Original Article
Bone Marrow Transplantation (2008) 41, S87–S89; doi:10.1038/bmt.2008.62
Non Malignant Diseases
The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update
A M Rovelli1
1BMT Unit, Paediatric Department of Milano-Bicocca University, San Gerardo Hospital, Monza, Italy
Correspondence: Dr AM Rovelli, Centro Trapianto di Midollo Osseo, Clinica Pediatrica dell'Università di Milano-Bicocca, Ospedale San Gerardo, Via Pergolesi 33, Monza 20052, Italy. E-mail: attilio.rovelli@pediatriamonza.it
Abstract
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.
Keywords:
haematopoietic cell transplantation, metabolic inborn errors, lysosomal diseases, mucopolysaccharidosis, leukodystrophy
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RESEARCH
Bone Marrow Transplantation Original Article
