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一名患有高危骨髓增生异常综合征的沃纳综合征患者成功进行脐血移植

Successful Cord Blood Transplantation in a Werner Syndrome Patient with High-risk Myelodysplastic Syndrome.

作者信息

Hayashi Kiyohito, Tasaka Taizo, Kondo Toshinori, Ishikawa Yuichi, Goto Makoto, Matsuhashi Yoshiko, Sadahira Yoshito, Sugihara Takashi, Wada Hideho

机构信息

Department of Hematology, Kawasaki Medical School, Japan.

Department of Transfusion Medicine, Saitama Medical Center, Saitama Medical University, Japan.

出版信息

Intern Med. 2019 Jan 1;58(1):109-113. doi: 10.2169/internalmedicine.0317-17. Epub 2018 Aug 24.

DOI:10.2169/internalmedicine.0317-17
PMID:30146558
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6367075/
Abstract

Werner syndrome (WS) confers a high risk of the development of neoplasias, including hematological malignancies, and curative treatment for these malignancies is difficult to achieve. A 44-year-old man with myelodysplastic syndrome was admitted to our hospital. He was diagnosed with mutation-proven WS. He underwent cord blood transplantation (CBT) following fludarabine, busulfan, and melphalan administration. A chimerism analysis of his marrow blood on day 62 showed a donor pattern >95%, which confirmed engraftment. The patient lived for 15 months while maintaining remission of MDS without treatment-related toxicity. Our case shows that CBT can be a treatment modality for WS patients with hematological malignancies.

摘要

沃纳综合征(WS)会使患肿瘤的风险大幅增加,包括血液系统恶性肿瘤,而治愈这些恶性肿瘤却很难实现。一名44岁的骨髓增生异常综合征男性患者入住我院。他被诊断为经突变证实的WS。在接受氟达拉滨、白消安和美法仑治疗后,他接受了脐血移植(CBT)。在第62天对其骨髓血进行的嵌合体分析显示供体模式>95%,这证实了植入成功。该患者存活了15个月,同时维持了骨髓增生异常综合征的缓解状态,且无治疗相关毒性。我们的病例表明,CBT可以作为患有血液系统恶性肿瘤的WS患者的一种治疗方式。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/61ef/6367075/f146748b801f/1349-7235-58-0109-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/61ef/6367075/f146748b801f/1349-7235-58-0109-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/61ef/6367075/f146748b801f/1349-7235-58-0109-g001.jpg

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本文引用的文献

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Hum Mutat. 2017 Jan;38(1):7-15. doi: 10.1002/humu.23128. Epub 2016 Oct 7.
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Biol Blood Marrow Transplant. 2016 Oct;22(10):1844-1850. doi: 10.1016/j.bbmt.2016.06.017. Epub 2016 Jun 21.
3
I.v. BU/fludarabine plus melphalan or TBI in unrelated cord blood transplantation for high-risk hematological diseases.
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Bone Marrow Transplant. 2015 Apr;50(4):607-9. doi: 10.1038/bmt.2014.316. Epub 2015 Jan 26.
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