The Placebo Response in Pediatric Clinical Trials
The Placebo Response in Pediatric Clinical Trials
There are few studies demonstrating the efficacy of drug treatment in children with functional gastrointestinal disease. To address this need, in 2009 Saps and coworkers conducted a multicenter, double-blind, placebo-controlled trial of amitriptyline in children with pain associated with a functional gastrointestinal disorder. Ninety children (ages 8–17 years) with irritable bowel syndrome, functional abdominal pain, or recurrent abdominal dyspepsia were enrolled in the 4-week trial. The children were randomized to receive amitriptyline (10 mg/day for those less than 35 kg or 20 mg/day for those 35 kg or more) or placebo. The primary outcome was pain as reported by the patient and overall sense of improvement, with secondary outcomes related to psychosocial traits and daily functioning. Improvement was noted by 63% of patients in the amitriptyline group and 57.5% of those given placebo (p = 0.63). The percentage reporting feeling worse during the study was 5% in the amitriptyline group and 2.5% in the placebo group. Logistic regression analysis revealed no difference between the groups in the numbers of subjects reporting an overall excellent, good, fair, or poor response. The only significant difference in response was a greater reduction in anxiety in the amitriptyline group (p < 0.001).
Although they found no clear benefit from amitriptyline, the authors viewed their findings as an important piece of information to add to studies on the beneficial response to placebo in children with gastrointestinal disorders. In an editorial accompanying the article, Benninga and Mayer echo the suggestion of the paper's authors that enrollment in the study, which was likely associated with increased education about disease management, reassurance, and allocation of time to discuss the patient's concerns, may have been the primary determinant of the strong placebo response and an indication of the importance of these factors in patient care.
Functional Gastrointestinal Disease
There are few studies demonstrating the efficacy of drug treatment in children with functional gastrointestinal disease. To address this need, in 2009 Saps and coworkers conducted a multicenter, double-blind, placebo-controlled trial of amitriptyline in children with pain associated with a functional gastrointestinal disorder. Ninety children (ages 8–17 years) with irritable bowel syndrome, functional abdominal pain, or recurrent abdominal dyspepsia were enrolled in the 4-week trial. The children were randomized to receive amitriptyline (10 mg/day for those less than 35 kg or 20 mg/day for those 35 kg or more) or placebo. The primary outcome was pain as reported by the patient and overall sense of improvement, with secondary outcomes related to psychosocial traits and daily functioning. Improvement was noted by 63% of patients in the amitriptyline group and 57.5% of those given placebo (p = 0.63). The percentage reporting feeling worse during the study was 5% in the amitriptyline group and 2.5% in the placebo group. Logistic regression analysis revealed no difference between the groups in the numbers of subjects reporting an overall excellent, good, fair, or poor response. The only significant difference in response was a greater reduction in anxiety in the amitriptyline group (p < 0.001).
Although they found no clear benefit from amitriptyline, the authors viewed their findings as an important piece of information to add to studies on the beneficial response to placebo in children with gastrointestinal disorders. In an editorial accompanying the article, Benninga and Mayer echo the suggestion of the paper's authors that enrollment in the study, which was likely associated with increased education about disease management, reassurance, and allocation of time to discuss the patient's concerns, may have been the primary determinant of the strong placebo response and an indication of the importance of these factors in patient care.
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