Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades
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Date
2022-09
Journal Title
Journal ISSN
Volume Title
Publisher
Indian Academy of Pediatrics
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.
Description
Keywords
Alternate donor HSCT, Gaucher disease, Leukodystrophy, Mucopolysaccharidosis.
Citation
Swaminathan Venkateswaran Vellaichamy, Meena Satishkumar, Varla Harika, Chandar Rumesh, Jayakumar Indira, Ramakrishnan Balasubramaniam, Uppuluri Ramya, Raj Revathi. Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades. Indian Pediatrics. 2022 Sept; 59(9): 699-702