A Pilot Study of Ketotifen in Patients Aged 8-17 Years with Functional Dyspepsia Associated with Mucosal Eosinophilia.

Creator(s)

Chance S. Friesen, Children's Mercy HospitalFollow
Valentina Shakhnovich
Paul C. Toren, Children's Mercy HospitalFollow
Brandon Retke, Children's Mercy Kansas CityFollow
Jennifer Verrill Schurman, Children’s Mercy Kansas CityFollow
Jennifer Colombo, Children's Mercy HospitalFollow
Amanda D. Deacy, Children's Mercy HospitalFollow
Craig A. Friesen, Children's Mercy HospitalFollow
Susan Abdel-Rahman

Document Type

Article

Publication Date

7-2024

Identifier

DOI: 10.1007/s40272-024-00628-8

Abstract

BACKGROUND AND OBJECTIVE: Mast cells have been implicated in abdominal pain-associated disorders of gut-brain interaction, such as functional dyspepsia. As such, ketotifen, a second-generation antihistamine and mast cell stabilizer, could represent a viable treatment option in these conditions. The primary aim of the current pilot study was to assess clinical response to ketotifen and assess pharmacokinetics in youth with functional dyspepsia.

METHODS: We conducted a pilot randomized, double-blind, placebo-controlled, cross-over trial of ketotifen in 11 youth with functional dyspepsia and duodenal mucosal eosinophilia with 4 weeks of active treatment at a dose of 1 mg twice daily. Global clinical response was graded on a 5-point Likert Scale. A single plasma sample was obtained at steady state for pharmacokinetic analysis.

RESULTS: Ketotifen was not superior to placebo with regard to global clinical response. Only 18% of patients demonstrated a complete or near-complete clinical response. The estimated half-life was 3.3 h.

CONCLUSIONS: While ketotifen was not superior to placebo, this study highlights several important challenges for developing drug trials for youth with chronic abdominal pain. Recommendations are made for designing a larger treatment trial for ketotifen in this patient group.

CLINICAL TRIAL REGISTRATION: This study was registered at ClinicalTrials.gov: NCT02484248.

Journal Title

Paediatric drugs

Volume

26

Issue

4

First Page

451

Last Page

457

MeSH Keywords

Humans; Ketotifen; Pilot Projects; Child; Adolescent; Dyspepsia; Double-Blind Method; Female; Cross-Over Studies; Male; Eosinophilia; Histamine H1 Antagonists; Intestinal Mucosa; Abdominal Pain; Treatment Outcome

Keywords

Ketotifen; Pilot Projects; Dyspepsia; Double-Blind Method; Cross-Over Studies; Eosinophilia; Histamine H1 Antagonists; Intestinal Mucosa; Abdominal Pain; Treatment Outcome

Recommended Citation

Friesen CS, Shakhnovich V, Toren P, et al. A Pilot Study of Ketotifen in Patients Aged 8-17 Years with Functional Dyspepsia Associated with Mucosal Eosinophilia. Paediatr Drugs. 2024;26(4):451-457. doi:10.1007/s40272-024-00628-8