Moving Away from Symptoms-Based Heart Failure Treatment: Misperceptions and Real Risks for Patients with Heart Failure

Although multiple new drug and device-based therapies have been approved for heart failure and reduced ejection fraction, outcomes for these patients remain suboptimal. There are over a million hospitalizations for worsening heart failure annually in the USA and in Europe. These hospitalizations are associated with ≈ 30% mortality risk within a year post-discharge and this trajectory has not changed in over two decades.1 Many patients are undertreated, including lack of prescription of life-saving drugs and devices and, when prescribed, they may be under-dosed. There is an ongoing inertia in optimizing treatment for patients with heart failure. This inertia is related to not only the physician taking care of the patients but, importantly, is also dependent on a misperception regarding heart failure treatment.2-4 It has been known for decades that heart failure is a progressive disease that may be reversible in a proportion of patients treated optimally. However, the treatment of these patients often remains unchanged and contributes to the poor outcomes for patients with heart failure. This may be largely related to a misperception of risk that can occur in both directions, i.e. under- and over-perception of risk related to symptoms, and both paradigms can lead to suboptimal care (Figure 1). Patients with New York Heart Association (NYHA) class III–IV symptoms are deemed to have a progressively worsening condition marked by recurrent hospitalizations that will inevitably terminate in a mortality event in the short to intermediate term. Advanced therapies or palliative care is important for a select group of patients who truly have end-stage disease. However, most patients with heart failure, despite symptoms, have dysfunctional but viable vulnerable myocardium that does not contract normally but has the potential to improve with optimizing guideline-directed therapy and to respond to novel experimental therapies.5 Neurohormonal antagonists are less well tolerated and difficult to administer and uptitrate in these true end-stage patients; however, they represent a minority of symptomatic heart failure patients. Most of these patients do not receive adequate doses and optimal treatment without clear reasons and this has an impact on outcomes. A perceived poor trajectory leads to nihilism and prematurely labelling of patients as having end-stage disease. Data do not support such an assertion. For example, although a small proportion of patients do have end-stage disease, the vast majority of hospitalized heart failure patients respond rapidly with improvement in symptoms, have relatively preserved haemodynamics, and have adequate end organ function—all features that argue against the notion of a terminal illness.6 Unlike other organ system failure, such as cirrhosis where the normal parenchyma is largely replaced by fibrosis, the majority of the dysfunctional myocardial segments in heart failure either have either no or minimal scarring, rendering them viable and potentially reversible to an active state with aggressive medical and device-based therapy.7 In the contrast, the stability of symptoms in patients with NYHA class II heart failure is taken as an indication of stability of the disease process. This under-appreciation of the risk of adverse outcomes in the presence of stable symptoms has reverse consequences. Many patients are not on optimal drug and device-based therapy, and even if they are receiving this therapy, the doses prescribed are less than those recommended by the guidelines.8 There is tendency with stable symptoms to leave patients alone as optimizing drugs and doses, or implanting devices, risks side-effects and the increased discomfort of multiple health-care interventions. Considering heart failure prognosis being worse than many forms of cancer, one would expect that the patient community to be active in advocacy for efforts towards more research to discover new therapies. However, such is not the case. Survey results show that patients with heart failure do not understand their prognosis.9, 10 Providers and patients tend to equate stability of symptoms with disease stability, despite the fact that almost one-third of the patients hospitalized for worsening heart failure die within a year of discharge, notwithstanding rapid improvement in symptoms during hospitalization.1 One may equate heart failure to being the 'cancer of the heart' but, unlike oncology, the culture of participation in clinical trials by either the patients or the providers is not as robust.11 Unlike other chronic diseases with poor prognosis such as cancer or chronic kidney disease that are treated primarily by specialists, the majority of heart failure patients are treated by generalists despite data suggesting better adherence to guidelines by specialists.3, 4 Heart failure care should not be based primarily on symptoms. It should be emphasized that NYHA class II patients may have sudden death without worsening symptoms and that they have a progressive disease whose progression can be slowed by appropriate treatment. However, NYHA class III–IV patients do not necessarily have end-stage disease and initiation of proven therapies, attempts to attain recommended doses, and implantation of appropriate devices should be tried regardless of symptoms. All therapies known to improve heart failure outcomes do so across the spectrum of patients ranging from NYHA class II–IV symptoms.12 Hospitalization for heart failure represents an especially important time-point as it marks a fundamental change in the trajectory of the disease process. Every effort must be made to optimize medical therapy, emphasize the importance of self-care behaviours, and treat comorbid conditions during hospitalization. Efforts in advocacy are needed in order to have better societal appreciation of the risks associated with heart failure. Investigators, physicians, individuals involved in regulatory agencies, and law makers must be aware that heart failure is a progressive disease leading to poor quality of life, recurrent hospitalizations, and early death, but at the same time it is also potentially reversible with considerable room for improvement in care across the spectrum of symptomatic patients with heart failure. Providers and patients have to embrace participation in clinical trials for heart failure, without which it will be impossible to discover effective therapies in a timely manner. No funding was received for this work. Conflicts of interest: J.B. reports receiving research support from the National Institutes of Health, and European Union, and serves as a consultant to Amgen, Bayer, Boehringer Ingelheim, Cardiocell, Celladon, Novartis, Trevena, Relypsa, Z Pharma, and Zensun. M.G. reports consulting relationships with Abbott, Astellas, AstraZeneca, Bayer, Cardiorentis, CorThera, Cytokinetics, CytoPherx, DebioPharm SA, Errekappa Terapeutici, GlaxoSmithKline, Ikaria, Intersection Medical, Johnson & Johnson, Medtronic, Merck, Novartis, Ono Pharmaceuticals, Otsuka Pharmaceuticals, Palatin Technologies, Pericor Therapeutics, Protein Design Laboratories, Sanofi-Aventis, Sigma Tau, Solvay Pharmaceuticals, Sticares InterACT, Takeda, and Trevena Therapeutics. M.M. has received consulting incomes from Bayer, Novartis, and Servier.

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