将免疫消融疗法和自体造血干细胞移植作为多发性硬化症患者的一线疾病调节疗法。

IF 0.7 4区 医学 Q4 MEDICINE, RESEARCH & EXPERIMENTAL Biomedical Papers-Olomouc Pub Date : 2024-03-01 Epub Date: 2023-06-16 DOI:10.5507/bp.2023.023
Martin Lachnit, Kamila Zondra Revendova, Pavel Hradilek, Radovan Bunganic, Zdenek Koristek, Tomas Jelinek, Monika Skutova, Radim Piza, Ondrej Volny, Roman Hajek, Michal Bar
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引用次数: 0

摘要

导言:免疫消融治疗后进行自体造血干细胞移植(AHSCT)是多发性硬化症(MS)患者可能采用的疾病改变疗法(DMT)之一。在本病例系列中,我们将介绍六名接受AHSCT作为一线DMT的多发性硬化症患者:2018 年至 2021 年期间,六名残疾进展迅速、伴有或不伴有复发的多发性硬化症患者在俄斯特拉发大学医院接受了 AHSCT 作为一线 DMT。AHSCT使用的调节方案是中强度方案BEAM(卡莫司汀、依托泊苷、胞磷胆碱、美法兰)和基于环磷酰胺的低强度方案。六名患者中有四名在接受 AHSCT 后出现了一些残疾进展,因此 AHSCT 只是减缓了多发性硬化症的快速进展。一名患者在 AHSCT 后三个月出现磁共振成像活动,两名患者在随访期间出现轻度复发。我们的患者中没有人出现四级非血液学毒性;所有感染都很轻微。一名患者出现了过敏反应,可能是二甲基亚砜引起的:我们对 6 例患者进行的病例分析表明,AHSCT 是一种很有前景的治疗方法,可减缓多发性硬化症患者临床残疾的快速进展,且安全性良好。
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Immunoablative therapy followed by autologous hematopoietic stem cell transplantation as the first-line disease-modifying therapy in patients with multiple sclerosis.

Introduction: Immunoablative therapy followed by autologous hematopoietic stem cell transplantation (AHSCT) is one of the possible disease-modifying therapies (DMTs) for patients with multiple sclerosis (MS). In this case series, we would like to present six patients with MS, who underwent AHSCT as the first-line DMT.

Case reports: Six MS patients with a rapid progression of disability with or without relapses underwent AHSCT as the first-line DMT at the University Hospital Ostrava between 2018 and 2021. The conditioning regimens for AHSCT used were a medium-intensity regime BEAM (Carmustine, Etoposid, Cytarabin, Melphalan) and low-intensity regime based on Cyclophosphamide. Four out of six patients showed some disability progression after AHSCT, so the rapid progression of MS was just slowed down by AHSCT. One patient developed activity on magnetic resonance imaging three months after AHSCT, and two experienced mild relapses during the follow-up period. None of our patients developed grade 4 non-hematological toxicity; all infections were mild. In one patient, an allergic reaction probably to dimethyl sulfoxide was observed.

Conclusion: Our case series of 6 patients shows that AHSCT is a promising therapeutic approach to slow down the rapid progression of clinical disability in MS patients with a good safety profile.

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来源期刊
Biomedical Papers-Olomouc
Biomedical Papers-Olomouc MEDICINE, RESEARCH & EXPERIMENTAL-
CiteScore
2.30
自引率
0.00%
发文量
74
审稿时长
6-12 weeks
期刊介绍: Biomedical Papers is a journal of Palacký University Olomouc, Faculty of Medicine and Dentistry, Olomouc, Czech Republic. It includes reviews and original articles reporting on basic and clinical research in medicine. Biomedical Papers is published as one volume per year in four issues.
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