The most promising areas are genotype-deep phenotype studies, advances in cardiac imaging to quantify extracellular volume as a measure of fibrosis; working with industry to develop better VADS/pre-transplant devices for small children, QOL studies in heart transplant children and their families, personalized medicine, pharmacogenomics, RCTs for new heart failure interventions and also cardiac rehabilitation, leverage and expand registries to support RCTs, molecular mechanism studies including studies of restrictive physiology, using genomic findings to directly inform clinical practice.

This is an area that needs to be studied, because if the macrolides provide the benefit suggested by many studies (about 20% mortality benefit), much morbidity and mortality would be prevented by universal macrolide use for CAP.  I am not aware of any studies being performed that would answer this question.

NTM lung disease is most common among older and female patients with estimated prevalences as high as 50-60 cases per 100,000 in this group and can be seen in this context as a women’s health issue.  While it may appear paradoxical, NTM lung disease studies would both profoundly change outcomes for a relatively rare condition overall, but would also benefit, in a potentially profound way, a large number of patients in a selected population (older female patients) in which the disease is not so rare.   To date there has been no significant funding for any aspect of NTM lung disease outside of limited support from the pharmaceutical industry.  The studies that have been done to date are largely single center/institution studies with relatively small numbers of patients.  Again, the few collaborative studies that have been done were accomplished with support from the pharmaceutical industry.  NTM lung diseases, and associated lung diseases such as bronchiectasis have not attracted the interest or support of major non-industry funding sources.

Essentially every aspect of Nontuberculous Mycobacterial (NTM) lung disease is associated with some level of controversy. The diagnostic guidelines were adopted primarily as a result of experience with common NTM pathogens such as Mycobacterium avium complex (MAC), M. kansasii , and M. abscessus. The relevance of the diagnostic guidelines for other potential NTM pathogens, especially rarely encountered organisms (in the U.S.) such as M. xenopi and organisms usually associated with specimen contamination such as M. fortuitum , is untested and unknown. The treatment of NTM lung disease is almost entirely based on case series, both retrospective and prospective, with consensus on basic treatment concepts. Treatment outcomes remain unsatisfactory for many pathogens including the most common, MAC, and prospective treatment trials are needed to establish not just optimal treatment regimens but the roles of agents in common use, such as clofazimine, the fluoroquinolones and inhaled amikacin, of unproven value.  Other basic studies including radiographic and pulmonary function evolution for treated and untreated disease and long term natural history of treated and untreated NTM lung disease patients are lacking.

Once such study is established, it can be used as a wellness model/control for many diseases and conditions. The longer we have the study, more useful it will become. This can help us to change or optimize our health care model, from treating diseases to maintain wellness.

This can be quite feasible as there are many frequent and long term donors. They care about others’ health as well as their own health. They are also very willing to support research. Follow-up can start from early adulthood.

With funding support this can be achieved.