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Comparative Study
. 2020 Jan 1;146(1):20-29.
doi: 10.1001/jamaoto.2019.3022.

Comparative Treatment Outcomes for Patients With Idiopathic Subglottic Stenosis

Alexander Gelbard  1 , Catherine Anderson  1 , Lynne D Berry  2 , Milan R Amin  3 , Michael S Benninger  4 , Joel H Blumin  5 , Jonathan M Bock  5 , Paul C Bryson  4 , Paul F Castellanos  6 , Sheau-Chiann Chen  2 , Matthew S Clary  7 , Seth M Cohen  8 , Brianna K Crawley  9 , Seth H Dailey  10 , James J Daniero  11 , Alessandro de Alarcon  12 , Donald T Donovan  13 , Eric S Edell  14 , Dale C Ekbom  15 , Sara Fernandes-Taylor  10 , Daniel S Fink  7 , Ramon A Franco  16 , C Gaelyn Garrett  1 , Elizabeth A Guardiani  17 , Alexander T Hillel  18 , Henry T Hoffman  19 , Norman D Hogikyan  20 , Rebecca J Howell  12 , Li-Ching Huang  2 , Lena K Hussain  2 , Michael M Johns 3rd  21 , Jan L Kasperbauer  15 , Sid M Khosla  12 , Cheryl Kinnard  1 , Robbi A Kupfer  20 , Alexander J Langerman  1 , Robert J Lentz  22 , Robert R Lorenz  5 , David G Lott  23 , Anne S Lowery  1 , Samir S Makani  24 , Fabien Maldonado  22 , Kyle Mannion  1 , Laura Matrka  25 , Andrew J McWhorter  26 , Albert L Merati  27 , Matthew C Mori  28 , James L Netterville  1 , Karla O'Dell  21 , Julina Ongkasuwan  13 , Gregory N Postma  29 , Lindsay S Reder  21 , Sarah L Rohde  1 , Brent E Richardson  30 , Otis B Rickman  22 , Clark A Rosen  31 , Michael J Rutter  12 , Guri S Sandhu  32 , Joshua S Schindler  33 , G Todd Schneider  34 , Rupali N Shah  35 , Andrew G Sikora  13 , Robert J Sinard  1 , Marshall E Smith  36 , Libby J Smith  37 , Ahmed M S Soliman  38 , Sigríður Sveinsdóttir  39 , Douglas J Van Daele  19 , David Veivers  40 , Sunil P Verma  41 , Paul M Weinberger  42 , Philip A Weissbrod  43 , Christopher T Wootten  1 , Yu Shyr  2 , David O Francis  4
Affiliations
Comparative Study

Comparative Treatment Outcomes for Patients With Idiopathic Subglottic Stenosis

Alexander Gelbard et al. JAMA Otolaryngol Head Neck Surg. .

Abstract

Importance: Surgical treatment comparisons in rare diseases are difficult secondary to the geographic distribution of patients. Fortunately, emerging technologies offer promise to reduce these barriers for research.

Objective: To prospectively compare the outcomes of the 3 most common surgical approaches for idiopathic subglottic stenosis (iSGS), a rare airway disease.

Design, setting, and participants: In this international, prospective, 3-year multicenter cohort study, 810 patients with untreated, newly diagnosed, or previously treated iSGS were enrolled after undergoing a surgical procedure (endoscopic dilation [ED], endoscopic resection with adjuvant medical therapy [ERMT], or cricotracheal resection [CTR]). Patients were recruited from clinician practices in the North American Airway Collaborative and an online iSGS community on Facebook.

Main outcomes and measures: The primary end point was days from initial surgical procedure to recurrent surgical procedure. Secondary end points included quality of life using the Clinical COPD (chronic obstructive pulmonary disease) Questionnaire (CCQ), Voice Handicap Index-10 (VHI-10), Eating Assessment Test-10 (EAT-10), the 12-Item Short-Form Version 2 (SF-12v2), and postoperative complications.

Results: Of 810 patients in this cohort, 798 (98.5%) were female and 787 (97.2%) were white, with a median age of 50 years (interquartile range, 43-58 years). Index surgical procedures were ED (n = 603; 74.4%), ERMT (n = 121; 14.9%), and CTR (n = 86; 10.6%). Overall, 185 patients (22.8%) had a recurrent surgical procedure during the 3-year study, but recurrence differed by modality (CTR, 1 patient [1.2%]; ERMT, 15 [12.4%]; and ED, 169 [28.0%]). Weighted, propensity score-matched, Cox proportional hazards regression models showed ED was inferior to ERMT (hazard ratio [HR], 3.16; 95% CI, 1.8-5.5). Among successfully treated patients without recurrence, those treated with CTR had the best CCQ (0.75 points) and SF-12v2 (54 points) scores and worst VHI-10 score (13 points) 360 days after enrollment as well as the greatest perioperative risk.

Conclusions and relevance: In this cohort study of 810 patients with iSGS, endoscopic dilation, the most popular surgical approach for iSGS, was associated with a higher recurrence rate compared with other procedures. Cricotracheal resection offered the most durable results but showed the greatest perioperative risk and the worst long-term voice outcomes. Endoscopic resection with medical therapy was associated with better disease control compared with ED and had minimal association with vocal function. These results may be used to inform individual patient treatment decision-making.

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Conflict of interest statement

Conflict of Interest Disclosures: Dr Gelbard reported receiving grants from the Patient-Centered Outcomes Research Institute (PCORI) and grants from the National Institutes of Health–National Heart, Lung, and Blood Institute during the conduct of the study. Dr Berry reported receiving grants from PCORI during the conduct of the study. Dr Bock reported receiving other support from Diversatek Healthcare outside the submitted work. Dr Cohen reported receiving other support from ZSquare and other support from Syneos outside the submitted work. Dr Daniero reported having a patent to the University of Virginia pending. Dr Garrett reported receiving grants from PCORI during the conduct of the study. Dr Hillel reported receiving personal fees from Olympus USA and from Ambu outside the submitted work. Dr Hoffman reported receiving other support from Iotamotion and other support from UpToDate outside the submitted work. Dr Hogikyan reported receiving grants from PCORI during the conduct of the study. Dr Hussain reported receiving grants from PCORI during the conduct of the study. Dr Johns III reported receiving royalties from Plural Publishing for 2 books about laryngology and receiving royalties from Medbridge for an online instructional course in laryngeal videostroboscopy. Dr Kasperbauer reported receiving grants from PCORI during the conduct of the study. Dr Kupfer reported receiving grants from PCORI during the conduct of the study. Dr Langerman reported receiving other support from ExplORer Surgical outside the submitted work. Dr Ongkasuwan reported receiving grants from PCORI during the conduct of the study. Dr Rickman reported receiving grants from the National Institutes of Health and PCORI during the conduct of the study. Dr Rutter reported having a suprastomal stent marketed with his name and declined associated royalties and having a patent on an airway balloon dilator and receiving licensing fees/royalties. Dr Sikora reported receiving nonfinancial support from Advaxis, receiving grants from Tessa Therapeutics, and receiving other support from Ovodex LLC outside the submitted work. Dr Weinberger reported nonfinancial and other support from SphereoFill LLC outside the submitted work. No other disclosures were reported.

Figures

Figure 1.
Figure 1.. Flowchart and Absolute Number of Participants Recruited
Figure 2.
Figure 2.. Kaplan-Meier Analysis of Disease Recurrence Among the 3 Treatment Groups
Figure 3.
Figure 3.. Longitudinal Mean Peak Expiratory Flow Rate Among Patients Without Recurrence in the 3 Treatment Arms
Loess smooth curve of mixed-effects model shows sustained peak expiratory flow rate (measured in liters per second during a single expiratory cycle and reported as percentage of matched normative data) among patients after successful treatment. Self-reported patient longitudinal peak expiratory flow rate was captured using an inexpensive portable handheld device and a free smartphone app created specifically for this study.
Figure 4.
Figure 4.. Secondary End Points of Patient-Reported Functional Outcome at 12 Months
COPD indicates chronic obstructive pulmonary disease; ERMT, endoscopic resection with adjuvant medical therapy.
See this image and copyright information in PMC

References

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