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Endocrinology

Impact of hematopoietic stem cell transplantation on growth outcomes in mucopolysaccharidosis: a systematic review

Farzaneh Abbasi, Asal Khalili Dehkordi, Reihaneh Mohsenipour

Clin Exp Pediatr. 2025;68(6):417-427. Published online March 11, 2025

Hematopoietic stem cell transplantation (HSCT) enhances the growth outcomes of pediatric patients with mucopolysaccharidosis, with early intervention leading to improved height, weight, and body mass index. However, achieving a standard adult height remains uncommon among these patients, even in cases of early HSCT. Growth hormone therapy provides short-term benefits but does not address long-term height deficits. Pubertal development is generally normal; however, precocious puberty and pubertal arrest may occur.

DOI: https://doi.org/10.3345/cep.2024.01725

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