Third Proceedings of The North American Airway Collaborative (NoAAC): Consensus Statement on Trial Design for Airway Stenosis.

Abstract

Importance: Airway stenosis is a rare but debilitating disorder that significantly degrades the quality of life in affected patients. Treatments are primarily surgical, and disease management lacks established medical therapies. The North American Airway Collaborative held its third symposium at The Johns Hopkins Hospital in Baltimore, Maryland, on April 15, 2024, focused on strategies to advance the care of these patients. The proceedings summarize the discussion of trial design in airway stenosis and the resulting North American Airway Collaborative consensus regarding clinical end points for rigorous study of novel therapies.

Observations: The lectures and panels centered on the translation of a growing body of preclinical data into therapeutic targets. Additionally, detailed discussion explored design of clinical trials to evaluate safety and efficacy of novel therapeutics. The need for a consensus regarding clinically meaningful end points in airway stenosis was identified to facilitate the comparison of outcomes across institutions and future multi-institutional trials.

Conclusions and relevance: The group achieved consensus regarding change in peak expiratory flow as the primary clinical end point in airway stenosis. Additional clinical measures, such as disease recurrence (identified as time to recurrent intervention), anatomical characterization of subglottic scar via axial computed tomography imaging, and patient-reported outcome measures (Clinical COPD Questionnaire [CCQ], Voice Handicap Index-10 [VHI-10], Eating Assessment Tool-10 [EAT-10], and 12-Item Short-Form Health Survey, version 2 [SF-12]) were identified as essential secondary outcomes.