Association and risk factors of pediatric pulmonary hypertension with obstructive sleep apnea: A national study utilizing the Kids' Inpatient Database (KID).

STUDY OBJECTIVE: Assess the prevalence of and risk factors for pediatric pulmonary hypertension (PH) in the 2016 Kids' Inpatient Database (KID), including obstructive sleep apnea (OSA) and obesity. METHODS: Retrospective cross-sectional cohort study utilizing 6,081,132 weighted pediatric discharges from the 2016 KID. Study variables included age, length of stay, mortality, gender, hospital region, primary payer, race, median household income for patient's ZIP code, OSA, central sleep apnea (CSA), obesity, Down syndrome, sickle cell disease (SCD), thalassemia, congenital heart disease (CHD), hypertension, asthma and chronic lung disease of prematurity (CLDP). PH was the primary outcome of interest. Bivariate and multivariable logistic regression models were utilized with odds ratios and 95 % confidence intervals. RESULTS: The mean age was 3.76 years, the mean hospital length of stay was 3.85 days, 48.9 % were male, 52.6 % had government health insurance, 51.0 % were White, 16.1 % were Black, 21.1 % were Hispanic, 5.0 % were Asian or Pacific Islander, 0.80 % were Native American and 6.1 % identified as "other". The prevalence of PH was 0.21 % (12,777 patients). There were 37,631 patients with OSA and the prevalence of PH among this cohort was 3.3 %, over 10x greater than the overall prevalence of PH in the 2016 KID (0.21 %). Risk factors associated with PH included CLDP, CHD, Down syndrome, asthma, OSA, CSA, hypertension, SCD, obesity, race/ethnicity, government insurance, age, male gender (p < 0.0001), and hospital region (p = 0.0002). CONCLUSIONS: Several risk factors were independently associated with PH, such as OSA, CSA, obesity, asthma, and insurance status. Prospective multi-institutional studies are needed to assess the relationships between these risk factors, severity metrics, and causative links in the development of PH; in addition to identifying children with OSA who are most likely to benefit from cardiopulmonary screening prior to adenotonsillectomy. LEVEL OF EVIDENCE: Level III.

Admission practices following pediatric tonsillectomy: A survey of ASPO members.

OBJECTIVES: Although evidence-based Clinical Practice Guidelines (CPGs) have specified postoperative admission criteria for pediatric tonsillectomy, there is substantial variation in guideline implementation and adherence among otolaryngologists in practice. We aimed to assess pediatric otolaryngologists' post-tonsillectomy admission practices and to examine patient and surgeon factors associated with differences in admission practices. METHODS: An electronic cross-sectional survey was distributed to members of the American Society of Pediatric Otolaryngology (ASPO) to determine current practices regarding admission practices following pediatric tonsillectomy. Chi-square and Fisher's exact tests were performed to compare differences in adherence to tonsillectomy CPGs by respondent characteristics. RESULTS: The survey was sent to 644 pediatric otolaryngologists with a response rate of 19.1%. 37% of respondents reported "always" and 60% "often" using the Academy of Otolaryngology-Head and Neck Surgery (AAO-HNS) CPG to guide decision for admission. Years in practice was the factor most strongly associated with admission practices, with 10 or fewer years in practice significantly correlated with stricter adherence to the AAO-HNS CPG of overnight observation when Apnea-Hypopnea Index (AHI) ≥10, age <3 years, or O2 nadir <80%) (OR 4.2, p <0.001), as well as specific individual criteria such as an AHI ≥10 (OR 4.1, p = 0.03). Respondents in an academic practice setting were more likely to admit children <3 years of age than those in private practice (OR 5.0, p = 0.01). CONCLUSION: Admission practices varied among pediatric otolaryngologist survey respondents, and strict AAO-HNS CPG adherence was associated with fewer years in practice and academic practice setting. These results suggest that further study investigating factors influencing guideline adherence and post-tonsillectomy admission practices is warranted.

Impact of a postgraduate year one (PGY-1) otolaryngology bootcamp on procedural skill development.

Background: To measure the impact of an intensive eight-week postgraduate year one (PGY-1) otolaryngology bootcamp on the acquisition and retention of otolaryngology residents' procedural skills compared to the traditional method of skill acquisition through clinical exposure. Methods: Residents at our institution were evaluated on their performance of flexible laryngoscopy, suture ligature, and rigid bronchoscopy setup at three time points: pre-bootcamp, one-week post-bootcamp, and one-year post-bootcamp. Video recordings were scored by two blinded faculty reviewers using a multipoint rating system. A control group of rising postgraduate year two (PGY-2) residents who did not participate in bootcamp were recorded performing these same skills. Scores in the three skills were compared between groups via t-tests. The eight-week bootcamp curriculum for PGY-1s was held at the Montefiore Einstein Center for Innovation in Simulation at Albert Einstein College of Medicine/Montefiore Medical Center. The participants were two classes of PGY-1 residents (n=8) at our institution who participated in a bootcamp at the beginning of residency, and one class of rising PGY-2 residents (n=3) who did not participate in a bootcamp (control group). Results: A comparison of pre-bootcamp scores to one-week post-bootcamp scores showed significant improvement in suture ligature ( P<0.05) and rigid bronchoscopy ( P<0.05), but no difference in flexible laryngoscopy ( P=0.54). Suture ligature ( P=0.09) and rigid bronchoscopy ( P=0.25) skills were not significantly different from one-week post-bootcamp to one-year post-bootcamp; however, a significant skill improvement was observed in flexible laryngoscopy ( P<0.05). By June of PGY1 year, the two bootcamp cohorts were similar to controls in all three skills: flexible laryngoscopy ( P=0.05), rigid bronchoscopy ( P=0.26), and suture ligature ( P=0.10). Conclusions: Participation in PGY-1 bootcamp was associated with improved acquisition and short-term retention of basic procedural skills, suggesting that bootcamps can be an effective arena to teach basic skills in otolaryngology. PGY-1 bootcamp is a promising arena for multi-institutional development.

Efficacy and Safety of Electromyography-Guided Injection of Botulinum Toxin to Treat Retrograde Cricopharyngeus Dysfunction.

OBJECTIVE: To report the efficacy and safety of electromyography-guided percutaneous botulinum toxin injection into the cricopharyngeus muscle in an office setting for treatment of the inability to belch and associated symptoms caused by retrograde cricopharyngeus dysfunction (R-CPD). STUDY DESIGN: Retrospective case series of treated patients. SETTING: Tertiary care laryngology clinic. METHODS: A retrospective review was performed on 18 consecutive patients who were diagnosed syndromically with R-CPD. The combined diagnostic test and treatment-specifically, botulinum toxin injection into the cricopharyngeus muscle-was accomplished in an office setting by a single surgeon using electromyography guidance. Items assessed are efficacy, safety, complications, and duration of benefit. RESULTS: All 18 patients (100%) treated in the in-office setting gained the ability to burp with improvement in the associated symptoms of R-CPD at initial follow-up. Of those who had the in-office procedure performed initially, 80% maintained the ability to burp at 6 months with relief of all the associated symptoms of R-CPD. No patients experienced permanent complications from the injection, but 7 patients experienced varying degrees of noisy breathing within 1 week after the procedure, which was managed with breathing techniques and resolved. CONCLUSION: In a case series of 18 patients with R-CPD, all patients gained the ability to burp with improvement in the majority of their symptoms of R-CPD at the time of their initial follow-up at 1 week. None experienced severe complications, and 7 experienced transient noisy breathing, which resolved.

Topical Capsaicin for the Treatment of Sensory Neuropathic Cough.

OBJECTIVE: To evaluate a novel treatment for sensory neuropathic cough (SNC): topical capsaicin. STUDY DESIGN: Retrospective review. SETTING: Tertiary care laryngology clinic. METHODS: A retrospective review was performed on 201 consecutive patients treated for SNC with capsaicin 0.02% to 0.04% applied topically to the upper aerodigestive tract, typically after failure of standard medications. Patients were asked to use the spray 4 times daily for 2 weeks prior to assessment of benefit. Items assessed included the percentage reduction of coughing, type of benefit noted, and side effects. RESULTS: Of the 201 patients who used the spray, 36.3% noted no benefit, whereas 63.7% (n = 128) had benefit in terms of cough reduction: 30.8% (n = 62) reported ≥75% reduction; 17.4% (n = 35), 50%-74% reduction; 7.0% (n = 14), 25%-49% reduction; and 8.5% (n = 17), 1%-24% reduction. Of all patients, 78.3% reported no side effects or complications. Of the remaining 21.7%, 1 patient noted a nosebleed after a single administration, and 1 patient noted transient wheezing after administration. The others reported unpleasant local effects, including throat/ear discomfort, voice change, sneezing, reflexive vomiting, and headache. CONCLUSION: In our group of 201 patients with SNC, most of which had failed to respond to standard treatments, 63.7% had some response to capsaicin spray, with 30.8% reporting ≥75% reduction. Minimal side effects of treatment were reported. Thus, we suggest that this therapy can be another treatment option for patients with SNC.

Intracellular calcium leak as a therapeutic target for RYR1-related myopathies.

RYR1 encodes the type 1 ryanodine receptor, an intracellular calcium release channel (RyR1) on the skeletal muscle sarcoplasmic reticulum (SR). Pathogenic RYR1 variations can destabilize RyR1 leading to calcium leak causing oxidative overload and myopathy. However, the effect of RyR1 leak has not been established in individuals with RYR1-related myopathies (RYR1-RM), a broad spectrum of rare neuromuscular disorders. We sought to determine whether RYR1-RM affected individuals exhibit pathologic, leaky RyR1 and whether variant location in the channel structure can predict pathogenicity. Skeletal muscle biopsies were obtained from 17 individuals with RYR1-RM. Mutant RyR1 from these individuals exhibited pathologic SR calcium leak and increased activity of calcium-activated proteases. The increased calcium leak and protease activity were normalized by ex-vivo treatment with S107, a RyR stabilizing Rycal molecule. Using the cryo-EM structure of RyR1 and a new dataset of > 2200 suspected RYR1-RM affected individuals we developed a method for assigning pathogenicity probabilities to RYR1 variants based on 3D co-localization of known pathogenic variants. This study provides the rationale for a clinical trial testing Rycals in RYR1-RM affected individuals and introduces a predictive tool for investigating the pathogenicity of RYR1 variants of uncertain significance.

Ryanodine receptor dysfunction in human disorders.

Regulation of intracellular calcium (Ca2+) is critical in all cell types. The ryanodine receptor (RyR), an intracellular Ca2+ release channel located on the sarco/endoplasmic reticulum (SR/ER), releases Ca2+ from intracellular stores to activate critical functions including muscle contraction and neurotransmitter release. Dysfunctional RyR-mediated Ca2+ handling has been implicated in the pathogenesis of inherited and non-inherited conditions including heart failure, cardiac arrhythmias, skeletal myopathies, diabetes, and neurodegenerative diseases. Here we have reviewed the evidence linking human disorders to RyR dysfunction and describe novel approaches to RyR-targeted therapeutics.