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

IF 1.9 4区医学 Q2 SURGERY

Clinical Transplantation Pub Date : 2025-06-13 DOI:10.1111/ctr.70188

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

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引用次数: 0

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.

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无放疗的粘多糖病患者造血干细胞移植的远期疗效

目的粘多糖病（Mucopolysaccharidosis， MPS）是一种遗传性溶酶体贮积障碍（LSD）亚类，由糖胺聚糖（glycosaminoglycans， GAG）内、外糖苷酶功能障碍引起，导致GAG积累。由于酶替代疗法（ERT）的局限性和挑战，HSCT被认为是一些MPS亚型的唯一标准治疗选择。方法对2016年9月至2023年12月有HSCT指征的MPS亚型I、II和VI患者进行横断面研究。所有患者均给予无放疗的清髓调节（MAC）方案。结果本组患者的总生存率为75.3%。考虑不同MPS亚型，MPS- i、II和VI患者的OS率分别为71.6%、62.5%和81.3%。使用PB供体细胞没有移植失败，所有存活的患者在移植后1年达到正常的酶活性。考虑到生长参数，我们发现MPS I患者比MPS VI患者更能从HSCT中获益。然而，MPS VI患者表现出更好的OS。结论不同MPS亚型的HSCT结果比较数据有限，MPS VI患者的HSCT结果多局限于病例报告。所有患者应考虑无放疗的调理方案，以减少hsct后并发症。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical Transplantation 医学-外科

CiteScore

3.70

自引率

4.80%

发文量

286

审稿时长

2 months

期刊介绍： Clinical Transplantation: The Journal of Clinical and Translational Research aims to serve as a channel of rapid communication for all those involved in the care of patients who require, or have had, organ or tissue transplants, including: kidney, intestine, liver, pancreas, islets, heart, heart valves, lung, bone marrow, cornea, skin, bone, and cartilage, viable or stored. Published monthly, Clinical Transplantation’s scope is focused on the complete spectrum of present transplant therapies, as well as also those that are experimental or may become possible in future. Topics include: Immunology and immunosuppression; Patient preparation; Social, ethical, and psychological issues; Complications, short- and long-term results; Artificial organs; Donation and preservation of organ and tissue; Translational studies; Advances in tissue typing; Updates on transplant pathology;. Clinical and translational studies are particularly welcome, as well as focused reviews. Full-length papers and short communications are invited. Clinical reviews are encouraged, as well as seminal papers in basic science which might lead to immediate clinical application. Prominence is regularly given to the results of cooperative surveys conducted by the organ and tissue transplant registries. Clinical Transplantation: The Journal of Clinical and Translational Research is essential reading for clinicians and researchers in the diverse field of transplantation: surgeons; clinical immunologists; cryobiologists; hematologists; gastroenterologists; hepatologists; pulmonologists; nephrologists; cardiologists; and endocrinologists. It will also be of interest to sociologists, psychologists, research workers, and to all health professionals whose combined efforts will improve the prognosis of transplant recipients.