THE SINGLE-SUBJECT RANDOMIZED TRIAL - A USEFUL CLINICAL TOOL FOR ASSESSING THERAPEUTIC EFFICACY IN PEDIATRIC PRACTICE

The purpose of a single-subject randomized trial is to assess objectiv ely the efficacy of a specific therapeutic intervention in an individu al patient. Treatment is randomly alternated with placebo over a numbe r 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 childr en. We present single-subject trials of two pediatric patients done to assess the effect of cisapride on symptoms arising from gastroesophag eal reflux. In the first patient, the drug affected neither vomiting n or gagging, although stool frequency increased. Since the symptoms of concern were unaffected, cisapride was discontinued. In the second pat ient, use of cisapride led to a significant decrease in vomiting and w heezing; the drug was therefore incorporated into the therapeutic regi men. Single-subject randomized trials are inexpensive and simple and c an 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.