Department of Neurology, Danish Headache Center, Copenhagen University Hospital - Rigshospitalet, Valdemar Hansens Vej 5, Glostrup, 2600, Copenhagen, Denmark.
Department of Clinical Medicine, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.
J Headache Pain. 2023 Jun 12;24(1):70. doi: 10.1186/s10194-023-01604-2.
BACKGROUND: Erenumab has demonstrated effectiveness for prevention of migraine attacks, but the treatment is costly, and a considerable proportion of patients do not respond to it. The Registry for Migraine study (REFORM) was initiated to discover biomarkers that can predict response to erenumab in patients with migraine. The specific objective was to investigate differences in erenumab efficacy based on clinical information, blood-based biomarkers, structural and functional magnetic resonance imaging (MRI), and response to intravenous infusion of calcitonin gene-related peptide (CGRP). In this first report of the REFORM study, we provide a comprehensive description of the study methodology, and present the baseline characteristics of the study population. METHODS: The REFORM study was a single-center, prospective, longitudinal cohort study in adults with migraine who were scheduled to receive preventive treatment with erenumab as part of a separate, open-label, single-arm phase IV trial. The study included four periods: a 2-week screening period (Weeks -6 to -5), 4-week baseline period (Week -4 to Day 1), 24-week treatment period (Day 1 to Week 24), and a 24-week follow-up period without treatment (Week 25 to Week 48). Demographic and clinical characteristics were recorded using a semi-structured interview, whilst outcome data were obtained using a headache diary, patient-reported outcomes, blood sampling, brain MRI, and responsiveness to intravenous infusion of CGRP. RESULTS: The study enrolled 751 participants, with a mean age ± SD of 43.8 ± 12.2 years, of which 88.8% (n = 667) were female. At enrollment, 64.7% (n = 486) were diagnosed with chronic migraine, and 30.2% (n = 227) had history of aura. The mean monthly migraine days (MMDs) was 14.5 ± 7.0. Concomitant preventive medications were used by 48.5% (n = 364) of the participants, and 39.9% (n = 300) had failed ≥ 4 preventive medications. CONCLUSION: The REFORM study enrolled a population with a high migraine burden and frequent use of concomitant medications. The baseline characteristics were representative of patients with migraine in specialized headache clinics. Future publications will report the results of the investigations presented in this article. TRIAL REGISTRATION: The study and sub-studies were registered on ClinicalTrials.gov (NCT04592952; NCT04603976; and NCT04674020).
背景：依瑞奈单抗已被证明可有效预防偏头痛发作，但治疗费用昂贵，且相当一部分患者对此治疗无反应。偏头痛登记研究（REFORM）旨在寻找可预测依瑞奈单抗治疗偏头痛患者反应的生物标志物。具体目标是基于临床信息、基于血液的生物标志物、结构和功能磁共振成像（MRI）以及对降钙素基因相关肽（CGRP）静脉输注的反应来研究依瑞奈单抗疗效的差异。在 REFORM 研究的第一份报告中，我们提供了研究方法的全面描述，并介绍了研究人群的基线特征。
方法：REFORM 研究是一项单中心、前瞻性、纵向队列研究，纳入计划接受依瑞奈单抗预防性治疗的偏头痛成年患者，该研究是一项单独的、开放标签、单臂 IV 期试验的一部分。该研究包括四个阶段：为期 2 周的筛选期（-6 周至-5 周）、为期 4 周的基线期（-4 周至第 1 天）、为期 24 周的治疗期（第 1 天至第 24 周）和为期 24 周的无治疗随访期（第 25 周至第 48 周）。使用半结构式访谈记录人口统计学和临床特征，而使用头痛日记、患者报告的结果、血液采样、脑 MRI 和对 CGRP 静脉输注的反应来获取结果数据。
结果：该研究共纳入 751 名参与者，平均年龄 ± 标准差为 43.8 ± 12.2 岁，其中 88.8%（n = 667）为女性。入组时，64.7%（n = 486）被诊断为慢性偏头痛，30.2%（n = 227）有先兆。每月偏头痛天数（MMD）平均为 14.5 ± 7.0。48.5%（n = 364）的参与者同时使用预防性药物，39.9%（n = 300）曾使用过≥4 种预防性药物治疗失败。
结论：REFORM 研究纳入了偏头痛负担较重且经常同时使用药物的人群。基线特征代表了专门的头痛诊所中偏头痛患者的情况。未来的出版物将报告本文介绍的研究结果。
试验注册：该研究及其子研究已在 ClinicalTrials.gov 上注册（NCT04592952；NCT04603976；和 NCT04674020）。
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