Two studies in the New England Journal of Medicine confirm that pneumothorax is a rare serious and poorly-defined syndrome. 1A strong genetic predisposition and complex medical history combined with limited oxygen supply to the brain make patients at increased risk for this condition. 1A 2014 study by David Lees of the University of Bristol in the UK and colleagues confirmed the genetic association with 15 years of observation. 2The lung injuries of yaws-an inherited neurovascular disorder with a risk for recurrent respiratory tract infections and respiratory failure-are a common denominator the authors say. 3Recent advances in sequencing technology which allows researchers to study or study the same organism at different times of life offer the promise of potential knowledge gained from investigating these diseases. The 2 in a Canadian encephalopathia study published in the New England Journal of Medicine are evidence for the potential value of this emerging field.
The study involved 472 patients who admitted to 10 hospitals with yaws a rare neurological disorder affecting only 7-10 of children and young adults in the U. K. across 2 specialties. The study documented 19 inflammatory pneumonia cases (64 pneumothorax). Yaws disease is an inherited neurological condition with symptoms that usually affect children between 5 10 years old. While these patients often recover to full mobility some have not survived five years.
The researchers said that the recent Rapid Challenges-World Class (R-D-W)-level trials which have been accelerating development of new precision medicine for yaws and lymphoma over the last 15 years could be a valuable tool to health care professionals searching for ways to prevent further more chronic complications amongst patients with yaws. Rather than being divided into subgroups based on differences in incidence the R-D-W trials constitute an objective tool for generating novel risk-stratified risk adjuvants using standard care the authors said.
Importantly the R-D-W trials are being used to evaluate the activity of a class of breast and prostate cancer drug development inhibitors known as Dabrafenib and Aspen which have shown benefit in efficacy and safety.
Organizers of the R-D-W trials considering inclusion or exclusion should be aware of risk variants targeting the babies and young adults recruited and evaluated in the trials they said. Leaders and clinical leadership of such therapies should evaluate clinical outcomes using the R-D-W trials.