PPI's efficacy and safety questioned for pediatric GERD

May 1, 2011

The effectiveness and safety of proton pump inhibitors for pediatric gastroesophageal reflux disease are far from proven, according to a new systematic review.

Key Points

The effectiveness and safety of proton pump inhibitors (PPIs) for pediatric gastroesophageal reflux disease (GERD) are far from proven, according to a new systematic review published on-line, ahead-of-print in Pediatrics.

Despite this, lead author Rachel van der Pol, MD, from the department of pediatric gastroenterology and nutrition, Emma Children's Hospital AMC, Amsterdam, Netherlands, said, "Use of PPIs for the treatment of GERD in children has increased enormously." Six-month costs of pediatric GERD are estimated to exceed $2,300 per patient.

Dr van der Pol and co-investigators conducted a systematic literature review of the PubMed, Embase, and Cochrane databases in an attempt to unearth all available randomized controlled trials investigating the efficacy and safety of PPIs in children (aged 0 to 18 years) with GERD. Ultimately, the investigators identified 12 eligible trials enrolling a total of 895 patients. PPIs evaluated in eligible studies included omeprazole, esomeprazole, pantoprazole, and lansoprazole.

Furthermore, the researchers noted that while there is a lack of placebo-controlled trials in children and adolescents, available data comparing PPIs to active controls such as alginate and histamine-2 receptor blockers suggests these anti-secretory agents are no more efficacious than comparators.

PPI safety in the pediatric population was also evaluated in this literature review. Authors commented that short-term PPI use appeared well tolerated, "but evidence to ensure safety is still lacking."

Taken together the findings prompted investigators to recommend, "...physicians should be careful when prescribing PPIs, medications that are not approved for infants and have potential adverse effects, unless there is documented disease or with careful monitoring."

The investigators highlighted that there was a paucity of methodologically rigorous, well-designed randomized controlled trials addressing this topic published in the medical literature, and that such trials had only meager sample sizes. The authors concluded by calling for larger randomized controlled trials to be conducted in pediatric patients of every age in order to address what they referred to as "a growing healthcare problem."