Showing 11 ideas for tag "treatment"

Goal 2: Reduce Human Disease

Elucidate the different causes of circadian disorders, and tailor the treatment to the cause

There are several possible physiological causes of Circadian Rhythm Sleep-Wake Disorders (CRSWDs), including lack of sensitivity to light, over-sensitivity to light, deficiencies in the ipRGC cells of the retina, lack of melatonin production, long elimination time of melatonin, long intrinsic circadian period, differences in timing of sleep relative to internal circadian rhythms, differences in tolerance to phase mismatch,... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

By analogy, a patient may complain of abdominal pain. This could be due to infection, food poisoning, ulcers, gall bladder, cancer, etc. Imagine if we treated all these patients by removing their gall bladder. There would be some successes, and many failures. Surveys of the literature would be unable to conclude that there's a well established successful treatment. That's the state of treatment of CRSWDs today.

CRSWDs such as Delayed Sleep-Wake Phase Disorder and Non-24-Hour Sleep-Wake Disorder cause suffering and inability to work normal hours, and often result in otherwise productive individuals being unable to find jobs and having to seek disability support. Those who force themselves into a conventional schedule often end up with chronic health problems, resulting in further cost to themselves and to society.

Feasibility and challenges of addressing this CQ or CC

A major challenge is to diagnose not only a CRSWD but also its underlying cause. Improved biomarkers are essential to this effort, and are the subject of other Questions/Challenges. So too are a better understanding of the effects of light and melatonin on patients with these disorders, with the differing underlying impairments studied separately. With improvements in genetic testing and analysis, and improved sensor technology and automated data analysis, we are at the threshold of being able to study these underlying causes of CRSWDs.

Name of idea submitter and other team members who worked on this idea Peter Mansbach

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47 net votes
58 up votes
11 down votes
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Goal 3: Advance Translational Research

Impact of care coordination programs on depression detection and treatment in COPD patients

Does a care coordination program improve the detection and treatment of co-morbid depression in patients with COPD?

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Name of idea submitter and other team members who worked on this idea Edna Shattuck, COPD Foundation Patient Advocate

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13 net votes
17 up votes
4 down votes
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Goal 2: Reduce Human Disease

RCT of stepped-care depression treatment on CV events & death

Does treating depression improve survival and reduce major adverse cardiac events in acute coronary syndrome patients?

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

A substantial evidence base now exists showing that depression is associated with a two-fold increased risk of death and recurrent CV events in cardiac patients, leading to a recent AHA scientific statement recommending its elevation to the status of a risk factor for adverse medical outcomes in patients with acute coronary syndrome (Lichtman et al., 2014). Yet there is currently no clinical trial evidence that reducing depression improves cardiac morbidity and mortality. A clinical trial, using new, more effective depression treatment methods, such as collaborative care approaches that combine psychological counseling with medication in stepped-care fashion, is needed to determine whether effective treatment of depression can improve survival and reduce clinical cardiovascular events in cardiac patients.

Feasibility and challenges of addressing this CQ or CC

Newer stepped-care treatments for depression, combining medication and psychotherapy, have recently been developed and found to more effectively reduce depression than earlier treatments. By using these newer treatment methods to substantially lower depression, we can better answer the question as to whether treating the newly acknowledged risk factor of depression in ACS patients can improve clinical outcomes in these patients.

Name of idea submitter and other team members who worked on this idea NHLBI Staff

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5 net votes
22 up votes
17 down votes
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Goal 2: Reduce Human Disease

Longitudinal Outcomes in Adults with Congenital Heart Disease

There is a critical need in congenital heart disease to assess longitudinal medical and psychosocial outcomes in older children and adults in the U.S. Current data in this area, particularly in the adult population, is limited to single center or small multicenter efforts or registry/population data from other countries. Understanding the long-term aspects of care is necessary not only to provide lifelong high quality... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

Opportunity: It is well known that there are now more adults than children living with congenital heart disease. It was estimated that there were approximately 1 million adults living with CHD in the United States in 2000. Yet, less than one-third of these patients receive specialized adult congenital cardiology subspecialty care. Despite reported gaps in care and published guidelines, there is no accurate estimate of this population in the United States. In the absence of a national registry for patients with CHD, there are other opportunities to reach a broader population for participation in research and for population-based studies using resources such as improved electronic health records and social media. These might include the development of methods to use large electronic health record networks. Moreover, established networks are often required to facilitate translating basic science observations to clinical outcomes. It is only with further mechanistic and populatio n-based data that we will begin to understand long term outcomes, develop new medical, transcatheter, and surgical strategies, improve quality of care and begin to plan for lifelong care of all people with CHD.

Feasibility and challenges of addressing this CQ or CC

Feasibility will remain difficult in the absence of associated registries or databases currently in existence. New electronic health records may make this more feasible than in the current system. Efforts to standardize definitions, classification and coding of CHD would also be required to link and compare current databases. Although the challenges described are certain, multicenter collaboration as demonstrated by the Alliance for Adult Research in Congenital Cardiology (AARCC); an established collaborative research network among 15 adult congenital heart centers, may help overcome these obstacles.

Name of idea submitter and other team members who worked on this idea Stephen Cook

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11 net votes
15 up votes
4 down votes
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Goal 3: Advance Translational Research

Addressing Unrecognized and Over Diagnosis of COPD

How can we create precision diagnostics for COPD in practice settings that will help inform the transition from screening to better diagnosis and treatment strategies and that will help identify patients or communities at highest risk for unrecognized or over diagnosed COPD.

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

About 12 million individuals are estimated to have undiagnosed COPD and in most cases patients aren’t diagnosed until they have lost over half of their lung function leading to worse outcomes short and long term. Conversely there are challenges with over and mis diagnosed COPD that can result in over treatment and incorrect treatment. Fine tuning screening, diagnostic and management tools can result in earlier and proper identification of disease, earlier initiation of risk mitigation and/or treatment strategies and improved health outcomes as well as improved efficiencies in the healthcare system.

Name of idea submitter and other team members who worked on this idea COPD Foundation, Nancy Leidy, COPDF MASAC

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11 net votes
12 up votes
1 down votes
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Goal 2: Reduce Human Disease

Reversing pulmonary fibrosis/interstitial lung disease

Can a multipronged approach to reversing/repairing scar tissue in pulmonary fibrosis be deployed as soon as possible?

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

Despite high mortality rates and frustrating disease management marked by exacerbations and uncertainty including chronic oxygen supplementation, pulmonary fibrosis patients lack evidence-based treatments to reverse and repair the fibrotic process. These patients comprise a comparatively smaller proportion of lung disease patients such as chronic obstructive pulmonary disease and asthma and owing to their smaller number, have not been studied in large-scale randomized controlled trials. Treatments that need to be critically studied include regenerating lung tissue through stem cell treatment, artificial/ mechanical lung (augmentation) technology, and new drugs to directly reverse scar tissue, not simply to arrest the fibrotic process as with the two new available drugs for IPF patients.

Feasibility and challenges of addressing this CQ or CC

Challenges are smaller numbers of available patients available for clinical trials compared with other lung conditions such as COPD and asthma and lack of research funding. Also the etiology of the disease is multifactorial and the categories of fibrosis difficult to accurately diagnose and understand.

Name of idea submitter and other team members who worked on this idea Mary Hand

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6 net votes
8 up votes
2 down votes
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Goal 3: Advance Translational Research

Create a National Action Plan for COPD

Lead a coordinated effort of government, patient advocacy organizations, professional organizations, payers and others to plan and implement a coordinated plan to improve COPD awareness, education for patients and healthcare professionals, treatment strategies, research and data collection, policies and public health infrastructure and programs.

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

Unlike most leading causes of death and disability, there is no coordinated effort to lower disease burden associated with COPD. Coordinated plans provide a forum for identifying the most pressing issues that must be tackled, for setting goals and convening partners from different disciplines and are a framework upon which policy change can be achieved. In order to make meaningful progress in the impact that COPD is having on patients, health systems and payers, coordinated planning and action is needed and NHLBI can lead the way but time is of the essence.

Feasibility and challenges of addressing this CQ or CC

There are proven models of multi-stakeholder, public and private partnerships to tackle disease burden and create national plans. There are also multiple national and regional organizations standing ready to assist in these efforts.

Name of idea submitter and other team members who worked on this idea COPD Foundation Board of Directors, COPDF MASAC, COPDF State Advocacy Captains

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6 net votes
6 up votes
0 down votes
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Goal 2: Reduce Human Disease

Treatment Options for Diabetics and Impact on Cardiovascular Health

As a clinician, over the years I have noted major differences in adverse cardiovascular outcomes in diabetics who are treated with insulin +/- oral agents compared to those only treated with oral agents. Cardiovascular events occur much less often and at a much later timeframe in diabetics ("Type 2/adult onset") treated with insulin as the primary method. Even with newer agents, there may be slight improvement, but... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

Delaying the onset of injury to vasculature in Type 2 diabetics would have a major impact on quality of life for the diabetic and costs to the healthcare system. Dialysis and related costs are around $200,000/yr and interfere with life and ability to work, leg ulcers are often chronic and in many cases result in amputation.

Feasibility and challenges of addressing this CQ or CC

This study could be performed in the clinical setting or could be completed with chart review to determine diabetics on insulin on insulin and review outcomes. If access to charts for patients continuously treated for five years or longer were available, this study could be done in a shorter timeframe and at less cost. A long term and costly alternative would be to begin a clinical trial. Medical claims data may be an alternative, but it would be important to identify onset of diabetes and treatment for a minimum of five years.

Name of idea submitter and other team members who worked on this idea Patricia Gladowski

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3 net votes
7 up votes
4 down votes
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Goal 2: Reduce Human Disease

Preventing hospitalization of asthma patients through agressive acute asthma management

Develop simplified agressive acute asthma treatment protocol and promote the implementation for primary care, ED, Urgent care, Internists, Pediatricians, Allergists, Pulmonologists, and Families. Goal is to prevent hospitalization of children and adults with asthma.
For example: 3 cycles of Duoneb and Albuterol, monitoring pulsoximetry, with oral / IM Dexamethasone dosage. Also initiation of controller medication at... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea D M Meier MD

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0 net votes
7 up votes
7 down votes
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Goal 2: Reduce Human Disease

Define the biological basis, new diagnostics and therapeutics for severe sarcoidosis phenotypes

Sarcoidosis affects individuals of all races and ages and both genders, although it tends to cause significant morbidity and mortality for people in the prime of their productive life. Women, minorities and underserved populations tend to be more affected. Recent studies suggest that sarcoidosis and its severe manifestations, such as cardiac, neurologic and end stage pulmonary disease' are increasing, While the current... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Critical Challenge (CC)

Details on the impact of addressing this CQ or CC

Increasing our understanding of the biological basis of sarcoidosis, its more severe phenotypes, and resolution of disease, will help improve detection and personalized management and treatment of this disease, ultimately reducing disease burden. With the current availability of epidemiologic, genetic, genomic and epigenetic tools and studies, as well as buy in from sarcoidosis patient groups, we are poised to address why some people develop more severe forms of this disease. While genetic variants are associated with sarcoidosis, the specific variants responsible for disease risk and that dictate disease activity are largely unknown. The results of studies to date suggest that other susceptibility factors or forms of genetic regulation must act in concert with exposure in the development of sarcoidosis. Growing data in other immune-mediated diseases suggests that epigenetic mechanisms in combination with genetic susceptibility and environment may help explain disease risk. By understanding these genetic, genomic and epigenetic factors, and better defining the natural history of sarcoidosis, interventions can be tested and undertaken to potentially prevent or treat the development and or progression of this devastating disease.

Feasibility and challenges of addressing this CQ or CC

The care and management of individuals with sarcoidosis is not well standardized. This has been hampered by a lack of understanding of the natural history, which appears to vary significantly. As a result, undertaking studies to define the pathobiology of this disease is biased based on the centers and researchers involved. There have been few limited multi-center studies of sarcoidosis, except for pharmaceutical trials. In the past few years, NHLBI has funded the GRADS study, a cross-sectional multi-center study, laying the ground work for needed longitudinal multi-center studies of the biological basis of disease. With involvement from a larger sarcoidosis research community and the ability to undertake large scale studies to not only define the epidemiology of this disease, but also the pathobiology of disease based on integrative Omics, new personalized diagnostics and therapeutics can be developed and tested to help address the burden of sarcoidosis.

Name of idea submitter and other team members who worked on this idea Lisa Maier, Nabeel Hamzeh, Tasha Fingerlin, Ivana Yang, Brian O'Connor, Elliott Crouser

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1 net vote
3 up votes
2 down votes
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Goal 1: Promote Human Health

dose-response relationship for behavioral treatment of obesity

As a member of the NHLBI panel updating the obesity treatment guidelines, it was clear that there has not been much systematic work on the relationship between the frequency of intervention contacts and the short and long-term effects on weight change and maintenance of weight change. The general finding that more frequent contacts with professional weight loss counselors, and a longer series of contacts, are more effective,... more »

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

The sad truth is that many short-term weight loss treatment programs are still being offered by health care organizations. Although experienced weight loss researchers agree that such programs are ineffective and therefore a waste of resources. the definitive research on the does-response relationship between number of intervention contacts, and duration of intervention programs has not been done.

Feasibility and challenges of addressing this CQ or CC

Such a study could easily be done, but would probably require an RFA mechanism to get past prejudices of some peer reviewers.

Name of idea submitter and other team members who worked on this idea Victor J. Stevens

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-2 net votes
3 up votes
5 down votes
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