pre- and post-HCT care has proven essential to increase the likelihood of a good outcome.
1BMT Unit, Paediatric Department of Milano-Bicocca University, San Gerardo Hospital, Monza, Italy. attilio.rovelli@pediatriamonza.it
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D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D016464:Lysosomal Storage Diseases / Q000628:therapy*; D045164:Mesenchymal Stem Cell Transplantation