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Modified umbilical cord-blood transplantation for pediatric patients with mucopolysaccharidosis

Bone Marrow Transplantation volume 58, pages 112–114 (2023)Cite this article

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Mucopolysaccharidoses (MPSs) are a group of inherited errors of metabolism caused by gene encoding mutation that affects the degradation of mucopolysaccharides or glycosaminoglycans (GAGs), resulting in the accumulation of glycosaminoglycans in various organs and tissues of patients with MPS leads to multi-systemic clinical picture with a broad range of clinical signs and symptoms [1]. Although the advent of enzyme replacement therapy (ERT) has paved the way for MPS treatment, the blood-brain barrier (BBB) has prevented patients with central nervous system manifestations from benefiting from ERT [2]. Therefore, allogenic bone marrow transplantation (HSCT) is still the only effective treatment option for patients with MPS [3, 4].

This retrospective study included 60 patients with MPS who underwent a new conditioning regimen based on a slightly modified position of ATG or the first use of ALG in patients with MPS from December 2018 to February 2022 treated at the Hospital of Hebei Pediatrics of Integrated Traditional Chinese and Western Medicine (n = 56) and the Hospital of Beijing Jingdu Pediatrics (n = 4). The study was approved by the institutional review board of the two centers. Written informed consent was obtained from the parents or legal guardians of the patients.

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Fig. 1: Conditioning regimens and overall survival after transplantation for patients with MPS.

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The data for this article are not publicly available. Requests to access the data should be directed to the corresponding authors.

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Acknowledgements

We thank all the patients who participated in the study.

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Author notes

  1. These authors contributed equally: Yan Yue, Guangming Qiao.

Authors and Affiliations

  1. Department of Hematology and Oncology, Hospital of Beijing Jingdu Pediatrics, Beijing, China

    Yan Yue, Zhouyang Liu, Fan Jiang, Shifen Fan, Jiao Chen & Yuan Sun

  2. Department of Hematology and Oncology, Hospital of Hebei Pediatrics of Integrated Traditional Chinese and Western Medicine, Hebei, China

    Guangming Qiao, Yaochen Zhang, Yajuan Shi, Xuexin Zhang & Xiaoyu Qi

  3. Department of Medical Affairs, Acornmed Biotechnology Co., Ltd., Tianjin, China

    Jiayue Qin

  4. Department of Hematology and Oncology, Hospital of Capital Institute of Pediatrics, Beijing, China

    Xiaodong Shi

Authors
  1. Yan Yue
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Contributions

YY, GQ, YS, and XS designed, wrote, and revised the manuscript. YY, GQ, ZL, YZ, YS, XZ, XQ, FJ, SF, JC collected data and provided clinical care. YY, GQ, and JQ analyzed the clinical data. All authors approved the final manuscript for publication.

Corresponding authors

Correspondence to Yuan Sun or Xiaodong Shi.

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The authors declare no competing interests.

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Yue, Y., Qiao, G., Liu, Z. et al. Modified umbilical cord-blood transplantation for pediatric patients with mucopolysaccharidosis. Bone Marrow Transplant 58, 112–114 (2023). https://doi.org/10.1038/s41409-022-01858-5

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