The fragility of statistically significant findings from randomized controlled trials in pediatric appendicitis: A
Tessa Robinson1, Noora Al-Shahwani2, Bethany Easterbrook3
1McMaster Pediatric Surgery Research Collaborative, McMaster University, Hamilton, Ontario, Canada; Division of Pediatric General Surgery, Department of Surgery, McMaster University, Hamilton, Ontario, Canada.
Insights
Pediatric appendicitis randomized controlled trials (RCTs) often have fragile results. A small change in patient outcomes could alter statistical significance, suggesting caution when changing clinical practice based on these studies.
Area of Science:
- Pediatric surgery
- Clinical trial methodology
- Evidence-based medicine
Background:
- Randomized controlled trials (RCTs) are crucial for evidence-based practice in pediatric appendicitis.
- The robustness and reliability of existing evidence from pediatric appendicitis RCTs are not well-established.
- Limited RCTs in this field necessitate an evaluation of their result stability.
Purpose of the Study:
- To assess the fragility of results in existing randomized controlled trials (RCTs) for pediatric appendicitis.
- To determine how sensitive the statistical significance of outcomes is to small changes in data.
- To inform clinical decision-making by understanding the reliability of current evidence.
Main Methods:
- Systematic literature search of Embase and MEDLINE databases.
- Inclusion criteria: two-armed RCTs, pediatric patients (0-17) with appendicitis, at least one statistically significant dichotomous outcome.
- Calculation of the Fragility Index (FI) for one significant outcome per trial using Fisher's exact test (p < 0.05).
Main Results:
- Six RCTs met the inclusion criteria.
- Median patient cohort size was 103 (IQR 86-127), with a median of 18 events (IQR 4.5-41.25).
- The median Fragility Index (FI) was 3 (IQR 0.75-4.25), indicating that altering just 3 patient outcomes could change a significant result to non-significant.
Conclusions:
- Results from pediatric appendicitis RCTs may be fragile and should be interpreted with caution.
- The Fragility Index (FI) offers valuable insight into result stability beyond p-values.
- Consideration of the FI is recommended for investigators and clinicians when evaluating evidence for practice changes.
Purpose:
Randomized controlled trials (RCT) in pediatric appendicitis remain limited, and the robustness of available evidence is unknown. The aim of this study was to determine the fragility of results in pediatric appendicitis RCTs.
Methods:
A systematic search of Embase and MEDLINE was performed. Eligible studies were two-armed RCTs that included at least one statistically significant dichotomous outcome, had parallel-group allocation, and assessed pediatric patients (0-17) with a primary diagnosis of appendicitis. The Fragility Index (FI) for one statistically significant outcome per trial was calculated using a Fisher's exact test, with statistical significance set at p < 0.05.
Results:
Six studies were identified for inclusion. Studies included a median of 103 patients (interquartile range [IQR] 86-127), with a median of 18 (IQR 4.5-41.25) events for analyzed outcomes. The primary outcome variable was included in analysis for 4(67%) studies. The median FI across studies was 3 (IQR 0.75-4.25), with results ranging from 0 to 5. Results indicate that overall, converting 3 patients from non-events to events in a single trial arm would change the significant dichotomous outcome to nonsignificant.
Conclusion:
The fragility of results in RCTs in pediatric appendicitis should be considered before clinical practice is changed. Investigators should consider reporting the FI alongside study results, as p-values alone may be misleading.
Type Of Study:
Randomized Controlled Trial.
Level Of Evidence:
Level I.
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