| Hematopoietic stem cell transplantation in pediatric patients with type VI mucopolysaccharidosis |
Vedat Uygun1 
, Koray Yalçın2, Hayriye Daloğlu3, Seda Öztürkmen3, Suna Çelen2, Suleimen Zhumatayev4, Gülsün Karasu3,4, Akif Yeşilipek3,4 |
1İstinye University, Faculty of Medicine, MedicalPark Antalya Hospital, Department Of Pediatric Bone Marrow Transplantation Unit, Antalya, Turkey
2Bahçeşehir University, Faculty of Medicine, MedicalPark Göztepe Hospital, Department Of Pediatric Bone Marrow Transplantation Unit, İstanbul, Turkey
3MedicalPark Antalya Hospital, Department of Pediatric Bone Marrow Transplantation Unit, Antalya, Turkey
4MedicalPark Göztepe Hospital, Department Of Pediatric Bone Marrow Transplantation Unit, İstanbul, Turkey |
Correspondence:
Vedat Uygun, Email: veddat@hotmail.com
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Received: 28 December 2024 • Revised: 5 February 2025 • Accepted: 20 February 2025 |
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| Abstract |
Background
It is uncertain whether hematopoietic stem cell transplantation (HSCT), versus standard enzyme replacement therapy (ERT), is effective for type VI mucopolysaccharidosis (MPS VI).
Purpose
New related advances in HSCT prompted an examination of the transplant procedures performed in a recent cohort.
Methods
This single-center retrospective study reviewed the medical records of 17 pediatric patients with MPS VI who underwent allogeneic HSCT in 2021–2023. All conditioning regimens were myeloablative. Engraftment days, complications, and survival data were recorded. As follow-up was short, we recorded only 6-min walk test distance before versus after HSCT.
Results
The patients underwent transplantation at a median of 6 years post-diagnosis. All were engrafted and had a full or mixed chimerism. Enzyme levels were within normal ranges. Walking tests of all evaluable patients improved at a median 9-month follow-up.
Conclusion
HSCT aims to improve the disease and provides a permanent solution at the enzyme level, eliminating enzyme replacement therapy. Our study showed that HSCT, a less expensive and permanent treatment option, should be offered to patients with MPS VI.
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| Key Words:
Mucopolysaccharidoses, Hematopoietic stem cell transplantation, Enzyme replacement therapy, Children, Maroteaux–Lamy syndrome |
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