Real-world integration of rimegepant into pediatric migraine care: treatment selection, persistence, and longitudinal trajectories

Abstract Background Rimegepant is increasingly entering pediatric migraine care, but little is known about how scheduled preventive use is selected, integrated with existing treatment, and subsequently adapted in routine practice. We characterized treatment selection, four-month clinical outcomes, and longitudinal treatment trajectories after initiation of scheduled rimegepant. Methods This multicenter retrospective real-world cohort study used data from the China Headache and Vertigo Registry Study. Within a 448-patient pediatric/adolescent source population, 109 patients were linked by original registry identifiers to clinically verified follow-up of at least four months; 64 initiated scheduled preventive rimegepant and 45 were managed without scheduled rimegepant. Potential selection into longitudinal follow-up was assessed using linked-versus-non-linked comparisons, including a center-conditioned sensitivity analysis. Among scheduled-rimegepant initiators, monthly headache days (MHD), response thresholds, background preventive treatment, treatment duration, and subsequent treatment trajectories were evaluated. Results Linked patients showed no major measured registry-baseline imbalance relative to non-linked patients, including in the analysis restricted to centers contributing longitudinal cases. Scheduled rimegepant was more often introduced in patients with chronic migraine and greater preventive-treatment complexity; chronic migraine (aOR 4.71, 95% CI 1.13–19.63) and concurrent amitriptyline use (aOR 3.29, 95% CI 1.40–7.75) were associated with treatment selection. Among 64 initiators, mean MHD decreased from 16.58 ± 10.62 to 5.82 ± 5.78 days/month, and 46/64 (71.9%) achieved a ≥ 50% reduction. At four months, 10/64 remained on scheduled treatment, 6/64 transitioned to as-needed (PRN) use, and 48/64 discontinued rimegepant. All 48 patients who discontinued rimegepant continued their background conventional preventive therapy. Of these discontinuations, 20 followed complete or marked improvement, 27 followed insufficient or absent effectiveness, and 1 was attributed to nausea. Improvement-associated de-escalation occurred in 26/64 (40.6%). Conclusions Scheduled rimegepant was preferentially integrated into more complex pediatric migraine care, while subsequent exposure was dynamically adapted within an ongoing preventive regimen. Clinical response, persistence, and therapeutic role were related but non-interchangeable dimensions of the longitudinal treatment course. These findings characterize a real-world treatment pathway rather than comparative rimegepant effectiveness.