Developing a Fit-for-Purpose Composite Symptom Score as a Symptom Burden Endpoint for Clinical Trials in Patients With Malignant Pleural Mesothelioma

Authors

Charles S Cleeland
Karen N Keating
Brian Cuffel
Cem Elbi
Jonathan M Siegel
Christoph Gerlinger
Tara Symonds
Jeff A Sloan
Amylou C Dueck
Andrew Bottomley
Xin Shelley Wang
Loretta A Williams
Tito R Mendoza

Publication Date

6-27-2024

Journal

Scientific Reports

DOI

10.1038/s41598-024-62307-5

PMID

38937473

PMCID

PMC11211485

PubMedCentral® Posted Date

6-27-2024

PubMedCentral® Full Text Version

Post-print

Abstract

We developed a composite symptom score (CSS) representing disease-related symptom burden over time in patients with malignant pleural mesothelioma (MPM). Longitudinal data were collected from an open-label Phase IIB study in which 239 patients completed the validated MD Anderson Symptom Inventory for MPM (MDASI-MPM). A blinded, independent review committee of external patient-reported outcomes experts advised on MDASI-MPM symptoms to include in the CSS. Through iterative analyses of potential symptom-item combinations, 5 MPM symptoms (pain, fatigue, shortness of breath, muscle weakness, coughing) were selected. The CSS correlated strongly with the full MDASI-MPM symptom set (0.92-0.94) and the Lung Cancer Symptom Scale-Mesothelioma (0.79-0.87) at each co-administration of the scales. The CSS also had good sensitivity to worsening disease and global quality-of-life ratings. The MDASI-MPM CSS can be used as an outcome in MPM clinical trials, including in responder analyses and at the individual patient level. It is brief enough to administer frequently, including electronically, to better capture symptom trajectories during and after a trial and in clinical practice. As a single score, the CSS addresses multiplicity issues that can arise when several symptoms increase due to worsening disease. Our process can be adapted to produce a CSS for other advanced-cancer trials.

Keywords

Humans, Mesothelioma, Malignant, Male, Female, Pleural Neoplasms, Aged, Middle Aged, Quality of Life, Lung Neoplasms, Mesothelioma, Patient Reported Outcome Measures, Fatigue, Symptom Assessment, Longitudinal Studies, Severity of Illness Index, Symptom Burden

Published Open-Access

yes

Recommended Citation

Cleeland, Charles S; Keating, Karen N; Cuffel, Brian; et al., "Developing a Fit-for-Purpose Composite Symptom Score as a Symptom Burden Endpoint for Clinical Trials in Patients With Malignant Pleural Mesothelioma" (2024). Faculty, Staff and Student Publications. 5090.
https://digitalcommons.library.tmc.edu/uthgsbs_docs/5090