CERSI Collaborators: Shannon Dunlay, MD (Mayo Clinic) (PI), Jennifer Ridgeway, PhD (Mayo Clinic), Erica S. Spatz, MD, MHS (Yale)

FDA Collaborators: Fraser Bocell, PhD, Brittany Caldwell, PhD, Anindita Saha, Michelle Tarver, MD, PhD

Project Start Date: October 2018

Project End Date: September 2022

Regulatory Science Challenge

Cardiovascular disease is the leading cause of death for women in the world. In recent years, approximately 1 in every 4 deaths in the United States results from heart disease, with similar rates among men and women. Patient-reported outcomes (PRO) are reports about health status that come directly from patients, usually in the form of a survey. PRO surveys can be used to gather information on health-related topics best described by patients themselves, such as quality of life. Heart failure (HF) PROs, like the Kansas City Cardiomyopathy Questionnaire (KCCQ), which gather information about patients’ physical limitations and symptoms, have been shown to predict hospitalization and death, and are regularly used as endpoints in clinical studies and patient care. The differences in presentation and symptoms of the disease in women and men are well documented; however, because the KCCQ was developed and validated on a sample that was majority men, it is unknown whether the KCCQ captures the range of symptoms experienced by women. This is important as differences in scores between men and women may not reflect differences in HF severity. Regulatory decisions being made upon the KCCQ results may not fully reflect all patients adequately.

Project Description and Goals

The goal of this project was to explore gender differences in the KCCQ PRO measure. This work addressed improved health communication by initiating the work to make KCCQ clear and applicable to women, potentially helping to improve both patient care and regulatory decisions.

Goal 1: Using qualitative interviews, describe if and how men and women think about health-related quality of life and interpret KCCQ questions differently using qualitative interviews.

Results/ Findings/ Publications

A total of 25 adults (13 women, 12 men, 22 years and older) with clinician-diagnosed HF and left ventricular ejection fraction ≤ 40% were recruited from two academic medical centers to participate in semi-structured interviews. All interviews were conducted over the phone/web and audio recorded. Interviews were transcribed and descriptive qualitative content analysis was used to summarize findings overall and by gender. The average age was 67 years (range: 25-88). Women attributed a wider variety of symptoms to HF than men. Some participants had difficulty differentiating whether their experiences were due to HF, side effects of their medications, or age. We found very little evidence that participants interpreted KCCQ items differently based on gender. Overall, our findings indicate that interpretation of the KCCQ items were similar in men and women. However, some modifications to items may improve clarity of interpretation for a wide range of patients.

An abstract was presented at the 28th Annual Conference of the International Society for Quality of Life Research (ISOQOL) (October 2021): Exploring Possible Interpretation Differences by Men and Women on the Kansas City Cardiomyopathy Questionnaire (KCCQ) via Qualitative Interviews (poster presentation).

The qualitative results were also published in a peer-reviewed journal (citation below):

Coles TM, Lucas N, McFatrich M, Henke D, Ridgeway JL, Behnken EM, Weinfurt K, Reeve BB, Corneli A, Dunlay SM, Spertus JA, Lin L, Piña IL, Bocell FD, Tarver ME, Dohse H, Saha A, Caldwell B. Investigating gender-based differential item functioning on the Kansas City Cardiomyopathy Questionnaire (KCCQ) using qualitative content analysis. Qual Life Res. 2023 Mar;32(3):841-852. doi: 10.1007/s11136-022-03276-y. Epub 2022 Nov 2. PMID: 36322269; PMCID: PMC9628332.