JAMA otolaryngology-- head & neck surgery最新文献

Importance: Retrograde cricopharyngeus dysfunction (R-CPD) is an emerging disorder associated with disabling symptoms. The origin of R-CPD remains unknown.

Objective: To investigate the development of symptoms, diagnosis approach, and therapeutic outcomes of R-CPD in patients treated with in-office botulinum toxin injection (BTI) into the cricopharyngeus.

Design, setting, and participants: This was a case series including patients with R-CPD who were consecutively and prospectively recruited from April 2022 to May 2024 in an academic hospital. Semistructured interviews were conducted to collect and analyze data on each patient's clinical history, potential causes or factors associated with R-CPD development, diagnostic approaches, and symptom presentation.

Intervention: Clinic-based (in-office) BTI into the cricopharyngeus.

Outcomes and measures: Associations with laryngopharyngeal reflux disease, patients' Reflux Symptom Score-12 (RSS-12), and BTI effectiveness, revisions, and complications were evaluated.

Results: The case series comprised 106 patients with R-CPD treated with BTI (52 females [49.1%] and 54 males [51.9%]). Their mean (SD) age at symptom onset was 13.6 (7.7) years, and at diagnosis, 30.4 (6.4) years. Sixty-eight patients (64.2%) had potential congenital R-CPD, according to the parents' testimonies. A family history was reported in 18 of 62 cases (29.0%). In 105 cases (99.1%), patients made the diagnosis themselves despite medical consultations (n = 162), empirical treatments (n = 113), and additional examinations (n = 92). The cumulative success rate of BTI was 90.6% (96 of 106 patients). In 26 cases (24.5%), additional injections were administered to address the symptoms. Family history of R-CPD was a negative predictor of single-BTI success. Dysphagia was the primary adverse effect occurring after 89 of 126 BTIs (70.6%) and lasted a mean (SD) of 16.3 (12.0) days. In 10 cases, operating-room BTI was administered after primary in-office BTI.

Conclusions and relevance: R-CPD is an emerging and poorly known disorder associated with high rates of ineffective consultations, additional examinations, and self-diagnosis by patients. In-office BTI was associated with a high rate of partial or total symptom relief and long-term effectiveness.

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.

Importance: The increasing use of glucagon-like peptide-1 receptor agonists (GLP-1RA) demands a better understanding of their association with thyroid cancer.

Objective: To estimate the risk of incident thyroid cancer among adults with type 2 diabetes being treated with GLP-1RA vs other common glucose-lowering medications.

Design, setting, and participants: This was a prespecified secondary analysis of a target trial emulation of a comparative effectiveness study using claims data for enrollees in commercial, Medicare Advantage, and Medicare fee-for-service plans across the US. Eligible participants were adults with type 2 diabetes at moderate risk for cardiovascular disease and without history of thyroid cancer who had newly filled prescriptions for GLP-1RA, sodium-glucose cotransporter 2 inhibitor (SGLT2i), dipeptidyl peptidase-4 inhibitor (DPP4i), or sulfonylurea from January 1, 2014, to December 31, 2021. Data were analyzed February 1 to October 31, 2024.

Main outcomes and measures: Overall and piecewise (<1, 1-2, and ≥2 years since treatment initiation) hazard ratios (HRs) for thyroid cancer with use of GLP-1RA vs the other 3 drug classes were estimated using inverse propensity score weighted Cox proportional hazards models. Modified intention-to-treat (mITT) (primary) and as-treated (sensitivity) analyses were performed.

Results: Of 351 913 patients (mean [SD] age, 65.3 [8.5] years; 173 391 [49.3%] females and 178 522 [50.7%] males), 41 112 started treatment with GLP-1RA; 76 093, with DPP4i; 43 499, with SGLT2i; and 191 209, with sulfonylurea therapy. The numbers of patients diagnosed with thyroid cancer were 69 (0.17%) in the GLP-1RA group, 172 (0.23%) in the DPP4i group, 72 (0.17%) in the SGLT2i group, and 381 (0.20%) in the sulfonylurea group. In the mITT analysis, GLP-1RA initiation was not significantly associated with increased overall risk for thyroid cancer compared to the other 3 diabetes drugs (HR, 1.24; 95% CI, 0.88-1.76). However, the risk for thyroid cancer was significantly higher within the first year after GLP-1RA initiation (HR, 1.85; 95% CI, 1.11-3.08) and was amplified in the overall as-treated analysis that censored patients when therapy was discontinued or another medication was added (HR, 2.07; 95% CI, 1.10-3.95).

Conclusions and relevance: This secondary analysis of a target trial emulation of a comparative effectiveness study found that despite the low absolute risk of thyroid cancer among patients receiving GLP-1RA therapy, there was an increased risk of new thyroid cancer diagnoses within the first year of GLP-1RA initiation compared to 3 other diabetes drugs. This finding may have been due to enhanced early detection; therefore, further research is necessary to understand the underlying causes of this association.