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

Background: Aprepitant 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.

Methods: A 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 Results: Of 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 Inferences: Aprepitant is a safe and effective prophylactic medication in adults with refractory CVS. Adequate insurance coverage is a major barrier preventing its use.