Background

Aprepitant is a neurokinin-1 receptor antagonist (NK1R) approved for the treatment of chemotherapy-induced nausea. We aimed to investigate the safety and efficacy of aprepitant in patients with subacute sclerosing panencephalitis (SSPE).

Methods

A randomized, double-blind, placebo-controlled study was conducted in patients with SSPE assigned to receive two courses of aprepitant 250 mg/day p.o. or placebo for 15 days with an interval of 2 months between courses. Primary endpoints were safety and tolerability and secondary endpoint, clinical improvement/stabilization assessed by SSPE Scoring System. Electroencephalography (EEG), brain magnetic resonance imaging (MRI) and cerebrospinal fluid measles-specific IgG index were evaluated before and after treatment.

Results

Sixty-two SSPE patients were allocated to aprepitant (n=31, median age 18 years) or placebo (n=31, median age 22 years) groups. Fifteen patients left the study within the first 6 months and 12 patients left between 6-12 months. Aprepitant was well tolerated and treatment–associated adverse events were similar to those described in the treatment of nausea. Clinical status at 6 and 12 months follow-up did not differ between aprepitant and placebo groups. Post-treatment EEG scores at 12 months were better in the aprepitant group (p=0.015). Cerebral atrophy on MRI increased in both groups while measles-specific IgG index decreased in the placebo group.

Conclusion

In this first clinical trial of aprepitant treatment in SSPE patients, the drug was safe and well tolerated. No clinical effect was observed. A modest improvement in EEG findings might justify trials for longer periods because EEG changes can precede clinical findings in SSPE.

中文翻译：

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阿瑞匹坦在治疗亚急性硬化性全脑炎中的作用：一项随机，双盲，安慰剂对照的研究。

背景

Aprepitant是一种神经激肽-1受体拮抗剂（NK1R），已被批准用于治疗化疗引起的恶心。我们旨在研究阿瑞匹坦在亚急性硬化性全脑炎（SSPE）患者中的安全性和有效性。

方法

在SSPE患者中进行了一项随机，双盲，安慰剂对照研究，他们被分配接受两个疗程的250毫克/天的阿瑞吡坦或安慰剂治疗15天，每个疗程之间的间隔为2个月。主要终点为安全性和耐受性，次要终点为SSPE评分系统评估的临床改善/稳定性。治疗前后评估脑电图（EEG），脑磁共振成像（MRI）和脑脊髓液麻疹特异性IgG指数。

结果

62例SSPE患者被分为阿瑞匹坦组（n = 31，中位年龄18岁）或安慰剂组（n = 31，中位年龄22岁）。在最初的6个月内有15例患者退出研究，而在6到12个月之间有12例患者退出研究。阿瑞匹坦耐受性良好，与治疗相关的不良事件与恶心治疗中描述的事件相似。阿瑞吡坦组和安慰剂组在6个月和12个月随访时的临床状况无差异。阿瑞吡坦组治疗后12个月的脑电图评分更好（p = 0.015）。两组的MRI脑萎缩均增加，而安慰剂组的麻疹特异性IgG指数降低。

结论

在SSPE患者中先兆蛋白治疗的第一项临床试验中，该药物安全且耐受良好。没有观察到临床效果。脑电图检查结果的适度改善可能需要较长时间进行试验，因为脑电图检查的改变可以先于SSPE的临床发现。