A survey of patients with laryngotracheal stenosis on future clinical trial design

Abstract

Background/aims: Laryngotracheal stenosis is a rare but devastating proximal airway fibrosis that restricts a patient's ability to breathe. Treatment is primarily surgical and to date, there has never been a multi-institutional, randomized, prospective, and interventional clinical trial for a medical therapy to treat laryngotracheal stenosis. Therefore, we aimed to obtain patient feedback to guide successful trial design, recruitment, retention, and for identifying potential barriers to study participation.

Methods: Over 1000 members of an international laryngotracheal stenosis online support community (the Living with Idiopathic Subglottic Stenosis Facebook group) were sent two questionnaires for a proposed interventional double-blinded, randomized, placebo-controlled clinical trial.

Results: A total of 317 and 558 participants responded to the first and second surveys, respectively. The majority of participants (77%) were willing to consider enrollment, regardless of having a 50% chance of receiving placebo versus treatment (78%). The majority (84%) of participants were willing to travel 200 miles to participate for up to six in-person visits over 50 days. Specific side effects, including anemia/thrombocytopenia (72%) or risk of infection (69.3%) had the greatest impact on clinical trial participation with other side effects (peripheral edema (53%), oral ulcers (51%), and gastrointestinal side effects (41%)) having less impact.

Conclusion: Patients with laryngotracheal stenosis possess nuanced insight into their disease and treatment options. As a group, they are extremely motivated for better therapies. Future laryngotracheal stenosis clinical trials should focus on providing excellent side effect -related education and utilizing feedback from online advocacy groups to optimize recruitment and retention.

Keywords: Laryngotracheal stenosis; clinical trial; cross-sectional survey; patient-guided trial design; placebo-controlled study; recruitment.

Figure 1.

Geographical distribution of survey participants. (a) The number of participants in the United States was listed per state. (b) Internationally, the number of participants was listed by continent. Darker blue color hue indicates a higher density of participants.

Figure 2.

Willingness to participate in an interventional clinical trial for laryngotracheal stenosis. Of survey respondents, 77.2% were either very likely (green) or likely (blue) to participate in a theoretical clinical trial for laryngotracheal stenosis. Unlikely, participation (yellow) was noted in 18.7% and 4.1% of respondents stated that they were very unlikely (red) to participate.

Figure 3.

Barriers to trial participation. Likelihood of participation was assessed based on willingness to travel up to 200 miles for six total visits over a 50-day period and willingness to pay out-of-pocket travel costs (survey 1). When listed together as a whole, side effects overall were likely or very likely to deter up to 43% of survey participants from trial participation.

Figure 4.

Use of a placebo and individual side effects. (a) Percentage of survey participants who were likely (54%) or very likely (24%) to participate in a randomized placebo-controlled study. (b) Impact of side effects individually on trial participation. Dotted line represents 50% point between participants who would or would not participate.