If NHLBI set research priorities for pediatric cardiomyopathy and heart failure, it would help investigators better align their application with institute goals.
During recent years, clinical research including well-organized randomized clinical trials in developed countries generated large database and human biological sample banks. These are valuable resources for human disease research. Mechanisms to encourage and facilitate international collaboration for genetic and metabolic research using database and human biological samples from specific human disease population of international... more »
Many pharma companies are developing inhaled antibiotics for patients with bronchiectasis, recognizing the increasing market (over 110,000 patients in the US). These drugs cost thousands of dollars per year and will be heavily marketed and heavily used, as there are limited options for these patients. It is unknown if rotating oral antibiotics (cost is a fraction of the inhaled ABx) would yield similar results. A Cochrane... more »
1) Phase III efficacy trials of new tuberculosis drugs (e.g., bedaquiline, delamanid, PA-824) that have shown promise in early phase studies for multidrug-resistant tuberculosis. 2) Phase III efficacy trials of new and existing tuberculosis drugs to development very short course regimens (3-4 months). 3) Phase III efficacy trials of new and existing drugs for treatment of latent tuberculosis infection in contacts of... more »
Community-acquired pneumonia (CAP) is responsible for over 1 million hospital admissions and about 100,000 deaths per year. We still do not know the best antibiotic regimen to treat CAP. Retrospective studies and cohort studies support giving macrolides, while randomized controlled studies (essentially all done by pharma) have not shown benefit of macrolides. Guidelines allow either a macrolide or a quinolone.
The true prevalence of Nontuberculous mycobacterial (NTM) lung infections remains incompletely understood, however several aspects of NTM lung disease prevalence are becoming more clear. NTM lung disease is currently more common in the U.S. than TB (by a factor of 3) and has consistently been shown to be increasing in prevalence. When viewed in the context of likely universal environmental NTM exposure this increasing... more »
1. Monotherapy with a quinolone vs combination therapy with a 3rd generation cephalosporin. The issue of the best antibiotic treatment for severe CAP has been a major area of contention now for a decade and it is the most common cause of infectious death in the United States. 2. Combination therapy vs monotherapy for pneumonia due to Pseudomonas. This is another major area of contention – for nearly 2 decades, and generates... more »
Blood donors (especially young donors) in general represent healthy populations. Longitudinal follow-up of frequent and long term blood donors can be useful to establish data and sample sources for the study of wellness, instead of disease (especially for blood diseases). Not only it can be used as healthy controls, it can also be used to predict the wellness factors such as genetic variation, life style, exercise patterns,... more »
How can we insure that there are sufficient numbers of clinical scientists over the next 20 years?
Would an NIH trans-IC office of critical care research improve coordination and strategic planning across?
Can novel therapeutics including cell-based therapy be tested in patients with severe acute lung injury (P/F <200) and shock (need for vasopressors) since these are the patients with the highest mortality (> 30%) based on NHLBI ARDS Network data?
Can novel extracorporeal devices that remove carbon dioxide be tested to limit or avoid positive pressure ventilation in patients with acute respiratory failure from COPD?
Will adaptive trial design improve clinical research for acute lung injury?
Does the addition of albumin to fluid conservative management of ALI (ARDSnet FACTT trial protocol, Wiedemann et al) further shorten ventilator time and/or improve survival?
Critical care medicine comprises a diffuse array of diseases, syndromes, illnesses and symptoms arising from those sources requiring advanced care by highly trained teams of interdisciplinary professionals. Research is sorely needed on generating evidence that is broadly applicable to a heterogeneous group of patients. This is a major challenge for researchers who enroll critically ill patients into their clinical trials.... more »