Hematopoietic stem cell transplantation in pediatric patients with type VI mucopolysaccharidosis.

IF 3.2 Q1 PEDIATRICS
Vedat Uygun, Koray Yalçın, Hayriye Daloğlu, Seda Öztürkmen, Suna Çelen, Suleimen Zhumatayev, Gülsün Karasu, Akif Yeşilipek
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引用次数: 0

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.

造血干细胞移植治疗小儿VI型粘多糖病。
背景:目前尚不清楚造血干细胞移植(HSCT)与标准酶替代疗法(ERT)相比,是否对6型粘多糖病(MPS VI)有效。目的:在最近的一项队列研究中,HSCT的新相关进展促使了对移植手术的检查。方法:本单中心回顾性研究回顾了2017 -2023年接受同种异体造血干细胞移植的17例小儿MPS VI患者的医疗记录。所有的调理方案都是清髓性的。记录移植天数、并发症和存活数据。由于随访时间较短,我们只记录了HSCT前后6分钟的步行测试距离。结果:患者在确诊后中位6年接受移植。所有的移植体都具有完全或混合嵌合。酶水平在正常范围内。在中位9个月的随访中,所有可评估患者的行走测试均有所改善。结论:HSCT旨在改善疾病,并在酶水平上提供永久性解决方案,消除了酶替代治疗。我们的研究表明,HSCT是一种较便宜的永久性治疗选择,应该提供给MPS VI患者。
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
CiteScore
8.00
自引率
2.40%
发文量
88
审稿时长
60 weeks
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