From waterfall plots to spaghetti plots in early oncology clinical development.
Pharm Stat
; 18(5): 526-532, 2019 10.
Article
em En
| MEDLINE
| ID: mdl-30942559
ABSTRACT
Waterfall plots are used to describe changes in tumor size observed in clinical studies. They are frequently used to illustrate the overall drug response in oncology clinical trials because of its simple representation of results. Unfortunately, this visual display suffers a number of limitations including (1) potential misguidance by masking the time dynamics of tumor size, (2) ambiguous labelling of the y-axis, and (3) low data-to-ink ratio. We offer some alternatives to address these shortcomings and recommend moving away from waterfall plots to the benefit of plots showing the individual time profiles of sum of lesion diameters (according to RECIST). The spider plot presents the individual changes in tumor measurements over time relative to baseline tumor burden. Baseline tumor size is a well-known confounding factor of drug effect which has to be accounted for when analyzing data in early clinical trials. While spider plots are conveniently correct for baseline tumor size, they cannot be presented in isolation. Indeed, percentage change from baseline has suboptimal statistical properties (including skewed distribution) and can be overly optimistic in favor of drug efficacy. We argued that plots of raw data (referred to as spaghetti plots) should always accompany spider plots to provide an equipoised illustration of the drug effect on lesion diameters.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Ensaios Clínicos como Assunto
/
Interpretação Estatística de Dados
/
Desenvolvimento de Medicamentos
/
Neoplasias
Tipo de estudo:
Prognostic_studies
Limite:
Humans
Idioma:
En
Revista:
Pharm Stat
Ano de publicação:
2019
Tipo de documento:
Article