EASD 2017: Study confirms safety of once-weekly exenatide in broad range of patients with diabetes

September 14, 2017 – A global team of researchers reported in the New England Journal of Medicine that the study did not meet its primary efficacy endpoint, but yielded important results for all-cause mortality.

Diabetes is a widespread and increasing problem currently affecting 30.3 million Americans, 4.5 million people living in the United Kingdom, and 422 million people worldwide. In 2015, an estimated 1.6 million deaths worldwide were directly caused by diabetes. There is a clear need to improve health outcomes, particularly for cardiovascular events, which represent the greatest burden in terms of morbidity and mortality.

The EXSCEL trial (EXenatide Study of Cardiovascular Event Lowering) was among the largest ever carried out in type 2 diabetes, setting out to evaluate the safety and efficacy of a once-weekly extended-release formulation of exenatide, a glucagon-like peptide-1 (GLP-1) receptor agonist. The drug works by mimicking a hormone in that reduces blood sugar levels after meals but its impact on heart disease is unknown.

Launched in 2009 and completed in April 2017, the EXSCEL trial was a Phase IIIB/IV, double-blind, placebo-controlled, global cardiovascular outcomes trial involving 14,752 patients with type 2 diabetes in 35 countries. Participants – who were eligible with or without additional cardiovascular risk factors or prior cardiovascular events – all received usual type 2 diabetes care and were randomized to receive subcutaneous injections of 2mg exenatide once-weekly, or a matching placebo. The trial compared the risk of major adverse cardiac events (MACE) – a composite endpoint of cardiovascular death, non-fatal myocardial infarction or non-fatal stroke – in the two groups of patients. The median length of follow-up was 3.2 years.

The trial met its primary safety objective of non-inferiority for MACE. The efficacy objective of a superior reduction in MACE did not reach statistical significance, although a prespecified analysis suggested all-cause mortality was lower with exenatide than placebo.

The results were presented today at the European Association for the Study of Diabetes (EASD) annual meeting in Lisbon, Portugal. They were also published simultaneously in the New England Journal of Medicine.

EXSCEL was run jointly by the DCRI and the University of Oxford Diabetes Trials Unit (DTU).

“The study results show that exenatide had no adverse effects on cardiovascular health, meaning that the drug can be used safely in people with type 2 diabetes who may have a wide range of existing cardiovascular conditions,” said the DTU’s Rury R. Holman, who co-led the study. “There did not seem to be any increase in the risk of hypoglycemia, acute pancreatitis, pancreatic cancer, or medullary thyroid carcinoma.”

“It’s encouraging for the field of diabetes to see these results in patients similar to what we see in clinical practice can have a potentially lower risk of death from all causes with the convenience of once-weekly dosing,” said the DCRI’s Adrian F. Hernandez, MD, MHS, Holman’s co-leader on the trial.

“This confirms the importance of carrying out large studies to evaluate impacts on cardiovascular outcomes. EXSCEL largely mirrored what we’ve learned from other studies of this class of medications – that they are safe and may have outcomes benefits.”

In addition to Holman and Hernandez, co-authors on the EXCSEL paper were Angelyn Bethel,MD, from the DTU, and Robert Mentz, MD; Vivian Thompson, MPH; Yuliya Lokhnygina, PhD; and Neha Pagidipati, MD, MPH, from Duke and DCRI.

Trial launches to learn how to prevent kidney stone recurrence

September 13, 2017 – The DCRI serves as the Scientific Data Research Center for the Urinary Stone Disease Research Network, whose researchers are conducting the study.

Researchers are recruiting participants for a two-year clinical trial to determine whether using a high-tech water bottle and encouraging people to drink more water, and therefore urinate, will reduce the recurrence of urinary stone disease, also known as kidney stones.

The trial, known as the Prevention of Urinary Stones with Hydration (PUSH) study, is being conducted as part of a five-year initiative by the National Institutes of Diabetes and Digestive and Kidney Diseases (NIDDK) to improve outcomes for patients with kidney stones.

The purpose of the initiative, the Urinary Stone Disease Research Network (USDRN), is to design and conduct clinical trials in adults and children who suffer from kidney stones. The DCRI serves as the network’s Scientific Data Research Center. Charles Scales, MD, and Hussein Al-Khalidi, PhD, serve as the research center’s principal investigators.

In addition to the data research center, the USDRN comprises clinical sites at the University of Pennsylvania, Children’s Hospital of Philadelphia, the University of Texas Southwestern Medical Center, the University of Washington at Seattle, and Washington University. Investigators at the DCRI and each clinical site will work collaboratively to plan, execute, and analyze USDRN studies.

Kidney stones are small, hard mineral deposits that form inside the kidneys and can cause kidney damage and excruciating pain. Kidney stones affect about 1 out of 11 Americans. The prevalence of kidney stones among Americans has nearly doubled in the last 15 years, and is increasing in both adults and children. According to the NIDDK, kidney stones cost an estimated $10 billion each year, making them the most expensive non-malignant urologic condition in the United States. A recent study estimates that the impact of obesity, diabetes and population rates will increase costs of kidney stones to $1.24 billion a year by 2030.

Kidney stones are a recurring condition for many patients. Data from the National Health and Nutrition Examination Survey show that 35 percent of participants had experienced two or more distinct episodes of urinary stones. Although the causes of kidney stone formation are relatively well understood, there has been little clinical emphasis on preventing recurrent kidney stones. Many physicians treat kidney stones as discrete events, rather than a chronic metabolic condition resulting in painful “stone attacks.”

“If we treated people with heart disease the way we treat patients with kidney stones, we would only be treating patients with chest pain or a heart attack,” Scales said. “We wouldn’t pay attention to risk factors like smoking or cholesterol. My hope is that through this research network, we can focus on stone prevention, and empower patients to make the necessary lifestyle changes to avoid recurrent kidney stones.”

PUSH will enroll 1,642 people, half in an intervention group and half in a control group. The study’s primary aim is to determine whether a program of financial incentives, receiving advice from a health coach, and using a smart water bottle called Hidrate Spark that monitors fluid consumption and connects to an app will result in reduced risk of USD recurrence over a two-year period.

Those in the intervention group will be asked to drink a specific quantity of fluids calculated based on each person’s urine output. They will also be given financial incentives if they achieve their fluid targets. They will also meet with a health coach who will help identify barriers to drinking more liquids, and help solve them. Study participants in both groups will receive the water bottles to monitor how much they drink and will be asked to try to achieve a goal of drinking enough to expel 2.5 liters of urine per day – about 10.5 cups.

“Urinary stones are painful and debilitating, and their treatment expensive. We hope that identifying the barriers to water intake, and helping people overcome those barriers individually, will be successful,” said Ziya Kirkali, MD, program director of urology clinical research and epidemiology, in NIDDK’s Division of Kidney, Urologic, and Hematologic Diseases. “If successful, the study could change management of kidney stones, and could result in healthcare systems incorporating problem solving and prevention strategies, perhaps using incentives to modify behavior in people with urinary stone disease.”

The study is supported by the NIDDK (DK110986).

A new approach to real-world evidence

September 8, 2017 – The Robert J. Margolis, MD, Center for Health Policy at Duke University has issued a white paper in advance of its upcoming one-day workshop on the use of real-world evidence.

Adrian Hernandez

As the demand for more use of real-evidence (RWE) grows, so does the need for a more nuanced and considered approach to it. That is the conclusion of a white paper issued by the Robert J. Margolis, MD, Center for Health Policy at Duke University in advance of its Sept. 13 workshop on real-world evidence.

The DCRI’s Adrian Hernandez, MD, MHS, is among the white paper’s authors.

In recent years the amount of patient data collected outside of clinical trials has become more plentiful and, with the advent of electronic health records, more easily accessible. Researchers and clinicians have recognized the potential uses of this information, but questions remain about how to best use it.

“Here, we advance the idea that RWE is the output from the combination of a variety of different RWD sources and methods for evidence development, and that the resultant applicability of RWE will necessarily vary across regulatory contexts of use,” the authors write. “Appropriately matching data and methods to potential uses will take time and input from all stakeholders involved in the development and use of medical products, but progress can be made if thoughtful and transparent collaborations can improve underlying challenges in data collection, study design, and shared infrastructure.”

The complete paper can be downloaded here.

Duke receives $12.5 million to study children with autism and ADHD

September 7, 2017 – The DCRI’s Scott Kollins, PhD, is a co-principal investigator for the trial.

Duke researchers will lead a $12.5 million, five-year program to study connections between autism and attention deficit and hyperactivity disorder (ADHD), joining five other universities as a National Institutes of Health Autism Center of Excellence.

Having both autism spectrum disorder (ASD) and ADHD can lead to more severe autism symptoms in young children, including tantrums, greater challenges at school and trouble making friends. There is little research on the estimated half of individuals with ASD who also have ADHD.

“Young children with autism who also have ADHD are diagnosed with autism at a much later age and have poorer outcomes,” said Geraldine Dawson, PhD, a co-principal investigator for the grant and director of the Duke Center for Autism and Brain Development. “Children with both conditions are 30 times more likely to receive a diagnosis of autism after age 6, which is a shame because we are able to diagnose autism reliably by 24 months. We want to understand why these children are being missed and help them get early interventions.”

Duke researchers across disciplines — including psychology, psychiatry, neuroscience, engineering, computer science and public policy — will launch three major projects designed to improve early detection and treatment of children with autism and ADHD.

The first project will follow about 9,000 infants and toddlers visiting Duke primary care clinics to identify those with symptoms of ASD, ADHD, or both. They will compare symptoms, progression and overall health outcomes, and test new screening tools. They will also probe racial and ethnic disparities in early diagnosis and strategies to reduce them.

A second effort will focus on understanding how brain dysfunction in ASD and ADHD are similar and how they differ. Researchers aim to identify signatures in brain activity or attention-related biomarkers that could predict risk for autism and ADHD in infancy.

The third project will evaluate a treatment that combines behavioral intervention and the use of Adzenys-XR-ODT, an FDA-approved ADHD drug. The researchers will study how the combined treatment affects autism and ADHD symptoms and patterns of brain activity.

“This research has the potential to significantly impact clinical practice,” said the DCRI’s Scott Kollins, PhD, co-principal investigator for the grant and director of the Duke ADHD Program (pictured). “We hope it will validate new approaches to early screening, specifically in pediatric primary care. It will also provide many children in North Carolina who have autism and ADHD with diagnostic and treatment services. We are grateful to NIH and to Duke for providing the opportunity to make a real difference both for families in our community and families everywhere.”

Data from Duke and all other Autism Centers of Excellence are included in a centralized NIH National Database for Autism Research, available to scientists and institutions around the world working to uncover the causes and develop the best treatments for ASD.

In addition to Dawson and Kollins, investigators leading the projects include Linmarie Sikich, Guillermo Sapiro, Scott Compton, Kenneth Dodge, Naomi Davis and Michael Murias.

NIH Health Care Systems Research Collaboratory launches comprehensive online resource on pragmatic clinical trials

September 5, 2017 – The new website shares the latest expert consensus on conducting efficient trials that generate evidence to improve healthcare.

The NIH Health Care Systems Research Collaboratory has launched a new website to serve as a primary resource for anyone wishing to learn how to conduct pragmatic clinical trials. Pragmatic trials are distinct from usual methods of research in that evidence is gathered from settings where healthcare is delivered every day, such as hospitals or clinics, often relying on electronic data that is already routinely captured. These trials have the opportunity to answer important research questions quickly and efficiently by applying novel research designs and tools—but employing new methods also comes with challenges.

Drawing on experiences from the NIH Collaboratory’s own pragmatic trials and community of experts, Rethinking Clinical Trials®: A Living Textbook of Pragmatic Clinical Trials explains how pragmatic trials are distinct and provides guidance on special considerations at each step of the research process, from trial design through applying results in the real world. The updated website can be accessed at www.rethinkingclinicaltrials.org.

“The new Living Textbook will help to change the way research is conducted by promoting best practices for pragmatic clinical trials. Easy access to this vast knowledge base will help expand the capabilities for conducting pragmatic research within health care systems, ultimately leading to advances in healthcare,” said the DCRI’s Kevin Weinfurt, PhD, editor-in-chief of the Living Textbook.

“The new website contains a wealth of knowledge from the past 5 years of conducting pragmatic trials in the NIH Collaboratory and will be continually updated as new information emerges. I look forward to seeing the impact the Living Textbook will have on the future of clinical research,” said the DCRI’s Adrian Hernandez, MD, MHS, who helps lead the NIH Collaboratory.

The NIH Collaboratory is supported by the National Institutes of Health Common Fund through a grant administered by the National Center for Complementary and Integrative Health (U54AT007748).

Cardiac arrests in black neighborhoods less likely to get CPR, defibrillation

August 30, 2017 – Study shows that delaying CPR is associated with worse survival.

Surviving cardiac arrest often seems like a matter of luck — whether a passerby knows CPR, or a defibrillator is close at hand or the ambulance arrives quickly.

But one surprising factor also plays a role: The racial makeup of your neighborhood.

Compared to people who live in predominantly white neighborhoods, those who live in predominantly black areas are much less likely to receive CPR or defibrillation from a bystander when their heart suddenly stops beating while they are at home or out in the community.

And that appears to translate into a worse chance of survival for people in black neighborhoods, according to a study published online Aug. 30 in JAMA Cardiology by researchers at the DCRI.

“We have known that there are differences in the rates of survival from cardiac arrest between blacks and whites, but it was surprising to see how the demographics of a neighborhood affected outcomes of residents who experience cardiac arrest,” said lead author Monique Starks, MD, a cardiologist and member of the DCRI. “This is absolutely a call to action to improve and expand CPR training and defibrillator access.”

Starks and colleagues — including senior author Graham Nichol, MD, director of the University of Washington-Harborview Center for Prehospital Emergency Care — analyzed cardiac arrest data from a registry that includes information from seven U.S. cities: Birmingham, Dallas-Fort Worth, Pittsburgh, Portland, Seattle, and Milwaukee. U.S. Census data were used to provide demographic information at the neighborhood level.

The researchers analyzed more than 22,000 cases in which cardiac arrest occurred in a non-hospital setting over a four-year period between 2008-11. Neighborhoods where out-of-hospital cardiac arrests occurred were classified by census tract, based on percentage of black residents, ranging from fewer than 25 percent, between 25–50 percent, between 51–75 percent, and more than 75 percent.

Compared with patients who experienced a cardiac arrest in mainly white neighborhoods, those in predominantly black neighborhoods were slightly younger, more frequently female, had lower rates of initial shockable rhythm, and less frequently experienced their heart event in a public location.

Overall, nearly 40 percent of people stricken with cardiac arrest outside of the hospital received bystander CPR. In primarily white neighborhoods, the rate was almost 47 percent, but in predominantly black neighborhoods, the rate was just 18 percent.

Use of an automated external defibrillator was similarly disproportionate by racial composition of the neighborhood. In mostly white neighborhoods, 4.5 percent of cardiac arrest victims received defibrillation from a bystander, compared to 0.9 percent in black neighborhoods.

Neighborhood make-up was also associated with survival. People with cardiac arrest in mixed- to majority- black neighborhoods had significantly lower adjusted survival rates at hospital discharge, the researchers found.

“We clearly see that treatments and outcomes for patients with cardiac arrest in black neighborhoods are worse than those in white neighborhoods. We then asked, does it matter if you were black or white within those neighborhoods?” Nichol said. “Our observations were reassuring. It actually does not matter if you are black or white within the neighborhood, but the neighborhood matters.”

That finding, the researchers noted, points to a lack of education about cardiac arrest and CPR in general among people living in black neighborhoods, along with a dearth of defibrillators in public spaces.

“This is something that can be addressed,” Starks said. “Organizations such as the American Heart Association and the American Red Cross, along with medical centers and public health departments, have a unique opportunity to address this issue with dedicated education programs that are particularly tailored for black neighborhood. This is a relatively low-cost solution that could save lives.”

In addition to Starks and Nichol, study authors include Robert H. Schmicker, Eric D. Peterson, Susanne May, Jason E. Buick, Peter J. Kudenchuk, Ian R. Drennan, Heather Herren, Jamie Jasti, Michael Sayre, Dion Stub, Gary M. Vilke, Shannon W.  Stephens, Anna M. Chang and Jack Nuttall on behalf of the Resuscitation Outcomes Consortium investigators.

The study was supported by the Resuscitation Outcomes Consortium, which was funded by the National Institutes of Health and American Heart Association. Starks also receives grant support from the National Institutes of Health Common Fund to promote diversity in health-related research (3U54AT007748-02S1).

As medical advances change the end of life for heart patients, new challenges emerge

August 28, 2017 – A new study finds cardiovascular disease (CVD) patients are more symptomatic, less likely to die at home and to receive high-quality palliative care than those with other diseases such as cancer.

While modern treatments and technologies have reduced both sudden and non-sudden cardiac deaths, many patients live longer but with the disabling symptoms of heart failure, according to an analysis of CDC Wonder data (Wide-ranging Online Data for Epidemiologic Research) by DCRI Fellow Haider Warraich, MD.

Warraich is the lead author of the study in the Sept. 5 issue of the Journal of the American College of Cardiology. The article was co-authored with the DCRI’s Adrian F. Hernandez, MD, MHS, and Larry A. Allen, MD, MHS, of the University of Colorado.

“Heart disease is extremely complex and challenging, especially in the last years of life,” said Warraich. “As physicians, we come into medicine as a calling, to save people from dying. In our training, we are taught to think of death as the enemy. But, as we deal with heart disease patients in the real world, we find it’s not that simple.”

The broad and comprehensive CDC data showed that while age-adjusted mortality of CVD has dropped in the past few decades, more people continue to die of heart disease than cancer or other diseases. And while new technologies help patients live longer, at some point, physician efforts switch to end-of-life care for both patients and caregivers.

“Cancer remains the template for that care, which often means dying at home and/or in hospice,” he said. “Heart disease is underrepresented in hospice care mostly because patient mortality is more difficult to predict, especially as hospice requires expected survival of less than six months.”

Longer lifespans mean more years with disability and a higher chance of dying in the hospital. With an aging and often obese population, the disability associated with CVD continues to rise. Medical advances have improved the care of heart disease patients over the years and patients don’t die as suddenly as they used to. Instead, they are more likely to survive previously life-threatening events such as myocardial infarction but be left with disabling heart failure.

“The clustering of illness in the last years of life is particularly pronounced in heart failure patients,” said Warraich, “and this is also placing a greater burden on caregivers.”

With advances in medical care have come new ethical challenges, especially when related to deactivation of cardiac devices, such as pacemakers, defibrillators, and mechanical circulatory support. Some patients prefer limits on the intensity of care at the end of life, but cardiologists rarely initiate such discussions with their patients – Warraich refers to guidelines suggesting that such preferences be discussed at least once a year.

The article makes several recommendations to improve end-of-life care for CVD patients. These include optimizing metrics to assess the quality of that care, overcoming and ameliorating disparities between types of patient care, enhancing physician education as well as research in palliative care, and innovating both palliative care delivery and reimbursement.

“But clear communication with patients is at the core, especially with health literacy being so variable,” Warraich said. “For example, some patients don’t want to be intubated but others want more care, including heroic measures, rather than less. They may want us to do everything, but may not understand what could happen if things don’t go as planned. I try to give them all the info they need to make the best decisions.”

Advances in medical science have increasingly made healthcare providers the gatekeepers of death and dying. While impressive progress has been made, Warraich said, patients with CVD continue to suffer disproportionately.

“How medicine is practiced and its effects on so many people are changing,” he said. “I hope this article will encourage cardiologists and other clinicians to reevaluate their relationship with heart disease patients and to carefully consider the many implications of advances in modern CVD care.”

Education and monitoring improves the use of stroke-prevention therapies

August 28, 2017 – Improving use of anticoagulation drugs is a key to preventing strokes

Only about half of patients with atrial fibrillation worldwide take anticoagulant drugs, despite the medications being highly effective in preventing strokes.

Increasing the use of anticoagulation therapies could prevent hundreds of thousands of strokes each year. A new study shows that education, measurement and feedback are effective approaches to increasing the use of anticoagulants, and demonstrate on a large scale how this improvement can be achieved.

In a large, international study led by the DCRI and five coordinating centers around the world, a multi-faceted informational campaign aimed at patients, families and physicians led to a 9-percent absolute increase in the use of anticoagulation therapies. The increased use of the drugs was accompanied by a small, but notable reduction in the risk of stroke.

“If this intervention could be broadly applied, which we believe is possible, the public health implications would be substantial,” said the DCRI’s Christopher Granger, MD (pictured), senior author of the IMProve treatment with AntiCoagulanTs in patients with Atrial Fibrillation (IMPACT-AF) trial presented at the European Society of Cardiology meeting. The findings were simultaneously published Aug. 28 in Lancet. “More than 33 million people worldwide have atrial fibrillation, which is a leading cause of stoke. Improving adherence to anticoagulation therapy would be a lifesaver.”

Granger and colleagues conducted a study in five countries — Argentina, Brazil, China, India and Romania — to test their intervention.

More than 2,200 patients were enrolled at 48 hospitals and monitored for a year. The centers were randomly divided to either provide standard care or a comprehensive educational effort — customized to each country — that explained the benefits of anticoagulant therapies, as well as their risks.

Patients were given brochures and shown videos, and then monitored at doctor visits to get their feedback and learn of any problems that kept them from being on the medication. Physicians received education on treatment of atrial fibrillation through articles and webinars. They also received audits for the specific treatment of each of their patients via regular phone calls.

The researchers reported that among patients at centers that received the educational intervention, the use of anticoagulation therapies rose by 11.7 percent, compared to a 2.6 percent rise in their usage in the non-intervention group. In the intervention group of patients not treated at baseline, 48 percent were on an anticoagulant at one year.

“Our study also found a reduction in strokes in the intervention group compared to the control group,” said Renato Lopes, MD, PhD, principal investigator for Brazil. “While this was a secondary outcome, it highlights the potential benefit of improved anticoagulation care.”

A limitation of the cluster-randomization design, in which recruited expert centers were assigned to one or the other arms of the study, was a potential overestimation of the baseline use of anticoagulants in the non-intervention sites.

Granger said additional studies are needed to better understand why such a large proportion of patients remains untreated. In the meantime, he said, applying the interventions tested in this study has been shown to improve care.

In addition to Granger and Lopes, study authors include Dragos Vinereanu, M. Cecilia Bahit, Denis Xavier, Jie Jiang, Hussein R. Al-Khalidi, Wensheng He, Ying Xian, Andrea O. Ciobanu, Deepak Y. Kamath, Kathleen A. Fox, Meena P. Rao, Sean D. Pokorney, Otavio Berwanger, Carlos Tajer, Pedro Barros, Mayme L. Roettig and Yong Huo.

The investigator-initiated study received support through education and research grants from pharmaceutical companies that market anticoagulation drugs, including Boehringer Ingelheim Pharmaceuticals Inc., Daiichi Sankyo, Bayer Pharmaceuticals, Bristol-Myers Squibb and Pfizer, Inc.

New study to establish evidence for myasthenia gravis treatments

August 25, 2017 – The DCRI will serve as the coordinating center for the study of the rare and debilitating condition.

For the first time, patients and clinicians may have empirical evidence for the best treatments for myasthenia gravis, thanks to a newly funded research project led by Duke’s Donald Sanders, MD, and Pushpa Narayanaswami, MD (Beth Israel Deaconess Medical Center/Harvard Medical School), and funded by the Patient-Centered Outcomes Research Institute (PCORI).

The DCRI will act as the primary research facility and coordinate the results from the various centers collecting data. In addition, other researchers, clinicians, patients with myasthenia gravis, advocacy groups such as the Myasthenia Gravis Foundation of America, and other stakeholders will be involved in the implementation of the study and dissemination of the results.

A variety of treatments exist for this rare but debilitating, potentially fatal condition. However, clinicians still don’t have strong evidence for how these treatments compare in alleviating symptoms or long-term management of the disease. In addition, the ideal treatment for an individual varies based on that person’s disease severity, age, gender, and other factors. The $2.5 million PCORI contract will allow Sanders and Narayanaswami to follow roughly 200 patients from across the United States and Canada for two years each.

“This study is the culmination of many years’ work with myasthenia gravis patients and their physicians, and will bring together members of an informal network of neurologists who share the common goal of improving the lives of their patients,” said Sanders. “It provides a unique opportunity to help patients–and their physicians–answer the question, ‘Given my diagnosis and disease severity, which treatment(s) will be the most effective for me, with the least side effects?’”

The prospective, observational study may also help myasthenia gravis patients receive coverage for the costs of their health care. Because no treatments for myasthenia gravis have FDA approval, insurance companies are sometimes reluctant to reimburse patients for treatment. Stronger evidence for the benefits of treatments may make insurers more likely to cover those costs, Sanders said.

The study will have two specific approaches. First, it will test the validity of the international consensus guidance statements for myasthenia gravis published last year. At the end of the three-year study period, researchers will be able to analyze whether patients receiving treatment meeting this guideline did better than patients who did not. Secondly, the project will also help clinicians to directly compare treatments to see if either is more effective than the other, or if both are equally effective.

The award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract. PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed healthcare decisions. For more information about PCORI’s funding, visit www.pcori.org.

Improving heart failure care and outcomes through behavioral economics

August 24, 2017 – Applying behavioral economic concepts and encouraging positive behaviors in heart failure patients may offer additional approaches to improving patient-centered outcomes, according to a new study by DCRI researchers.

An in-depth review by DCRI researchers published this week in Circulation, the official journal of the American Heart Association, highlights how behavioral economics concepts can help overcome barriers to change, encourage positive behaviors and offer novel approaches to improving patient outcomes for heart failure patients.

“The biggest challenge of living with heart failure is that it is something that must be dealt with around the clock, every single day, with no days off,” said the DCRI’s Adam DeVore, MD (pictured), the article’s first author. “When every meal you consume has the potential to make you sick, disciplined daily routines and consistently healthy behaviors become not just necessary, but crucial to a patient’s wellbeing and longevity.”

Heart failure affects 5.7 million American adults, costing the U.S. health system $30.7 billion annually, and is associated with high morbidity and mortality. The strong relationship between the disease and the patient’s daily lifestyle choices make it difficult to manage, yet these same challenges make it an ideal disease to target with well-designed “nudges.”

“Heart failure serves as a model prototype to explore the potential role of behavioral economics in managing chronic diseases,” DeVore said.

Their paper provides a brief explanation of core behavioral economics concepts that can predict and affect human behavior including various types of bias, loss aversion and anticipated regret, which apply to heart failure patients. It also provides guidelines on how to craft these concepts into tools, such as financial incentives and social networks that may improve the management of heart failure patients.

“While offering financial incentives or commitment contracts provides short-term gratification to reward behaviors consistent with a positive health outcome, social incentives combined with financial incentives have the potential to have a more powerful and longer-lasting effect on heart failure patients,” DeVore said.

Although there are various tools available for the effective management of heart failure, especially prescription medications, physicians struggle with being able to give those therapies to patients in an efficient way, where patients can remember to regularly take them on a timely basis.

To explore different ways to help patients with various aspects of self-care, DeVore and his colleagues are in the process of conducting the Care Optimization through Patient and Hospital Engagement Clinical Trial for Heart Failure (CONNECT-HF). CONNECT-HF is currently enrolling patients and will explore behavioral economics in relation to HF besides examining the impact of two mobile applications utilizing non-financial incentives and rituals.

“Our aim is to try and learn what different incentives we can use to try and help promote healthy behavior change,” DeVore said.

Acknowledging the work of co-author, Dan Ariely, MD, James B. Duke Professor of Psychology and Behavioral Economics at Duke University and his group, the Center for Advanced Hindsight, who also helped design and execute the CONNECT-HF trial, DeVore said that by incorporating behavioral knowledge into medical practice, low-cost, large-scale policy interventions can be developed to encourage positive and persistent behavioral change in heart failure patients.

In addition to DeVore and Ariely, other contributing authors included Leslie Chang, Bradi Granger, Zubin Eapen and Adrian Hernandez.

To learn more about the CONNECT-HF trial visit http://connectheartfailure.org.