Showing 27 ideas for tag "disease"

Goal 3: Advance Translational Research

Bone Marrow Stem Cell Transplant in Peds sibling matched SCD

There is a need to improve accessibility of Bone Marrow Stem Cell Transplantation (BMSCT) for Sickle Cell Disease patients who are most likely to benefit from this treatment option.

1. Building a culture of trust between and among primary care providers, specialists, patients/families, and other stakeholders

2. Consensus building around BMSCT as an acceptable treatment alternative (as opposed to another research endeavor)... 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
  1. It could potentially decrease the prevalence of SCD and significantly decrease the overall morbidity and mortality associated with SCD in children with matched sibling donors.
  2. It could increase the awareness of health professionals who have a low awareness of the role of BMSCT in the treatment and cure of SCD (i.e., those in rural areas)
  3. It can improve patient/family access to information and communications to facilitate informed discussion and choice for all SCD treatment options
  4. It could open the gateway for more therapeutic applications for other genetic diseases
Feasibility and challenges of addressing this CQ or CC
  1. The science in this has evolved substantially such that BMSCT is a viable therapeutic option with reduced morbidity and mortality in the sibling matched population
  2. There is an opportunity to broaden current collaborations with other agencies and the BMSCT community to expand the accessibility of their research forward.
  3. Other agencies are emphasizing work in the area of BMSCT particularly for hemoglobinopathies.

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

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52 net votes
80 up votes
28 down votes
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Goal 3: Advance Translational Research

Increasing Regenerative Medical Strategies in Pulmonary Arterial Hypertension

Pulmonary arterial hypertension (PAH) is a complex, progressive condition characterized by high blood pressure in the lungs and restriction of flow through the pulmonary arterial system. Current PAH therapies mainly act of the vasoconstrictive component of the disease; however there is a widely accepted view that another contributor to the disease is an abnormal overgrowth of cells that line the pulmonary arteries, which... 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

In the past twenty years, 12 PAH targeted-therapies have been approved by the FDA. This increase in disease state awareness and in the treatment armamentarium have contributed to an increase in average survival from 2.8 years to an estimated 8-10 years. However, current treatments primarily address the vasoconstrictive component of the disease and do not address the now accepted theory of post-apoptotic overgrowth of hyperproliferative cells of the pulmonary vessels. A number of circulating stem and progenitor cells, derived from the bone marrow, have been identified that could have roles in repair of the pulmonary vascular system when interacting with the quickly, abnormally growing cells in the lung vessels. Work in this area has been named as a future research opportunity in the NHLBI-ORDR Strategic Plan for Lung Vascular Research (Erzurum S, et al. 2010).

Feasibility and challenges of addressing this CQ or CC

Basic and translational research support is needed—including high-throughput approaches such as phage display and large-scale proteomic analysis—to better understand the relationship between circulating bone marrow-derived cells, lung-resident stem and progenitor cells, and endothelial cells of the pulmonary arterial system.

Name of idea submitter and other team members who worked on this idea Pulmonary Hyeprtension Association, Michael Gray, Katie Kroner

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71 net votes
81 up votes
10 down votes
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Goal 3: Advance Translational Research

Financial and psycosocial impact of transition in sickle cell disease

What are the financial and social implications for individuals living with SCD from adolescence transitioning into adult care? What impact does this transition have on the caregiver and family as a whole?

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

Details on the impact of addressing this CQ or CC

It is very difficult for many living with SCD to maintain employment (many people have to rely on disability benefits to survive) finish school, even maintain relationships. Greater understanding of the financial and social impact would enable advocacy and direct patient service organizations to better prepare the transitioning population, reducing the financial strain on the individual and the growing medical debt incurred by hospitals and government social service programs.

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

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30 net votes
38 up votes
8 down votes
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Goal 3: Advance Translational Research

psychosocial care in sickle cell disease

What are the most effective trans-disciplinary and multi-level strategies for accelerating psychosocial care with sickle cell disease and how psychosocial factors impact families?

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

Details on the impact of addressing this CQ or CC

Since sickle cell has multiple layers in medical treatment strategies, how can the same thought process happen when it comes to psychosocial matters? How can the NHLBI develop effective patient engagement trans-disciplinary and multi-level strategies that work with medical strategies to deal with psychosocial matters for individuals and families?

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

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38 net votes
46 up votes
8 down votes
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Goal 3: Advance Translational Research

Implementation science research to reduce adverse effects of SCD

From various publications and reports, we have characterized the risks associated with sickle cell disease (SCD) and understand many of the barriers for treatment of SCD in LMICs. How can implementation science research be used to reduce the negative outcomes of SCD in low/middle income countries?

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

Details on the impact of addressing this CQ or CC

• Reduction of deaths and negative outcomes associated with SCD and in LMICs
• Provide the evidence base that supports culturally relevant implementation strategies that reduce deaths associated with SCD in LIMCs

Feasibility and challenges of addressing this CQ or CC

• Yes
, this is feasible
• Common goals and deliverables between NHLBI and partners will need to be identified
• Partnerships can be with international organizations, Ministries of Health and other partners

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

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18 net votes
31 up votes
13 down votes
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Goal 3: Advance Translational Research

Enhancing Cardiovascular Health in Childhood Through Adulthood

To enhance or maintain ideal cardiovascular health (CVH) in children and adolescents, what novel and long-term interventions can be implemented using multi-level (i.e., targeting individual, family, community, and built environment) and sustainable approaches?

Would implementation and translation of the AHA 2020 impact goals in children and adolescents enhance their CVH through adulthood?

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

Details on the impact of addressing this CQ or CC

Broad impact on the health of children and adolescents and ultimately, the health of the nation.

Feasibility and challenges of addressing this CQ or CC

Because there are some proven modalities in small-scale studies for improving cardiovascular health in children. Most are short-term or tested mostly in adults. This CQ focuses on trials that could span 10 years from Childhood into adulthood.
NHLBI has supported numerous large-scale trials (e.g., Girls health Enrichment Multisite Studies-GEMS, PATHWAYS, Child and Adolescent Trials for Cardiovascular Health-CATCH, Trial of Activity in Adolescent Girls-TAAG, and other investigated interventions in children and adolescents along with NICHD and other ICs) that could be harnessed to support this initiative.). NHLBI is supporting multi-level trials such as the Childhood Obesity Prevention and Treatment Research (COPTR) Consortium) that could provide modalities to enhance CVH in youth. Currently, there are no long-term trials spanning childhood through young adulthood in the US on this topic. An example of such a study is The Special Turku Coronary Risk Factor Intervention Project for Children [STRIP] study in Finland. Pahkala et al., Circulation. 2013;127:2088-2096.

A major challenge is cost, retention in trial and long-term adherence to intervention modalities. These could be mitigated using public-private funds (cost), incentives and/or clinical trial methodologies to enhance participation and adherence. Ability to motivate children and adolescents throughout their growth could be a challenge.

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

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17 net votes
34 up votes
17 down votes
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Goal 3: Advance Translational Research

Sickle cell disease and fatigue

What factors, other than hemoglobin count, are involved in the extreme fatigue experienced by individuals living with sickle cell disease?

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

Details on the impact of addressing this CQ or CC

Many in the sickle cell community report continuous fatigue that interrupts their daily activities in spite of normal, or above baseline, hemoglobin levels

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

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17 net votes
29 up votes
12 down votes
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Goal 3: Advance Translational Research

Ranking Impact of Dietary Changes on Reducing cardiovascular disease Risk

Of the components of DASH diet, which dietary changes have the greatest impact on reducing cardiovascular disease risk factors?

How can we rank the relative health effects of individual dietary changes such as reducing sodium, increasing fiber, reducing saturated/trans fats, etc. to identify where to target interventions for promoting initial dietary changes?

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

Details on the impact of addressing this CQ or CC

This will inform intervention, education, and outreach efforts.

Feasibility and challenges of addressing this CQ or CC

Similar studies have been conducted in that time frame.
Strong evidence shows that following a DASH-style dietary pattern can lower blood pressure and improve blood lipids, and thus reduce risk for cardiovascular disease. The DASH dietary pattern is high in vegetables, fruits, low-fat dairy products, whole grains, poultry, fish, and nuts and low in sweets, sugar-sweetened beverages, and red meats. In terms of nutrients, the DASH dietary pattern is low in saturated fat, total fat, and cholesterol and rich in potassium, magnesium, and calcium, as well as protein and fiber.

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

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11 net votes
21 up votes
10 down votes
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Goal 3: Advance Translational Research

Follow-up care for newborns diagnosed with sickle cell trait or disease

• There is a need to develop and support formal programs to provide follow-up care for newborns who test positive for the sickle cell trait or sickle cell disease upon screening. While newborn screening programs exist nationwide, healthcare providers report that often, screening is conducted only upon request (likely related to cost) and there is usually no follow-up afterwards. Interventions are also needed further... 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 International Association of Sickle Cell Nurses and Physician Assistants, Inc.

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14 net votes
17 up votes
3 down votes
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Goal 3: Advance Translational Research

Implementation Science to Improve Care in Sickle Cell Disease

There are approximately 100,000 individuals living with sickle cell disease in the US, however study after study has shown that many lack access to the few existing evidence based interventions such as hydroxyurea. We need to investigate novel ways to increase acess to hematology care and disease modifying therapies.

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

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12 net votes
14 up votes
2 down votes
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Goal 3: Advance Translational Research

Establish COPD Biomarkers

Specific biomarkers to monitor COPD disease activity are needed.

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

Details on the impact of addressing this CQ or CC

Much is understood about the pathogenesis of COPD at the cellular and biochemical level. There is no established way these insights can be used to test or implement new medicines. An explicit and economical set of procedures need to be established that will facilitate the development of new medicines and guide their clinical use.

Feasibility and challenges of addressing this CQ or CC

Many very small studies have identified biomarkers that relate to COPD activity in large groups of patients. There is no clear regulatory path for these insights to lead to tests that can be used with confidence in the development and implementation of novel treatments. Realistic regulatory pathways and criteria should be adopted to facilitate the development biomarkers of disease activity in COPD.

Name of idea submitter and other team members who worked on this idea COPD Foundation, COPD Biomarkers Qualification Consortium

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15 net votes
17 up votes
2 down votes
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Goal 3: Advance Translational Research

Safety and effectiveness of new direct oral anticoagulants

What is the optimal use of new direct oral anticoagulants?

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

Details on the impact of addressing this CQ or CC

Vitamin K antagonist, warfarin, has traditionally been the mainstay of anticoagulation therapy. It requires frequent monitoring to maintain safe and effective dose and is associated with many food and drug interactions. A new generation of direct oral anticoagulants has been developed to overcome such shortcomings.
Two main classes of new direct oral anticoagulants are available: factor Xa inhibitors and factor IIa (thrombin) inhibitors. Their mechanism of action involves direct inhibition of a single factor within the coagulation cascade to exert an anticoagulant effect. The industry is positioning them as monitoring-free universal warfarin replacement products. However, use of new direct oral anticoagulants introduces two major clinical issues: majority of new generation anticoagulants do not have developed dose-monitoring assay and do not have antidotes to rapidly restore blood coagulation properties in patients with trauma, emergent surgery, or anticoagulation overdose. Addressing these issues would positively impact cardiovascular, pulmonary, benign hematology, and orthopedic services worldwide.
While the idea of a universal, low-maintenance, “one dose fits all” anticoagulant is highly appealing to both patients and physicians, it may be feasible to consider more targeted approach, where each new anticoagulant would be assessed for most plausible effect in the specific patient population with consideration to genetic s, sex, race, age, thrombosis history, and obesity

Feasibility and challenges of addressing this CQ or CC

Rapid advancement in the field of new generation direct oral anticoagulants and multiplicity of new drugs introduce opportunity to conduct comparative effectiveness research and assess how different characteristics of new products may be appropriate for different patients. Increased use of new direct oral anticoagulants requires expedited development of assays and antidotes for safe and efficient therapy of millions of Americans.

 

A challenge is that the majority of the clinical trials for new direct oral anticoagulants were conducted by the industry with the main goal of demonstrating superiority, or non-inferiority to warfarin. Secondary analyses of these trials’ data for efficacy in specific patient population may be difficult. Prospective clinical studies in this area may require large sample size and establishing collaboration between hematologists and other involved clinicians.

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

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4 net votes
31 up votes
27 down votes
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Goal 3: Advance Translational Research

Improving Community-Based Care for Sickle Cell Disease

Sickle cell treatment centers are located throughout the United States, primarily in urban areas, and play an invaluable role. However, there is a critical need to identify and educate primary care providers who can provide routine and preventive care, but will also know when to consult with/refer to hematologists and other appropriate providers when necessary.

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

Details on the impact of addressing this CQ or CC

For the first time, comprehensive guidelines addressing the management of sickle cell disease were issued in 2014 by the NHLBI. (Previous guidelines were not comprehensive.) The 2014 guidelines, which address issues such as health maintenance and the treatment of acute and chronic complications, are based on a systematic review of available evidence; consensus of an expert panel; and published, vetted guidelines by other organizations when evidence was unavailable or insufficient. These guidelines can provide a solid overview of the knowledge essential for the care of people with sickle cell disease.

Identifying primary care providers who can provide routine and preventive care but at the same time are knowledgeable about sickle cell disease, should be a more efficient, less costly method of provide important health services to people with sickle cell disease and should ultimately improve the health and well being of this population, particularly for people who do not live near a sickle cell center.

Feasibility and challenges of addressing this CQ or CC

Addressing this question is very feasible. However, for a variety of reasons, including misconceptions about patients with the condition, it may be challenging to recruit large numbers of primary care providers.

Name of idea submitter and other team members who worked on this idea The Sickle Cell Association of New Jersey

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9 net votes
9 up votes
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Goal 3: Advance Translational Research

Advancing Translational Research

Ensuring that basic science is translated into clinical practice is essential. While there have been great strides in ensuring that babies born with congenital heart defects (CHD) are identified and repaired, we know that there are lifelong implications for those with CHDs that require continued follow-up and treatment. As the proportion of those with CHDs as adults continues to outpace the pediatric population, we urge... more »

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

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2 net votes
4 up votes
2 down votes
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