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


BACKGROUND
ClinicalTrials.gov, NCT01595932.
Pediatric Gastroenterology and Liver Unit, Sapienza - University of Rome, Rome, Italy. salvatore.cucchiara@uniroma1.it.
Giovanni
G
Di Nardo
Salvatore
S
Oliva
Federica
F
Ferrari
Saverio
S
Mallardo
Giovanni
G
Barbara
Cesare
C
Cremon
Marina
M
Aloi
Salvatore
S
Cucchiara
D015746:Abdominal Pain / Q000188:drug therapy* / Q000209:etiology; D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D005260:Female; D005414:Flatulence / Q000150:complications / Q000188:drug therapy*; D005740:Gases; D005765:Gastrointestinal Agents / Q000627:therapeutic use*; D006801:Humans; D007422:Intestines; D008297:Male; D010147:Pain Measurement; D016896:Treatment Outcome; D000519:alpha-Galactosidase / Q000627:therapeutic use*
D005740:Gases; D005765:Gastrointestinal Agents; D000519:alpha-Galactosidase