Development of a novel self-assessment tool following endoscopic sinus surgery in the management of chronic rhinosinusitis.

OBJECTIVE: To propose a novel quality metric tool for retrospectively examining ESS performed on chronic rhinosinusitis (CRS) patients, ultimately to facilitate clinician self-assessment and optimize care provision within this population. DESIGN: Quality improvement study. SETTING: Multi-center. PARTICIPANTS: Observational, prospective research database of adult patients with medically recalcitrant CRS, presenting to seven North-American academic rhinology centers, who underwent ESS between 2011 and 2021. Participant characteristics, comorbidities, and preoperative study measures were collected. MAIN OUTCOMES AND MEASURES: A simple ratio of preoperative Lund-Mackay (LM) score to the number of sinus regions operated on during the course of ESS was determined for each participant and dichotomized into ratios of >1.0 or <1.0. RESULTS: 828 study participants with medically recalcitrant CRS met final study inclusion, of which 47.8 % were male with an average age of 49.0 years. Approximately 50.9 % of participants had a history of previous ESS. Overall mean ratio between preoperative LM scores and numbers of surgically addressed sinuses for all patients with CRS (n = 828) was 1.61 (range: 0.00-6.00), with a minority of subjects (n = 108; 13.0 %) found to have ratios below 1.00. Mean ratios between patients who underwent primary ESS versus revision ESS were not statistically different (2.00 [±0.83] vs 1.98 [±0.88]; 0.02 %, 95 % CI -0.10, 0.14; P = 0.76), whereas differences in mean ratios between CRSsNP patients (without nasal polyposis) and CRSwNP patients (with nasal polyposis) were statistically significant (1.78 [±0.93] vs 2.26 [±0.67]; 0.48 %, 95 % CI 0.37, 0.59; P ≤ 0.001). CONCLUSIONS AND RELEVANCE: This quality metric ratio represents a simple operational means for clinicians to integrate qualitative methodology into self-reflection when evaluating the extent of ESS performed on CRS patients. Its use as a clinical tool for retrospective self-reflection enables the surgeon to identify areas for improvement, assess situational specifics, and hone their craft.

The Global Macroeconomic Burden of Burn Injuries.

BACKGROUND: Standardized estimates of global economic losses from burn injuries are lacking. The primary objective of this study was to determine the global macroeconomic consequences of burn injuries and their geographic distribution. METHODS: Using the Institute of Health Metrics and Evaluation database (2009 and 2019), mean and 95% uncertainty interval (UI) data on incidence, mortality, and disability-adjusted life-years (DALYs) from injuries caused by fire, heat, and hot substances were collected. Gross domestic product (GDP) data were analyzed together with DALYs to estimate macroeconomic losses globally using a value of lost welfare approach. RESULTS: There were 9 million global burn cases (95% UI, 6.8 to 11.2 million) and 111,000 deaths from burns (95% UI, 88,000 to 132,000 deaths) in 2019, representing a total of 7.5 million DALYs (95% UI, 5.8 to 9.5 million DALYs). This represented welfare losses of $112 billion (95% UI, $78 to $161 billion), or 0.09% of GDP (95% UI, 0.06% to 0.13%). Welfare losses as a share of GDP were highest in low- and middle-income countries (LMICs) of Oceania (0.24%; 95% UI, 0.09% to 0.42%) and Eastern Europe (0.24%; 95% UI, 0.19% to 0.30%) compared with high-income country regions such as Western Europe (0.06%; 95% UI, 0.04% to 0.09%). Mortality-incidence ratios were highest in LMIC regions, highlighting a lack of treatment access, with southern sub-Saharan Africa reporting a mortality-incidence ratio of 40.1 per 1000 people compared with 1.9 for Australasia. CONCLUSIONS: Burden of disease and resulting economic losses because of burn injuries are substantial worldwide and are disproportionately higher in LMICs. Possible effective solutions include targeted education, advocacy, and legislation to decrease incidence and investing in existing burn centers to improve treatment access.

Global, Regional, and National Economic Consequences of Stroke.

BACKGROUND: An understanding of global, regional, and national macroeconomic losses caused by stroke is important for allocation of clinical and research resources. The authors investigated the macroeconomic consequences of stroke disease burden in the year 2019 in 173 countries. METHODS: Disability-adjusted life year data for overall stroke and its subtypes (ischemic stroke, intracerebral hemorrhage, and subarachnoid hemorrhage) were collected from the GBD study (Global Burden of Disease) 2019 database. Gross domestic product (GDP, adjusted for purchasing power parity [PPP]) data were collected from the World Bank; GDP and disability-adjusted life year data were combined to estimate macroeconomic losses using a value of lost welfare (VLW) approach. All results are presented in 2017 international US dollars adjusted for PPP. RESULTS: Globally, in 2019, VLW due to stroke was $2059.67 billion or 1.66% of the global GDP. Global VLW/GDP for stroke subtypes was 0.78% (VLW=$964.51 billion) for ischemic stroke, 0.71% (VLW=$882.81 billion) for intracerebral hemorrhage, and 0.17% (VLW=$212.36 billion) for subarachnoid hemorrhage. The Central European, Eastern European, and Central Asian GBD super-region reported the highest VLW/GDP for stroke overall (3.01%), ischemic stroke (1.86%), and for subarachnoid hemorrhage (0.26%). The Southeast Asian, East Asian, and Oceanian GBD super-region reported the highest VLW/GDP for intracerebral hemorrhage (1.48%). CONCLUSIONS: The global macroeconomic consequences related to stroke are vast even when considering stroke subtypes. The present quantification may be leveraged to help justify increased spending of finite resources on stroke in an effort to improve outcomes for patients with stroke globally.

A Multicenter, Propensity Score-Matched Assessment of Endoscopic Versus Microscopic Approaches in the Management of Pituitary Adenomas.

BACKGROUND AND OBJECTIVES: There is considerable controversy as to which of the 2 operating modalities (microsurgical or endoscopic transnasal surgery) currently used to resect pituitary adenomas (PAs) is the safest and most effective intervention. We compared rates of clinical outcomes of patients with PAs who underwent resection by either microsurgical or endoscopic transnasal surgery. METHODS: To independently assess the outcomes of each modality type, we sought to isolate endoscopic and microscopic PA surgeries with a 1:1 tight-caliper (0.01) propensity score-matched analysis using a multicenter, neurosurgery-specific database. Surgeries were performed between 2017 and 2020, with data collected retrospectively from 12 international institutions on 4 continents. Matching was based on age, previous neurological deficit, American Society of Anesthesiologists (ASA) score, tumor functionality, tumor size, and Knosp score. Univariate and multivariate analyses were performed. RESULTS: Among a pool of 2826 patients, propensity score matching resulted in 600 patients from 9 surgery centers being analyzed. Multivariate analysis showed that microscopic surgery had a 1.91 odds ratio (OR) ( P = .03) of gross total resection (GTR) and shorter operative duration ( P < .01). However, microscopic surgery also had a 7.82 OR ( P < .01) for intensive care unit stay, 2.08 OR ( P < .01) for intraoperative cerebrospinal fluid (CSF) leak, 2.47 OR ( P = .02) for postoperative syndrome of inappropriate antidiuretic hormone secretion (SIADH), and was an independent predictor for longer postoperative stay (ß = 2.01, P < .01). Overall, no differences in postoperative complications or 3- to 6-month outcomes were seen by surgical approach. CONCLUSION: Our international, multicenter matched analysis suggests microscopic approaches for pituitary tumor resection may offer better GTR rates, albeit with increased intensive care unit stay, CSF leak, SIADH, and hospital utilization. Better prospective studies can further validate these findings as matching patients for outcome analysis remains challenging. These results may provide insight into surgical benchmarks at different centers, offer room for further registry studies, and identify best practices.

Impact of Medicaid Expansion on Rhinologic Cancer Presentation, Treatment, and Outcomes.

OBJECTIVE: The United States Patient Protection and Affordable Care Act allocated funds for states to expand Medicaid coverage. However, several states declined expansion. We aim to determine whether Medicaid expansion is associated with healthcare coverage, cancer stage at diagnosis, treatment, and survival among patients with rhinologic cancer. Rhinologic cancer was defined to include cancer of the nasal cavity, paranasal sinus, nasopharynx, or olfactory nerve. STUDY DESIGN: Cohort study. METHODS: Patients diagnosed with primary rhinologic malignancies between 2007 to 2016 were extracted from the National Cancer Institute Surveillance, Epidemiology, End Results (SEER) registry. Patients were grouped by diagnosis before and after 2014 (when Medicaid expansion became effective) and whether their state had expanded Medicaid. Multivariable logistic regression controlling for age, sex, race, ethnicity, and income/education was utilized to examine associations between Medicaid expansion/insurance status and stage at diagnosis, treatment, and survival. Overall and disease-specific survival were examined using Kaplan-Meier analysis. RESULTS: Analysis included 10,164 patients. The proportion of uninsured patients decreased after 2014 (2.4%) compared to before 2014 (4.8%, P < .001). After 2014, patients in nonexpanded states were more likely to be diagnosed with advanced stage disease compared to patients in expanded states (N = 2,364; OR = 1.27, 95% CI 1.01-1.60). Being uninsured in any state was associated with advanced stage disease at diagnosis (OR = 1.75, 95% CI 1.41-2.22) and increased risk of disease-specific death (HR = 1.54, 95% CI 1.32-1.82). Survival measures were not associated with diagnosis before versus after 2014 or Medicaid expansion. CONCLUSIONS: Patients lacking insurance or residing in nonexpanded states may be more likely to present with advanced stage rhinologic cancer. Longitudinal studies should validate these findings. LEVEL OF EVIDENCE: 3 Laryngoscope, 133:43-50, 2023.

A national stratification of the global macroeconomic burden of central nervous system cancer.

OBJECTIVE: Country-by-country estimates of the macroeconomic disease burden of central nervous system (CNS) cancers are important when determining the allocation of resources related to neuro-oncology. Accordingly, in this study the authors investigated macroeconomic losses related to CNS cancer in 173 countries and identified pertinent epidemiological trends. METHODS: Data for CNS cancer incidence, mortality, and disability-adjusted life years (DALYs) were collected from the Global Burden of Disease 2019 database. Gross domestic product data were combined with DALY data to estimate economic losses using a value of lost welfare approach. RESULTS: The mortality-to-incidence ratio of CNS cancer in 2019 was 0.60 in high-income regions compared to 0.82 in Sub-Saharan Africa and 0.87 in Central Europe, Eastern Europe, and Central Asia. Welfare losses varied across both high- and low-income countries. Welfare losses attributable to CNS cancer in Japan represented 0.07% of the gross domestic product compared to 0.23% in Germany. In low- and middle-income countries, Iraq reported welfare losses of 0.20% compared to 0.04% in Angola. Globally, the DALY rate in 2019 was the same for CNS cancer as for prostate cancer at 112 per 100,000 person-years, despite a 75% lower incidence rate, equating to CNS cancer welfare losses of 182 billion US dollars. CONCLUSIONS: Macroeconomic losses vary across high- and low-income settings and appear to be region specific. These differences may be explained by differences in regional access to screening and diagnosis, population-level genetic predispositions, and environmental risk factors. Mortality-to-incidence ratios are higher in low- and middle-income countries than in high-income countries, highlighting possible gaps in treatment access. Quantification of macroeconomic losses related to CNS cancer can help to justify the spending of finite resources to improve outcomes for neuro-oncological patients globally.

Building a practice-based research network for healthcare integration: a protocol paper for a mixed-method project.

INTRODUCTION: Practice-based research networks (PBRNs) are sustained collaborations between healthcare professionals, researchers and members of the community that develop, conduct and report on research relevant to local needs. While PBRNs have traditionally been focused towards primary care practices and their patients, there has been increasing interest in how they may help facilitate healthcare integration. Yet, little is known on the ways in which PBRNs can best integrate with the broader healthcare system, in particular Advanced Health Research and Translation Centres. The overall project aim is to build a sustainable collaboration between a PBRN and an Advanced Health Research and Translation Centre to generate a research platform suitable for planning, undertaking and translating research to improve care across the healthcare continuum. METHODS AND ANALYSIS: We will use a developmental evaluation design. Our iterative approach will be informed by a programme logic model and consists of: preparation work (pre-implementation assessment, literature review, community and stakeholder engagement), adaptation and building for a sustainable collaboration (strategy for recruitment and sustainment of members) and planning for network action (designing and implementing priority initiatives, monitoring and follow-up). ETHICS AND DISSEMINATION: This project was approved by the Monash Health ethics committee (ERM Reference Number: 76281; Monash Health Ref: RES-21-0000-392L) and the Monash University Human Research ethics committee (Reference Number: 29786). Dissemination will take place via various channels, including relevant national and international committees and conferences, peer-reviewed journals and social media. Continuous dissemination to and communication with all participants in this project as well as other relevant stakeholders will help strengthen and sustain the network.

Telemedicine and the right to health: A neurosurgical perspective.

Neurosurgical task force is limited and unevenly distributed. Telemedicine has become increasingly popular, and could help neurosurgical centers meet patient right to care. This scoping review aims to evaluate the impact and feasibility of telemedicine on the right to neurosurgical care, using the AAAQ toolbox. The AAAQ toolbox consists of Availability, Accessibility, Acceptability and Quality. Neurosurgical availability is limited by the number of neurosurgeons, but by using task shifting and -sharing via telemedicine, the number of patients receiving neurosurgical care could increase without increasing the number of neurosurgeons. Telemedicine can improve geographic accessibility to neurosurgical care, but may also introduce technological literacy barriers. Acceptability of telemedicine is a double-edged sword; while a useful service, telemedicine also creates ethical concerns regarding privacy and confidentiality. Regulations and adaptations for vulnerable patient groups are key considerations for deploying telemedicine. Finally, there is emerging evidence that the quality of remote neurosurgical diagnostics and care can keep high standards. Overall, telemedicine has the potential of taking neurosurgery one step closer to meeting patient right to health, globally.

Economic Burden and Healthcare Resource Use of Alopecia Areata in an Insured Population in the USA.

INTRODUCTION: Comparative data on the economic burden of alopecia areata relative to the general population are limited. The objective of this retrospective database analysis was to evaluate healthcare resource utilization and direct medical costs among patients with alopecia areata from the US payer perspective compared with matched controls. METHODS: Validated billing codes were used to identify patients with alopecia areata from the IQVIA PharMetrics Plus (2016-2018) who had continuous pharmacy and medical enrollment for 365 days both before (baseline period) and after (evaluation period) the index date. Demographic and clinical characteristics were characterized, and baseline comorbidities were assessed with the Quan Charlson Comorbidity Index. RESULTS: Using the exact matching feature from Instant Health Data, 14,340 patients with alopecia areata were matched with 42,998 control patients aged ≥ 12 years. Patients with alopecia areata had higher healthcare resource utilization and adjusted total all-cause mean medical costs versus matched controls ($8557 versus $6416; p < 0.0001), because of higher inpatient costs, emergency department visits, ambulatory visits, number of prescriptions and prescription costs, and other costs such as durable medical equipment and home healthcare. The number of inpatient visits did not significantly differ between the two groups. Mean ambulatory costs were $3640 for patients with alopecia areata and $2062 for controls, and mean pharmacy costs were $3287 and $1843, respectively (p < 0.0001 for both). Pharmacy costs related to immunologic agents represented 50.0% of the total difference in pharmacy spending between patients with alopecia areata and controls. Surgery on the integumentary system accounted for 9.5% of the total difference in ambulatory costs. CONCLUSION: Alopecia areata is associated with significant incremental healthcare resource utilization and costs relative to matched controls due to increased spending in areas such as surgical procedures and psychological and pharmacological interventions. Costs are primarily driven by ambulatory and pharmacy spending.

International consensus statement on allergy and rhinology: Olfaction.

BACKGROUND: The literature regarding clinical olfaction, olfactory loss, and olfactory dysfunction has expanded rapidly over the past two decades, with an exponential rise in the past year. There is substantial variability in the quality of this literature and a need to consolidate and critically review the evidence. It is with that aim that we have gathered experts from around the world to produce this International Consensus on Allergy and Rhinology: Olfaction (ICAR:O). METHODS: Using previously described methodology, specific topics were developed relating to olfaction. Each topic was assigned a literature review, evidence-based review, or evidence-based review with recommendations format as dictated by available evidence and scope within the ICAR:O document. Following iterative reviews of each topic, the ICAR:O document was integrated and reviewed by all authors for final consensus. RESULTS: The ICAR:O document reviews nearly 100 separate topics within the realm of olfaction, including diagnosis, epidemiology, disease burden, diagnosis, testing, etiology, treatment, and associated pathologies. CONCLUSION: This critical review of the existing clinical olfaction literature provides much needed insight and clarity into the evaluation, diagnosis, and treatment of patients with olfactory dysfunction, while also clearly delineating gaps in our knowledge and evidence base that we should investigate further.

Financial Hardship Impacts Depression and Anxiety Among U.S. Patients with Sinusitis.

BACKGROUND: Mental health conditions are common in the United States, and recent efforts have examined the development of mental health conditions among patients with sinusitis. OBJECTIVES: The purpose of this study was to investigate the association between depression, anxiety, and financial hardship among patients with sinusitis. METHODS: Cross-sectional study using the 2018 National Health Interview Survey (NHIS). Data regarding demographics, perceived financial hardship, self-reported depression and anxiety, mental healthcare utilization, and treatment compliance were obtained. RESULTS: Among patients with sinusitis (N = 28 million adults), 9% reported depression and 24% reported anxiety. Sinusitis patients with depression and anxiety reported an increased severity of financial insecurity (p < 0.001). On multivariable logistic regression, worsening financial insecurity increased the odds of depression and anxiety. Patients reporting the highest financial insecurity severity had the highest odds of depression (OR = 3.88, 95% CI = 3.84-3.93, p < 0.001) and anxiety (OR = 2.09, 95% CI = 2.08-2.10, p < 0.001) among measures of financial stress. Specific financial stressors were independently associated with patient-reported depression and anxiety. Sinusitis patients with increased financial insecurity were more likely to require mental health services and treatment (p < 0.001), but were also more likely to report cost-related treatment noncompliance (p < 0.001) and reduced access to mental healthcare due to costs (p < 0.001). CONCLUSION: Perceived financial hardship is associated with self-reported depression and anxiety among patients with sinusitis. Sinusitis patients with financial hardship also face challenges in accessing and maintaining mental health services and treatment due to costs. Understanding the burden of financial insecurity on mental health and access to treatment may improve quality of care through the development of screening tools and individualized treatment strategies.

Retrospective Database Analysis: Dose Escalation and Adherence in Patients Initiating Biologics for Ulcerative Colitis.

BACKGROUND: Biologic therapies are often used in patients with ulcerative colitis (UC) who are nonresponsive to conventional treatments. However, nonresponse or loss of response to biologics often occurs, leading to dose escalation, combination therapy, and/or treatment switching. We investigated real-world treatment patterns of biologic therapies among patients with UC in the USA. METHODS: This study analyzed data from the IBM® MarketScan® Commercial and Medicare Supplemental Databases (medical/pharmacy claims for >250 million patients in the USA) to identify patients with UC initiating a biologic therapy (adalimumab, infliximab, golimumab, or vedolizumab) with 12 months of follow-up post-initiation. Key measures were patient baseline characteristics, dose escalation (average maintenance dose >20% higher than label), adherence (proportion of days covered), and ulcerative colitis-related healthcare costs in the 12 months following biologic therapy initiation. RESULTS: Of 2,331 patients included in the study (adalimumab [N = 1,291], infliximab [N = 810], golimumab [N = 127], and vedolizumab [N = 103]), 28.1% used concomitant immunosuppressant therapy within 12 months post-initiation. Overall, 23.6% (adalimumab), 34.8% (infliximab), 9.9% (golimumab), and 39.2% (vedolizumab) of patients dose escalated within 12 months. Patients who dose escalated incurred USD 20,106 higher total UC-related healthcare costs over 12 months than those who did not. Adherence (covariate-adjusted proportion of days covered) ranged from 0.63 to 0.73, and 39.3% of patients discontinued within 12 months (median treatment duration = 112 days). CONCLUSION: Dose escalation was common, and incurred higher costs, in patients with UC initiating biologic therapies. Suboptimal adherence and/or discontinuation within 12 months of initiation occurred frequently, highlighting the challenges in managing these patients.

Perceived Financial Insecurity Impacts Healthcare Decision-Making Among Patients With Sinusitis.

OBJECTIVES/HYPOTHESIS: The economic burden of sinusitis is significant, and socioeconomic factors can impact patient decision-making. The purpose of this study was to examine the impact of perceived financial insecurity on healthcare decision-making and treatment compliance among sinusitis patients. STUDY DESIGN: Cross-sectional study using the 2018 National Health Interview Survey. METHODS: Survey responses to nine questions regarding financial stressors and nine questions regarding cost-saving healthcare actions were recorded, which included seeking lower cost medication, medication noncompliance, and avoiding care visits due to costs. RESULTS: There was a total weighted sample size of 28.9 million patients who self-reported a diagnosis of sinusitis (12% of the U.S. population). Sinusitis patients who reported cost-saving actions had an increased severity of perceived financial insecurity than those without cost-saving actions (P < .001). Sinusitis patients with perceived financial insecurity had the highest odds of at least one cost-saving action (odds ratio [OR] = 5.94, 95% CI = 5.911-5.970, P < .001), followed by lack of health insurance (OR = 5.13, 95% CI = 5.107-5.159, P < .001), and poor self-reported health status (OR = 2.81, 95% CI = 2.792-2.822, P < .001). Increasing the number of financial stressors increased the odds of at least one cost-saving action (P < .001). Across all financial stressors, the most commonly performed cost-saving action was asking for lower cost medication. CONCLUSIONS: Perceived financial insecurity is associated with cost-saving healthcare actions among sinusitis patients, including treatment noncompliance. Interventions to assess financial insecurity among sinusitis patients may facilitate shared decision-making for optimal, individualized treatment plans that may lead to improved outcomes and quality of life. LEVEL OF EVIDENCE: NA Laryngoscope, 131:2403-2412, 2021.

Clinical and Economic Burden of Mild-to-Moderate Atopic Dermatitis in the UK: A Propensity-Score-Matched Case-Control Study.

INTRODUCTION: The burden of mild-to-moderate atopic dermatitis (AD) in the United Kingdom (UK) is not well understood. Long-lasting AD flares may lead to systemic inflammation resulting in reversible progression from mild to more severe AD. This study aimed to assess the clinical and economic burden of mild-to-moderate AD in the UK. METHODS: AD patients were identified in the Health Improvement Network (THIN) from 2013 to 2017 and propensity score matched to non-AD controls by demographics. Patients were identified based on continuous disease activity using validated algorithms and sufficient patient status to fully validate data integrity for the entire period. Mild-to-moderate AD patients were identified by using treatment as a surrogate. Demographics, clinical characteristics and healthcare resource use (HCRU) were obtained from THIN. Literature reviews were conducted to obtain additional outcomes. A cost-of-illness model was developed to extrapolate the burden in 2017 to the UK population and in subsequent years (2018-2022). RESULTS: In 2017, the prevalence of mild-to-moderate AD in THIN was 1.28%. These patients reported higher comorbidity rates and significantly higher (p < 0.0001) HCRU, encompassing mean general practitioner visits (5.57 versus 3.59), AD-related prescriptions (5.85 versus 0.68) and total referrals (0.97 versus 0.82) versus matched non-AD controls. The model projected total HCRU and drug excess costs of €462.99M over the 5 years. The excess cost decreased to €417.35M after excluding patients on very potent topical corticosteroids, who most likely had at least moderate disease. The excess costs increased to €1.21B and €7.06B when considering comorbidity burden and productivity losses, respectively. CONCLUSION: Mild-to-moderate AD patients had higher comorbidity burden, HCRU and cost compared with matched non-AD controls. Overall, UK country-based economic burden was high given partly the high prevalence of this disease. Moreover, productivity burden and comorbidities had considerable impact on the economic burden, which further suggests the importance of optimal disease management.

The epidemiology and management patterns of pediatric pituitary tumors in the United States.

PURPOSE: Hypothalamic-pituitary axis dysfunction and mass effect symptoms in the pediatric population can indicate a pituitary region tumor. Herein, we evaluate the epidemiology and management of this rare entity. METHODS: Pediatric patients (≤ 21yo) who presented from 2004 to 2017 with a pituitary tumor were evaluated from the U.S. National Cancer Database. The distributions and management patterns of pituitary tumors were assessed by patients' tumor type, age, sex, race/ethnicity, tumor size, and insurance status. RESULTS: 19.7% of intracranial tumors in the pediatric population originated in the pituitary region. 7653 pediatric patients with pituitary region tumors were identified, 68.2% of whom were female, with the tumors predominantly occurring in early adolescence (46.9%) and late adolescence (34.8%). The majority of pediatric pituitary region tumors were pituitary adenomas (77.9%), followed by craniopharyngiomas (18.1%) and germ cell tumors (1.6%). Girls demonstrated higher proportions of pituitary adenomas across all ages than boys. Asian/Pacific Islander patients were independently more likely to present at younger ages (mean 13.9yrs) and with germ cell tumors than patients of other races/ethnicities. Only 5.5% of patients were uninsured (referent), but they were independently more likely to present at older ages (mean 17.9yrs) and less likely to undergo surgery than patients with private insurance (OR = 1.93, 95% CI = 1.47-2.52, p < 0.001) or Medicaid (OR = 1.51, 95% CI = 1.14-2.00, p = 0.004). CONCLUSION: Pituitary region tumors comprise a significant fraction of intracranial pediatric tumors, particularly in adolescent girls. The differential diagnosis of pituitary tumor types differed significantly by patients' age, sex, and race/ethnicity. Uninsured patients were associated with delays in care and less surgical management.

Should Oral Corticosteroids be Used in Medical Therapy for Chronic Rhinosinusitis? A Risk Analysis.

OBJECTIVES: Oral corticosteroid (OCS) as a part of appropriate medical therapy (AMT) (formerly maximal medical therapy) in chronic rhinosinusitis remains controversial. While the risks of OCS are well known, the benefit remains unclear due the absence of a standardized prescribing regimen. Consequently, it is difficult to characterize whether the risks of OCS and its ability to avert endoscopic sinus surgery (ESS) are helpful in AMT. When OCS is highly effective at averting surgery, the lesser risks of OCS would be justified because it can avoid the greater risks of ESS. When OCS is poorly effective at averting ESS, the risks of OCS would not be justified because many patients will be exposed to both risks. This study seeks to identify the threshold effectiveness of OCS at averting ESS that would minimize risk exposure to patients. METHODS: A probabilistic risks-based decision analysis was constructed from literature reported incidences and impacts of adverse events of OCS and ESS. Monte Carlo analysis was performed to identify the minimum effectiveness required to avoid further intervention (MERAFI) for chronic sinusitis without nasal polyp (CRSsNP) and chronic sinusitis with nasal polyp (CRSwNP). RESULTS: The analysis showed MERAFI results of 20.8% (95% CI 20.7-20.9%) for CRSsNP and 16.8% (95% CI 16.7-16.9%) for CRSwNP. CONCLUSIONS: Given reported OCS effectiveness in the range of 34-71% in CRSsNP and 46-63% in CRSwNP, this analysis suggests that the inclusion of OCS in AMT may be the lower risk strategy. LEVEL OF EVIDENCE: N/A Laryngoscope, 131:473-481, 2021.

Real-World Evidence to Contextualize Clinical Trial Results and Inform Regulatory Decisions: Tofacitinib Modified-Release Once-Daily vs Immediate-Release Twice-Daily for Rheumatoid Arthritis.

INTRODUCTION: Tofacitinib is an oral Janus kinase inhibitor for the treatment of rheumatoid arthritis (RA). To provide additional clinical evidence in regulatory submissions for a modified-release (MR) once-daily (QD) tofacitinib formulation, we compared real-world adherence and effectiveness between patients initiating the MR QD formulation and patients initiating an immediate-release (IR) twice-daily (BID) formulation. METHODS: Two noninterventional cohort studies were conducted. First, adherence and two effectiveness proxies were compared between patients with RA who newly initiated tofacitinib MR 11 mg QD or IR 5 mg BID in the IBM® MarketScan® Commercial and Medicare Supplemental US insurance claims databases (March 2016-October 2018). Second, using data collected in the Corrona US RA Registry (February 2016-August 2019), two Clinical Disease Activity Index (CDAI)-based measures of effectiveness were compared between tofacitinib MR 11 mg QD and IR 5 mg BID, and against noninferiority criteria derived from placebo-controlled clinical trials of the tofacitinib IR formulation. Multiple sensitivity analyses of the registry data were conducted to reassure regulators of consistent results across different assumptions. RESULTS: In each study, approximately two-thirds of patients initiated the MR formulation. In the claims database study, improved adherence and at least comparable effectiveness were observed with tofacitinib MR vs IR over 12 months, particularly in patients without prior advanced therapy. In the registry study, the noninferiority of tofacitinib MR vs IR was demonstrated for both CDAI outcomes at ~6 months; this finding was robust across multiple sensitivity analyses. CONCLUSION: These results demonstrate the value of real-world evidence from complementary data sources in understanding the impact of medication adherence with a QD formulation in clinical practice. These analyses were suitable for regulatory consideration as an important component of evidence for the comparability of tofacitinib MR 11 mg QD vs IR 5 mg BID in patients with RA. TRIAL REGISTRATION: Claims database study: ClinicalTrials.gov identifier NCT04018001, retrospectively registered July 12, 2019. Corrona US RA Registry study: ClinicalTrials.gov identifier NCT04267380, retrospectively registered February 12, 2020.