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Original Article

Hematology

Hematopoietic stem cell transplantation in pediatric patients with type VI mucopolysaccharidosis

Vedat Uygun, Koray Yalçın, Hayriye Daloğlu, Seda Öztürkmen, Suna Çelen, Suleimen Zhumatayev, Gülsün Karasu, Akif Yeşilipek

Clin Exp Pediatr. 2025;68(8):601-607. Published online March 11, 2025

Question: Could hematopoietic stem cell transplantation (HSCT) be an alternative to enzyme replacement therapy (ERT) for type VI mucopolysaccharidosis (MPS VI)?
Finding: HSCT is generally not offered due to reports of high toxicity and mortality. However, we detected fewer complications and graft-versus-host disease cases and no deaths with HSCT.
Meaning: HSCT is both less expensive than ERT and permanent; thus, it should be considered an alternative treatment for MPS VI.

DOI: https://doi.org/10.3345/cep.2024.02033

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