Showing 12 ideas for tag "children"

Goal 2: Reduce Human Disease

How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging

Clinical research, especially randomized pharmaceutical clinical trials, poses many unique challenges compared to research in adult subjects. In pulmonary arterial hypertension, a disease characterized by high blood pressure of the lungs with increased pulmonary vascular resistance leading to right ventricular failure, there are 12 FDA-approved PAH-targeted therapies for adults. None of these medications are currently... 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

Pulmonary arterial hypertension is a heterogeneous condition generally characterized by high blood pressure in the lungs and increased pulmonary vascular resistance that leads to right heart failure if left untreated. Though some causes of PAH are seen in both adult and pediatric populations, some etiologies are seen exclusively in pediatric populations, including persistent pulmonary hypertension of the newborn, bronchopulmonary dysplasia, lung hypoplasia, and alveolar capillary dysplasia. Despite these differences in disease etiology, and known physiologic differences in pediatric populations, inhaled nitric oxide (iNO) in the acute setting is the only approved medication for PAH treatment in children. A number of issues have decreased pediatric PAH pharmaceutical research, including protection of the pediatric population as vulnerable subjects, principle of scientific necessity, balance of risk and potential benefit, parental consent/child assent, and feasibility of pediatric clinical trial design and implementation. Encouraging clinical trials of existing adult medications and potentially emerging, novel agents specifically for pediatrics—either through direct sponsorship or regulatory incentives—would not only lead to better outcomes for pediatric PAH patients, but potentially to a better and more comprehensive characterization of the developing pulmonary vascular system and right ventricle.

Feasibility and challenges of addressing this CQ or CC

Several challenges exist for addressing this critical challenge. First, there are a number of differences between conducting clinical research in pediatric populations compared to adult populations. This not only includes the broad items referenced above, but items as noted by Rose and colleagues related to clinical trial design and analysis including (1) accepted age-matched normal ranges for laboratory values; (2) requirements for the validation of clinical endpoints for the assessment of efficacy and safety; and (3) standards for long-term safety monitoring and pharmacovigilance (Rose K, et al. NEJM 2005). Sponsorship of this type of clinical research is a second concern, which could either be mitigated by direct support from the National Institutes of Health of pediatric PAH clinical trials or in regulatory changes incentivizing pediatric clinical research in rare diseases.

Name of idea submitter and other team members who worked on this idea Katherine Kroner, Michael Patrick Gray, PHA

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Goal 2: Reduce Human Disease

Understanding the Genetic & Epigenetic Basis of Congenital Heart Disease?

Over the last thirty years, our fundamental understanding of the genetics and pathogenesis of congenital heart disease has lagged the tremendous advances in the surgical and clinical care of infants with this group of disorders. We need to close this gap with investigation into the genetic basis of congenital heart malformations to develop new models of disease. The goall is translate an improved molecular genetic and... 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

Congenital heart disease (CHD) is the most common congenital malformation and the most common cause of mortality during the first year of life. Approximately 70% of cases occur sporadically without a strong family history or identifiable genetic syndrome, and the primary heritable basis of most non-syndromic CHD has yet to be identified. Studies of affected kindreds, syndromic disease, and more recently genome wide association studies (GWAS) have shed light on a handful of causal loci, while exome sequencing and studies of structural variation uncovering rare de novo variants in trios have yielded only an 8-10% rate of diagnosis in cohorts with CHD. Despite the application of contemporary techniques and study design to genetic discovery in CHD, the majority of the genetic risk for human cardiac malformations remains unexplained.

Feasibility and challenges of addressing this CQ or CC

One key challenge is that many of the stakeholders including those affected with congenital heart disease (children), along with the physicians make a diagnosis and referral (obstetricians, neonatologists, general pediatricians), are generally funded by other agencies (NICHD). Trans-agency collaboration and cooperation is necessary to improve the translational research structures necessary to improve disease.

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Goal 3: Advance Translational Research

Can We Successfully Reduce the Risk Factor Burden and Atherosclerosis in Younger Individuals?

There is growing evidence that risk factors beginning in childhood and young adult life, e.g. from such studies as CARDIA, Bogalusa, etc., are primary determinants of risk of CHD in adults.

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 levels of BP, lipoproteins, ApoB, obviously obesity, cigarette smoking, and growing risk of early age type 2 diabetes, are epidemic in the young United States population. This may result in a continued high burden of CHD in future generations in the United States. Unless something is done about controlling risk factors in younger individuals, a continued epidemic of CHD is likely.

Feasibility and challenges of addressing this CQ or CC

The NHLBI should begin clinical trials to determine the best approaches to reducing risk factors in the younger United States population. This should include various studies of environmental modifications, better nutrition studies, improved exercise programs, and identification of host genetic susceptibility. To date, the programs have been remarkably unsuccessful and different approaches are required, especially in lower income and minority populations. The NHLBI should also begin to consider the evaluation of drug therapies for elevated lipids and BP, new drug therapies for obesity and even bariatric surgery in high risk young individuals with initial evaluation of the reduction of subclinical atherosclerotic disease, evidence of myocardial injury, e.g. elevated hs-cTnT, NT-proBNP, etc., in these high risk populations. There is little that the current approaches are very effective.

Name of idea submitter and other team members who worked on this idea Lewis H. Kuller, MD, DrPH

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46 up votes
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Goal 2: Reduce Human Disease

Understand the Impact of Thrombosis in Children with Cancer

CC: Despite the potential impact that venous thrombotic events (VTE) have on children with cancer, several unresolved issues remain. To date, we are yet to understand:
- incidence/prevalence of VTE according to cancer type/staging
- ideal imaging modalities to diagnose/follow VTE
- thromboprophylaxis according to thrombosis risk stratification (development of VTE predictors)
- efficacy/safety to anticoagulate children... 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

Venous thrombotic events (VTE) are now occurring in 1/200 children admitted to a tertiary pediatric facility. In around 70-90% of cases, VTE occurs in children with an underlying condition, amongst which cancer represents up to 1/3 of patients. Within this group of patients, the thrombotic complications are associated with a higher morbidity (e.g. higher recurrence rates, high rate of CNS events in acute leukemia) and mortality. Nevertheless, the clinical challenges highlighted in the itemized Critical Challenge Section illustrate the lack of basic science, translational and clinical research available, as well as the paucity of evidence-based medicine recommendations necessary to acoount for the increasing number of patients with this complication.
On the other hand, pediatric oncology is one of the areas of pediatric care where the medical progresses of the last decades have drastically changed the natural history of cancer in children. In light of much higher survival rates for almost all types of pediatric cancer, the focus has now shifted towards decreasing treatment-related, as well as disease-related morbidities, increasing the quality of life of the many survivors. Because VTE is now recognized as one of the significant remaining complications within this patient population, addressing the list summarized herein would contribute to further improve the care of children with cancer.

Feasibility and challenges of addressing this CQ or CC

The infrastructure that is already in place under the Children's Oncology Group (COG), where almost any new clinical and/or translational idea related to the care of children with cancer becomes part of a clinical trial, could be rolled over to explore many of the items listed under the CC Section.
As a principle, VTE in children with cancer develop due to: a) host-related factors; b) chemotherapy/treatment-related factors; and c) disease-related issues. Therefore, protocol- and disease-specific studies could address, under the auspices of COG, the prevalence of VTE according to cancer type in a prospective manner. Similarly, high risk groups for VTE could be submitted to standardized imaging and/or biomarker investigation prospectivelly, in addition to collection of outcome data related to VTE and to anticoagulation protocols. Furthermore, tumor specimens/genetic markers could be evaluated and correlated to the study outcomes. The challenges of reaching consensus during protocol development would allow identification of equipoise for certain clinical scenarios, obviating the need of trials, or the use of consensus techniques, before diagnostic/therapeutic protocols could be adopted.
In conclusion, the develoment of a multidisciplinary task force (i.e. pediatric radiologists, oncologists, hematologists, molecular biology experts), which, for the most part, is already in place (i.e. COG), would be instrumental to foster research on this extremely clinically relevant area.

Name of idea submitter and other team members who worked on this idea Leonardo R. Brandao, MD, MSc;

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38 up 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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34 up votes
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Goal 2: Reduce Human Disease

Optimal hemoglobin threshold for transfusion in children with ARDS?

Do different hemoglobin transfusion thresholds alter outcomes in children with ARDS? What is the optimal *minimum* transfusion threshold for children with ARDS? What patient-centered outcomes can be affected by transfusion strategies: ventilator free days, time to organ function recovery, duration of intensive care stay, survival?

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 Society of Critical Care Medicine Executive Committee/Council

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8 net votes
9 up votes
1 down votes
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Goal 2: Reduce Human Disease

Asthma Disparities in Low income children

What are the most effective methods to reduce asthma disparities in low income children?

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 advances in care, asthma inequities are persistent among low income children. What are the most effective measures to close the gap?

Feasibility and challenges of addressing this CQ or CC

There are multiple strategies but what are the most effective treatment options in low income children who have higher morbidity and mortality?

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4 net votes
9 up votes
5 down votes
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Goal 1: Promote Human Health

THE RELEVANCE OF PREVENTION TRIALS

Prevention trials, implemented to reduce or delay progression to overt disease in a population at risk to the disease, are an important approach to health promotion.
Therapies shown to reduce disease severity in patients with a specific disease are obvious, but not the only, candidates for a prevention trial in populations at high risk for prevalent diseases (such as heart failure, diabetes, COPD, asthma in children).... 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

The impact of implementing such trials is considerable. They will clearly address an important component of NHLBI’s mission with respect to effectiveness of therapies and behavioral interventions, and it has minimal and clearly definable overlap with commercial trials of specific therapeutic products. It will also provide an important public health focus – preventing disease or reducing the impact of disease processes, thus potentially reducing chronic care costs and increasing years of useful life.

Feasibility and challenges of addressing this CQ or CC

The biggest challenge in designing and implementing prevention trials is identifying the target, “at risk” population most likely to develop the clinical disease from known biomarkers or early signs/symptoms. Increasing availability of large, population-based registries or databases maintained for other purposes provides a very cost-efficient mechanism to electronically screen and identify “at risk” individuals. The same mechanism may also facilitate implementation of pragmatic, electronically managed, cost efficient trials.

Name of idea submitter and other team members who worked on this idea Sonja McKinlay other Team Members: Susan Assmann and Paul Stark

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Goal 3: Advance Translational Research

Screening for SDB and Sleep Disorders in School-Aged Chidren by School Nurses

Can school nurses effectively screen for SDB and Sleep Disorders in school aged children? Who else in the school setting could provide such screening? Should such screening be limited to "at risk" children who display identified markers, or be open to all children? What is the role of teachers to "identify" children in need of such screening?

What role will such screening serve to mitigate learning, behavioral, developmental... 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 impact of screening at risk children for sleep disorders and sleep disordered breathing, including subsequent referrals and professional treatment, may serve to mitigate future health, learning, developmental and behavioral risks/issues for children by addressing these issues in early childhood. Research based protocols will be accessed and used for screening.

Feasibility and challenges of addressing this CQ or CC

Additional considerations:
Does the nurse refer an at risk child to the Pediatrician? Dentist? ENT?
What questionnaires or other identifiers would be used for screening? Is there a bio test to assess risk for a SD or SDB?
What should the target age level be for children undergoing proposed screening?

How can sleep education and training be integrated with this screening process to promote good sleeping habits/hygiene at home, for all children, parents and caretakers, as well as teachers. Who creates and provides the educational material? Who does the teaching?

Parental involvement- KEY!

Name of idea submitter and other team members who worked on this idea Nancy Rothstein

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5 net votes
6 up votes
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Goal 3: Advance Translational Research

Clinical Tools for Pediatric CVD Risk Reduction and Asthma Treat

What are effective strategies and clinical decision support tools that can maximize pediatric care providers’ adoption of evidence-based recommendations for assessment and treatment of cardiovascular risk factors and/or asthma?

• Clinical recommendations and associated implementation tools are often incorporated into electronic medical records (EMRs). Currently there is no standard EMR format and therefore it is difficult... 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

• Cardiovascular disease (CVD) remains the leading cause of death and disability in North America. There is extensive evidence documenting the initiation of the atherosclerotic process, the pathologic basis for clinical heart disease, in childhood. Additionally, asthma a chronic condition that affects more than 7 million children in the U.S. and leading to numerous emergency visits.
• Among the major factors that are associated with increased clinical recommendation use are ease of access and feasibility. A common obstacle that providers face is the availability of proper information at the point of care.
• The Community Preventive Services Task Force recommends clinical decision-support systems for prevention of cardiovascular disease based on sufficient evidence of effectiveness in improving screening for CVD risk factors and practices for CVD-related preventive care services, clinical tests, and treatments. Mobile solutions may help to further facilitate this process.
• Successful implementation of clinical recommendations for prevention and treatment of CVD pediatric risk factors and asthma could greatly reduce the number of youth moving into adulthood at increased risk for CVD and could improve health outcomes for children with asthma.

Feasibility and challenges of addressing this CQ or CC

• Most care providers have mobile devices or computers for use in the clinical setting. There is good evidence that clinical decision support tools (and other implementation tools) can help facilitate adoption of clinical recommendation.
• It is important to test strategies in a large scale intervention that will measure clinical CVD outcomes.

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

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Goal 2: Reduce Human Disease

Life-Course Approach to Science

We encourage the NHLBI to include research focused on children as a priority in their strategic vision. We encourage them to consider the implications of a life-course approach, where childhood presents a unique opportunity to set the trajectory for health risk as an adult. This also implies that interventions targeting children may have the greatest impact on the population as a whole in the long term.

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 American Society of Pediatric Nephrology (ASPN)

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Goal 2: Reduce Human Disease

Effect of obesity on recovery of lung function in pediatric survivors of critical illness

What are the determinants of persistent respiratory failure in children? Are obese children at greater risk for prolonged mechanical ventilation than non-obese children? Does BMI affect the time to recovery of lung function in obese children with ARDS? What is the pathogenesis and molecular contributors of obesity on respiratory failure in critical illness?

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 Society of Critical Care Medicine Executive Committee/Council

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1 net vote
3 up votes
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