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Indian Journal of Pediatrics

19-04-2024 | Stem Cell Transplantation | Special Article

Hematopoietic Stem Cell Transplantation for Storage Disorders: Present Status

Authors: Soumalya Chakraborty, Aditya Kumar Gupta, Neerja Gupta, Jagdish Prasad Meena, Rachna Seth, Madhulika Kabra

Published in: Indian Journal of Pediatrics

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Abstract

Storage disorders are a group of inborn errors of metabolism caused by the defective activity of lysosomal enzymes or transporters. All of these disorders have multisystem involvement with variable degrees of neurological features. Neurological manifestations are one of the most difficult aspects of treatment concerning these diseases. The available treatment modalities for some of these disorders include enzyme replacement therapy, substrate reduction therapy, hematopoietic stem cell transplantation (HSCT) and the upcoming gene therapies. As a one-time intervention, the economic feasibility of HSCT makes it an attractive option for treating these disorders, especially in lower and middle-income countries. Further, improvements in peri-transplantation medical care, better conditioning regimens and better supportive care have improved the outcomes of patients undergoing HSCT. In this review, we discuss the current evidence for HSCT in various storage disorders and its suitability as a mode of therapy for the developing world.
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Metadata
Title
Hematopoietic Stem Cell Transplantation for Storage Disorders: Present Status
Authors
Soumalya Chakraborty
Aditya Kumar Gupta
Neerja Gupta
Jagdish Prasad Meena
Rachna Seth
Madhulika Kabra
Publication date
19-04-2024
Publisher
Springer India
Published in
Indian Journal of Pediatrics
Print ISSN: 0019-5456
Electronic ISSN: 0973-7693
DOI
https://doi.org/10.1007/s12098-024-05110-4