艾拉戈利对比地诺孕素治疗中重度子宫内膜异位症疼痛：III期非劣效研究
该多中心、双盲、双模拟、随机、阳性对照III期非劣效研究在印度19个中心纳入230例子宫内膜异位症中重度疼痛女性，1:1接受口服艾拉戈利150mg每日一次或地诺孕素2mg每日一次治疗24周。第85天NRS疼痛评分较基线变化组间差异0.04（95%CI -0.3, 0.37，p=0.9747），95%CI上限低于预设非劣效界值1.5，证实艾拉戈利非劣效于地诺孕素；第169天整体疼痛、痛经及非经期盆腔痛改善两组相当，不良事件发生率相似，无严重治疗中出现的不良事件或停药，骨密度未见显著变化。
要点
- 艾拉戈利150mg每日一次治疗24周在改善子宫内膜异位症相关疼痛方面非劣效于地诺孕素2mg每日一次。
- 第169天整体疼痛、痛经及非经期盆腔痛改善两组相当，补救药物使用和患者总体印象变化无差异。
- 不良事件发生率艾拉戈利14.8%、地诺孕素19.1%，无严重治疗中出现的不良事件或停药，骨密度未见显著变化。
核心结论
- 艾拉戈利对中重度子宫内膜异位症相关疼痛的疗效非劣效于地诺孕素，且安全性和耐受性可接受。
研究设计与样本
- 多中心、双盲、双模拟、随机、平行组、阳性对照、非劣效III期研究，在印度19个临床中心开展。
- 筛选340例，随机230例（每组115例），年龄18-49岁，诊断为子宫内膜异位症且伴中重度疼痛。
- 参与者1:1随机接受口服艾拉戈利150mg每日一次或地诺孕素2mg每日一次，治疗24周。
主要发现
- 第85天NRS疼痛评分较基线变化组间差异0.04（95%CI -0.3, 0.37），p=0.9747。
- 第169天整体疼痛、痛经和非经期盆腔痛较基线改善两组相当，p值分别为0.9372、0.8884和0.9616。
- 补救药物使用和患者总体印象变化两组可比。
- 不良事件发生率艾拉戈利14.8%、地诺孕素19.1%，无严重治疗中出现的不良事件或停药。
- 未观察到骨密度显著变化。
关键数值与推荐
- 主要终点为非劣效设计，预设非劣效界值为1.5，艾拉戈利组与地诺孕素组第85天NRS变化差异的95%CI上限低于1.5。
临床怎么用
- 艾拉戈利150mg每日一次可作为地诺孕素2mg每日一次之外治疗中重度子宫内膜异位症相关疼痛的选项。
- 治疗24周内需关注不良事件，但本研究中无严重治疗中出现的不良事件或停药，骨密度未见显著变化。
局限性
- 研究仅在印度19个中心开展，样本量230例，结论外推需谨慎。
- 随访至第169天（约24周），缺乏更长期疗效和安全性数据。
以上解读依据论文摘要（全文未开放获取或许可不允许翻译转载）。
原文
OBJECTIVE: To compare the efficacy, safety and tolerability of elagolix with dienogest in women with moderate-to-severe endometriosis-associated pain. DESIGN: A multicentre, double-blind, double-dummy, randomised, parallel-group, active-controlled, non-inferiority phase III study. SETTING: Nineteen clinical centres across India. STUDY POPULATION: Women (18-49 years) diagnosed with endometriosis and experiencing moderate-to-severe pain. METHODS: Participants were randomised (1:1) to receive oral elagolix (150 mg once daily) or dienogest (2 mg once daily) for 24 weeks. OUTCOME MEASURES: The primary outcome was change in endometriosis-related pain (Numeric Rating Scale [NRS]) from baseline to Day 85. Secondary outcomes included changes in NRS (Day 169), dysmenorrhoea, non-menstrual pelvic pain (NMPP) scores (Days 85 and 169), rescue medication use, patient global impression of change (PGIC), adverse events and bone mineral density. RESULTS: Of 340 patients screened, 230 were randomised (115 per group). At Day 85, both arms showed similar reductions in NRS pain scores with a treatment difference of 0.04 (95% CI: -0.3, 0.37) [p = 0.9747] demonstrating non-inferiority as upper 95% CI was below pre-specified margin of 1.5. At Day 169, both arms showed comparable improvements in overall pain, dysmenorrhoea and NMPP from baseline (p = 0.9372, p = 0.8884, and p = 0.9616, respectively). Rescue medication use and PGIC were comparable between treatment arms. Adverse event incidence was similar (elagolix: 14.8%; dienogest: 19.1%), with no serious TEAEs or discontinuations. No significant bone mineral density changes were observed. CONCLUSIONS: Elagolix demonstrated non-inferiority to dienogest with an acceptable safety and tolerability profile, supporting its use in managing endometriosis-associated pain. TRIAL REGISTRATION: ClinicalTrials.gov identifier: CTRI/2023/01/049292.
引用
- Bhoir S, Patel J, Palve T, et al. Efficacy and Safety of Elagolix Versus Dienogest for Treatment of Moderate-to-Severe Endometriosis Pain: A Phase III, Multicentric, Double-Blind, Active-Controlled, Non-Inferiority Study. BJOG. 2026. PMID: 42116589. doi:10.1111/1471-0528.70256
供执业医师工作参考，不替代临床判断；数值与分级以指南原文为准。