Longitudinal graphics of patient-reported physical function in patients treated for hematologic malignancies.

ABSTRACT:

Background

The US Food and Drug Administration (FDA) released a draft guidance document detailing core patient-reported outcomes in cancer clinical trials, including physical function (PF). The objectives of this study were to develop analytic methods and visualizations of patient-reported PF in patients with cancer.

Methods

We applied an estimand framework to a patient-reported tolerability endpoint to develop data summaries cross-sectionally and over time, along with visualizations. We accomplished this through iterative feedback with clinicians, statisticians, and FDA stakeholders using three clinical trial datasets in hematologic malignancies. Graphical approaches were applied to three datasets in hematologic malignancies: (1) patients with myeloproliferative neoplasms e

SUBMITTER: Thanarajasingam G 

PROVIDER: S-EPMC12329971 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature