Hematopoietic Stem Cell Transplantation for Storage Disorders: Present Status.
Indian J Pediatr
; 91(8): 830-838, 2024 Aug.
Article
em En
| MEDLINE
| ID: mdl-38639861
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.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Doenças por Armazenamento dos Lisossomos
/
Transplante de Células-Tronco Hematopoéticas
Limite:
Humans
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article