Comparative Treatment Outcomes for Patients With Idiopathic Subglottic Stenosis.

To access publisher's full text version of this article, please click on the hyperlink in Additional Links field or click on the hyperlink at the top of the page marked DownloadImportance: 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.Patient-Centered Outcomes Research Institute - PCOR

The Indoor Environment and Otitis Media among Australian Children: A National Cross-Sectional Study

The association between the indoor environment and lifetime prevalence of otitis media (OM) in Australian children was assessed. We analysed data from a cross-sectional study of children, aged 7–11 years, performed in twelve Australian cities during 2007–2008. The main outcome was a parental report of their child’s diagnosis with OM by a doctor. Information on the indoor environment (energy sources used for heating, cooling, and cooking, pets, and second-hand smoke exposure), in the first year of life and at present, was collected from parents by a questionnaire. Multi-level logistic regression models were used to adjust for individual- and area-level confounders. Our analysis comprised 2872 children (51% female, mean age: 10.0 (SD 1.2)). Of those, 1097 (39%) were reported to have OM. Exposure to gas heating in the first year of life was significantly associated with higher odds of OM in adjusted models (OR:1.22; 95% CI: 1.00,1.47), as was current exposure to reverse-cycle air conditioning (OR: 1.52, 95% CI: 1.27,1.82). Ownership of a cat or dog at any time was also associated with high odds of OM (OR: 1.50; 95% CI: 1.17,1.92). No other significant associations were observed. In this national study of Australian children, indoor environmental exposures associated with the lifetime prevalence of OM were gas heating, reverse-cycle air conditioning and pet ownership. Exposures in both early life and later childhood may both play a role in OM

Atypical Pharyngeal Pouch Arising Bilaterally between the Hyoid Bone and Thyroid Cartilage

Introduction. Pharyngoesophageal diverticuli are a common cause of dysphagia; they are associated with various morbidities and a decreased quality of life. There are several different types of the diverticuli, and they are divided based on the anatomical location of origin relative to the cricopharyngeal muscle; these include Zenker’s, Killian-Jamieson’s, and Laimer’s diverticula. The authors present a unique case of pharyngoesophageal diverticulum that has not been previously described. Case Presentation. A 65-year-old male presented with a 12-month history of dysphagia and odynophagia for solids. Barium swallow revealed bilateral moderately sized diverticuli that altered in size during the different phases of swallow. CT scan of the neck with oral contrast further identified the anatomy of the diverticuli, arising between the hyoid bone and thyroid cartilage. Discussion. An external transcervical approach was utilised to successfully repair the diverticuli. Subsequent cricopharyngeal spasm was treated with botulinum toxin, and the patient recovered with no ongoing symptoms. The barium swallow study is a commonly utilised initial investigation as it is easy to perform and safe and has good diagnostic value. Definitive management usually involves either endoscopic or open surgery. This case depicts a unique case of a pharyngeal diverticulum arising between the hyoid bone and thyroid cartilage

Intensity-modulated radiotherapy using simultaneous-integrated boost for definitive treatment of locally advanced mucosal head and neck cancer: Outcomes from a single-institution series

Introduction: The study aims to report outcomes for patients treated using intensity-modulated radiotherapy (IMRT) with simultaneous-integrated boost and weekly cisplatin for American Joint Committee on Cancer stage III/IV mucosal head and neck squamous cell carcinomas (HNSCCs). Methods: Records for 67 patients treated definitively with IMRT for HNSCC were reviewed. By including only those treated with weekly cisplatin, 45 patients were eligible for analysis. Treatment outcomes, effect of patient, tumour and treatment characteristics on disease recurrence were analysed. Results: All patients completed IMRT to 7000 cGy in 35 fractions, with concurrent weekly cisplatin 40 mg/m2 (median 6 cycles). Median follow-up was 28 months for living patients. Two-year loco-regional recurrence-free, metastasis-free and overall survival were 85.4, 81.0 and 87.4%, respectively. Local recurrence occurred in three patients, and distant recurrence in eight patients. Conclusions: Our results show efficacy of IMRT and weekly cisplatin in the treatment of stage III/IV HNSCC at our institution with respect to loco-regional control

A patient-derived stem cell model of hereditary spastic paraplegia with SPAST mutations

SUMMARY Hereditary spastic paraplegia (HSP) leads to progressive gait disturbances with lower limb muscle weakness and spasticity. Mutations in SPAST are a major cause of adult-onset, autosomal-dominant HSP. Spastin, the protein encoded by SPAST, is a microtubule-severing protein that is enriched in the distal axon of corticospinal motor neurons, which degenerate in HSP patients. Animal and cell models have identified functions of spastin and mutated spastin but these models lack the gene dosage, mutation variability and genetic background that characterize patients with the disease. In this study, this genetic variability is encompassed by comparing neural progenitor cells derived from biopsies of the olfactory mucosa from healthy controls with similar cells from HSP patients with SPAST mutations, in order to identify cell functions altered in HSP. Patient-derived cells were similar to control-derived cells in proliferation and multiple metabolic functions but had major dysregulation of gene expression, with 57% of all mRNA transcripts affected, including many associated with microtubule dynamics. Compared to control cells, patient-derived cells had 50% spastin, 50% acetylated α-tubulin and 150% stathmin, a microtubule-destabilizing enzyme. Patient-derived cells were smaller than control cells. They had altered intracellular distributions of peroxisomes and mitochondria and they had slower moving peroxisomes. These results suggest that patient-derived cells might compensate for reduced spastin, but their increased stathmin expression reduced stabilized microtubules and altered organelle trafficking. Sub-nanomolar concentrations of the microtubule-binding drugs, paclitaxel and vinblastine, increased acetylated α-tubulin levels in patient cells to control levels, indicating the utility of this cell model for screening other candidate compounds for drug therapies

Additional file 1: Table S1. of Systemic inflammation is an independent predictive marker of clinical outcomes in mucosal squamous cell carcinoma of the head and neck in oropharyngeal and non-oropharyngeal patients

Patient demographic differences between p16 tested and non-tested oropharyngeal patients. (XLSX 11 kb