Ubrogepant可有效治疗偏头痛，这一成果由美国梅奥医学中心David W. Dodick团队取得。这一研究成果发表在2019年12月5日出版的国际学术期刊《新英格兰医学杂志》上。

Ubrogepant是一种口服小分子降钙素基因相关肽受体拮抗剂，可用于治疗急性偏头痛。

研究组进行了一项随机试验，以评估Ubrogepant的疗效、安全性和副作用。共招募了1672名先兆型或无先兆偏头痛患者，按1：1：1随机分组，其中559名接受安慰剂治疗，556名接受50mg Ubrogepant治疗，557名接受100mg Ubrogepant治疗。

安慰剂组有11.8%的患者在2小时内疼痛消失，显著低于50mg Ubrogepant组（19.2%）和100mg Ubrogepant组（21.2%）。安慰剂组有27.8%的患者在2小时内摆脱了最烦人症状，显著少于50mg Ubrogepant组（38.6%）和100mg Ubrogepant组（37.7%）。

安慰剂组有12.8%的患者在首次或第二次给药48小时内出现不良事件，50mg Ubrogepant组和100mg Ubrogepant组分别有9.4%和16.3%。最常见的不良反应是恶心、嗜睡和口干，且在100mg Ubrogepant组中更常见。30天内发生的严重不良事件主要包括阑尾炎、自然流产、心包积液和癫痫发作，但在给药48小时内均未发生。

总之，服用Ubrogepant与安慰剂相比可显著减少2小时疼痛与最烦人的症状。最常见的不良反应是恶心、嗜睡和口干。仍需进一步试验来确定Ubrogepant治疗急性偏头痛的持久性和安全性。

附：英文原文

Title: Ubrogepant for the Treatment of Migraine

Author: David W. Dodick, M.D.,, Richard B. Lipton, M.D.,, Jessica Ailani, M.D.,, Kaifeng Lu, Ph.D.,, Michelle Finnegan, M.P.H.,, Joel M. Trugman, M.D.,, and Armin Szegedi, M.D.

Issue&Volume: 2019-12-04

Abstract:

Background

Ubrogepant is an oral, small-molecule calcitonin gene–related peptide receptor antagonist for acute migraine treatment.

Methods

We conducted a randomized trial to evaluate the efficacy, safety, and side-effect profile of ubrogepant. We assigned adults with migraine, with or without aura, in a 1:1:1 ratio to receive an initial dose of placebo, ubrogepant at a dose of 50 mg, or ubrogepant at a dose of 100 mg for treatment of a single migraine attack, with the option to take a second dose. The coprimary efficacy end points were freedom from pain at 2 hours after the initial dose and absence of the most bothersome migraine-associated symptom at 2 hours. Secondary end points included pain relief (at 2 hours), sustained pain relief (from 2 to 24 hours), sustained freedom from pain (from 2 to 24 hours), and absence of symptoms associated with migraine (photophobia, phonophobia, and nausea) at 2 hours.

Results

A total of 1672 participants were enrolled; 559 were assigned to receive placebo, 556 to receive 50 mg of ubrogepant, and 557 to receive 100 mg of ubrogepant. The percentage of participants who had freedom from pain at 2 hours was 11.8% in the placebo group, 19.2% in the 50-mg ubrogepant group (P=0.002, adjusted for multiplicity, for the comparison with placebo), and 21.2% in the 100-mg ubrogepant group (P<0.001). The percentage of participants who had freedom from the most bothersome symptom at 2 hours was 27.8% in the placebo group, 38.6% in the 50-mg ubrogepant group (P=0.002), and 37.7% in the 100-mg ubrogepant group (P=0.002). Adverse events within 48 hours after the initial or optional second dose were reported in 12.8% of participants in the placebo group, in 9.4% in the 50-mg ubrogepant group, and in 16.3% in the 100-mg ubrogepant group. The most common adverse events were nausea, somnolence, and dry mouth (reported in 0.4 to 4.1%); these events were more frequent in the 100-mg ubrogepant group (reported in 2.1 to 4.1%). Serious adverse events reported within 30 days in the ubrogepant groups included appendicitis, spontaneous abortion, pericardial effusion, and seizure; none of the events occurred within 48 hours after the dose.

Conclusions

A higher percentage of participants who received ubrogepant than of those who received placebo had freedom from pain and absence of the most bothersome symptom at 2 hours after the dose. The most commonly reported adverse events were nausea, somnolence, and dry mouth. Further trials are needed to determine the durability and safety of ubrogepant for acute migraine treatment and to compare it with other drugs for migraine.

DOI: 10.1056/NEJMoa1813049

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1813049