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Efficacy of aprepitant as a prophylactic medication in adults with cyclic vomiting syndrome
Milan Patel1, Omeed Partovi1, Harrison Mooers2
1Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Insights
Aprepitant effectively reduces symptoms and healthcare use in adults with cyclic vomiting syndrome (CVS). However, cost and insurance coverage are significant barriers to its use for CVS treatment.
Area of Science:
- Gastroenterology
- Pharmacology
Background:
- Cyclic Vomiting Syndrome (CVS) is a debilitating condition.
- Neurokinin-1 receptor antagonists, like aprepitant, are recommended for CVS prophylaxis.
- Limited data exist on aprepitant's efficacy in adults with CVS.
Purpose of the Study:
- To evaluate the efficacy of aprepitant in adult patients with moderate-to-severe CVS.
- To assess the impact of aprepitant on symptom reduction and healthcare utilization in CVS patients.
Main Methods:
- Retrospective review of CVS patients diagnosed using Rome criteria.
- Intent-to-treat (ITT) analysis of patients prescribed aprepitant.
- Drug response defined as >50% reduction in symptoms and/or healthcare utilization.
Main Results:
- 71.4% of patients experienced a global symptom response to aprepitant.
- Significant reductions observed in CVS episodes, emergency department visits, and hospital admissions.
- Aprepitant was generally well-tolerated, with only 10% discontinuing due to minor side effects.
Conclusions:
- Aprepitant demonstrates safety and efficacy as a prophylactic medication for refractory CVS in adults.
- High cost and inadequate insurance coverage present major obstacles to aprepitant accessibility for CVS treatment.
- Further research may be needed to address cost barriers and expand aprepitant's use in CVS management.
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.
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