Top

Published in:

Open Access 01-10-2020 | Heart Failure

Development of a conceptual model and patient-reported outcome measures for assessing symptoms and functioning in patients with heart failure

Authors: Olga Moshkovich, Katy Benjamin, Katie Hall, Ryan Murphy, Robyn von Maltzahn, Boris Gorsh, Vanja Sikirica, Rajnish Saini, Dennis Sprecher

Published in: Quality of Life Research | Issue 10/2020

Abstract

Purpose

Heart failure (HF) is a common condition that places considerable burden on patients. We aimed to develop a patient-reported outcome (PRO) measure to assess the symptoms and impacts of HF.

Methods

Phase 1: a targeted literature review, expert interviews, and concept elicitation (CE) interviews with patients with HF (n = 26) were used to develop a conceptual model of the core symptoms and impacts of HF. To capture these concepts, three new fit-for-purpose PRO questionnaires were constructed in accordance with US Food and Drug Administration PRO guidance. Phase 2: three ‘waves’ of cognitive interviews were conducted with patients with HF (n = 28) to validate and refine the questionnaires.

Results

Three key symptoms—shortness of breath, oedema, and fatigue—were identified across the literature review, expert interviews and CE interviews. Several additional symptoms, cognitive changes and impacts of HF were reported in the CE interviews and included in the conceptual model. A 10-item symptom questionnaire (Heart Failure-Daily Symptom Diary) was constructed; cognitive testing showed that the final PRO measure was easy to understand/complete and relevant to patients with HF, confirming content validity. Two HF impact questionnaires were developed (Assessing Dyspnoea’s Impact on Mobility and Sleep and Heart Failure-Functional Status Assessment), but required refinement to ensure patient understanding.

Conclusions

Patient input contributed to the development of a PRO instrument for assessing physical and cognitive symptoms important to patients with HF using novel measurement strategies. Inclusion of daily metrics offers differentiation from other qualified instruments and may provide clinical insight for improving lifestyles. Additionally, two draft PRO measures may, after further validation, be useful to assess the impacts of HF.

Available only for authorised users

Inamdar, A. A., & Inamdar, A. C. (2016). Heart failure: Diagnosis, management and utilization. Journal of Clinical Medicine, 5(7), 62. https://doi.org/10.3390/jcm5070062.CrossRefPubMedCentral

Benjamin, E. J., Virani, S. S., Callaway, C. W., Chamberlain, A. M., Chang, A. R., Cheng, S., et al. (2018). Heart disease and stroke statistics—2018 update: A report from the American Heart Association. Circulation, 137(12), e67–e492. https://doi.org/10.1161/cir.0000000000000558.CrossRefPubMed

Dunlay, S. M., Shah, N. D., Shi, Q., Morlan, B., VanHouten, H., Long, K. H., et al. (2011). Lifetime costs of medical care after heart failure diagnosis. Circulation: Cardiovascular Quality and Outcomes, 4(1), 68–75. https://doi.org/10.1161/CIRCOUTCOMES.110.957225.CrossRef

Heidenreich, P. A., Albert, N. M., Allen, L. A., Bluemke, D. A., Butler, J., Fonarow, G. C., et al. (2013). Forecasting the impact of heart failure in the United States: A policy statement from the American Heart Association. Circulation: Heart Failure, 6(3), 606–619. https://doi.org/10.1161/HHF.0b013e318291329a.CrossRef

Jeon, Y. H., Kraus, S. G., Jowsey, T., & Glasgow, N. J. (2010). The experience of living with chronic heart failure: A narrative review of qualitative studies. BMC Health Services Research, 10, 77. https://doi.org/10.1186/1472-6963-10-77.CrossRefPubMedPubMedCentral

Zambroski, C. H., Moser, D. K., Bhat, G., & Ziegler, C. (2005). Impact of symptom prevalence and symptom burden on quality of life in patients with heart failure. European Journal of Cardiovascular Nursing, 4(3), 198–206. https://doi.org/10.1016/j.ejcnurse.2005.03.010.CrossRefPubMed

Cowie, M. R., Anker, S. D., Cleland, J. G., Felker, G. M., Filippatos, G., Jaarsma, T., et al. (2014). Improving care for patients with acute heart failure. Retrieved May, 2018, from https://www.oxfordhealthpolicyforum.org/reports/acute-heart-failure/improving-care-for-patients-with-acute-heart-failure.

Roger, V. L. (2013). Epidemiology of heart failure. Circulation Research, 113(6), 646–659. https://doi.org/10.1161/CIRCRESAHA.113.300268.CrossRefPubMedPubMedCentral

Psotka, M. A., von Maltzahn, R., Anatchkova, M., Agodoa, I., Chau, D., Malik, F. I., et al. (2016). Patient-reported outcomes in chronic heart failure: Applicability for regulatory approval. JACC Heart Failure, 4(10), 791–804. https://doi.org/10.1016/j.jchf.2016.04.010.CrossRefPubMed

10.

US Department of Health and Human Services. (2009). Guidance for industry. Patient-reported outcome measures: Use in medical product development to support labeling claims. Retrieved May, 2018, from https://www.fda.gov/downloads/drugs/guidances/ucm193282.pdf.

11.

Green, C. P., Porter, C. B., Bresnahan, D. R., & Spertus, J. A. (2000). Development and evaluation of the Kansas City Cardiomyopathy Questionnaire: A new health status measure for heart failure. Journal of the American College of Cardiology, 35(5), 1245–1255. https://doi.org/10.1016/s0735-1097(00)00531-3.CrossRefPubMed

12.

Chauvet-Gelinier, J. C., & Bonin, B. (2017). Stress, anxiety and depression in heart disease patients: A major challenge for cardiac rehabilitation. Annals of Physical and Rehabilitation Medicine, 60(1), 6–12. https://doi.org/10.1016/j.rehab.2016.09.002.CrossRefPubMed

13.

Food and Drug Administration. (2016). MEDICAL DEVICE DEVELOPMENT TOOL (MDDT) QUALIFICATION DECISION SUMMARY FOR MINNESOTA LIVING WITH HEART FAILURE QUESTIONNAIRE (MLHFQ). Retrieved January, 2020, from https://www.fda.gov/media/112157/download.

14.

Food and Drug Administration. (2016). MEDICAL DEVICE DEVELOPMENT TOOL (MDDT) QUALIFICATION DECISION SUMMARY FOR KANSAS CITY CARDIOMYOPATHY QUESTIONNAIRE (KCCQ). Retrieved January, 2020, from https://www.fda.gov/media/108301/download.

15.

Patrick, D. L., Burke, L. B., Gwaltney, C. J., Leidy, N. K., Martin, M. L., Molsen, E., et al. (2011). Content validity–establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part 1—Eliciting concepts for a new PRO instrument. Value Health, 14(8), 967–977. https://doi.org/10.1016/j.jval.2011.06.014.CrossRefPubMed

16.

Patrick, D. L., Burke, L. B., Gwaltney, C. J., Leidy, N. K., Martin, M. L., Molsen, E., et al. (2011). Content validity–establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: Part 2—Assessing respondent understanding. Value Health, 14(8), 978–988. https://doi.org/10.1016/j.jval.2011.06.013.CrossRefPubMed

17.

Rector, T. S., Kubo, S. H., & Cohn, J. N. (1993). Validity of the minnesota living with heart failure questionnaire as a measure of therapeutic response to enalapril or placebo. American Journal of Cardiology, 71(12), 1106–1107. https://doi.org/10.1016/0002-9149(93)90582-w.CrossRefPubMed

18.

Rector, T., Kubo, S., & Cohn, J. (1987). Patients' self-assessment of their congestive heart failure—Part 2: Content, reliability and validity of a new measure, the Minnesota Living with Heart Failure Questionnaire. Heart Failure, 3, 198–209.

19.

Heo, S., Lennie, T. A., Okoli, C., & Moser, D. K. (2009). Quality of life in patients with heart failure: Ask the patients. Heart and Lung, 38(2), 100–108. https://doi.org/10.1016/j.hrtlng.2008.04.002.CrossRefPubMed

20.

US Food and Drug Administration (2017). Medical Device Development Tool (MDDT) Qualification Decision Summary for Kansas City Cardiomyopathy Questionnaire (KCCQ). Retrieved November 29, 2019, from https://www.fda.gov/media/108301/download.

21.

US Food and Drug Administration (2018). Medical Device Development Tool (MDDT) Qualification Decision Summary for Minnesota Living with Heart Failure Questionnaire (MLHFQ). Retrieved December 17, 2019, from https://www.fda.gov/media/112157/download.

22.

Ahmad, F. S., Kallen, M. A., Schifferdecker, K. E., Carluzzo, K. L., Yount, S. E., Gelow, J. M., et al. (2019). Development and initial validation of the PROMIS(R)-Plus-HF profile measure. Circulation: Heart Failure, 12(6), e005751. https://doi.org/10.1161/circheartfailure.118.005751.CrossRef

23.

Bui, A. L., Horwich, T. B., & Fonarow, G. C. (2011). Epidemiology and risk profile of heart failure. Nature Reviews. Cardiology, 8(1), 30–41. https://doi.org/10.1038/nrcardio.2010.165.CrossRefPubMed

24.

Braunstein, J. B., Anderson, G. F., Gerstenblith, G., Weller, W., Niefeld, M., Herbert, R., et al. (2003). Noncardiac comorbidity increases preventable hospitalizations and mortality among Medicare beneficiaries with chronic heart failure. Journal of the American College of Cardiology, 42(7), 1226–1233.CrossRef

25.

Javaheri, S., Parker, T. J., Liming, J. D., Corbett, W. S., Nishiyama, H., Wexler, L., et al. (1998). Sleep apnea in 81 ambulatory male patients with stable heart failure. Types and their prevalences, consequences, and presentations. Circulation, 97(21), 2154–2159. https://doi.org/10.1161/01.cir.97.21.2154.CrossRefPubMed

26.

Sin, D. D., Fitzgerald, F., Parker, J. D., Newton, G., Floras, J. S., & Bradley, T. D. (1999). Risk factors for central and obstructive sleep apnea in 450 men and women with congestive heart failure. American Journal of Respiratory and Critical Care Medicine, 160(4), 1101–1106. https://doi.org/10.1164/ajrccm.160.4.9903020.CrossRefPubMed

Title: Development of a conceptual model and patient-reported outcome measures for assessing symptoms and functioning in patients with heart failure
Authors: Olga Moshkovich
Katy Benjamin
Katie Hall
Ryan Murphy
Robyn von Maltzahn
Boris Gorsh
Vanja Sikirica
Rajnish Saini
Dennis Sprecher
Publication date: 01-10-2020
Publisher: Springer International Publishing
Keyword: Heart Failure
Published in: Quality of Life Research / Issue 10/2020
Print ISSN: 0962-9343
Electronic ISSN: 1573-2649
DOI: https://doi.org/10.1007/s11136-020-02537-y

At a glance: The STEP trials

Springer Medicine

Development of a conceptual model and patient-reported outcome measures for assessing symptoms and functioning in patients with heart failure

Abstract

Purpose

Methods

Results

Conclusions

At a glance: The STEP trials

Springer Medicine

Abstract

Purpose

Methods

Results

Conclusions

Please log in to get access to this content

Other articles of this Issue 10/2020

Genetic polymorphisms and haplotypes of BRCA1 gene associated with quality of life and survival among patients with non-small-cell lung cancer

Potentially inappropriate medications use and its association with health-related quality of life among elderly cardiac patients

The complexity of assessing health-related quality of life among sarcoma patients

Registered Reports at “Quality of Life Research”

The association of stroke severity with health-related quality of life in survivors of acute cerebrovascular disease and their informal caregivers during the first year post stroke: a survey study

Factors associated with poor-to-moderate quality of life among pregnant women with gestational diabetes mellitus: a cross-sectional study in Malaysia