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The single-subject randomized trial. A useful clinical tool for assessing therapeutic efficacy in pediatric practice
J C Langer1, A L Winthrop, R M Issenman
1Department of Surgery, McMaster University, Hamilton, Ontario, Canada.
Insights
Single-subject randomized trials can assess pediatric gastroesophageal reflux treatments. Cisapride effectively reduced symptoms in one child, demonstrating personalized therapeutic assessment.
Area of Science:
- Pediatric Gastroenterology
- Clinical Trial Design
Background:
- Single-subject randomized trials (SSRTs) objectively assess therapeutic efficacy in individual patients.
- SSRTs involve alternating treatments with placebos and blind outcome measurement.
- While common in adults, SSRTs are rarely used in pediatric populations.
Observation:
- Two pediatric patients with gastroesophageal reflux (GER) underwent SSRTs to evaluate cisapride's effectiveness.
- The first patient showed no improvement in vomiting or gagging, with only increased stool frequency.
Findings:
- Cisapride was discontinued in the first patient due to lack of efficacy for target GER symptoms.
- The second patient experienced significant reductions in vomiting and wheezing with cisapride use.
- Cisapride was incorporated into the second patient's ongoing therapeutic regimen based on positive SSRT results.
Implications:
- SSRTs offer a simple, cost-effective method for physicians to personalize pediatric treatment strategies.
- These trials facilitate the rational application of effective therapies and discontinuation of ineffective ones in pediatric practice.
- The study highlights the utility of SSRTs in evaluating drug efficacy for conditions like pediatric GER.
Abstract:
The purpose of a single-subject randomized trial is to assess objectively the efficacy of a specific therapeutic intervention in an individual patient. Treatment is randomly alternated with placebo over a number of study periods. Specific outcome measures are recorded blindly and later compared via paired statistical analysis. Single-subject trials have long been successfully performed in adults, but rarely in children. We present single-subject trials of two pediatric patients done to assess the effect of cisapride on symptoms arising from gastroesophageal reflux. In the first patient, the drug affected neither vomiting nor gagging, although stool frequency increased. Since the symptoms of concern were unaffected, cisapride was discontinued. In the second patient, use of cisapride led to a significant decrease in vomiting and wheezing; the drug was therefore incorporated into the therapeutic regimen. Single-subject randomized trials are inexpensive and simple and can be used by the family physician, pediatrician, or pediatric surgeon in daily practice. They permit the rational use of effective therapy and the abandonment of ineffective measures.