Endpoints in Heart Failure Drug Development

Fiuzat, Mona; Lowy, Naomi; Stockbridge, Norman; Sbolli, Marco; Latta, Federica; Lindenfeld, Jo Ann; Lewis, Eldrin F.; Abraham, William T.; Teerlink, John R.; Walsh, Mary Norine; Heidenreich, Paul A.; Bozkurt, Biykem; Starling, Randall C.; Solomon, Scott D.; Felker, G. Michael; Butler, Javed; Yancy, Clyde W.; Stevenson, Lynne Warner; O’Connor, Christopher M.; Unger, Ellis F.; McMurray, John J.V.

doi:10.1016/j.jchf.2019.12.011

Cited by 39 publications

(36 citation statements)

References 25 publications

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“…Furthermore, the day-to-day unpredictability of HF (i.e., acute exacerbations and hospitalizations), medical management issues such as pill burden and side effects can cause distress and feelings of hopelessness and helplessness in both HF patients and their caregivers [10]. While there are successful HF therapies which have demonstrated reductions in mortality and morbidity, very few HF drugs have been indicated for improving HRQoL in HF patients in addition to physical function, or symptoms [11].…”

Section: Introductionmentioning

confidence: 99%

The impact of heart failure on patients and caregivers: A qualitative study

et al. 2021

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Background Heart failure is rising in prevalence but relatively little is known about the experiences and journey of patients and their caregivers. The goal of this paper is to present the symptom and symptom impact experiences of patients with heart failure and their caregivers. Methods This was a United States-based study wherein in-person focus groups were conducted. Groups were audio recorded, transcribed and a content-analysis approach was used to analyze the data. Results Ninety participants (64 patients and 26 caregivers) were included in the study. Most patients were female (52.0%) with mean age 59.3 ± 8 years; 55.6% were New York Heart Association Class II. The most commonly reported symptoms were shortness of breath (81.3%), fatigue/tiredness (76.6%), swelling of legs and ankles (57.8%), and trouble sleeping (50.0%). Patients reported reductions in social/family interactions (67.2%), dietary changes (64.1%), and difficulty walking and climbing stairs (56.3%) as the most common adverse disease impacts. Mental-health sequelae were noted as depression and sadness (43.8%), fear of dying (32.8%), and anxiety (32.8%). Caregivers (mean age 55.5 ± 11.2 years and 52.0% female) discussed 33 daily heart failure impacts, with the top three being reductions in social/family interactions (50.0%); being stressed, worried, and fearful (46.2%); and having to monitor their “patience” level (42.3%). Conclusions There are serious unmet needs in HF for both patients and caregivers. More research is needed to better characterize these needs and the impacts of HF along with the development and evaluation of disease management toolkits that can support patients and their caregivers.

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Section: Introductionmentioning

confidence: 99%

The impact of heart failure on patients and caregivers: A qualitative study

et al. 2021

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“…Evolving evidence for new effective preventive and treatment strategies in HF will require clarity in the different stages and/or ejection fraction (EF) subgroups of HF, 15,16 along with an increased emphasis on performance measures with a need for accuracy in patient diagnoses and treatment indications, [17][18][19] a need for improved communication and understanding of the definition of HF with patients and for shared decision-making and transitions of care between different levels of care and health care professionals, 3 and an increased recognition and emphasis of standard diagnoses and end points in the settings of research, and clinical trials and registries. 20,21 The objectives of this document are to provide a universal definition of HF that is clinically relevant, simple but conceptually comprehensive, with the ability to subclassify and to encompass stages within; with universal applicability globally, and with prognostic and therapeutic validity and acceptable sensitivity and specificity. We envision the proposed universal definition and classifications to be used in a standardized fashion across scientific societies and guidelines, used by clinicians, and used in research studies.…”

Section: Preamblementioning

confidence: 99%

Universal Definition and Classification of Heart Failure

Bozkurt¹,

Coats²,

Tsutsui³

et al. 2021

Journal of Cardiac Failure

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501

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In this document, we propose a universal definition of heart failure (HF) as the following: HF is a clinical syndrome with symptoms and or signs caused by a structural and/or functional cardiac abnormality and corroborated by elevated natriuretic peptide levels and or objective evidence of pulmonary or systemic congestion. We propose revised stages of HF as follows. At-risk for HF (Stage A), for patients at risk for HF but without current or prior symptoms or signs of HF and without structural or biomarkers evidence of heart disease. Pre-HF (stage B), for patients without current or prior symptoms or signs of HF, but evidence of structural heart disease or abnormal cardiac function, or elevated natriuretic peptide levels. HF (Stage C), for patients with current or prior symptoms and/or signs of HF caused by a structural and/or functional cardiac abnormality. Advanced HF (Stage D), for patients with severe symptoms and/or signs of HF at rest, recurrent hospitalizations despite guideline-directed management and therapy (GDMT), refractory or intolerant to GDMT, requiring advanced therapies such as consideration for transplant, mechanical circulatory support, or palliative care. Finally, we propose a new and revised classification of HF according to left ventricular ejection fraction (LVEF). The classification includes HF with reduced EF (HFrEF):

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“…The two common endpoints in outcome analysis after TMVr that this article focuses on are acute procedural failure (APF) and all-cause and/or cardiac mortality [21][22][23][24] . The Mitral Valve Academic Research Consortium (MVARC) differentiates between technical, device, procedural, and patient success [24] .…”

Section: Endpoints and Patient Cohortmentioning

confidence: 99%