Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades
Indian Pediatr
; 2022 Sept; 59(9): 699-702
Article
| IMSEAR
| ID: sea-225368
ABSTRACT
Objective:
We present outcome data on hematopoietic stem cell transplantation (HSCT) in children with inborn errors of metabolism (IEM).Methods:
We retrospectively analyzed data on children up to 18 years of age, diagnosed with IEM, who underwent HSCT between January, 2002 and December, 2020.Results:
24 children, (mucopolysaccharidosis – 13, Gaucher disease – 4, X-linked adrenoleukodystrophy – 4, metachromatic leukodystrophy – 2, Krabbe disease – 1) were included. Donors were matched family donors in 24%, matched unrelated donors in 34%, and haploidentical fathers in 42% of the transplants, with engraftment in 91% of children. Overall survival was 72% (55-100%) with a median followup of 76.5 (10-120 ) months, and progression-free survival of 68% (MPS-76%, X-ALD - 60%, Gaucher disease – 50%, and 100% in MLD and Krabbe disease).Conclusion:
HSCT is an available curative option, and early age at HSCT prevents end-organ damage.
Full text:
1
Index:
IMSEAR
Journal:
Indian Pediatr
Year:
2022
Type:
Article