Institute of Neuroimmunology and Multiple Sclerosis, University Medical Centre Hamburg-Eppendorf, Falkenried 94, Hamburg, 20251, Germany.
Department of Neurology, University Medical Centre Hamburg-Eppendorf, Martinistraße 52, Hamburg, 20246, Germany.
Ann Clin Transl Neurol. 2021 Jun;8(6):1269-1278. doi: 10.1002/acn3.51366. Epub 2021 May 5.
Autologous hematopoietic stem cell transplantation (aHSCT) is increasingly recognized as a potential therapy for patients with highly active multiple sclerosis (MS). This study aims to assess outcome differences in disease activity in MS patients treated either with aHSCT or alemtuzumab.
We conducted a monocentric registry-based cohort study by recording the clinical course (EDSS and relapses), MRI parameters (new T2 lesions), and neuropsychological assessment in all 19 MS patients receiving aHSCT, and all 21 patients receiving alemtuzumab between 2007 and 2018. We used survival analyses of no evidence of disease activity (NEDA) as the primary objective which was defined by no EDSS progression, no relapse, and no new T2 lesion on MRI. Secondary objectives were EDSS improvement and neurocognitive performance.
Both treatment groups were similar in respect of age, gender, disability, and neurocognitive performance except for significantly longer disease duration in the alemtuzumab group. Mean follow-up was 58.8 [range 29-140] months in the aHSCT group compared to 27.6 [range 11-52] months in the alemtuzumab-treated group. We observed significantly more patients maintaining NEDA in the aHSCT group (p = 0.048) compared to the alemtuzumab-treated patients. Furthermore, 37% of the aHSCT patients showed an improvement of EDSS compared to none in the alemtuzumab-treated group (p = 0.033). It is of note that cognitive function was significantly improved in the aHSCT-treated patients.
aHSCT suppresses inflammatory activity more effectively than alemtuzumab and might enable improvement of overall disability and cognition in MS.
自体造血干细胞移植（aHSCT）越来越被认为是治疗高度活跃性多发性硬化症（MS）患者的一种潜在疗法。本研究旨在评估接受 aHSCT 或阿仑单抗治疗的 MS 患者在疾病活动方面的结果差异。
我们通过记录 19 名接受 aHSCT 的 MS 患者和 2007 年至 2018 年间接受阿仑单抗治疗的 21 名患者的临床病程（EDSS 和复发）、MRI 参数（新 T2 病变）和神经心理学评估，进行了一项基于单中心登记的队列研究。我们使用无疾病活动证据（NEDA）的生存分析作为主要目标，该目标定义为 EDSS 无进展、无复发和 MRI 上无新 T2 病变。次要目标是 EDSS 改善和神经认知表现。
除了阿仑单抗组的疾病持续时间明显更长外，两组在年龄、性别、残疾和神经认知表现方面相似。aHSCT 组的平均随访时间为 58.8 [范围 29-140] 个月，而阿仑单抗治疗组为 27.6 [范围 11-52] 个月。我们观察到在 aHSCT 组中维持 NEDA 的患者明显更多（p=0.048），而阿仑单抗治疗组中维持 NEDA 的患者则较少。此外，与阿仑单抗治疗组相比，aHSCT 组中有 37%的患者 EDSS 得到改善（p=0.033），而阿仑单抗治疗组中没有患者 EDSS 得到改善。值得注意的是，aHSCT 治疗的患者认知功能显著改善。
aHSCT 比阿仑单抗更有效地抑制炎症活动，可能改善 MS 的总体残疾和认知功能。
