A randomized, double-blinded, placebo-controlled study of the use of viscous oral cromolyn sodium for the treatment of eosinophilic esophagitis.

BACKGROUND: There is a need for effective, nonsteroid pharmacologic therapies for eosinophilic esophagitis (EoE). Cromolyn sodium offers an option because mast cells have been implicated in the symptomatology of EoE and cromolyn has been shown to have some anti-eosinophilic properties.

OBJECTIVE: To evaluate the efficacy of cromolyn in decreasing esophageal eosinophilia in patients with EoE. Secondary outcomes included symptom improvement and adverse effects.

METHODS: We conducted a randomized, double-blinded, placebo-controlled trial of viscous oral cromolyn for EoE in pediatric patients. Subjects were randomized to 100 mg (2-11 years of age) or 200 mg (12-17 years of age) of cromolyn or placebo 4 times daily. The medications were mixed with powdered sugar at home to make them viscous.

RESULTS: Sixteen subjects (50% boys, median age 11.4 years) were enrolled. Nine were randomized to cromolyn and 7 were randomized to placebo. Cromolyn decreased the peak eosinophil count from 62.1 to 57.3 eosinophils per high-powered field (P = .78) and placebo decreased the peak eosinophil count from 87.0 to 71.3 eosinophils per high-powered field (P = .62) One subject randomized to cromolyn and none in the placebo arm had complete resolution of eosinophilia. Cromolyn decreased symptoms scores from a mean baseline score of 37.8 to a mean post-therapy score of 17.5, which was a 54% decrease (P = .04). Placebo decreased the symptom scores from a baseline score of 32.2 to a post-therapy score of 23.3, which was a 28% decrease (P = .05).

CONCLUSION: Cromolyn, when mixed into a viscous preparation, did not significantly decrease esophageal eosinophilia. However, it did decrease symptom scores (although not significantly more than placebo) and led to complete resolution in 1 subject.