在多发性硬化症患者中，aHSCT 相较于阿仑单抗的获益不仅在于残疾和复发：认知和生活质量持续改善。
Benefits of aHSCT over alemtuzumab in patients with multiple sclerosis besides disability and relapses: Sustained improvement in cognition and quality of life.
机构信息
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.
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; APHM, Hopital de la Timone, CEMEREM, Marseille, France; Aix Marseille Univ, CNRS, CRMBM, UMR 7339, Marseille, France.
出版信息
Mult Scler Relat Disord. 2024 Feb;82:105414. doi: 10.1016/j.msard.2023.105414. Epub 2023 Dec 27.
BACKGROUND
Autologous hematopoietic stem cell transplantation (aHSCT) exhibits promising results for multiple sclerosis (MS) in the short term. We investigated the long-term outcome differences in disease progression and cognitive impairment after aHSCT and alemtuzumab treatment.
METHODS
20 patients receiving aHSCT and 21 patients treated with alemtuzumab between 2007 and 2020 were included in this monocentric observational cohort study. The primary objective was to compare the outcome of both groups with regards to achieving No Evidence of Disease Activity (NEDA-3), defined by the absence of relapses, EDSS progression, and MRI activity. Secondary endpoints in the study included the assessment of neurocognitive functioning, quality of life (QoL), Multiple Sclerosis Functional Composite (MSFC), and EDSS improvement.
RESULTS
Baseline characteristics between both groups were comparable, except for a longer disease duration in the alemtuzumab group of 11.3 years compared to 5.4 years in aHSCT-treated patients (p = 0.002) and a longer mean follow-up time in the aHSCT cohort of 9.0 (range 2.8-15.7) years compared to 5.9 years (range 0.9-9.2) in alemtuzumab patients. NEDA-3 was more frequently observed in the aHSCT group with 75.0 % and 55.0 % at five and 10 years, respectively, than in the alemtuzumab group with only 40.0 % at five years (p = 0.012). Relapse free survival was higher in the aHSCT group (p < 0.001). None of the aHSCT-treated patients showed new T2-lesions six months after therapy initiation until the end of the observational period in contrast to 35.0 % of the alemtuzumab-treated patients showing new T2-lesions (95 %CI 14.2-98.9, p = 0.002). aHSCT-treated patients showed significantly improved cognitive performance in five out of 12 cognitive tests whereas alemtuzumab treated patients deteriorated in four out of 12 tests. Quality of life remained on a constant level for up to 10 years in patients receiving aHSCT with improved scores for the subscale fatigue (p = 0.013).
CONCLUSION
aHSCT seems to be superior to alemtuzumab in maintaining long-term NEDA-3 status, improving cognition and stabilizing quality of life for up to 10 years.
背景
自体造血干细胞移植（aHSCT）在短期内对多发性硬化症（MS）显示出良好的效果。我们研究了 aHSCT 和阿仑单抗治疗后疾病进展和认知障碍的长期结果差异。
方法
本研究纳入了 2007 年至 2020 年间接受 aHSCT 和 21 例阿仑单抗治疗的 20 例患者，为单中心观察队列研究。主要目的是比较两组在实现无疾病活动证据（NEDA-3）方面的结果，NEDA-3 定义为无复发、扩展残疾状况量表（EDSS）进展和 MRI 活动。研究的次要终点包括评估神经认知功能、生活质量（QoL）、多发性硬化功能复合（MSFC）和 EDSS 改善。
结果
两组患者的基线特征相似，除了阿仑单抗组的疾病持续时间较长，为 11.3 年，而 aHSCT 治疗组为 5.4 年（p=0.002），以及 aHSCT 组的平均随访时间较长，为 9.0 年（范围 2.8-15.7），而阿仑单抗组为 5.9 年（范围 0.9-9.2）。NEDA-3 在 aHSCT 组更常见，5 年和 10 年时分别为 75.0%和 55.0%，而阿仑单抗组分别为 40.0%（p=0.012）。aHSCT 组的无复发生存时间更高（p<0.001）。在治疗开始后 6 个月至观察结束期间，没有任何接受 aHSCT 治疗的患者出现新的 T2 病变，而阿仑单抗治疗组有 35.0%的患者出现新的 T2 病变（95%CI 14.2-98.9，p=0.002）。与接受阿仑单抗治疗的患者相比，接受 aHSCT 治疗的患者在 12 项认知测试中的 5 项测试中认知表现显著改善，而阿仑单抗治疗的患者在 12 项测试中的 4 项测试中认知表现恶化。接受 aHSCT 治疗的患者在长达 10 年内生活质量保持稳定，亚量表疲劳的评分提高（p=0.013）。
结论
aHSCT 在维持长期 NEDA-3 状态、改善认知和稳定生活质量方面似乎优于阿仑单抗，长达 10 年。
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