Goal 2: Reduce Human Disease

To extend our knowledge of the pathobiology of heart, lung, blood, and sleep disorders and enable clinical investigations that advance the prediction, prevention, preemption, treatment, and cures of human disease.

Goal 2: Reduce Human Disease

Sleep Apnea and Metabolic Complications

Does treating sleep apnea benefit metabolic complications including non-alcoholic fatty liver disease?

Submitted by (@nhlbiforumadministrator)

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 : ATS Member

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Goal 2: Reduce Human Disease

Sleep Paralysis must be better known to doctors, therapists, and the public

Sleep Paralysis (SP) is a very common (up to 40% of all people), yet little-known condition that is terrifying, and potentially traumatizing, especially to people who are unaware of this condition. It is critical that SP is better known by all doctors, therapists, and the public. Too many people are mistreated and misdiagnosed as psychotic or even demon possessed when they do not understand SP, or they hide the experience ...more »

Submitted by (@kendraz)

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

Details on the impact of addressing this CQ or CC :

Worldwide, all cultures have created elaborate spiritual explanations for SP, many of which unnecessarily foster fear and superstition. Both fear and superstition could be greatly reduced by an objective understanding of SP and its remedies.

 

Examples of sleep paralysis being little known: In the current Coursera course, Sleep: Neurobiology, Medicine, and Society at the University of Michigan, which has many medical doctors and Phds as students, all the rare forms of sleep disorders were listed and discussed, except common Sleep Paralysis, which was never even mentioned.

 

When I personally experienced chronic SP 13 years ago, none of my doctors had heard of it. I had to do my own research to discover what it was and how to reduce it. In the meantime, when I shared the experiences of seeing fearful presences in my bedroom with my best friend, she became convinced I was possessed by an evil spirit and urged me to undergo a shamanic depossession ritual. Even after I explained to her that SP was common, she completely cut off all relationship with me out of her fear.

Feasibility and challenges of addressing this CQ or CC :

making Sleep Paralysis better known as a first step should be easily accomplished by requiring education on this topic for all medical personnel. In addition, a campaign to inform the public is needed.

 

Reducing stress and disturbed sleep are the baseline for reducing SP. These simple remedies should be easily communicated to the public.

Name of idea submitter and other team members who worked on this idea : kendra zoa

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15 up votes
5 down votes
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Goal 2: Reduce Human Disease

What types of questions are most likely to improve the health of the public? The importance of discovery science.

Congressional eagerness to see research funding translate into improvements in health care may make studies that address “how-to-deliver-care-questions” seem attractive. But the answers to “how–questions” are often so context dependent that the findings are neither generalizable nor durable. The answers to “how–questions” too often become obsolete when the health-care system, the electronic medical record, or the insurance ...more »

Submitted by (@psaty0)

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

Details on the impact of addressing this CQ or CC :

Improvement in the health of the public.

Feasibility and challenges of addressing this CQ or CC :

It is easily a matter of focus.

Name of idea submitter and other team members who worked on this idea : Psaty & Tracy

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22 net votes
29 up votes
7 down votes
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Goal 2: Reduce Human Disease

What causes the structural abnormalities that cause sleep apnea, and how can they be prevented?

It is estimated that up to 28% of the population suffer from sleep apnea, which impairs functioning and reduces quality of life, while increasing risk of accidents and a variety of cardiovascular, metabolic, and neuropsychiatric diseases. A large portion of sleep apnea cases are caused by abnormal oro-nasal-maxillo-mandibular features that result in crowding of the upper airway, making it vulnerable to collapsing or ...more »

Submitted by (@bmdixon)

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

Details on the impact of addressing this CQ or CC :

Obstructive sleep apnea (OSA) is a common condition, which causes chronic fatigue and daytime sleepiness, as well as cognitive impairments affecting learning, concentration, and memory. Over the long term, it increases many health risks, including accidents, cardiovascular disease, and depression. OSA is characterized by partial, or complete, blockage of the airway during sleep, so that breathing repeatedly pauses or airflow is limited, causing repeated arousals from sleep. It is usually secondary to a narrow, or collapsible, airway due to either 1) obesity or overweight, or 2) abnormal morphology of the mandible or maxilla bones, which crowds facial structures, such as the tongue and nose, narrowing the pharynx. The causes of obesity are already being well studied, but there is relatively little research on the etiology of the structural abnormalities involved in OSA. Abnormalities of facial structure are widespread in the population causing, not only OSA, but also orthodontic problems that require many to get braces or have wisdom teeth extracted, and widespread temporomandibular joint (TMJ) problems. However, multiple studies have documented that these abnormalities are almost completely absent from populations living a preindustrial, agrarian or forager, lifestyle, making them a “disease of civilization”. In particular, the abnormalities are associated with consumption of a modern diet of processed foods during prenatal, infant, and early childhood development.

Feasibility and challenges of addressing this CQ or CC :

Current evidence implicates three factors in the development of these structural abnormalities: prenatal maternal nutrition (especially vitamin K2 status), breastfeeding vs. bottle-feeding, and frequency of consumption of tough foods after weaning (which provides exercise to the jaw). We need to form a large cohort and study orthodontic development prospectively from fetal development through mid-childhood, with data on diet, feeding practices, and physiological measures of nutrient status. Measurement methods are available using existing technologies to collect the necessary data on each of these measures. Determining the causes responsible for these structural abnormalities will enable further research to demonstrate effective methods of preventing them. Given that many patients with OSA are rendered so miserable by it that they undergo maxillomandibular advancement surgery to correct it, an expensive procedure with a lengthy recovery period, prevention would be a far better solution. This research will move us a big step closer to a future without sleep apnea and its formidable collection of negative effects on health and functioning.

Name of idea submitter and other team members who worked on this idea : Bonnie Dixon

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Goal 2: Reduce Human Disease

What is the optimal management of coronary artery disease in patients with chronic kidney disease

Patients with chronic kidney disease (CKD) have an extremely high risk of death from cardiovascular cause. The prognosis of patients with chronic kidney disease who also have coronary artery disease is worse than certain cancer. While great strides have been made to create awareness about breast cancer, there is paucity of knowledge about the cardiovascular risk of CKD patients among both physicians and patients. Moreover, ...more »

Submitted by (@sripal.bangalore)

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

Details on the impact of addressing this CQ or CC :

Patients with chronic kidney disease tend to be undertreated (from CV perspective), underrepresented (in clinical trials) and underdiagnosed with less referral for stress testing, even though cardiovascular cause is the leading cause of death in these patients. Patients with chronic kidney disease is a growing cohort and with increase in obesity and diabetes, the prevalence is exploding exponentially. The decision as to what is the best treatment option for patients with coronary artery disease who also have kidney disease- revascularization or medical therapy-is important and can be a paradigm shift in the way we treat such patients

Feasibility and challenges of addressing this CQ or CC :

Randomized clinical trials are urgently needed to answer this question.

Name of idea submitter and other team members who worked on this idea : Sripal Bangalore

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Goal 2: Reduce Human Disease

Evidence based approaches to Red Blood Cell transfusion

What are the optimal RBC transfusion thresholds for adult and pediatric cancer patients undergoing chemotherapy regimens that may improve functional status and quality of life?

Submitted by (@nareg.roubinian)

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

Details on the impact of addressing this CQ or CC :

Cancer patients undergo intensive medical and surgical therapies to treat their underlying disease. Treatment commonly results in anemia requiring RBC and platelet transfusions to support the patient through the hypoproliferative phase of chemotherapy. This is particularly true for those patients requiring hematopoetic stem cell transplantation (HSCT). Following therapy, cancer outpatients commonly receive RBC transfusions for weeks to months to maintain their functional status.

 

Common causes of death in patients with hematological malignancies and other cancers are infections and bleeding. A meta-analysis of clinical trials suggested that liberal transfusion is associated with greater risk of infection. Conversely, restrictive transfusion could adversely affect quality of life and functional status in oncology populations. In addition, pre-clinical and clinical studies support that concomitant anemia and thrombocytopenia significantly compound bleeding risk, and that hemostasis can be optimized in thrombocytopenia by maintaining a higher hematocrit. Although bleeding risks in relation to platelet transfusion thresholds are well studied in patients with hematological malignancy, optimal hemoglobin levels in thrombocytopenic patients are not known. Despite the significant allocation of blood components to cancer patients as a whole, RBC transfusion practices are not well studied within this group.

Feasibility and challenges of addressing this CQ or CC :

Randomized controlled clinical trials and other studies investigating optimal transfusion thresholds and other measures of practice are required to provide health care providers with evidence to guide one of the most common therapies administered in the setting of malignancy. The clinically important end points of well-designed studies could include: 1) quality of life and functional status for both inpatients and outpatients; 2) neurocognitive development in pediatric populations; 3) bleeding events / bleeding scores; 4) impact on immunity including immunomodulation and infection; 5) reconstitution of hematopoiesis; and 6) survival and/or recurrence of disease.. Besides a generalizable study population, certain target populations of interest are those with high risk disease, HSCT patients, patients undergoing radiation therapy, and pediatric patients.

 

There are >1.6 million new cases of cancer annually in the USA, including >50,000 with leukemia and >6,000 with HSCT. Cancer therapies are rapidly advancing in the era of genomics and immunotherapy. Capitalizing on the tradition of research in cancer, single and multicenter studies of RBC transfusion are feasible using randomized controlled designs in conjunction with clinical trials of chemotherapeutic regimens. The results of these studies will impact a large patient population’s quality of life, and may ultimately impact healthcare cost and blood demand.

Name of idea submitter and other team members who worked on this idea : Nareg Roubinian, MD and Naomi Luban, MD for the 2015 NHLBI State of the Science in Transfusion Medicine

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Goal 2: Reduce Human Disease

Oral Magnesium Supplementation in Asthmatic Populations

Do asthmatics and/or those at risk for developing asthma benefit from oral Magnesium supplementation?

Submitted by (@nhlbiforumadministrator1)

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

Details on the impact of addressing this CQ or CC :

Possible reduction of asthma symptoms, bronchial reactivity, bronchodilator use, and increased quality of life in asthmatic population particularly those with higher asthma burden. Oral Mg supplementation could be effective adjunct therapy.

Feasibility and challenges of addressing this CQ or CC :

Oral Mg supplementation is inexpensive and appears to be safe in Asthma populations studied so far. Careful consideration of asthma endpoint(s) to be measured, Mg status measure, and mechanisms of action may be possible at this time considering previous studies in this area. Equipoise appears to exist based on the literature, for finding definitive answer about effectiveness/efficacy (and safety) of oral Mg supplementation.

 

Thoughtful consideration of asthma endpoint(s) and Mg status measure, and the population to be studied so that the trial provides definitive answers. Feasibility for enrolling the population with the chosen characteristics, in sufficient numbers without interfering with institute-initiated Asthma studies, may need to be demonstrated.

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

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-21 net votes
3 up votes
24 down votes
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Goal 2: Reduce Human Disease

Early COPD

What does early COPD actually look like. This is defined as severe COPD 30 years prior to its manifestation.

Submitted by (@davidmannino)

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

Details on the impact of addressing this CQ or CC :

Prevention programs in COPD either target smoking or those with established disease. Better understanding the factors that lead to the development of COPD (both in ever and never smokers) is critical to improved disease prevention.

Feasibility and challenges of addressing this CQ or CC :

We need to revisit long term studies in novel ways- and look at new cohorts. Better biomarkers need to be developed.

Name of idea submitter and other team members who worked on this idea : Dave Mannino

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22 net votes
31 up votes
9 down votes
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Goal 2: Reduce Human Disease

Impact of intrauterine environment on obesity

How does maternal obesity before or during pregnancy affect the intrauterine environment and increase the risk of overweight/obesity in the offspring?

How does maternal dietary intake during pregnancy impact weight in the offspring?

What types of interventions are most effective for preventing excessive gestational weight gain among high risk groups?

Submitted by (@nhlbiforumadministrator1)

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

Details on the impact of addressing this CQ or CC :

Developing effective interventions during pregnancy can help reduce the risk of obesity in the offspring and mother.

Feasibility and challenges of addressing this CQ or CC :

Addressing how the intrauterine environment impacts obesity is feasible because there is already existing preliminary data that shows pregnancy can have an impact on obesity in offspring. More work needs to be done to better understand the mechanism and how best to intervene.

There is emerging evidence that the intrauterine environment can have an impact on obesity.

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

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33 net votes
66 up votes
33 down votes
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Goal 2: Reduce Human Disease

Mental health and wellness in sickle cell disease

A growing concern among the sickle cell community surrounds the lack of mental health and wellness services. Many in the community deal with anxiety and depression. It is well known how intricately connected mental and physical health are. So if we know that stress can trigger a psychological crisis which in turn triggers a physical pain crisis, why do we not automatically include mental health services within patient ...more »

Submitted by (@sicklecellwarrior)

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

Details on the impact of addressing this CQ or CC :

Many in the SCD community feel like providers do not take a proactive approach to mental health. A comprehensive approach to developing mental health and wellness services and programs provides an opportunity to address factors contributing to morbidity, and perhaps mortality, in the SCD community, outside of the hospital walls.

Name of idea submitter and other team members who worked on this idea : Sickle Cell Warriors, Inc. community members

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25 net votes
38 up votes
13 down votes
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Goal 2: Reduce Human Disease

Personalized therapy of HCT complications

Can biomarkers make all the use of new predictive biomarkers enable earlier and more effective treatment of acute GVHD? Can biomarkers accurately guide reduction in therapy for patients who will respond to standard steroid treatment? Can biomarkers enable earlier and thus more effective therapy for high risk GVHD? Can new biomarkers (proteomic, genomic or a combination) also predict patients who are risk of relapse?

Submitted by (@james.ferrara)

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 enormous advances in immunology over the past several decades, there are no validated new therapies for acute GVHD, the major complication of allogeneic HCT. Several biomarkers have now been identified at the onset of GVHD that can predict response to treatment and provide a personalized profile of patients. But these biomarkers have not yet been used to guide therapy, and definitive clinical trials are needed to answer this question

Feasibility and challenges of addressing this CQ or CC :

Need for consistent and accurate biomarker determination available for a large number of centers

Name of idea submitter and other team members who worked on this idea : John Levine and James Ferrara

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74 net votes
99 up votes
25 down votes
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Goal 2: Reduce Human Disease

Extending gene editing and iPS/cell technologies to high throughput formats

Can we extend gene editing and iPS/cell technologies to high throughput formats for the evaluation of genetic variants identified from GWAS in the development and progression of disease?

Submitted by (@nhlbiforumadministrator)

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 : NHLBI Staff

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-15 net votes
3 up votes
18 down votes
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