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Eptinezumab in treatment-resistant chronic migraine: Four-year real-world effectiveness and treatment persistence

Hsieh WJ, Nicol K, Ng CC, Hou TW, Phan P, Keith SW, Heckel B, Marmura MJ et al.

Cephalalgia : an international journal of headache, 2026

112 participants

Abstract

AimTo evaluate 48-month real-world outcomes of eptinezumab users in chronic migraine (CM), including longitudinal effectiveness, treatment persistence, reasons for discontinuation, and post-discontinuation anti-calcitonin gene-related peptide (CGRP) treatment patterns.MethodsWe conducted a retrospective cohort study of 112 CM subjects initiating eptinezumab at a tertiary headache center between 2019 and 2021. Monthly headache days (MHD), migraine disability assessment (MIDAS), and average pain intensity (API) were extracted at baseline and every 6 months for up to 48 months using a predefined outcome form at each visit. Longitudinal changes were analyzed using generalized estimating equations (GEE) with an unstructured correlation matrix, adjusting for age, sex, and body mass index (BMI). Analyses were performed in four predefined analytic cohorts: all initiators, on-treatment, continuation, and discontinuation. Estimated marginal means (EMMs) quantified adjusted mean changes from baseline at each timepoint. No imputation or censoring for treatment discontinuation was applied. Treatment persistence, reasons for discontinuation, factors associated with discontinuation, and post-discontinuation anti-CGRP treatment patterns were summarized descriptively.ResultsAmong 112 patients (mean age 50.2 ± 13.7; 77.7% female; baseline MHD median 30 [IQR 25-30]), most had failed prior anti-CGRP therapies (90.2% ≥ 1 failure; 70.6% ≥ 2 failures). In all-initiators, significant reductions in…

How strong is the evidence?

Evidence Hub reads this as a cohort study, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 112 participants or records.

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