Hematopoietic Stem Cell Transplantation for Storage Disorders: Present Status.

Publisher: Dr. K. C. Chaudhuri Foundation, co-published by Springer India Country of Publication: India NLM ID: 0417442 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 0973-7693 (Electronic) Linking ISSN: 00195456 NLM ISO Abbreviation: Indian J Pediatr Subsets: MEDLINE

Publication: [Mumbai] : Dr. K. C. Chaudhuri Foundation, co-published by Springer India
Original Publication: New Delhi : All India Institute Of Medical Sciences

Hematopoietic Stem Cell Transplantation* ; Lysosomal Storage Diseases*/therapy; Humans

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.
(© 2024. The Author(s), under exclusive licence to Dr. K C Chaudhuri Foundation.)

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Keywords: Hematopoietic stem cell transplantation; Lysosomal Storage Disorders (LSDs); Peroxisomal disorders

Date Created: 20240419 Date Completed: 20240715 Latest Revision: 20240715

20240715

10.1007/s12098-024-05110-4

38639861