Efficacy of aprepitant as a prophylactic medication in adults with cyclic vomiting syndrome

Abstract

AbstractBackgroundAprepitant is a neurokinin‐1 receptor antagonist, and recent guidelines by the American Neurogastoenterology and Motility Society recommend its use as prophylaxis in moderate‐to severe cyclic vomiting syndrome (CVS). Data are limited to small studies in children. We aimed to determine its efficacy in adults with CVS.MethodsA retrospective review of CVS patients diagnosed using Rome criteria at a tertiary referral center was conducted. Drug response was defined as >50% reduction in symptoms and/or healthcare utilization. An intent‐to‐treat (ITT) analysis was conducted.Key ResultsOf 96 patients prescribed aprepitant, 26 (27%) were unable to start due to cost/lack of insurance coverage. Of 70 receiving therapy, mean age was 33 ± 11 years; 51 (73%) were female and 56 (80%) Caucasian. The majority (93%) were refractory to other prophylactic medications. Aprepitant was taken thrice weekly in 51 (73%), daily in 16 (23%) and a few times a month in 3 (4%) due to cost. Fifty (71.4%) had a global symptom response to aprepitant. There was significant reduction in the number of CVS episodes (14.5 ± 11.7 to 6.2 ± 8.0, p < 0.0001), emergency department visits (4.2 ± 7.7 to 1.8 ± 3.4, p = 0.006), and hospital admissions (1.6 ± 3.9 to 0.8 ± 2.1, p = 0.02) in patients treated with aprepitant. Seven (10%) discontinued the drug due to minor side effects.Conclusions and InferencesAprepitant is a safe and effective prophylactic medication in adults with refractory CVS. Adequate insurance coverage is a major barrier preventing its use.