Long‐Term Outcomes of Hematopoietic Stem Cell Transplantation in Mucopolysaccharidoses Patients Without Radiation
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Wiley
2025
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| author | Mohammad Jahanpanah Leila Jafari Maryam Behfar Nadia Alipour Farah Ahad Rashin Mohseni Reihaneh Mohsenipour Aria Setoodeh Morteza Heidari Mahmoud Reza Ashrafi Reza Shervin Badv Mina Mokaram Shirin Eshghi Amir Ali Hamidieh |
| author_facet | Mohammad Jahanpanah Leila Jafari Maryam Behfar Nadia Alipour Farah Ahad Rashin Mohseni Reihaneh Mohsenipour Aria Setoodeh Morteza Heidari Mahmoud Reza Ashrafi Reza Shervin Badv Mina Mokaram Shirin Eshghi Amir Ali Hamidieh Mohammad Jahanpanah Leila Jafari Maryam Behfar Nadia Alipour Farah Ahad Rashin Mohseni Reihaneh Mohsenipour Aria Setoodeh Morteza Heidari Mahmoud Reza Ashrafi Reza Shervin Badv Mina Mokaram Shirin Eshghi Amir Ali Hamidieh |
| collection | Wiley Open Access |
| contents | Long‐Term Outcomes of Hematopoietic Stem Cell Transplantation in Mucopolysaccharidoses Patients Without Radiation Mohammad Jahanpanah Leila Jafari Maryam Behfar Nadia Alipour Farah Ahad Rashin Mohseni Reihaneh Mohsenipour Aria Setoodeh Morteza Heidari Mahmoud Reza Ashrafi Reza Shervin Badv Mina Mokaram Shirin Eshghi Amir Ali Hamidieh Clinical Transplantation 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. 10.1111/ctr.70188 http://onlinelibrary.wiley.com/termsAndConditions#vor |
| doi_str_mv | 10.1111/ctr.70188 |
| format | Artículo Open Access |
| id | wiley_oa_10_1111_ctr_70188 |
| institution | Wiley Open Access |
| license_str_mv | http://onlinelibrary.wiley.com/termsAndConditions#vor |
| publishDate | 2025 |
| publisher | Wiley |
| record_format | wiley_oa |
| spellingShingle | Long‐Term Outcomes of Hematopoietic Stem Cell Transplantation in Mucopolysaccharidoses Patients Without Radiation Mohammad Jahanpanah Leila Jafari Maryam Behfar Nadia Alipour Farah Ahad Rashin Mohseni Reihaneh Mohsenipour Aria Setoodeh Morteza Heidari Mahmoud Reza Ashrafi Reza Shervin Badv Mina Mokaram Shirin Eshghi Amir Ali Hamidieh Clinical Transplantation Long‐Term Outcomes of Hematopoietic Stem Cell Transplantation in Mucopolysaccharidoses Patients Without Radiation Mohammad Jahanpanah Leila Jafari Maryam Behfar Nadia Alipour Farah Ahad Rashin Mohseni Reihaneh Mohsenipour Aria Setoodeh Morteza Heidari Mahmoud Reza Ashrafi Reza Shervin Badv Mina Mokaram Shirin Eshghi Amir Ali Hamidieh Clinical Transplantation 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. 10.1111/ctr.70188 http://onlinelibrary.wiley.com/termsAndConditions#vor |
| title | Long‐Term Outcomes of Hematopoietic Stem Cell Transplantation in Mucopolysaccharidoses Patients Without Radiation |
| topic | Clinical Transplantation |
| url | https://onlinelibrary.wiley.com/doi/10.1111/ctr.70188 |