The single-subject randomized trial. A useful clinical tool for assessing therapeutic efficacy in pediatric practice

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A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
00:04

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition

Published on: September 20, 2019

J C Langer1, A L Winthrop, R M Issenman

  • 1Department of Surgery, McMaster University, Hamilton, Ontario, Canada.

Clinical Pediatrics
|November 1, 1993
PubMed

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.

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