Assessment of Accuracy of Waterfall Plot Representations of Response Rates in Cancer Treatment Published in Medical Journals

Autor: Vinay Prasad, Myung S. Kim
Jazyk: angličtina
Rok vydání: 2019
Předmět:
Zdroj: JAMA Network Open
ISSN: 2574-3805
Popis: Key Points Question How accurate are waterfall plots in representing overall response rates reported in clinical trials? Findings In this cross-sectional study of 126 studies published in 6 journals, waterfall plots showed visual response rates that were 6.1% higher compared with response rates based on investigator review and 12.0% higher compared with response rates based on central review. Use of waterfall plots has increased from 0% of original articles in 2004 to 7% in 2018. Meaning This study suggests that waterfall plots are used more frequently over time and exaggerate the visual estimate of the response rate.
This cross-sectional study of oncology articles in high–impact factor journals examines the frequency with which waterfall plots are used and measures the accuracy of these plots in representing response rate compared with investigator-assessed and centrally assessed rates.
Importance Response rates are a well-recognized outcome of clinical trials and provide an objective measure of drug activity. Objectives To quantify the difference between objective response rate and visual representation of response in waterfall plots in recent articles in major medical journals and to assess the change in frequency over time with which waterfall plots are used. Design, Setting, and Participants In a cross-sectional study, original articles of 6 top journals between July 2016 and June 2018 were manually reviewed to identify articles including a waterfall plot to describe a treatment effect of cancer therapy. Response rates visually represented in waterfall plots were compared with response rates reported as study outcomes. The number of original articles with a waterfall plot as a percentage of total original articles was evaluated, sampling articles from January, February, and March for the years 2004, 2008, 2012, 2016, and 2018. Main Outcomes and Measures Difference between response rates depicted in waterfall plots and response rates reported as study outcomes. Results One hundred twenty-six articles were selected for analysis. Of the 97 articles reporting investigator-assessed response rates, waterfall plots showed response rates a median (interquartile range) of 6.1% (1.8%-12.0%) higher than rates derived from investigator assessment. Forty-two articles reported response rates based on central assessment as an outcome, and waterfall plots showed response rates a median (interquartile range) of 12.0% (7.7%-18.5%) higher compared with centrally assessed response rates. The estimated percentage of original articles using waterfall plots increased from 0% in 2004 to 7% in 2018. Conclusions and Relevance This study suggests that waterfall plots are becoming more common in oncology literature. Waterfall plots provide a visual overestimate of response rate of cancer therapies and should be evaluated with caution.
Databáze: OpenAIRE