Showing 3 ideas for tag "adaptive"

Goal 2: Reduce Human Disease

Triggers of cellular and molecular pathway decompensation during pulmonary exacerbations.

What triggers decompensation of cellular and molecular pathways during exacerbations of chronic lung diseases?

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

Details on the impact of addressing this CQ or CC

Elucidation of molecular and cellular pathways driving and sustaining exacerbations in chronic lung diseases. Specifically, (1) discover what perturbs antecedent conditions precipitating mal(adaptive) compensatory mechanisms leading to pulmonary exacerbation including impact of heterogeneous resilience and concomitant chronic diseases and (2) clarify response heterogeneity of longitudinal molecular and cellular signature during treatment and recovery.

Feasibility and challenges of addressing this CQ or CC

Longitudinal pulmonary exacerbation research nested with clinical care can be initiated within 1-2 years. As part of clinical care leveraging digital education/data (electronic health/medical records and attendant meaningful use requirements), an N-of-one research design could become self-sustaining within health care systems.
Pulmonary exacerbations exhibit multiple pathogenic pathways various concurrent pathophysiological pathways, and diverse clinical manifestations. Prevention and treatment of pulmonary exacerbations is hampered by this complex biology that is dynamic and appears to vary during the course of exacerbations. Progress toward precision medicine for exacerbations may require organization of a new taxonomy for disease, which reflects a set of clinically meaningful and exploitable similarities and differences between disease traits (exacerbation polypathomics).

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Goal 2: Reduce Human Disease

Improving patient-centered outcome assessments in HLBS studies

What types of newer patient-centered quality of life assessment tools can be employed in heart, lung, blood and sleep studies so that they can be validated and refined to improve our measurement of quality of life outcomes in populations of interest to NHLBI?

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

Details on the impact of addressing this CQ or CC

Improving our ability to precisely measure heart, lung, blood, and sleep patients' quality of life can enable evaluation of a treatment's impact on patient-centered outcomes such as overall quality of life and its components -- pain, symptoms, and a patients' social, psychological, and physical functioning.

Feasibility and challenges of addressing this CQ or CC

New tools have been developed, notably through the PROMIS common fund project, that allow potentially more precise, reliable, valid and sensitive measurement of Q of L outcomes, with less patient burden. These tools are available but require validation in HLBS populations to allow widespread adoption and routine use in NHLBI-supported clinical trials and population studies.
Advances in biomedical science mean we are living longer with chronic diseases, and the goal of treatment increasingly focuses on disease management, maximizing function, and improving quality of life, not just lengthening life. In addition, patient-centered approaches to health care encourage a view of patients as “whole persons” with emphasis on function and capturing the "patient's voice," not just mortality/morbidity outcomes. Functional and quality of life outcomes, e.g., assessment of pain, symptoms, emotional distress, physical & social functioning, are critically important outcomes to many HLBS patients, but their measurement requires self-reports of patient experiences and thus pose challenges to precise, valid and reliable assessment.

Assessment tools using computerized adaptive testing (CAT), such as those developed in the Patient-Reported Outcomes Measurement Information System (PROMIS) project, have been shown to be precise, valid, sensitive to change and easier to administer than traditional Q of L measures in a limited number of studies, but they require validation in HLBS patient populations before they can be used more widely in NHLBI-funded studies.

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

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