|
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. |