多发性硬化症的自体造血干细胞移植：来自挪威的长期随访数据。
Autologous hematopoietic stem cell transplantation for multiple sclerosis: Long-term follow-up data from Norway.
机构信息
Department of Neurology, Haukeland University Hospital, Bergen, Norway.
Haematology Section, Department of Medicine, Haukeland University Hospital, Bergen, Norway.
出版信息
Mult Scler. 2024 May;30(6):751-754. doi: 10.1177/13524585241231665. Epub 2024 Feb 12.
BACKGROUND
Autologous hematopoietic stem cell transplantation (HSCT) is a potent treatment option for patients with aggressive relapsing-remitting multiple sclerosis (RRMS).
OBJECTIVE
To evaluate long-term outcomes of HSCT in MS.
METHODS
National retrospective single-center observational study of patients with aggressive RRMS that underwent HSCT in Norway from January 2015 to January 2018. Criteria for receiving HSCT included at least two clinical relapses the last year while on disease modifying treatment (DMT).
RESULTS
In total, 29 patients, with a mean follow-up time of 70 months (standard deviation:14.3), were evaluated. Twenty patients (69%) had sustained no evidence of disease activity (NEDA-3) status, 24 (83%) were relapse-free, 23 (79%) free of magnetic resonance imaging (MRI) activity, and 26 (90%) free of progression. Number of patients working full-time increased from 1 (3%), before HSCT, to 10 (33%) after 2 years and 15 (52%) after 5 years.
CONCLUSION
HSCT offers long-term disease-free survival with successively increasing work participation in patients with aggressive MS resistant to DMTs.
背景
自体造血干细胞移植（HSCT）是治疗侵袭性复发缓解型多发性硬化症（RRMS）患者的有效治疗选择。
目的
评估 HSCT 在多发性硬化症中的长期疗效。
方法
这是一项在挪威进行的全国性回顾性单中心观察性研究，纳入了 2015 年 1 月至 2018 年 1 月期间接受 HSCT 的侵袭性 RRMS 患者。接受 HSCT 的标准包括在疾病修正治疗（DMT）期间的过去一年中至少有两次临床复发。
结果
共评估了 29 名患者，平均随访时间为 70 个月（标准差：14.3）。20 名患者（69%）达到持续无疾病活动（NEDA-3）状态，24 名患者（83%）无复发，23 名患者（79%）无磁共振成像（MRI）活动，26 名患者（90%）无进展。全职工作的患者人数从 HSCT 前的 1 名（3%）增加到 2 年后的 10 名（33%）和 5 年后的 15 名（52%）。
结论
HSCT 为对 DMT 耐药的侵袭性 MS 患者提供了长期无疾病生存，并逐渐增加了患者的工作参与度。
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