Abstract 4383: Development and validation of the Lung Cancer-Health Index (LC-HI), a clinically-relevant, disease-specific patient-reported outcome measure
Authors: Varma Anika , Rosero Spencer , Seabury Jamison , Weinstein Jennifer , Engebrecht Charlotte , Zizzi Christine , Dilek Nuran , Heatwole John , Baumgart Megan , Mulford Deborah , Maggiore Ronald , Conrow Lainie , King Jennifer , Wiens Jacinta , Heatwole Chad 
Published: April 2023

Abstract
Study Aim: The goal of this research is to develop and validate a novel, disease-specific patient-reported outcome measure (PRO) for use in clinical monitoring and therapeutic trials in Lung Cancer (LC).
Methods: LC currently accounts for the greatest number of cancer deaths worldwide, driving an increased need for novel therapeutic development in LC. To bolster clinical trial infrastructure, it is critical to have a comprehensive, sensitive and reliable PRO that accurately tracks LC multifactorial disease burden. This research describes the development and validation of the Lung Cancer-Health Index (LC-HI) as an efficient mechanism for assessing how patients with LC feel and function in response to therapeutic treatment. We conducted semi-structured, qualitative interviews with 15 individuals diagnosed with LC and collected 653 direct participant quotes to determine the most frequent and important symptoms in LC. Based upon participant responses, we designed a survey that inquired about 162 symptoms from 14 symptomatic themes. This survey was implemented in a cross-sectional study with 139 participants with LC. The collected data was used to generate the first version of the LC-HI, which contained symptom questions showing the highest impact to the population and the potential to respond to therapeutic intervention. Symptom questions were grouped into distinct subscales using factor analysis. We beta-tested the LC-HI with 15 patients with LC to evaluate its usability, clarity, and applicability. Based on patient feedback, we modified the instrument and evaluated its test-retest reliability with 22 individuals with LC. Known groups testing was performed with the final instrument.
Results: The final version of the LC-HI contains 45 symptom questions grouped into 10 subscales that measure the following areas of LC health: 1) fatigue, 2) physical function, 3) emotional health, 4) sleep and daytime sleepiness, 5) activity participation, 6) breathing function, 7) gastrointestinal function, 8) cognitive function, 9) social performance, and 10) pain. Total LC disease burden is measured using a weighted composite of these subscale scores. During beta interviews, individuals with LC deemed the instrument to be easy to use, clear, relevant, and comprehensive. Statistical and psychometric analyses confirmed that the LC-HI is reliable, valid, sensitive, specific, of high internal consistency, and able to differentiate between groups with known varying levels of disease burden (based on disability status and smoking history).
Conclusions: Our research demonstrates the validity of the LC-HI as a marker of the multifaceted disease burden in LC. This disease-specific PRO provides researchers and clinicians with a reliable tool to use in patient monitoring, clinical trials, and to support FDA drug labeling claims.
Citation Format: Anika Varma, Spencer Rosero, Jamison Seabury, Jennifer Weinstein, Charlotte Engebrecht, Christine Zizzi, Nuran Dilek, John Heatwole, Megan Baumgart, Deborah Mulford, Ronald Maggiore, Lainie Conrow, Jennifer King, Jacinta Wiens, Chad Heatwole. Development and validation of the Lung Cancer-Health Index (LC-HI), a clinically-relevant, disease-specific patient-reported outcome measure. [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2023; Part 1 (Regular and Invited Abstracts); 2023 Apr 14-19; Orlando, FL. Philadelphia (PA): AACR; Cancer Res 2023;83(7_Suppl):Abstract nr 4383.