Efficacy of the neurokinin-1 receptor antagonist aprepitant in children with cyclical vomiting syndrome

Aliment Pharmacol Ther. 2014 Aug;40(3):309-17. doi: 10.1111/apt.12822. Epub 2014 Jun 5.


Background: Aprepitant (Emend, Merck Sharp & Dohme Ltd, Haarlem, the Netherlands), a neurokinin-1 receptor antagonist, prevents vomiting in a range of conditions. No data are available on its use in children with cyclical vomiting syndrome (CVS).

Aim: We investigated the efficacy of aprepitant as prophylactic treatment or acute intervention in CVS children refractory to conventional therapies.

Methods: Forty-one children (median age: 8 years) fulfilling NASPGHAN criteria treated acutely (RegA) or prophylactically (RegP) with aprepitant were retrospectively reviewed. Primary outcome was the clinical response (decrease in frequency and intensity of CVS episodes). Secondary outcomes were: number of CVS episodes/year, number of hospital admissions/year, CVS episode duration, number of vomits/h, symptom-free interval length (days), and school attendance percentage. The follow-up period was 18-60 months.

Results: Sixteen children received RegP and 25 RegA. One child on RegP stopped treatment due to severe migraine. At 12-months on intention-to-treat analysis, 13 children on RegP (81%) achieved either complete (3/16, 19%) or partial (10/16, 62%) clinical response. On RegA, 19 children (76%) had either complete (3/25, 12%) or partial (16/25, 64%) response (P = 0.8 vs. RegP). In both RegP and RegA, there was a significant decrease in CVS episodes/year, hospital admission number/year, CVS episode length, number of vomits/h, as well as an increase in symptom-free interval duration and school attendance percentage. Side effects were reported only in RegP (5/16, 31%) including hiccough (3/16, 19%), asthenia/fatigue (2/16, 12.5%), increased appetite (2/16, 12.5%), mild headache (1/16, 6%) and severe migraine (1/16, 6%).

Conclusion: Aprepitant appears effective for both acute and prophylactic management of paediatric cyclical vomiting syndrome refractory to conventional therapies.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Antiemetics / adverse effects
  • Antiemetics / therapeutic use*
  • Aprepitant
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Male
  • Morpholines / adverse effects
  • Morpholines / therapeutic use*
  • Neurokinin-1 Receptor Antagonists / adverse effects
  • Neurokinin-1 Receptor Antagonists / therapeutic use*
  • Treatment Outcome
  • Vomiting / drug therapy*
  • Vomiting / prevention & control


  • Antiemetics
  • Morpholines
  • Neurokinin-1 Receptor Antagonists
  • Aprepitant

Supplementary concepts

  • Familial cyclic vomiting syndrome