Familial hypercholesterolemia care by Dutch pediatricians-mind the gaps.

PURPOSE: Familial hypercholesterolemia (FH) leads to elevated low-density lipoprotein cholesterol levels, which increases the risk of premature atherosclerotic cardiovascular disease (ASCVD). Since the first functional and morphologic changes of the arterial wall occur in childhood, treatment should start early in childhood to mitigate the elevated risk of ASCVD. Pediatricians play an important role in the detection and care of children with FH. In this study, we aim to explore potential gaps in FH care amongst Dutch pediatricians, in order to enhance their knowledge and awareness of detecting and treating children with FH. METHODS: An anonymous online survey, deployed using Google Forms, including 26 closed and semi-closed questions on FH care in children was distributed by the Dutch Association of Pediatrics via a newsletter to which the majority of the practicing Dutch pediatricians subscribe. In addition, we requested that the pediatric departments of all Dutch hospitals in the Netherlands distribute this survey personally among their employed pediatricians. Respondents were instructed to answer the questions without any help or use of online resources. RESULTS: Between September 1st, 2023 and November 1st, 2023, 158 (an estimated 11% response rate) Dutch pediatricians completed the survey. They reported a median (IQR) of 15.0 (6.0-22.0) years of experience as a pediatrician, and 34 (21.5%) were working in academic hospitals. The majority (76.6%) of pediatricians correctly identified a typical FH lipid profile but 68 (43.0%) underestimated the true prevalence of FH (1:300). Underestimation and unawareness of the increased risk of FH patients for ASCVD were reported by 37.3% and 25.9% of pediatricians, respectively. Although 70.9% of the pediatricians correctly defined FH, only 67 (42.4%) selected statins and ezetimibe to treat severe hypercholesterolemia. CONCLUSIONS: The results of this study suggest significant gaps in knowledge and awareness of FH in children among Dutch pediatricians. FH care in children needs improvement through educational and training initiatives to mitigate the life-long risk of ASCVD from early life. WHAT IS KNOWN: • Familial hypercholesterolemia (FH) leads to elevated LDL-cholesterol levels, which increases the risk of premature atherosclerotic cardiovascular disease (ASCVD). • The process of atherosclerosis starts in childhood • Pediatricians play an important role in the detection and treatment of children with FH. WHAT IS NEW: • Our results highlight significant gaps in care for children with FH amongst pediatricians and this may lead to suboptimal detection and treatment. • FH care in children needs improvement by educational initiatives to ultimately prevent ASCVD in adulthood.

Birthweight measurement processes and perceived value: qualitative research in one EN-BIRTH study hospital in Tanzania.

BACKGROUND: Globally an estimated 20.5 million liveborn babies are low birthweight (LBW) each year, weighing less than 2500 g. LBW babies have increased risk of mortality even beyond the neonatal period, with an ongoing risk of stunting and non-communicable diseases. LBW is a priority global health indicator. Now almost 80% of births are in facilities, yet birthweight data are lacking in most high-mortality burden countries and are of poor quality, notably with heaping especially on values ending in 00. We aimed to undertake qualitative research in a regional hospital in Dar es Salaam, Tanzania, observing birthweight weighing scales, exploring barriers and enablers to weighing at birth as well as perceived value of birthweight data to health workers, women and stakeholders. METHODS: Observations were undertaken on type of birthweight scale availability in hospital wards. In-depth semi-structured interviews (n = 21) were conducted with three groups: women in postnatal and kangaroo mother care wards, health workers involved in birthweight measurement and recording, and stakeholders involved in data aggregation in Temeke Hospital, Tanzania, a site in the EN-BIRTH study. An inductive thematic analysis was undertaken of translated interview transcripts. RESULTS: Of five wards that were expected to have scales, three had functional scales, and only one of the functional scales was digital. The labour ward weighed the most newborns using an analogue scale that was not consistently zeroed. Hospital birthweight data were aggregated monthly for reporting into the health management information system. Birthweight measurement was highly valued by all respondents, notably families and healthcare workers, and local use of data was considered an enabler. Perceived barriers to high quality birthweight data included: gaps in availability of precise weighing devices, adequate health workers and imprecise measurement practices. CONCLUSION: Birthweight measurement is valued by families and health workers. There are opportunities to close the gap between the percentage of babies born in facilities and the percentage accurately weighed at birth by providing accurate scales, improving skills training and increasing local use of data. More accurate birthweight data are vitally important for all babies and specifically to track progress in preventing and improving immediate and long-term care for low birthweight children.

Expert Consensus on Nail Procedures and Selection of CPT Codes.

BACKGROUND: Dermatologists specialize in treating conditions of the skin, hair, and nails; however, it is our experience that the field of nail diseases is the least discussed facet of dermatology. Even less acknowledged is the complexity of nail procedures and how best to accurately code for these procedures. OBJECTIVE: To convene a panel of experts in nail disease to reach consensus on the most accurate and appropriate Current Procedural Terminology (CPT) codes associated with the most commonly performed nail procedures. METHODS: A questionnaire including 9 of the most commonly performed nail procedures and potential CPT codes was sent to experts in the treatment of nail disease, defined as those clinicians running a nail subspecialty clinic and performing nail procedures with regularity. A conference call was convened to discuss survey results. RESULTS: Unanimous consensus was reached on the appropriate CPT codes associated with all discussed procedures. LIMITATIONS: Although this article details the most commonly performed nail procedures, many were excluded and billing for these procedures continues to be largely subjective. This article is meant to serve as a guide for clinicians but should not be impervious to interpretation in specific clinical situations. CONCLUSION: Billing of nail procedures remains a practice gap within our field. The authors hope that the expert consensus on the most appropriate CPT codes associated with commonly performed nail procedures will aid clinicians as they diagnose and treat disorders of the nail unit and encourage accurate and complete billing practices.

Hepatitis C Virus (HCV) Care Continuum Outcomes and HCV Community Viral Loads Among Patients in an Opioid Treatment Program.

BACKGROUND: Hepatitis C virus (HCV) remains endemic among people who use drugs (PWUD). Measures of HCV community viral load (CVL) and HCV care continuum outcomes may be valuable for ascertaining unmet treatment need and for HCV surveillance and control. METHODS: Data from patients in an opioid treatment program during 2013-2016 were used to (1) identify proportions of antibody and viral load (VL) tested, linked-to-care, and treated, in 2013-2014 and 2015-2016, and pre- and postimplementation of qualitative reflex VL testing; (2) calculate engaged-in-care HCV CVL and "documented" and "estimated" unmet treatment need; and (3) examine factors associated with linkage-to-HCV-care. RESULTS: Among 11 267 patients, proportions of HCV antibody tested (52.5% in 2013-2014 vs 73.3% in 2015-2016), linked-to-HCV-care (15.7% vs 51.8%), and treated (12.0% vs 44.7%) all increased significantly. Hispanic ethnicity was associated with less linkage-to-care, and Manhattan residence was associated with improved linkage-to-care. The overall engaged-in-care HCV CVL was 4 351 079 copies/mL (standard deviation = 7 149 888); local HCV CVLs varied by subgroup and geography. Documented and estimated unmet treatment need decreased but remained high. CONCLUSIONS: After qualitative reflex VL testing was implemented, care continuum outcomes improved, but gaps remained. High rates of unmet treatment need suggest that control of the HCV epidemic among PWUD will require expansion of HCV treatment coverage.

Unmet needs in the international neuroendocrine tumor (NET) community: Assessment of major gaps from the perspective of patients, patient advocates and NET health care professionals.

Due to the increasing incidence and prevalence of neuroendocrine tumors (NETs), there is a need to assess any gaps in awareness and care. A survey was undertaken in 2017 to identify perceived unmet needs from the perspectives of patients/families, patient advocates and health care professionals (HCPs). The survey consisted of 33-37 questions (depending on type of respondent) across four areas: information, care, treatments and research. In total, 443 participants from 26 countries responded: 338 patients/families, 35 advocates and 70 HCPs. Perceived unmet needs regarding provision of information at diagnosis differed between groups. While 59% of HCPs believed they provided sufficient information, informational needs were mostly/fully met for only 30% of patients and 18% of advocates. Additionally, 91% of patients and 97% of advocates felt that patients had to search for information themselves. Availability of Gallium-68-Dotatate PET/CT scan was limited for the majority of patients (patients: 73%; advocates: 85%; HCP: 86%), as was access to treatments, particularly peptide receptor radionuclide therapy (patients: 42%; advocates: 95%; HCPs: 77%). All groups felt that standards of care, including psychological needs and diagnosis of mental health, were not fully met. Although about two-thirds of patients were managed by a multidisciplinary team, 14% of patients reportedly did not have enough contact. All groups supported more patient involvement in research; patients and advocates prioritized improvement in diagnosis and HCPs focused on clinical trials. This survey revealed significant unmet needs but differing perceptions regarding these among the groups. There is a need for investigation and collaboration to improve standards of care for NET patients.

A Survey of Trauma Surgeon Perceptions of Resources for Patients With Psychiatric Comorbidities.

BACKGROUND: Psychiatric illness is an independent risk factor for trauma and recidivism and is often comorbid in the trauma population. There is no current standard for the delivery of mental health services in trauma care. The purpose of this study was to gauge trauma surgeon perceptions of needed and currently available resources for this patient population at level 1 trauma centers in the United States. MATERIALS AND METHODS: A 10-question survey was developed to capture the estimated volume of psychiatric patients admitted to level 1 trauma centers, their available psychiatric services, and perceived need for resources. It was sent to 27 trauma surgery colleagues at different level 1 trauma centers across the United States using a public survey tool. Descriptive analyses were performed. RESULTS: Twenty-two of 27 trauma surgeons responded (81% response rate). Ten centers (48%) estimated admitting 1-5 patients with preexisting serious mental illness weekly, whereas others admitted more. Eight (36%) reported not having acute situational support services available. Ten respondents (46%) did not know how many psychiatric consultants were available at their institution. Twelve surgeons (55%) reported no designated outpatient follow-up for psychiatric issues. Sixteen trauma surgeons (73%) stated that expanded psychiatric services are needed at their trauma center. CONCLUSIONS: Trauma patients frequently present with preexisting serious mental illness and many struggle with psychological sequelae of trauma. Over half of the surveyed surgeons reported no outpatient follow-up for these patients, and almost three quarters perceived the need for expansion of psychiatric services. In addition to a lack of resources, these findings highlight an overlooked gap in high-quality patient-centered trauma care.

Predictors of Care Gaps in Home Dialysis: The Home Dialysis Virtual Ward Study.

BACKGROUND: Home dialysis patients may be at an increased risk of adverse events after transitional states. The home dialysis virtual ward (HDVW) trial was conducted in Canadian dialysis centers and aimed to evaluate potential care gaps and patient satisfaction during the HDVW. METHODS: The HDVW was a multicenter single-arm trial including peritoneal dialysis and home hemodialysis patients after 4 different events (hospital discharge, medical procedure, antibiotics, completion of training). Telephone-led interviews using a standardized assessment tool were performed over a 2-week period to assess a patient's care and adjust treatment as required. Upon completion, patients were surveyed to evaluate their perceived impact on domains of care using a rating scale; 1 not satisfied to 10 completely satisfied. RESULTS: The HDVW trial included 193 patients with a median number of potential care gaps/interventions of 1 (0-2) per patient. Patients admitted to the HDVW after hospital discharge were at a higher risk of potential gaps in care (OR 2.16, 95% CI 1.29-3.62), while longer dialysis vintage was -associated with a lower number of gaps/interventions (OR 0.97 per year, 95% CI 0.95-0.98). A total of 105/193 (54%) patients completed satisfaction surveys. Patients were highly satisfied with the HDVW (median rating scale score 8, IQR 2) and felt it had a positive impact (rating scale score ≥7) on their overall health, understanding of treatment and access to a nephrologist. CONCLUSION: The HDVW was effective at identifying several potential care gaps, and patients were satisfied across several domains of care. This intervention may be valuable in supporting home dialysis patients during care transitions.

Patterns of endocrine therapy in a national cohort of early stage HER2-positive breast cancer patients.

PURPOSE: Guidelines recommend ≥5 years of endocrine therapy for hormone receptor-positive breast cancer patients, but nonadherence and treatment discontinuation are common. We examined adherence trajectories and early discontinuation of endocrine therapy over 5 years from treatment initiation. METHODS: Our retrospective cohort study used a national sample of Australian women dispensed publicly subsidised trastuzumab for early HER2-positive breast cancer. We included women initiating endocrine therapy between April 2007 and June 2011, followed until June 2016 (n = 2656). We used group-based trajectory modelling and Kaplan-Meier analysis to examine patterns of adherence and time to discontinuation and restarting. RESULTS: We identified five adherence trajectories: quick decline (10.4%), moderate decline (8.6%), quick decline then stable (10.3%), stable with late decline (23.6%), and high and stable (47.2%). Women in the high and stable trajectory group were older and more likely to initiate therapy with anastrozole than women in other groups. Time periods after first 6 months, 1.5, and 4 years from initiation seemed critical in terms of remaining adherent on endocrine therapy; 45.8% of the cohort discontinued endocrine therapy with a median time to discontinuation of 2.6 years (interquartile range 1.0-4.4), and 45.8% of the women discontinuing restarted treatment (median time 182.0, interquartile range 133.0-279.0 days). CONCLUSIONS: Our study highlights evidence-practice gaps in the use of endocrine therapy, with half of our sample experiencing suboptimal adherence or persistence. Trajectory modelling provided detailed information about patterns of nonadherence and critical time periods for adherence to endocrine therapy. This information is important for developing targeted interventions to improve adherence.

Pharmacist documentation of gaps in care identified during diabetes coaching.

OBJECTIVES: 1) To identify the number of gaps, type of gaps, and contributing factors to gaps in diabetes care reported by pharmacists in the Diabetes Gaps in Care Portal (Gaps Portal). 2) To understand how pharmacists used the Gaps Portal and the implications for quality diabetes care. DESIGN: The Alliance for Patient Medication Safety developed an online Gaps Portal as a tool for pharmacists to identify and document gaps in care when managing patients with diabetes. SETTING: Pharmacists from across the United States in community pharmacy and ambulatory care settings who were engaged in diabetes coaching activities participated. MAIN OUTCOME MEASURES: Pharmacists entered gaps in diabetes care into the Gaps Portal from April 2016 to June 2017. Descriptive statistics were used to determine the type and number of gaps and contributing factors. A qualitative analysis of pharmacist interviews was conducted to identify themes related to pharmacist perceptions of the portal. RESULTS: Ten pharmacists entered 528 gaps in diabetes care (n = 469; 565 encounters). The most common category of gaps in diabetes care was drug therapy (n = 420/528; 79.6%). Of the drug therapy gaps reported, the most common gap occurred in patients with or at risk for atherosclerotic cardiovascular disease and not on a high-intensity statin (122/420), followed by those not on a moderate statin (106/420). Themes focused on during interviews included integration of diabetes coaching and documentation, impact of Gaps Portal on workflow, and prioritization of gaps. CONCLUSION: Pharmacists most commonly identified drug therapy gaps, predominantly pertaining to statin use, which coincides with a change in the guidelines for statin use in diabetics. Telephone interviews indicated that the Gaps Portal template served as a useful checklist to identify gaps in care during diabetes coaching activities and that the Gaps Portal was useful for reporting and monitoring purposes.

Treatment Approaches for Opioid Use Disorder Offered in US Substance Use Treatment Facilities.

This study compares medication and psychosocial treatments for opioid use disorder, as well as treatments offered at opioid and nonopioid treatment program facilities for commonly co-occurring substance use disorders and mental disorders.

Clinical audit effectively bridges the evidence-practice gap in chronic subdural haematoma management.

BACKGROUND: Placement of a subdural drain after drainage of chronic subdural haematoma (CSDH) has been shown to reduce the rate of recurrence in several randomised controlled trials (RCT). The most recently published RCT was from Cambridge, UK, in 2009. Despite class I evidence for the use of subdural drains, it is unclear whether these results have been translated into clinical practice. In this clinical audit we review the use of subdural drains in our institution before and after the publication of the 2009 RCT results. METHODS: A longitudinal retrospective study was performed on all adults having burr holes for CSDH between January 2009 and January 2014. Case notes were analysed to determine subdural drain use, re-operation for CSDH recurrence and post-operative complications. The audit loop was closed with data collected from August 2015 to January 2016. RESULTS: Thirty-one per cent of patients had subdural drains placed at operation. Drain placement was associated with lower reoperation rates (8% vs. 17%, p = 0.021) without increasing complication rates. Drain usage doubled after publication of the Santarius et al. (2009) trial but we observed persisting and significant variability in drain utilisation by supervising consultants. The use of drains in the department increased from 35% to 75% of all cases after presentation of these results. CONCLUSIONS: The use of subdural drains in our unit reduced recurrence rates following drainage of CSDH and reproduced the results of a 2009 clinical trial. Although the use of subdural drains doubled in the post-trial epoch, significant variability remains in practice. Clinical audit provided an effective tool necessary to drive the implementation of subdural drain placement in our unit.

Gaps in standardized postoperative pain management quality measures: A systematic review.

BACKGROUND: The goal of this study was an assessment of availability postoperative pain management quality measures and National Quality Forum-endorsed measures. Postoperative pain is an important clinical timepoint because poor pain control can lead to patient suffering, chronic opiate use, and/or chronic pain. Quality measures can guide best practices, but it is unclear whether there are measures for managing pain after surgery. METHODS: The National Quality Forum Quality Positioning System, Agency for Healthcare Research and Quality Indicators, and Centers for Medicare and Medicaid Services Measures Inventory Tool databases were searched in November 2019. We conducted a systematic literature review to further identify quality measures in research publications, clinical practice guidelines, and gray literature for the period between March 11, 2015 and March 11, 2020. RESULTS: Our systematic review yielded 1,328 publications, of which 206 were pertinent. Nineteen pain management quality measures were identified from the quality measure databases, and 5 were endorsed by National Quality Forum. The National Quality Forum measures were not specific to postoperative pain management. Three of the non-endorsed measures were specific to postoperative pain. CONCLUSION: The dearth of published postoperative pain management quality measures, especially National Quality Forum-endorsed measures, highlights the need for more rigorous evidence and widely endorsed postoperative pain quality measures to guide best practices.

Epidemiologic Research of Rare Cancers: Trends, Resources, and Challenges.

BACKGROUND: The goals of this project were to assess the status of NCI's rare cancer-focused population science research managed by the Division of Cancer Control and Population Sciences (DCCPS), to develop a framework for evaluation of rare cancer research activities, and to review available resources to study rare cancers. METHODS: Cancer types with an overall age-adjusted incidence rate of less than 20 cases per 100,000 individuals were identified using NCI Surveillance, Epidemiology and End Results (SEER) Program data. SEER data were utilized to develop a framework based on statistical commonalities. A portfolio analysis of DCCPS-supported active grants and a review of three genomic databases were conducted. RESULTS: For the 45 rare cancer types included in the analysis, 123 active DCCPS-supported rare cancer-focused grants were identified, of which the highest percentage (18.7%) focused on ovarian cancer. The developed framework revealed five clusters of rare cancer types. The cluster with the highest number of grants (n = 43) and grants per cancer type (10.8) was the cluster that included cancer types of higher incidence, average to better survival, and high prevalence (in comparison with other rare cancers). Resource review revealed rare cancers are represented in available genomic resources, but to a lesser extent compared with more common cancers. CONCLUSIONS: This article provides an overview of the rare cancer-focused population sciences research landscape as well as information on gaps and opportunities. IMPACT: The findings of this article can be used to develop efficient and comprehensive strategies to accelerate rare cancer research.See related commentary by James V. Lacey Jr, p. 1300.

Clinical Practices in Head and Neck Cancer: A Speech-Language Pathologist Practice Pattern Survey.

OBJECTIVE: Clinical practices of speech-language pathologists (SLP) treating head and neck cancer (HNC) patients range widely despite literature trending toward best practices. This survey study was designed to identify current patterns and assess for gaps in clinical implementation of research evidence. METHOD: A web-based survey was distributed to SLPs via listserv and social media outlets. Descriptive statistics and group calculations were completed to identify trends and associations in responses. RESULTS: Of 152 received surveys, the majority of respondents were hospital-based (86%) and had greater than 5 years of experience (65%). There was group consensus for the use of prophylactic exercise programs (95%), recommendations for SLP intervention during HNC treatment (75%), and use of maintenance programs post-treatment (97%). Conversely, no group consensus was observed for use of pre-treatment swallow evaluations, frequency of service provision, and content of therapy sessions. Variation in clinical decision making was noted in use of prophylactic feeding tubes and number of patients taking nothing by mouth during treatment. No associations were found between years of experience and decision-making practices, nor were any associations found between practice setting and clinical decision making. CONCLUSION: Despite the growing body of literature outlining evidence-based treatment practices for HNC patients, clinical practice patterns among SLPs continue to vary widely resulting in inconsistent patient care across practice settings. As compared to prior similar data, increased alignment with best practices was observed relative to early referrals, implementation of prophylactic intervention programs, and intervention with the SLP during the period of HNC treatment.

Temporal Trends in Revision Total Hip and Knee Arthroplasty from 2008 to 2018: Gaps and Opportunities.

BACKGROUND: An overall assessment of how patient demographic characteristics and comorbidities are improving or worsening can allow better understanding of the value of revision total joint arthroplasty (TJA). Therefore, the purpose of this study was to identify patient demographic characteristics and comorbidities trends and episode-of-care outcome trends from 2008 to 2018 in patients undergoing revision TJA. METHODS: The National Surgical Quality Improvement Program database was queried to identify patient demographic characteristics, comorbidities, and episode-of-care outcomes in patients undergoing revision TJA from 2008 to 2018 (n = 45,706). Pairwise t tests and pairwise chi-square tests were performed on consecutive years with Bonferroni correction. Trends were assessed using the 2-tailed Mann-Kendall test of the temporal trend. RESULTS: Among patients undergoing revision TJA, there was no clinically important difference, from 2008 to 2018, in age, body mass index (BMI), percentages with >40 kg/m2 BMI, diabetes (18.8% to 19%), chronic obstructive pulmonary disease (4.1% to 5.4%), congestive heart failure within 30 days (0% to 1%), or acute renal failure (0% to 0.2%). However, modifiable comorbidities including smoking status (14.7% to 12.0%; p = 0.01), hypertension (66% to 26.0%; p = 0.02), anemia (34.5% to 26.3%; p < 0.001), malnutrition (10.4% to 9.3%; p = 0.004), and overall morbidity or mortality probability have improved, with a decrease in the hospital length of stay and 30-day readmission and a significant increase in home discharge (p < 0.001 for all). CONCLUSIONS: Time-difference analysis demonstrated that the overall health status of patients undergoing revision TJA improved from 2008 to 2018. However, formal time-trend analysis demonstrated improvements to a lesser degree. The multidisciplinary effort to improve value-based metrics including patient comorbidity optimization and episode-of-care outcomes for primary TJA has been shown to potentially have an impact on revision TJA. LEVEL OF EVIDENCE: Prognostic Level IV. See Instructions for Authors for a complete description of levels of evidence.

Outpatient healthcare utilization and prescribing patterns for herpes zoster in United States adults.

Little is known about health resource utilization and treatment patterns for herpes zoster (HZV) after the introduction of HZV virus vaccination. The objective of this study is to characterize trends in HZV utilization, racial disparities, and treatment patterns in the United States. Data from the 1993-2015 National Ambulatory Medical Care Survey were analyzed, including 15,400,000 weighted primary acute HZV visits in adults. Overall, the weighted frequency (95% confidence interval) of HZV visits increased from 1993-1998 to 2007-2015 (1,269,815 [565,455-1,974,175]-8,017,911 [6,424,491-9,611,331], P = 0.0001). HZV visits were associated with African-American (38.8% [35.8-41.7%] vs. 8.2% [7.4-9.0%]) and Hispanic race/ethnicity (12.6% [6.6-18.5%] vs. 8.0% [7.3-8.5%]), public insurance (42.7% [36.6-49.2%] vs. 33.7% [32.2-35.2%]) in comparison with all other visits. Oral antiviral agents were prescribed in 64.3% (58.1-70.1%) of HZV visits. HZV visits were associated with higher rates of opioid prescriptions compared to all other visits (18.4% [14.0-23.9%] vs. 6.1% [5.6-6.6%], P < 0.0001). The limitation is no data on HZV severity. HZV visits increased over time, even after introduction of HZV vaccines. There were significant racial/ethnic and healthcare disparities of, and high rates of opiate and corticosteroid prescriptions at HZV visits. Future efforts are needed to address these practice gaps, and encourage vaccination and evidence-based prescribing in HZV.

Undertreatment of primary hyperparathyroidism in a privately insured US population: Decreasing utilization of parathyroidectomy despite expanding surgical guidelines.

BACKGROUND: Primary hyperparathyroidism is associated with substantial morbidity, including osteoporosis, nephrolithiasis, and chronic kidney disease. Parathyroidectomy can prevent these sequelae but is poorly utilized in many practice settings. METHODS: We performed a retrospective cohort study using the national Optum de-identified Clinformatics Data Mart Database. We identified patients aged ≥35 with a first observed primary hyperparathyroidism diagnosis from 2004 to 2016. Multivariable logistic regression was used to determine patient/provider characteristics associated with parathyroidectomy. RESULTS: Of 26,522 patients with primary hyperparathyroidism, 10,101 (38.1%) underwent parathyroidectomy. Of the 14,896 patients with any operative indication, 5,791 (38.9%) underwent parathyroidectomy. Over time, there was a decreasing trend in the rate of parathyroidectomy overall (2004: 54.4% to 2016: 32.4%, P < .001) and among groups with and without an operative indication. On multivariable analysis, increasing age and comorbidities were strongly, inversely associated with parathyroidectomy (age 75-84, odds ratio 0.50 [95% confidence interval 0.45-0.55]; age ≥85, odds ratio 0.21 [95% confidence interval 0.17-0.26] vs age 35-49; Charlson Comorbidity Index ≥2 vs 0 odds ratio 0.62 [95% confidence interval 0.58-0.66]). CONCLUSION: The majority of US privately insured patients with primary hyperparathyroidism are not treated with parathyroidectomy. Having an operative indication only modestly increases the likelihood of parathyroidectomy. Further research is needed to address barriers to treatment and the gap between guidelines and clinical care in primary hyperparathyroidism.

Epidemiology and treatment patterns for locally advanced or metastatic urothelial carcinoma: a systematic literature review and gap analysis.

BACKGROUND: Several immuno-oncology (IO) agents targeting programmed death-1 or programmed death-ligand 1 (PD-1/L1) are approved second-line therapy options for patients with locally advanced or metastatic urothelial carcinoma (la/mUC) previously treated with platinum-based chemotherapy or first-line options in patients ineligible for cisplatin whose tumors express PD-L1 or for any platinum-based chemotherapy regardless of PD-L1 expression levels. However, literature on the epidemiology of la/mUC is limited, and real-world treatment patterns are not well established, especially with respect to therapies used following IO. OBJECTIVES: To (a) report the epidemiology of urothelial carcinoma (UC) and la/mUC; (b) identify and summarize the published literature on la/mUC treatment patterns, including IO and post-IO treatment; and (c) identify evidence gaps. METHODS: A systematic literature review was conducted using Cochrane dual-reviewer methodology and the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols. Literature databases and selected congress abstracts (2017-2018) were searched for retrospective studies published January 2013-August 2018 in English reporting epidemiological and treatment data (all lines of therapy) for adult patients with la/mUC. RESULTS: Among 6,584 database references and 1,832 congress abstracts screened, 45 publications (29 manuscripts, 1 poster, 15 abstracts; reporting 37 unique studies) were retained. All studies related to treatment patterns, and the majority were from the United States (n = 17), Japan (n = 8), and the United Kingdom (n = 5). Epidemiological data were not identified among the searches thus online registries were leveraged. Among the identified publications, 21 (20 unique) reported on cisplatin versus non-cisplatin regimens, 14 (8 unique) on IO, and 9 (7 unique) on vinflunine. Cisplatin use varied both within and among countries (ranging from 18.4% in 1 U.S. study to 87.9% in 1 Japanese study). The use of IO was higher in later lines of therapy, ranging from 1.4% to 7.9% as first-line therapy to 57.8% as second-line and 64.4% as third-line therapy. Among studies reporting IO discontinuation rates, 41.4%-71% of patients were reported to discontinue IO across the studies, and the median time to discontinuation ranged from 2.7 to 5.8 months. Only 25%-35.5% of patients received subsequent therapy following IO discontinuation; post-IO treatments varied widely. CONCLUSIONS: Additional published data on the country-specific epidemiology of UC and la/mUC are needed, including rates of progression from early-stage disease to la/mUC. There was large variation in treatment rates, particularly cisplatin use, within and across countries. The few published real-world IO studies reported high levels of discontinuation with only a small percentage of patients receiving subsequent therapy. As IO therapies continue to be granted regulatory approval in countries outside the United States and novel therapies gain approval in the post-IO setting, the treatment paradigm for patients with la/mUC is shifting, and future studies with more recent data will be required. DISCLOSURES: This study was funded by Astellas/Seagen. Hepp is an employee of and owns stock in Seagen. Shah was a contractor for Astellas Pharma at the time of the study and owns stock in Pfizer. Smoyer is an employee and shareholder of Envision Pharma Group, paid consultants to Seagen. Vadagam was an employee of Envision Pharma Group, paid consultants to Seagen, at the time of the study. Parts of these data have been presented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 2019 Annual Meeting; May 18-22, 2019; New Orleans, LA.