Aprepitant Use in Pediatric Cyclical Vomiting Syndrome: A Systematic Review
Abstract
Background: Cyclical vomiting syndrome (CVS) is a disorder of gut–brain interaction characterized by recurrent, stereotyped episodes of intense nausea and vomiting with return to baseline in between. Pediatric CVS often leads to frequent hospitalizations, comorbidities, and poor quality of life. Standard abortive therapies include 5-HT₃ antagonists (e.g. ondansetron) and
triptans, while prophylaxis may involve cyproheptadine or amitriptyline. Aprepitant, an oral neurokinin-1 (NK1) receptor antagonist approved for chemotherapy-induced nausea, is increasingly used off-label in refractory pediatric CVS. Its efficacy and safety in this setting remain unclear.
Methods: We conducted a PRISMA-style systematic review of studies reporting on aprepitant in pediatric CVS. We searched PubMed, Embase, Scopus, Web of Science, and the Cochrane Library from inception to 15 January 2025 for any study (randomized trials, cohorts, case series, reports)
of aprepitant in children (<18) with CVS. Two reviewers independently screened records, extracted data, and assessed risk of bias using standard tools. Outcomes were patient-reported response (episode frequency/intensity), healthcare utilization (episodes/year, admissions), and
adverse events. Due to heterogeneity, results are summarized narratively.
Results: No randomized controlled trials were identified. Evidence consisted of one retrospective controlled cohort study, one retrospective single-center case series, and one case report, with…
How strong is the evidence?
Evidence Hub reads this as a systematic review, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 1,775 participants or records.
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