Frequent fragility of randomized controlled trials for HCC treatment

BMC Cancer. 2021 Apr 9;21(1):389. doi: 10.1186/s12885-021-08133-8.

Abstract

Background: The fragility index (FI) of trial results can provide a measure of confidence in the positive effects reported in randomized controlled trials (RCTs). The aim of this study was to calculate the FI of RCTs supporting HCC treatments.

Methods: A methodological systematic review of RCTs in HCC treatments was conducted. Two-arm studies with randomized and positive results for a time-to-event outcome were eligible for the FI calculation.

Results: A total of 6 trails were included in this analysis. The median FI was 0.5 (IQR 0-10). FI was ≤7 in 4 (66.7%) of 6 trials; in those trials the fragility quotient was ≤1%.

Conclusion: Many phase 3 RCTs supporting HCC treatments have a low FI, which challenges the confidence in concluding the superiority of these drugs over control treatments.

Keywords: Endpoint; Fragility index; Randomized controlled trials.

Publication types

  • Meta-Analysis
  • Systematic Review

MeSH terms

  • Carcinoma, Hepatocellular / therapy*
  • Clinical Trials, Phase III as Topic
  • Humans
  • Liver Neoplasms / therapy*
  • Models, Statistical*
  • Outcome Assessment, Health Care / methods*
  • Randomized Controlled Trials as Topic* / methods
  • Randomized Controlled Trials as Topic* / standards