Efficacy and tolerability of α-galactosidase in treating gas-related symptoms in children: a randomized, double-blind, placebo controlled trial

Abstract

Background: Gas-related symptoms represent very common complaints in children. The reduction of gas production can be considered as a valuable target in controlling symptoms. α-galactosidase has been shown to reduce gas production and related symptoms in adults. To evaluate the efficacy and tolerability of α-galactosidase in the treatment of gas-related symptoms in pediatric patients.

Methods: Single center, randomized, double-blind, placebo-controlled, parallel group study performed in tertiary care setting. Fifty-two pediatric patients (32 female, age range 4-17) with chronic or recurrent gas-related symptoms were randomized to receive placebo (n = 25) or α-galactosidase (n = 27). Both treatments were given as drops or tablets, according to body weight for 2 weeks. The primary endpoint was the reduction in global distress measured by the Faces Pain Scale-Revised (FPS-R) at the end of treatment compared to baseline. Secondary endpoints were the reduction in severity and frequency of gas-related symptoms as recorded by parents and/or children.

Results: α-galactosidase significantly reduced global distress (p = 0.02) compared to placebo. The digestive enzyme decreased the number of days with moderate to severe bloating (p = 0.03) and the proportion of patients with flatulence (p = 0.02). No significant differences were found for abdominal spasms and abdominal distension. No adverse events were reported during treatment.

Conclusions: Although larger and longer trials are needed to confirm this result, α-galactosidase seems to be a safe, well tolerated and effective treatment for gas-related symptoms in the pediatric population.

Trial registration: ClinicalTrials.gov, NCT01595932.

Figure 1

Study flow chart.

Figure 2

Change of FPS-score in any patient before and after the treatment.

Figure 3

Physician overall evaluation at end of treatment and at follow-up.