title: Efficacy of the neurokinin-1 receptor antagonist aprepitant in children with cyclical vomiting syndrome.
abstract: 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).
  
  
  OBJECTIVE
  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%).
  
  
  CONCLUSIONS
  Aprepitant appears effective for both acute and prophylactic management of paediatric cyclical vomiting syndrome refractory to conventional therapies.
authors.affiliation: Department of Gastroenterology, Great Ormond Street Hospital, Institute of Child Health, London, UK.
authors.forename: F
authors.initials: F
authors.lastname: Cristofori
authors.forename: N
authors.initials: N
authors.lastname: Thapar
authors.forename: E
authors.initials: E
authors.lastname: Saliakellis
authors.forename: N
authors.initials: N
authors.lastname: Kumaraguru
authors.forename: M
authors.initials: M
authors.lastname: Elawad
authors.forename: F
authors.initials: F
authors.lastname: Kiparissi
authors.forename: J
authors.initials: J
authors.lastname: Köglmeier
authors.forename: P
authors.initials: P
authors.lastname: Andrews
authors.forename: K J
authors.initials: KJ
authors.lastname: Lindley
authors.forename: O
authors.initials: O
authors.lastname: Borrelli
mesh_terms: D000293:Adolescent; D000932:Antiemetics / Q000009:adverse effects / Q000627:therapeutic use*; D000077608:Aprepitant; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008297:Male; D009025:Morpholines / Q000009:adverse effects / Q000627:therapeutic use*; D064729:Neurokinin-1 Receptor Antagonists / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D014839:Vomiting / Q000188:drug therapy* / Q000517:prevention & control
chemical_list: D000932:Antiemetics; D009025:Morpholines; D064729:Neurokinin-1 Receptor Antagonists; D000077608:Aprepitant