Indian pediatrics · 2022
Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades.
СтатьяНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
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Ошибки и ограничения
История · было → стало
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Swaminathan Venkateswaran Vellaichamy, Meena Satishkumar, Varla Harika, Chandar Rumesh, Jayakumar Indira, Ramakrishnan Balasubramaniam, Uppuluri Ramya, Raj Revathi
Аннотация
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 follow-up 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.
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 follow-up 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.