Treatment Alternatives in iSGS (NoAAC PR-02 Study)
- Conditions
- Idiopathic Subglottic Stenosis (iSGS)
- Interventions
- Procedure: Endoscopic dilation of subglottic stenosisProcedure: Endoscopic resection of the stenosisProcedure: Tracheal Resection
- Registration Number
- NCT02481817
- Lead Sponsor
- Vanderbilt University Medical Center
- Brief Summary
The study is aimed at answering the questions; (1) How well do the most commonly used treatments in iSGS work? and (2) What quality-of-life trade-offs are associated with each approach?
With the results of this trial, the investigators hope to provide information to iSGS patients: (1) Given my personal characteristics, conditions, and preferences, what should I expect will happen to me? and (2) What are my options, and what are the potential benefits and harms of these options?
- Detailed Description
Idiopathic subglottic stenosis (iSGS) is a rare disease in which the trachea narrows for no known reason. Although uncommon (with an estimated incidence of 1:400,000 persons per year), both the disease and its therapies profoundly affect patients' ability to breathe, communicate and swallow. Breathing difficulties (i.e. dyspnea) is the hallmark symptom and the primary cause of death and disability. However, patients can also experience debilitating voice changes and swallowing problems due to the condition or its treatment.
People with this disease often require several surgeries per year. A variety of treatments have been advanced to manage iSGS but are generally categorized into: 1) endoscopic dilation of the tracheal stenosis (accomplished with rigid instruments or inflatable balloons); 2) endoscopic resection of the stenosis (with prolonged medical therapy after surgery); or 3) open neck surgery with resection of the affected tracheal segment with end-to-end anastomosis. Each patient can require repeated surgeries to keep their trachea open, which increases odds of treatment side effects and complications. All approaches have unique and often disabling associated side effects, which can significantly affect a patient's quality of life.
Because the disease is rare, it is difficult for patients to find good information so that they can understand the spectrum of treatment options. This is particularly difficult because most patients present with severe breathing trouble and need treatment quickly, limiting their ability to explore options. Additionally, there is a general lack of high-quality, reliable, and accessible data to inform individual patient decision-making. Imperfect information and limited evidence on treatment outcomes complicate patient decision-making as they try to balance survival, symptoms, and quality of life considerations.
Beyond the gaps in understanding the relative effectiveness of the different treatments available, no studies have explored functional outcomes in iSGS (i.e. how well patients breathe, speak, and swallow after treatments). These endpoints are important to patients and are arguably a primary determinant in decision-making. Direct engagement with patients is critical to understand these quality of life considerations, since patient and physician perspectives aren't always the same. For example, results show that endoscopic dilation is associated with a higher rate of disease recurrence and thus need for repeated surgery. Meanwhile, open tracheal resection is a major surgery with significant immediate perioperative risks and has been associated with alterations in voice and swallowing. Open tracheal resection appears to reduce the risk of disease recurrence, but the degree of benefit, and the trade-offs associated with this approach are unanswered questions.
The proposed study from the North American Airway Collaborative (NoAAC PR- 02) is designed to fill this void. Our prospective study will directly compare the effectiveness of standard of care treatments and assess their associated quality of life tradeoffs in iSGS patients.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 1239
- Greater than 18 years of age.
- The lesion must involve the subglottis.
- Less than 18 years of age
- Patients without capacity to consent for themselves
- History of significant laryngotracheal traumatic injury.
- History of endotracheal intubation or tracheotomy within 2 years of presentation.
- Major anterior neck surgery.
- History of neck irradiation.
- History of caustic or thermal injuries to the laryngotracheal complex.
- History of a clinically diagnosed vasculitis or collage vascular disease.
- Positive antinuclear cytoplasmic antibody titers.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description iSGS patients Endoscopic dilation of subglottic stenosis Participants will receive standard of care treatment at the respective center and will be followed longitudinally for symptom changes, need for further treatment, complications, and will have Patient-reported outcomes (PROs) administered at a priori determined intervals. iSGS patients Tracheal Resection Participants will receive standard of care treatment at the respective center and will be followed longitudinally for symptom changes, need for further treatment, complications, and will have Patient-reported outcomes (PROs) administered at a priori determined intervals. iSGS patients Endoscopic resection of the stenosis Participants will receive standard of care treatment at the respective center and will be followed longitudinally for symptom changes, need for further treatment, complications, and will have Patient-reported outcomes (PROs) administered at a priori determined intervals.
- Primary Outcome Measures
Name Time Method Treatment Effectiveness: Time to recurrent procedure 3 years Treatment Effectiveness: Need for tracheostomy 3 years
- Secondary Outcome Measures
Name Time Method QOL 3 years Patient quality of life assessment: general quality of life (SF-12)
Patient Reported Outcome 3 years Non-traditional PRO focused on participatory decision-making style
Trial Locations
- Locations (41)
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
Harvard Medical School -Massachusetts Eye and Ear Infirmary
🇺🇸Boston, Massachusetts, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of Miami
🇺🇸Miami, Florida, United States
Augusta University
🇺🇸Augusta, Georgia, United States
University of Southern California
🇺🇸Los Angeles, California, United States
Bastian Voice Institute
🇺🇸Downers Grove, Illinois, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
University of Wisconsin
🇺🇸Madison, Wisconsin, United States
Stanford University
🇺🇸Palo Alto, California, United States
University of California Irvine
🇺🇸Irvine, California, United States
New York University Medical Center
🇺🇸New York, New York, United States
University of California San Diego
🇺🇸San Diego, California, United States
University of Rochester
🇺🇸Rochester, New York, United States
Loma Linda University Health Care
🇺🇸Loma Linda, California, United States
Emory University School of Medicine
🇺🇸Atlanta, Georgia, United States
University of California Los Angeles
🇺🇸Los Angeles, California, United States
Johns Hopkins
🇺🇸Baltimore, Maryland, United States
University of Washington
🇺🇸Seattle, Washington, United States
Louisiana State University
🇺🇸Baton Rouge, Louisiana, United States
University of Cincinatti
🇺🇸Cincinnati, Ohio, United States
Temple University
🇺🇸Philadelphia, Pennsylvania, United States
Charing Cross Hospital, Imperial College London
🇬🇧London, United Kingdom
University of Texas Southwestern
🇺🇸Dallas, Texas, United States
University of Sydney
🇦🇺St Leonards, Australia
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
University of Virginia
🇺🇸Charlottesville, Virginia, United States
Landspitali University Hospital
🇮🇸Reykjavik, Iceland
The University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
University of California San Francisco
🇺🇸San Francisco, California, United States
University of Colorado
🇺🇸Denver, Colorado, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
University of Nebraska
🇺🇸Omaha, Nebraska, United States
University of North Carolina
🇺🇸Chapel Hill, North Carolina, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Duke University
🇺🇸Durham, North Carolina, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States