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.

Treatment of glottal papillomatosis and dysplasia was mirror-guided and performed in surgeons' offices in the 19th century. It migrated to the operating room in the 20th century to accommodate direct laryngoscopic surgery, which required assistants to administer anesthesia and procedural support. Presently, the primary treatment goals, which are disease regression and voice restoration or maintenance, are tempered by the morbidity of general anesthesia and potential treatment-induced vocal deterioration. In fact, general anesthesia has been appropriately considered to be an acceptable source of morbidity for the promise of a precise procedure, which usually ensures airway safety and an optimal vocal outcome. However, patients with recurrent glottal papillomatosis and keratosis with dysplasia are typically monitored with various degrees of watchful waiting until there is a subjective judgment (on the part of the patient and surgeon) that the disease is more of a liability than is the procedure to treat it. Innovations in the 585-nm pulsed dye laser delivery system have allowed for its use in the clinic with local anesthesia through the working channel of a flexible fiberoptic laryngoscope. A prospective assessment was done on 51 patients in 82 cases of recurrent glottal papillomatosis (30) and dysplasia (52). All individuals had previously undergone microlaryngoscopic management with histopathologic evaluation. Five procedures could not be completed because of impaired exposure (2) or discomfort (3). Of those patients who could be treated, there was at least a 50% disease involution in 68 of 77 cases (88%) and 25% to 50% disease regression in the remaining 9 (12%). Patient self-assessment of the voice revealed that 34 of 77 were improved, 39 were unchanged, 4 were slightly worse, and none were substantially worse. These data confirm that diseased mucosa can be normalized without resection or substantial loss of vocal function. The putative mechanisms, which vary according to the fluence (energy) delivered by the laser, are photoangiolysis of sublesional microcirculation. denaturing of epithelial basement membrane linking proteins, and cellular destruction. Furthermore, this relatively safe, effective technique allowed for treatment of many patients (in a clinic setting) in whom classic surgery-related morbidity would have often delayed intervention.

The caregivers of 61 eligible children (6 months to 12 years old) completed a 20-item (OSA-20) health-related quality-of-life survey after polysomnography was performed to psychometrically validate the OSA-20. Excellent test-retest reliability was obtained for the individual survey items (R>0.74). Construct validity was shown by significant correlation of the mean survey score with the respiratory distress index (R = 0.43) and adenoid size (R = 0.43). Two items with poor validity were dropped, reducing the survey to 18 items (OSA-18). The relationship between the OSA-18 summary score and respiratory distress index remained significant when adjusted for tonsil size, adenoid size, body mass index, and child age. On the basis of the total survey score, the impact of OSAS on quality of life was small for 20 children (33%), moderate for 19 (31%), and large for 22 (36%). The OSA-18 is a practical means of office-based determination of quality-of-life impact for obstructive sleep apnea syndrome in children.

Phonosurgical management of early glottic cancer has evolved considerably, but objective vocal outcome data are sparse. A prospective clinical trial was done on 32 patients with unilateral cancer (T1a in 28 and T2a in 4) who underwent ultranarrow-margin resection; 15 had resection superficial to the vocal ligament, and 17 deep to it. The subepithelial infusion technique facilitated selection of these patients for the appropriate procedure. All are cancer-free without radiotherapy or open surgery. Involvement of the anterior commissure (22/32) or the vocal process (15/32) of the arytenoid cartilage did not influence local control. Nine of 17 patients had resection of paraglottic musculature, and all underwent medialization reconstruction by lipoinjection and/or Gore-Tex laryngoplasty. Eight of the 17 had resections deep to the vocal ligament, but without vocalis muscle, and 1 of the 8 underwent medialization. Posttreatment vocal function measures were obtained for all patients. A clear majority of the patients displayed normal values for average fundamental frequency (72%) during connected speech, and normal noise-to-harmonics ratio (75%) and average glottal airflow (91%) measures during sustained vowels. Smaller majorities of patients displayed normal values for average sound pressure level (SPL; 59%) during connected speech and for maximum ranges for fundamental frequency (56%) and SPL (59%). Fewer than half of the patients displayed normal values for sustained vowel measures of jitter (45%), shimmer (22%), and maximum phonation time (34%). Almost all patients had elevated subglottal pressures and reduced values for the ratio of SPL to subglottal pressure (vocal efficiency). There were significant improvements in a majority of patients for most vocal function measures after medialization reconstruction. Normal or near-normal conversation-level voices were achieved in most cases, regardless of the disease depth, by utilization of a spectrum of resection and reconstruction options. These favorable results are based on establishing aerodynamic glottal competency and preserving the layered microstructure of noncancerous glottal tissue.

4. Laryngoscope, 128:610-617, 2018.