Effective treatment for Heart Failure with Preserved Ejection Fraction (HFpEF) currently does not exist. Lack of understanding of underlying mechanism(s) probably contributed to this lack of treatment. The well studied neural-hormonal blockade will not work for HFpEF because down stream kinase targets of adrenergic stimulation enhances myocardial relaxation. Consequently, sustain research outside current main stream thinking... more »

We need much more support for critical basic research to understand and develop transformative therapies for this enormous health care burden. This is not simply a question of epidemiology and large multicenter population data bases. We really need hard core science. It is impossible to know where the next breakthrough will come, and setting aside funds for hot button things - stem cells, or iPS, or gene editing per... more »

Three chelators are presently available in the US and much of the world: parenteral deferoxamine, and oral deferasirox, as well as oral deferiprone. Monotherapy is unsuccessful in a significant minority of patients, due to side effects or inadequate response at tolerable doses. Taking a page from enormously successful strategies for combination oral therapy in hypertension, led by NHLBI and others over the past four... more »

Thanks to elegant work since the 1990s, many details of the role of iron regulation and metabolism have been elucidated. Recent efforts in academic and pharmaceutical laboratories aim to translate these discoveries into therapies that may alleviate anemia in iron-refractory states (including anemia of inflammation and congenital disorders), and may have important therapeutic effects in non-transfusion-dependent thalassemia,... more »

While the primary pathophysiology of thalassemia is related to globin gene mutations and unbalanced globin chain expression, the downstream consequences are manifold. Chronic pain turns out to be one of the most important factors identified by patients with transfusion dependent thalassemia major in health-related quality of life surveys and patient-reported outcome measures. Even as therapies aimed and gene editing,... more »

There needs to be a rehab type maintenance study for COPD patients to determine if such a program would slow progression, improve health and reduce exacerbations/hospitalizations going forward. Thus, reducing costs to patient/hospital/public aid programs.

Baby boys and girls can respond quite differently to replacement lung surfactants given in response to neonatal respiratory distress syndrome. There are also variations in efficacy between babies of different races, in addition to the generally increased risk of NRDS in minority populations overall to lack of prenatal care or access to neonatal intensive care units. Increasing the availability of surfactant treatment... more »

We have a number of highly effective evidence-based interventions that have been shown to reduce ASCVD events - statins, BP drugs, aspirin, acute care. Yet large proportions of high risk population groups are not taking evidence-based treatment. Numerous interventions have been tried at multiple levels from the individual patient to the federal government payors. What are the best practices? How can we systematically... more »

The critical challenge is to develop a standards-based BMT electronic medical record (EMR) and integrate research capacity into the architecture of EMR systems. The ultimate goal would be to build de-identified complete data-sets which can be used to support observational studies and clinical trials, improve transplant outcomes and inform public policy.

We are focusing a lot on the genes that may be protective or harmful to our lives. But what about the regulation of genes in response to external stimulations, such as psychosocial and/or environmental, that are probably more accountable for whether we live healthier or not.

The DLD, NHLBI workshops conducted and published (Reference 1, 2, 3) had as their purpose to stimulate research that would identify still obscure or novel cellular components of the human lung to determine cell function in promoting respiratory tract development and in health that contributes to disease, so that better therapy might result. With robust technologies now available, especially genomic advances, how much... more »

Sepsis is the leading cause of death in critically ill patients in the USA, affecting particularly young children and the elderly. A hallmark of septic shock patients upon diagnosis is peripheral blood cytopenia. This persistent cytopenia commonly affects myeloid, lymphoid and erythroid lineages resulting in immunosuppression and is a well-established predictor of fatal outcome. Clinical trials targeting the production... more »

There should be a ramp up in research to address both treatments and management strategies for patient with CF.

Patients seen by doctors often relay a specific health issue and are treated for that particular complaint using a standard list of treatment regimens that do not consider comorbid conditions as a significant beneficial means to optimize positive outcomes. Other than allergy considerations, immediate cost often appears to drive treatments.

Women are more likely to have insomnia and they develop the condition at an earlier age than men. Hormone changes and menopause are two key factors but little is known about the mechanisms, which will inform the treatment. This is an understudied area of scientific focus with vast potential public health impact.