Background: There is growing evidence that aprepitant can be helpful for patients with cyclic vomiting syndrome (CVS) and cannabinoid hyperemesis syndrome (CHS), but experience in children remains limited. Our objective is to review the efficacy and safety of aprepitant in the treatment of children with CVS and CHS.
Methods: We performed a retrospective review of children ages 0-18 years with a diagnosis of CVS or CHS based on Rome IV criteria who were treated with aprepitant for nausea or vomiting from 2018-2023. Demographic information, medical history, prior treatment, dose and duration of aprepitant treatment, side effects, and response were recorded. Response was assessed based on physician assessment and patient report documented in the electronic medical record. For patients who received aprepitant multiple times, data was collected from the first instance.
Results: We included 88 children with CVS or CHS who were treated with aprepitant and had follow-up data. 58 (66%) children had CVS and 30 (34%) had CHS. Patient characteristics are shown in Table 1. Children with CHS were older than those with CVS. Treatments given prior to aprepitant are shown in Table 2. Children with CVS were more likely to have been treated with neuromodulators and cyproheptadine, while those with CHS were more likely to have used topical capsaicin. Of the 58 CVS patients, 37 (64%) were treated with aprepitant as an inpatient, 9 (16%) as an outpatient, and 12 (21%) received a dose of aprepitant in the emergency department before discharge. The majority (90%) were prescribed a 3-day course. 49 (84%) patients experienced overall improvement in nausea and vomiting with aprepitant. Two patients were subsequently prescribed aprepitant to be used twice weekly as prophylaxis due to success with the initial response. Two patients reported a side effect of increased fatigue and one patient developed a rash. Of the 30 CHS patients, 29 (97%) were treated with aprepitant as an inpatient and 1 (3%) received a dose of aprepitant in the emergency department before discharge. The majority (77%) were prescribed a 3-day course, although course duration ranged from a single dose to a 5-day course. 29 (97%) patients experienced overall improvement. No side effects were reported. The likelihood of improvement with aprepitant was similar between CVS and CHS groups.
Conclusion: In a large cohort of children with CVS or CHS treated with aprepitant, we found that aprepitant was similarly effective for nausea and vomiting caused by either disorder. Side effects were rare. Prospective studies are needed to further our understanding of the role of aprepitant in the management of children with these disorders.

<b>Table 1: </b>Patient characteristics by diagnosis.

Table 1: Patient characteristics by diagnosis.

<b>Table 2: </b>Prior treatments by diagnosis.

Table 2: Prior treatments by diagnosis.

Nicole Schottenstein

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