Migraine affects approximately 10% of children and adolescents and is often disabling, but evidence-based options for acute treatment and prevention remain limited. A high placebo response has also complicated pivotal pediatric trials. Therapies targeting the calcitonin gene-related peptide (CGRP) pathway have reshaped adult migraine care, and the approval of one CGRP monoclonal antibody for pediatric prevention has validated this target in pediatric patients. Gepants, small-molecule oral or intranasal CGRP receptor antagonists, are of particular interest because, unlike injectable antibodies, they offer acute or preventive treatment without vasoconstrictive activity. This review summarizes the pharmacology and adult evidence for the four approved gepants, their emerging pediatric use, and ongoing clinical trials. Depending on the agent, gepants have established efficacy for acute treatment or prevention in adults, with favorable long-term safety and no new hepatic or cardiovascular safety signals. Pediatric evidence is limited to small retrospective studies of off-label use in patients whose migraine is refractory to standard therapy. These reports suggest acceptable tolerability and possible benefit in some patients, but controlled trials are needed to determine efficacy. Phase 3 randomized trials are evaluating ubrogepant for acute treatment and atogepant and rimegepant for the prevention of episodic and chronic migraine in pediatric patients aged 6–17 years for acute treatment and episodic migraine prevention, and 12–17 years for chronic migraine prevention, with estimated completion expected between 2027 and 2032. Until controlled data become available, gepant use should be individualized and restricted to patients who have not responded to standard therapies. Pediatric dosing, long-term safety, and the lower age limit of benefit remain key questions.
Migraine is a representative type of primary headache and a common chronic neurological disease that accounts for a large proportion of headaches in children, adolescents, and adults. Unlike migraine in adulthood, pediatric migraine occurs when brain development is not yet complete. This characteristic may require a new perspective for the treatment and management of pediatric migraine. Dietary therapies, mainly the ketogenic diet and its variants, can have positive effects on pediatric migraine. Several recent studies have revealed that dietary therapies, such as the classic ketogenic diet, modified Atkins diet, and low glycemic index diet, improve various neurological diseases by improving dysbiosis of microbiota, reducing proinflammatory cytokines, and increasing mitochondrial function. Nonetheless, the mechanism through which active dietary therapy affects pediatric migraine requires further research. To achieve this, an important role is played by the neuro-nutritional team, which can develop and manage tolerable diets for pediatric migraine patients through mutual collaboration among pediatric neurologists, nurses, and nutritionists.
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