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Grant spurs study of rare breathing condition in women

May. 7, 2015, 10:37 AM

A research team at Vanderbilt University Medical Center has been approved for a $2.7 million funding award by the Patient-Centered Outcomes Research Institute (PCORI) to study idiopathic subglottic stenosis (iSGS), a rare condition that inexplicably causes middle-aged women to struggle to breathe.

Alexander Gelbard, M.D., and David Francis, M.D., both assistant professors of Otolaryngology, are leading the effort. Vanderbilt will be the coordinating center for 30 academic medical centers studying the disease, said Ron Eavey, M.D., Guy M. Maness Professor and chair of Otolaryngology and director of the Vanderbilt Bill Wilkerson Center.

Alexander Gelbard, M.D.

“The grant success speaks loudly about the reputation of Vanderbilt and the world-acknowledged Voice Center,” Eavey said. “We are proud our young faculty members who have worked so diligently to demonstrate that the new funding mechanism has value and that these investigators take seriously our role as the coordinating center.”

Idiopathic subglottic stenosis is characterized by an unexplained and often recurrent narrowing of the upper trachea and affects almost exclusively middle-aged women. The disease is chronic, progressive, disabling and potentially fatal. It limits everyday activities, such as walking or climbing stairs, and makes it difficult to communicate or even swallow. Patients often end up with a social or emotional disability, leading to isolation as they struggle to maintain normal lives.

David Francis, M.D.

People with iSGS often require several surgeries each year just to be able to breathe.

Each of the three most common surgical approaches have unique and often disabling side effects, but there is no information available now that compares the options.

The study will evaluate the effectiveness of the three approaches and how each impacts a patient’s quality of life.

“The study is designed to understand what treatments work, what type of patients they work best in and what are the ultimate trade-offs in decisions they make between these different therapies,” Gelbard said.

Added Francis, “Not much is known about why people get this condition. It tends to affect women in their middle age who are otherwise healthy.”

Gelbard said the award would not have been possible without Vanderbilt’s infrastructure, including the Center for Health Services Research and the Center for Quantitative Sciences.

The study is one of 46 proposals that PCORI approved for funding on April 21 to advance the field of comparative clinical effectiveness research (CER).

The award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract to Vanderbilt.

“This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to fill an important gap in our health knowledge and give people information to help them weigh the effectiveness of their care options,” said PCORI Executive Director Joe Selby, M.D., MPH. “We look forward to following the study’s progress and working with Vanderbilt to share the results.”

Vanderbilt’s study and the other projects approved for PCORI funding were selected through a highly competitive review process in which patients, clinicians and other stakeholders joined clinical scientists to evaluate the proposals.

Applications were assessed for scientific merit, how well they will engage patients and other stakeholders, and their methodological rigor among other criteria.

PCORI is an independent, non-profit organization authorized by Congress in 2010 to fund comparative clinical effectiveness research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed health and health care decisions. PCORI is committed to seeking input from a broad range of stakeholders to guide its work.

PCORI has approved $854 million to support 399 research studies and initiatives since it began funding research in 2012. Go here for more information about PCORI funding.

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