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免疫消融治疗继自体造血干细胞移植作为多发性硬化症患者的一线疾病修正治疗。

Immunoablative therapy followed by autologous hematopoietic stem cell transplantation as the first-line disease-modifying therapy in patients with multiple sclerosis.

机构信息

Department of Hematooncology, University Hospital Ostrava, Czech Republic.

Department of Neurology, University Hospital Ostrava, Czech Republic.

出版信息

Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub. 2024 Mar;168(1):50-54. doi: 10.5507/bp.2023.023. Epub 2023 Jun 16.

DOI:10.5507/bp.2023.023
PMID:37337857
Abstract

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.

摘要

介绍

免疫消融治疗继自体造血干细胞移植(AHSCT)是多发性硬化症(MS)患者可能的疾病修正治疗(DMT)之一。在本病例系列中,我们想介绍 6 例接受 AHSCT 作为一线 DMT 的 MS 患者。

病例报告

2018 年至 2021 年,在奥斯特拉瓦大学医院,6 例有或无复发的残疾快速进展的 MS 患者接受 AHSCT 作为一线 DMT。AHSCT 使用的预处理方案为中强度方案 BEAM(卡莫司汀、依托泊苷、阿糖胞苷、马法兰)和基于环磷酰胺的低强度方案。6 例患者中有 4 例在 AHSCT 后出现了一些残疾进展,因此 AHSCT 只是减缓了 MS 的快速进展。1 例患者在 AHSCT 后 3 个月出现磁共振成像活动,2 例患者在随访期间出现轻度复发。我们的患者均未发生 4 级非血液学毒性;所有感染均为轻度。在 1 例患者中,观察到可能与二甲基亚砜有关的过敏反应。

结论

我们的 6 例患者的病例系列表明,AHSCT 是一种很有前途的治疗方法,可以减缓 MS 患者临床残疾的快速进展,具有良好的安全性。

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