A programmatic initiative that would transform the NHLBI for rare diseases is to host some international studies.
What are the risk factors and components of clinical course associated with progression to restrictive lung disease, and what approaches to treatment can limit this progression?
Does screening for pulmonary hypertension (PH) of sickle cell disease (SCD) alter disease outcomes?
Does the treatment of gastroesophageal reflux disease improve outcomes in patients with IPF?
Does anticoagulation with warfarin improve outcomes (time to clinical worsening, qualtiy of life, exercise capacity) in patients with pulmonary arterial hypertension treated with current oral/inhaled therapies? There are substantial "unknowns" and practice variation in anticoagulation in PAH. Resource utilization is also a factor here. We may either be helping patients (or hurting them with side effects) by using anticoagulation. ...more »
What are the risk factors and treatment options for pulmonary hypertension related to diastolic dysfunction in Sickle Cell Disease (SCD)?
Does treatment with spironolactone improve outcomes in patients with pulmonary arterial hypertension (and/or pulmonary hypertension associated with diffuse parenchymal lung disease or COPD)? Spironolactone has been shown beneficial in CHF and many of the same mechanisms are at plan in RV failure from pulmonary hypertension. Again, no clear evidence whether this is a useful treatment or not, and no evidence to guide ...more »
What role does venous thromboembolic disease (VTE) play as a disease modulator in Sickle Cell Disease?
What is the effectiveness and safety of treatment of the co-morbid condition of asthma with medications known to improve asthma outcomes in individuals without SCD?
Complex diseases such as interstitial lung disease and pulmonary fibrosis requires a collaborative effort to effectively characterize, appropriately diagnose, and efficient evaluate novel therapies. Similarly, basic, translational and clinical research in this field requires the integration of clinical phenotypes with biologic specimens. We propose the expanded development of the Care Center Network and Patient Registry ...more »
Chronic Kidney Disease (CKD) affects millions in the US, and is one of the new diseases on the rise globally. New therapies to slow CKD and to repair and regenerate failing kidneys are drastically needed to reduce health care costs and improve lives.
We plan to find the role of APOL1 in increasing the risk of kidney failure in African Americans and translate this knowledge into preventing end-stage kidney disease and the need for kidney replacement therapy, dialysis or kidney transplantation.
Much is still unknown about the lymphatic system. No medical specialist owns it. NHLBI and NCI could work together on understanding lymphatic disease in the direction of understanding how metastasis spreads disease. NHLBI can help by establishing a unit to organize research interest in such an important body system in health and disease.
How do patients learn? What is the educational model to promote disease self‐management?
What are the hits/triggers that occur early in life that predispose to lung disease later in life?