Showing 11 ideas for tag "pediatrics"

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

Transfusion strategies in pediatric and neonatal populations

What are the optimal strategies for transfusion of blood products in pediatric and neonatal population?

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

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Blood is the most prescribed drug in the PICU, and 95% of all neonates are transfused during their stay in the NICU. Currently, there are no evidence-based guidelines for the optimal hemoglobin levels or platelet counts for these populations. There is a balance that must be achieved between hemostasis and thrombosis for this vulnerable population.

Feasibility and challenges of addressing this CQ or CC

Clinical trials have begun to assess the optimal hemoglobin levels in neonates, but there are no trial to asses the optimal platelet count. Neonatologists, pediatric intensivists, and transfusion medicine physicians are beginning to come together to work on solutions to these problems.

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

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52 up votes
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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

It’s not just about adults…

Given the lack of outcomes data in the pediatric population, there is a need to develop a repository of data, including integrated electronic clinical data (outpatient, inpatient, laboratory, imaging, prescription), on all CVD risk factors (e.g. hypertension, dyslipidemia) in children. One approach is to start with leveraging health care systems that have existent electronic clinical data, infrastructure in place for... more »

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

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Fill a huge void of information on CVD risk and outcomes in the pediatric population. Provide essential information for prevention, determining events, tailoring interventions, facilitate targeted screening, and much more. Provide longitudinal information.

Feasibility and challenges of addressing this CQ or CC

We have the technology of EHRs and many are ripe for this pursuit.
This could provide a wealth of information. Imagine if we did this repository in health care systems where they can track a decent proportion of these kids into their adult years?

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

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32 up votes
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Goal 2: Reduce Human Disease

Pediatric heart failure

What is the best way to use what we have learned about the pediatric myocardium and cardiac-pulmonary interactions in congenital heart disease to develop new pathways for treating pediatric heart failure?

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

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Improved treatment for pediatric patients suffering from heart failure.

Feasibility and challenges of addressing this CQ or CC

n/a
Pediatric heart failure is almost always different from adult heart failure, due to varying mechanisms and underlying malformations.

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

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13 net votes
23 up votes
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Goal 1: Promote Human Health

Hypertension in children and adolescents - diagnosis and long term outcomes in large populations

Can we improve the thresholds for defining hypertension in children and adolescents based on risks for future adverse cardiovascular sequelae? In addition, can we to better understand how early identification of hypertension impacts long term health outcomes?

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

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Current thresholds for diagnosing hypertension in children and adolescents are based on the distribution of BP in general populations but are not linked to clinical outcomes. Most elevated BP in children and adolescents remains unrecognized, and there is little population level data on the benefits of early idnetification and treatment. There is a critical need for longitudinal data on BP with considerations that thresholds for diagnosing hypertension, evaluating for secondary causes and treating in this age group shoudl be linked to risks for long term cardiovasular sequelae. Important to consider is that labeling a child with mildly elevated BP as having hypertension could adversely impact family functioning and childhood health.

Feasibility and challenges of addressing this CQ or CC

This could be addressed through a population-based, observational cohort study, spanning childhood through young adulthood.

Name of idea submitter and other team members who worked on this idea Elyse Kharbanda

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16 up votes
4 down votes
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Goal 2: Reduce Human Disease

Inflammation and outcomes following pediatric cardiac operations

What is the contribution of the inflammatory response to postoperative recovery following pediatric cardiac operations and what strategies can improve outcomes?

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

Details on the impact of addressing this CQ or CC

Congenital heart disease is the most common cause of birth defects, with about 40,000 new cases born per year in the US. Affected individuals experience morbidity and mortality that generate health and economic consequences significantly out of proportion to their numbers. An estimated 10,000 of these patients will undergo cardiac surgery involving cardiopulmonary bypass (CPB). Furthermore, it is estimated that over 300,000 children in the US under age 21 have congenital cardiovascular disease and that 38% of these children will have had one or more surgical procedures. The use of CPB in neonates in particular has increased steadily over the past two decades. Further, neonates are generally sicker and consume more resources, including postoperative mechanical ventilation, ICU stay and hospital stay. Consequently, reducing the deleterious effects of CPB will have the largest impact in this group of patients.

Feasibility and challenges of addressing this CQ or CC

Research has begun to assess the inflammatory response to cardiopulmonary bypass in pediatrics. However, the magnitude and importance of its contribution to complicating postoperative recovery remains elusive. Clinical trials have begun to assess the efficacy of generalized anti-inflammatory therapies, typically steroids, with conflicting results. No therapy has been recognized as the standard of care. It’s critical that we improve our understanding of the molecular and cellular mechanisms of this inflammatory response and resulting derangements in vascular permeability and develop novel treatment strategies for infants and children undergoing cardiopulmonary bypass.

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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)

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• 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

Hypertension in the Pediatric Population

We also wish to draw attention to the rise in the prevalence of hypertension in the pediatric population, mostly as a consequence of the childhood obesity epidemic.

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

Details on the impact of addressing this CQ or CC

Additionally, hospitalizations related to pediatric hypertension have doubled over the past decade. These phenomena have clear and profound implications for the future cardiovascular health of the American population. The NHLBI has been instrumental in supporting studies in pediatric hypertension in the past, and we encourage a continued focus on pediatric populations for future hypertension research.

Name of idea submitter and other team members who worked on this idea American Society of Pediatric Nephrology (ASPN)

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