Long-Term Outcomes of Hematopoietic Stem Cell Transplantation in Mucopolysaccharidoses Patients Without Radiation Publisher Pubmed



M Jahanpanah MOHAMMAD ; L Jafari LEILA ; M Behfar MARYAM ; N Alipour NADIA ; F Ahad FARAH ; R Mohseni RASHIN ; R Mohsenipour REIHANEH ; A Sotoudeh ARYA ; M Heidari MORTEZA ; Mr Ashrafi Mahmoud REZA
Source: Clinical Transplantation Published:2025

Abstract

Aim: Mucopolysaccharidosis (MPS) is an inherited lysosomal storage disorder (LSD) subcategory caused by the glycosaminoglycans (GAG) endo- and exo-glycosidases malfunction or dysfunction, leading to GAG accumulation. Due to the enzyme replacement therapy's (ERT's) limitations and challenges, HSCT is considered the only standard curative option in some MPS subtypes. Methods: The cross-sectional study was conducted on MPS subtypes I, II, and VI patients with an indication for HSCT between September 2016 and December 2023. A myeloablative conditioning (MAC) regimen without radiation was administered to all patients. Results: In this study, the OS was 75.3% for all patients. Considering different MPS subtypes, the OS rate was 71.6%, 62.5%, and 81.3% for MPS-I, II, and VI patients, respectively. There was no graft failure using PB donor cells, and all alive patients reached normal enzyme activity 1 year post-HSCT. Considering growth parameters, we showed that MPS I patients could benefit from HSCT more than MPS VI patients. However, MPS VI patients showed better OS. Conclusions: Data regarding the comparison of HSCT outcomes in different MPS subtypes are limited, and HSCT outcomes in MPS VI patients are mostly limited to case reports. A conditioning regimen without radiation should be considered in all patients to reduce post-HSCT complications. © 2025 Elsevier B.V., All rights reserved.