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AbstractCompared with the general population, hematopoietic cell transplantation (HCT) survivors are at elevated risk for developing solid subsequent neoplasms (SNs). The Center for International Blood and Marrow Transplant Research (CIBMTR) is a key resource for quantifying solid SN incidence following HCT, but the completeness of SN ascertainment is uncertain. Within a cohort of 18 450 CIBMTR patients linked to the California Cancer Registry (CCR), we evaluated the completeness of solid SN data reported to the CIBMTR from 1991 to 2018 to understand the implications of using CIBMTR data alone or combined with CCR data to quantify the burden of solid SNs after HCT. We estimated the cumulative incidence of developing a solid SN, accounting for the competing risk of death. Within the cohort, solid SNs were reported among 724 patients; 15.6% of these patients had an SN reported by CIBMTR only, 36.9% by CCR only, and 47.5% by both. The corresponding cumulative incidence of developing a solid SN at 10 years following a first HCT was 4.0% (95% confidence interval [CI], 3.5-4.4) according to CIBMTR data only, 5.3% (95% CI, 4.9-5.9) according to CCR data only, and 6.3% (95% CI, 5.7-6.8) according to both sources combined. The patterns were similar for allogeneic and autologous HCT recipients. Linking detailed HCT information from CIBMTR with comprehensive SN data from cancer registries provides an opportunity to optimize SN ascertainment for informing follow-up care practices and evaluating risk factors in the growing population of HCT survivors.

ImportanceSignificant insurance status disparities have been demonstrated in head and neck cancer (HNC) outcomes. The effects of insurance status on HNC outcomes may be explained by differential access to high-quality care.ObjectiveTo evaluate the association of insurance status with the quality of the treating hospital and receipt of guideline-compliant care among patients with HNC.Design, setting, and participantsThis retrospective cohort study of data from the California Cancer Registry dataset linked with discharge records and hospital characteristics from the California Department of Health Care Access and Information included adult patients with HNC diagnosed between January 1, 2010, and December 31, 2019. Data were analyzed from May 10, 2023, to March 25, 2024.ExposuresInsurance status: commercial, Medicare, Medicaid, uninsured, other, or unknown.Main outcomes and measuresQuality of the treating hospital (tertiles), receipt of National Comprehensive Cancer Network guideline-compliant care, and overall survival.ResultsA total of 23 933 patients (mean [SD] age, 64.8 [12.3] years; 75.3% male) met the inclusion criteria. Treatment in top-tertile hospitals (hazard ratio, 0.87; 95% CI, 0.79-0.95) was associated with improved overall survival compared with treatment in bottom-tertile hospitals. Medicare (odds ratio [OR], 0.78; 95% CI, 0.73-0.84), Medicaid (OR, 0.60; 95% CI, 0.54-0.66), and uninsured (OR, 0.38; 95% CI, 0.29-0.49) status were associated with lower likelihood of treatment in high-quality hospitals compared with commercial insurance. Among patients with advanced disease, Medicaid (OR, 0.72; 95% CI, 0.62-0.83) and uninsured (OR, 0.64; 95% CI, 0.44-0.93) patients were less likely to receive dual-modality therapy. Among patients with surgically resected advanced disease, Medicaid coverage (OR, 0.73; 95% CI, 0.58-0.93) was associated with lower likelihood of receiving adjuvant radiotherapy.Conclusions and relevanceThis study found significant insurance disparities in quality of care among patients with HNC. These findings highlight the need for continued health insurance reform in the US to improve the quality of insurance coverage, in addition to expanding access to health insurance.

Clonal hematopoiesis (CH) is an age-associated phenomenon leading to an increased risk of both hematologic malignancy and nonmalignant organ dysfunction. Increasingly available genetic testing has made the incidental discovery of CH clinically common yet evidence-based guidelines and effective management strategies to prevent adverse CH health outcomes are lacking. To address this gap, the prospective CHIVE (clonal hematopoiesis and inflammation in the vasculature) registry and biorepository was created to identify and monitor individuals at risk, support multidisciplinary CH clinics, and refine taxonomy and standards of practice for CH risk mitigation. Data from the first 181 patients enrolled in this prospective registry recapitulate the molecular epidemiology of CH from biobank-scale retrospective studies, with DNMT3A, TET2, ASXL1, and TP53 as the most commonly mutated genes. Blood counts across all hematopoietic lineages trended lower in patients with CH. In addition, patients with CH had higher rates of end organ dysfunction, in particular chronic kidney disease. Among patients with CH, variant allele frequency was independently associated with the presence of cytopenias and progression to hematologic malignancy, whereas other common high-risk CH clone features were not clear. Notably, accumulation of multiple distinct high-risk clone features was also associated with cytopenias and hematologic malignancy progression, supporting a recently published CH risk score. Surprisingly, ∼30% of patients enrolled in CHIVE from CH clinics were adjudicated as not having clonal hematopoiesis of indeterminate potential, highlighting the need for molecular standards and purpose-built assays in this field. Maintenance of this well-annotated cohort and continued expansion of CHIVE to multiple institutions are underway and will be critical to understanding how to thoughtfully care for this patient population.

BackgroundChildhood cancers are associated with high mortality and morbidity, and some maternal prescription drug use during pregnancy has been implicated in cancer risk. There are few studies on the effects of hypertension, preeclampsia, and the use of antihypertensives in pregnancy on children's cancer risks.ObjectiveThis population-based cohort study analyzed the relationship between hypertension, preeclampsia, and antihypertensives taken during pregnancy and the risks of childhood cancers in the offspring.MethodsData on all children born in Taiwan between 2004 and 2015 (N = 2,294,292) were obtained from the Maternal and Child Health Database. This registry was linked with the National Health Insurance Database and Cancer Registry to get the records of maternal use of diuretics or other antihypertensives in pregnancy and records of children with cancer diagnosed before 13 years. We used Cox proportional hazard modeling to estimate the influence of maternal health conditions and antihypertensive drug exposure on the risks of developing childhood cancers.ResultsOffspring of mothers with hypertension (chronic or gestational) had a higher risk of acute lymphocytic lymphoma [hazard ratio (HR) = 1.87, 95% Confidence Interval (CI) 1.32 - 2.65] and non-Hodgkin's lymphoma (HR = 1.96, 95% CI 1.34 - 2.86). We estimated only a weak increased cancer risk in children whose mothers used diuretics (HR = 1.16, 95% CI 0.77 - 1.74) or used antihypertensives other than diuretics (HR = 1.15, 95% CI 0.86 - 1.54) before birth.ConclusionsIn this cohort study, children whose mothers had chronic and gestational hypertension had an increased risk of developing childhood cancer.

The Get With The Guidelines-Stroke program which, began 20 years ago, is one of the largest and most important nationally representative disease registries in the United States. Its importance to the stroke community can be gauged by its sustained growth and widespread dissemination of findings that demonstrate sustained increases in both the quality of care and patient outcomes over time. The objectives of this narrative review are to provide a brief history of Get With The Guidelines-Stroke, summarize its major successes and impact, and highlight lessons learned. Looking to the next 20 years, we discuss potential challenges and opportunities for the program.

To accelerate and facilitate clinical trials, the Ataxia Global Initiative (AGI) was established as a worldwide research platform for trial readiness in ataxias. One of AGIs major goals is the harmonization and standardization of outcome assessments. Clinical outcome assessments (COAs) that describe or reflect how a patient feels or functions are indispensable for clinical trials, but similarly important for observational studies and in routine patient care. The AGI working group on COAs has defined a set of data including a graded catalog of COAs that are recommended as a standard for future assessment and sharing of clinical data and joint clinical studies. Two datasets were defined: a mandatory dataset (minimal dataset) that can ideally be obtained during a routine clinical consultation and a more demanding extended dataset that is useful for research purposes. In the future, the currently most widely used clinician-reported outcome measure (ClinRO) in ataxia, the scale for the assessment and rating of ataxia (SARA), should be developed into a generally accepted instrument that can be used in upcoming clinical trials. Furthermore, there is an urgent need (i) to obtain more data on ataxia-specific, patient-reported outcome measures (PROs), (ii) to demonstrate and optimize sensitivity to change of many COAs, and (iii) to establish methods and evidence of anchoring change in COAs in patient meaningfulness, e.g., by determining patient-derived minimally meaningful thresholds of change.

BACKGROUND: Recent clinical trials established the benefit of dual antiplatelet therapy with aspirin and clopidogrel (DAPT-AC) in early-presenting patients with minor ischemic stroke. However, the impact of these trials over time on the use and outcomes of DAPT-AC among the patients with nonminor or late-presenting stroke who do not meet the eligibility criteria of these trials has not been delineated. METHODS AND RESULTS: In a multicenter stroke registry, this study examined yearly changes from April 2008 to August 2022 in DAPT-AC use for stroke patients ineligible for CHANCE/POINT (Clopidogrel in High-Risk Patients with Acute Nondisabling Cerebrovascular Events/Platelet-Oriented Inhibition in New TIA and Minor Ischemic Stroke) clinical trials due to National Institutes of Health Stroke Scale >4 or late arrival beyond 24 hours of onset. A total of 32 118 patients (age, 68.1±13.1 years; male, 58.5%) with National Institutes of Health Stroke Scale of 4 (interquartile range, 1-7) were analyzed. In 2008, DAPT-AC was used in 33.0%, other antiplatelets in 62.7%, and no antiplatelet in 4.3%. The frequency of DAPT-AC was relatively unchanged through 2013, when the CHANCE trial was published, and then increased steadily, reaching 78% in 2022, while other antiplatelets decreased to 17.8% in 2022 (Ptrend

ObjectivesVedolizumab (VDZ) and ustekinumab (UST) are second-line treatments in pediatric patients with ulcerative colitis (UC) refractory to antitumor necrosis factor (anti-TNF) therapy. Pediatric studies comparing the effectiveness of these medications are lacking. Using a registry from ImproveCareNow (ICN), a global research network in pediatric inflammatory bowel disease, we compared the effectiveness of UST and VDZ in anti-TNF refractory UC.MethodsWe performed a propensity-score weighted regression analysis to compare corticosteroid-free clinical remission (CFCR) at 6 months from starting second-line therapy. Sensitivity analyses tested the robustness of our findings to different ways of handling missing outcome data. Secondary analyses evaluated alternative proxies of response and infection risk.ResultsOur cohort included 262 patients on VDZ and 74 patients on UST. At baseline, the two groups differed on their mean pediatric UC activity index (PUCAI) (p = 0.03) but were otherwise similar. At Month 6, 28.3% of patients on VDZ and 25.8% of those on UST achieved CFCR (p = 0.76). Our primary model showed no difference in CFCR (odds ratio: 0.81; 95% confidence interval [CI]: 0.41-1.59) (p = 0.54). The time to biologic discontinuation was similar in both groups (hazard ratio: 1.26; 95% CI: 0.76-2.08) (p = 0.36), with the reference group being VDZ, and we found no differences in clinical response, growth parameters, hospitalizations, surgeries, infections, or malignancy risk. Sensitivity analyses supported these findings of similar effectiveness.ConclusionsUST and VDZ are similarly effective for inducing clinical remission in anti-TNF refractory UC in pediatric patients. Providers should consider safety, tolerability, cost, and comorbidities when deciding between these therapies.

BACKGROUND: Myocardial infarction secondary to spontaneous coronary artery dissection (SCAD) can be traumatic and potentially trigger posttraumatic stress disorder (PTSD). In a large, multicenter, registry-based cohort, we documented prevalence of lifetime and past-month SCAD-induced PTSD, as well as related treatment seeking, and examined a range of health-relevant correlates of SCAD-induced PTSD. METHODS AND RESULTS: Patients with SCAD were enrolled in the iSCAD (International SCAD) Registry. At baseline, site investigators completed medical report forms, and patients reported demographics, medical/SCAD history, psychosocial factors (including SCAD-induced PTSD symptoms), health behaviors, and health status via online questionnaires. Of 1156 registry patients, 859 patients (93.9% women; mean age, 52.3 years) completed questionnaires querying SCAD-induced PTSD. Nearly 35% (n=298) of patients met diagnostic criteria for probable SCAD-induced PTSD in their lifetime, and 6.4% (n=55) met criteria for probable past-month PTSD. Of 811 patients ever reporting any SCAD-induced PTSD symptoms, 34.8% indicated seeking treatment for this distress. However, 46.0% of the 298 patients with lifetime probable SCAD-induced PTSD diagnoses reported never receiving trauma-related treatment. Younger age at first SCAD, fewer years since SCAD, being single, unemployed status, more lifetime trauma, and history of anxiety were associated with greater past-month PTSD symptom severity in multivariable regression models. Greater past-month SCAD-induced PTSD symptoms were associated with greater past-week sleep disturbance and worse past-month disease-specific health status when adjusting for various risk factors. CONCLUSIONS: Given the high prevalence of SCAD-induced PTSD symptoms, efforts to support screening for these symptoms and connecting patients experiencing distress with empirically supported treatments are critical next steps. REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifier: NCT04496687.

BACKGROUND: Approximately half of patients who achieve successful reperfusion do not achieve functional independence. The present study sought to investigate the clinical outcomes and safety of intraarterial or intravenous tirofiban as adjunct therapy in patients with acute basilar artery occlusion who had achieved successful recanalization with endovascular treatment. METHODS AND RESULTS: In the national, prospective BASILAR (Endovascular Treatment for Acute Basilar Artery Occlusion Study) registry, 458 patients who met inclusion criteria were divided into 3 groups based on tirofiban administration (no tirofiban, n=262; intravenous tirofiban, n=101; intraarterial+intravenous tirofiban, n=95). Their clinical outcomes were compared with 90-day modified Rankin Scale scores. Adjusted odds ratios (aORs) and 95% CIs were obtained by logistic regression models and propensity score matching. Safety outcomes included any intracranial hemorrhage (ICH), symptomatic ICH, and mortality. Among 458 included patients, 184 (40.2%) achieved a favorable outcome (modified Rankin Scale score 0-3). There were no differences between the intravenous tirofiban group and the no tirofiban group in terms of safety and clinical outcomes (all P>0.05). Compared with the no tirofiban group, the intraarterial+intravenous tirofiban group had higher odds of 90-day modified Rankin Scale score 0 to 3 (aOR, 2.44 [95% CI, 1.30-4.64], P=0.006) and lower 3-month mortality (aOR, 0.38 [95% CI, 0.19-0.71], P=0.002) without an increase in any ICH (aOR, 0.34 [95% CI, 0.09-1.01], P=0.07) or symptomatic ICH (aOR, 0.23 [95% CI, 0.03-0.90], P=0.05). Similar results of intraarterial+intravenous tirofiban on improving clinical outcomes were detected in novel cohorts constructed by propensity score matching. CONCLUSIONS: Intraarterial+intravenous rather than intravenous tirofiban improved clinical outcomes without increasing the frequency of symptomatic ICH among patients with basilar artery occlusion after successful endovascular treatment. Further studies are needed to delineate the roles of intraarterial+intravenous tirofiban in patients with basilar artery occlusion receiving endovascular treatment.

INTRODUCTION AND OBJECTIVE: The ConcePTION project aims to improve the way medication use during pregnancy is studied. This includes exploring the possibility of developing a distributed data processing and analysis infrastructure using a common data model that could form a foundational platform for future surveillance and research. A prerequisite would be that data from various data access providers (DAPs) can be harmonised according to an agreed set of standard rules concerning the structure and content of the data. To do so, a reference framework of core data elements (CDEs) recommended for primary data studies on drug safety during pregnancy was previously developed. The aim of this study was to assess the ability of several public and private DAPs using different primary data sources focusing on multiple sclerosis, as a pilot, to map their respective data variables and definitions with the CDE recommendations framework. METHODS: Four pregnancy registries (Gilenya, Novartis; Aubagio, Sanofi; the Organization of Teratology Information Specialists [OTIS]; Aubagio, Sanofi; the Dutch Pregnancy Drug Register, Lareb), two enhanced pharmacovigilance programmes (Gilenya PRIM, Novartis; MAPLE-MS, Merck Healthcare KGaA) and four Teratology Information Services (UK TIS, Jerusalem TIS, Zerifin TIS, Swiss TIS) participated in the study. The ConcePTION primary data source CDE includes 51 items covering administrative functions, the description of pregnancy, maternal medical history, maternal illnesses arising in pregnancy, delivery details, and pregnancy and infant outcomes. For each variable in the CDE, the DAPs identified whether their variables were: identical to the one mentioned in the CDE; derived; similar but with a divergent definition; or not available. RESULTS: The majority of the DAP data variables were either directly taken (85%, n = 305/357, range 73-94% between DAPs) or derived by combining different variables (12%, n = 42/357, range 0-24% between DAPs) to conform to the CDE variables and definitions. For very few of the DAP variables, alignment with the CDE items was not possible, either because of divergent definitions (1%, n = 3/357, range 0-2% between DAPs) or because the variables were not available (2%, n = 7/357, range 0-4% between DAPs). CONCLUSIONS: Data access providers participating in this study presented a very high proportion of variables matching the CDE items, indicating that alignment of definitions and harmonisation of data analysis by different stakeholders to accelerate and strengthen pregnancy pharmacovigilance safety data analyses could be feasible.

BACKGROUND: Oral anti-coagulants (OAC) are the intervention for the prevention of stroke, which consistently improve clinical outcomes and survival among patients with atrial fibrillation (AF). The main purpose of this study is to identify problems in OAC utilization among hospitalized patients with AF in China. METHODS: Using data from the Improving Care for Cardiovascular Disease in China-Atrial Fibrillation (CCC-AF) registry, guideline-recommended OAC use in eligible patients was assessed. RESULTS: A total of 52,530 patients with non-valvular AF were enrolled from February 2015 to December 2019, of whom 38,203 were at a high risk of stroke, 9717 were at a moderate risk, and 4610 were at a low risk. On admission, only 20.0% (6075/30,420) of patients with a diagnosed AF and a high risk of stroke were taking OAC. The use of pre-hospital OAC on admission was associated with a lower risk of new-onset ischemic stroke/transient ischemic attack among the diagnosed AF population (adjusted odds ratio: 0.54, 95% confidence interval: 0.43-0.68; P  70% of AF patients were at a high risk of stroke. Although poor performance on guideline-recommended OAC use was found in this study, over time the CCC-AF project has made progress in stroke prevention in the Chinese AF population.Registration:ClinicalTrials.gov, NCT02309398.

BackgroundSince September 2005 the International Committee of Medical Journal Editors has required that trials be registered in accordance with the World Health Organization (WHO) minimum dataset, in order to be considered for publication. The objective is to evaluate registries' and individual trial records' compliance with the 2006 version of the WHO minimum data set.MethodsA retrospective evaluation of 21 online clinical trial registries (international, national, specialty, pharmaceutical industry and local) from April 2005 to February 2007 and a cross-sectional evaluation of a stratified random sample of 610 trial records from the 21 registries.ResultsAmong 11 registries that provided guidelines for registration, the median compliance with the WHO criteria were 14 out of 20 items (range 6 to 20). In the period April 2005-February 2007, six registries increased their compliance by six data items, on average. None of the local registry websites published guidelines on the trial data items required for registration. Slightly more than half (330/610; 54.1%, 95% CI 50.1% - 58.1%) of trial records completed the contact details criteria while 29.7% (181/610, 95% CI 26.1% - 33.5%) completed the key clinical and methodological data fields.ConclusionWhile the launch of the WHO minimum data set seemed to positively influence registries with better standardisation of approaches, individual registry entries are largely incomplete. Initiatives to ensure quality assurance of registries and trial data should be encouraged. Peer reviewers and editors should scrutinise clinical trial registration records to ensure consistency with WHO's core content requirements when considering trial-related publications.

BACKGROUND: Subjective cognitive decline (SCD) is defined as an individuals perception of sustained cognitive decline compared to their normal state while still performing within boundaries for normal functioning. Demographic, psychosocial and medical factors have been linked to age-related cognitive decline, and Alzheimers dementia (AD). However, their relation to risk for SCD remains unclear. This study aims to identify demographic factors, psychosocial and cardiovascular health associated with SCD within the Brain Health Registry (BHR) online cohort. METHODS: Participants aged 55+ (N=27,596) in the BHR self-reported SCD measured using the Everyday Cognition Scale (ECog) and medical conditions, depressive symptoms, body mass index, quality of sleep, health, family history of AD, years of education, race, ethnicity and gender. Multivariable linear regression was used to examine whether SCD was associated with demographic, psychosocial, and medical conditions. RESULTS: We found that advanced age, depressive symptoms, poorer sleep quality and poorer quality of health were positively associated with more self-reported SCD in all models. No race or ethnicity differences were found in association with SCD. Males who reported alcohol and tobacco use or underweight BMI had higher ECog scores compared with females. CONCLUSION: In addition to well-established risk factors for cognitive decline, such as age, our study consistently and robustly identified a strong association between psychosocial factors and self-reported cognitive decline in an online cohort. These findings provide further evidence that psychosocial health plays a pivotal role in comprehending the risk of SCD and early-stage cognitive ageing. Our findings emphasise the significance of psychosocial factors within the broader context of cardiovascular and demographic risk factors.

IntroductionWe elicited Vietnamese Americans' perspectives on culturally appropriate recruitment into a new research registry: Collaborative Approach for Asian Americans, Native Hawaiians, and Pacific Islanders (AANHPIs) Research and Education (CARE).MethodsThree focus groups were conducted with 21 Vietnamese Americans. Topics included knowledge about and experiences with research, outreach and recruitment methods for research participation and registry enrollment, and views about research incentives. Focus group transcripts were analyzed thematically.ResultsMean age of participants was 41 years (range 18-73), 57% were male, 86% were non-US born, and 81% had never participated in a research study. Themes that emerged included (1) motivations to participate in research to gain knowledge: for oneself, for family's benefit, and for the Vietnamese American community as a whole; (2) necessity of trustworthy and credible individuals/spokespersons to promote the research initiative; (3) recruitment strategies that are age-specific and culturally appropriate, and (4) importance of monetary incentives.ConclusionFindings from this study will be used to guide recruitment into and engagement with CARE among Vietnamese Americans but are also relevant for other registries aiming to diversify their participants.

BACKGROUND: Early identification of deficits in our ability to perceive odors is important as many normal (i.e., aging) and pathological (i.e., sinusitis, viral, neurodegeneration) processes can result in diminished olfactory function. To realistically enable population-level measurements of olfaction, validated olfaction tests must be capable of being administered outside the research laboratory and clinical setting. AIM: The purpose of this study was to determine the feasibility of remotely testing olfactory performance using a test that was developed with funding from the National Institutes of Health as part of a ready-to-use, non-proprietary set of measurements useful for epidemiologic studies (NIH Toolbox Odor ID Test). MATERIALS AND METHODS: Eligible participants older than 39 years and active (within 6 months) in the Brain Health Registry (BHR), an online cognitive assessment platform which connects participants with researchers, were recruited for this study. Interested participants were mailed the NIH Toolbox Odor ID Test along with instructions on accessing a website to record their responses. Data obtained from subjects who performed the test at home was compared to the normative data collected when the NIH Toolbox Odor ID Test was administered by a tester in a research setting and validated against the Smell Identification Test. The age-range and composition of the population ensured we had the ability to observe both age-related decline and gender-related deficits in olfactory ability, as shown in the experimental setting. RESULTS: We observed that age-associated olfactory decline and gender-associated performance was comparable to performance on the administered test. Self-administration of this test showed the age-related loss in olfactory acuity, F(4, 1156)=14.564, p

BACKGROUND: Unsupervised online cognitive assessments have demonstrated promise as an efficient and scalable approach for evaluating cognition in aging, and Alzheimers disease and related dementias. OBJECTIVES: The aim of this study was to evaluate the feasibility, usability, and construct validity of the Paired Associates Learning task from the Cambridge Neuropsychological Test Automated Battery® in adults enrolled in the Brain Health Registry. DESIGN, SETTING, PARTICIPANTS, MEASUREMENTS: The Paired Associates Learning task was administered to Brain Health Registry participants in a remote, unsupervised, online setting. In this cross-sectional analysis, we 1) evaluated construct validity by analyzing associations between Paired Associates Learning performance and additional participant registry data, including demographics, self- and study partner-reported subjective cognitive change (Everyday Cognition scale), self-reported memory concern, and depressive symptom severity (Patient Health Questionnaire-9) using multivariable linear regression models; 2) determined the predictive value of Paired Associates Learning and other registry variables for identifying participants who self-report Mild Cognitive Impairment by employing multivariable binomial logistic regressions and calculating the area under the receiver operator curve; 3) investigated feasibility by looking at task completion rates and statistically comparing characteristics of task completers and non-completers; and 4) evaluated usability in terms of participant requests for support from BHR related to the assessment. RESULTS: In terms of construct validity, in participants who took the Paired Associates Learning for the first time (N=14,528), worse performance was associated with being older, being male, lower educational attainment, higher levels of self- and study partner-reported decline, more self-reported memory concerns, greater depressive symptom severity, and self-report of Mild Cognitive Impairment. Paired Associates Learning performance and Brain Health Registry variables together identified those with self-reported Mild Cognitive Impairment with moderate accuracy (areas under the curve: 0.66-0.68). In terms of feasibility, in a sub-sample of 29,176 participants who had the opportunity to complete Paired Associates Learning for the first time in the registry, 14,417 started the task. 11,647 (80.9% of those who started) completed the task. Compared to those who did not complete the task at their first opportunity, those who completed were older, had more years of education, more likely to self-identify as White, less likely to self-identify as Latino, less likely to have a subjective memory concern, and more likely to report a family history of Alzheimers disease. In terms of usability, out of 8,395 received requests for support from BHR staff via email, 4.4% (n=374) were related to PAL. Of those, 82% were related to technical difficulties. CONCLUSIONS: Our findings support moderate feasibility, good usability, and construct validity of cross-sectional Paired Associates Learning in an unsupervised online registry, but also highlight the need to make the assessment more inclusive and accessible to individuals from ethnoculturally and socioeconomically diverse communities. A future, improved version could be a scalable, efficient method to assess cognition in many different settings, including clinical trials, observational studies, healthcare, and public health.

Background: India is a vast and diverse country with existing variations in the frequency and distribution of cancers across its various parts. In regions lacking population-based cancer registries (PBCRs) in a vast country like India, hospital-based cancer registry (HBCR) data become an important source of information on the trends and patterns of a region. To determine the numerical trends of cases of the top ten cancer sites reporting to HBCR of a tertiary care cancer center in Bihar from 2014 to 2021. Materials and Methods: The details of all histopathologically confirmed cancer cases registered in the HBCR department of radiation oncology, State Cancer Institute, Indira Gandhi Institute of Medical Sciences, Patna, India between January 2014 and December 2021 were included in this retrospective observational study. All these cases were grouped site-wise and listed in descending order of the total number of cases reported in each group. Cross-tabulation with age and sex distribution was done. The frequency distribution of the top ten leading cancers for every consecutive calendar year was plotted in line diagrams for time trend analysis. Statistical Package for the Social Sciences (IBM SPSS Statistics for Windows, Version 20.0. Armonk, NY: IBM Corp.). was used for analysis. Annual percent change (APC) was determined for the number of cases of all ten cancer sites using joinpoint regression analysis (Joinpoint Regression Software, Version 4.0.4--May 2013; Statistical Methodology and Applications Branch, Surveillance Research Program of the US National Cancer Institute; Bethesda, MD, USA). Results: Out of 32,057 total cancer cases registered between Jan 2014 and Dec 2021, 21,848 patients (68.2%) cases constituted the top ten cancers. The top ten cancers among both sexes were cancer gallbladder (n = 4204, 13.1%), head and neck (n = 3395, 10.6%), breast (n = 3392, 10.6%), lung (n = 2069, 6.5%), cervix (n = 2039, 6.4%), hematolymphoid (n = 1930, 6.0%), liver (n = 1572, 4.9%), stomach (n = 1116, 3.5%), ovary (n = 1103, 3.4%), and colon-rectum (n = 1028, 3.2%). Except for cervical and hematolymphoid cancers, the rest all showed a rising trend over consecutive years. Conclusion: Cancer of the gallbladder continues to be among the most common cancers in the region. Focused research in all aspects of this deadly disease is needed. Strengthening of prevention and screening programs for common cancers and upliftment of the existing infrastructure for diagnosis and treatment of cancer in the region are necessitated. [ABSTRACT FROM AUTHOR]

Burn registers are an important source of surveillance data on injury intent. These data are considered essential to inform prevention activities. In South Asia, intentional burn injuries are thought to disproportionately affect women. Assessment of injury intent is difficult because it is influenced by personal, family, social, and legal sensitivities. This can introduce misclassification into data, and bias analyses. We conducted a descriptive, hypothesis generating study to explore misclassification of injury intent using data from a newly digitised single centre burn register in south India. Data from 1st February 2016 to 28th February 2022 were analysed. All patients in the data set were included in the study (n = 1930). Demographic and clinical characteristics for patients are described for each classification of injury intent. All data cleaning and analyses were completed using RStudio. Injury intent data were missing for 12.6% of cases. It was the most commonly missing variable in the data set. "Accidental" injuries had a similar distribution over time, age, and total body surface area (TBSA) for males and females. "Homicidal" injuries were more common in females. Injuries reported as "Suicidal" affected men and women equally. A decrease in reporting of "Suicidal" injuries in females corresponded to an increase in high TBSA injuries classified as 'Other' or with missing data. Overwriting of injury intent was present in 1.5% of cases. The overwritten group had a greater proportion of females (62.1% vs. 48.5%) and higher median TBSA (77.5% vs. 27.5%) compared to the group where intent was not overwritten. Our findings indicate that some subgroups, such as females with high TBSA burns, appear to be more likely to be misclassified and should be the focus of future research. They also highlight that quality of surveillance data could be improved by recording of clinical impression, change in patient reported intent, and use of a common data element for intent to standardise data collection. We also recommend that injury intent is recorded as a unique variable and should not be mixed with other elements of injury causation (e.g. mechanism). Although this is a single centre study, the methods will be of interest to those who utilise routinely collected data and wish to reduce misclassification of this important variable. • We explored misclassification of injury intent using burn register data from India. • Injury intent was the most commonly missing variable in the data set (12.6%). • Patients with overwriting of intent predominantly included females and large burns. • Females with burns categorised as 'other' or with missing data may be misclassified. • These findings highlight opportunities to improve quality of surveillance data. [ABSTRACT FROM AUTHOR]

The advancement of medical toxicology knowledge has traditionally relied on case reports and case series because of the ethical challenges involved in studying poisoned patients. The growing availability of several large databases and registries now allows researchers to describe and analyze patterns in poisoned patients who share a particular exposure, outcome, or condition. A large database or registry can be useful in generating hypotheses, supporting extramural funding applications, and planning more rigorous studies. Knowing how to access and interpret data in registries such as NPDS, NHAMCS, and HCUP is essential for all stakeholders engaged in medical toxicology research. This review describes the strengths and limitations of different toxicology-relevant registries and databases and how to leverage these powerful tools to advance the science in the field of medical toxicology. [ABSTRACT FROM AUTHOR]

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Copyright of Innere Medizin (2731-7080) is the property of Springer Nature and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

Recurrence after colorectal cancer resection is rarely documented in the general population while a key clinical determinant for patient survival. We identified 8785 patients with colorectal cancer diagnosed between 2010 and 2013 and clinically followed up to 2020 in 15 cancer registries from seven European countries (Bulgaria, Switzerland, Germany, Estonia, France, Italy, and Spain). We estimated world age‐standardized net survival using a flexible cumulative excess hazard model. Recurrence rates were calculated for patients with initially resected stage I, II, or III cancer in six countries, using the actuarial survival method. The proportion of nonmetastatic resected colorectal cancers varied from 58.6% to 78.5% according to countries. The overall 5‐year net survival by country ranged between 60.8% and 74.5%. The absolute difference between the 5‐year survival extremes was 12.8 points for stage II (Bulgaria vs Switzerland), 19.7 points for stage III (Bulgaria vs. Switzerland) and 14.8 points for Stage IV and unresected cases (Bulgaria vs. Switzerland or France). Five‐year cumulative rate of recurrence among resected patients with stage I–III was 17.7%. As compared to the mean of the whole cohort, the risk of developing a recurrence did not differ between countries except a lower risk in Italy for both stage I/II and stage III cancers and a higher risk in Spain for stage III. Survival after colorectal cancer differed across the concerned European countries while there were slight differences in recurrence rates. Population‐based collection of cancer recurrence information is crucial to enhance efforts for evidence‐based management of colorectal cancer follow up. [ABSTRACT FROM AUTHOR]

AbstractObjectiveMethodsResultsConclusionsTo evaluate the impact of sociodemographic and clinical sex/gender-associated factors on dementia severity at the time of diagnosis.Retrospective, cross-sectional study using 2007–2020 data from the Registry of Dementia of Girona (ReDeGi), collecting information from new dementia diagnoses in the seven hospitals of the Health Region of Girona (Northeast region of Catalonia, Spain). Sociodemographic and clinical variables were compared by sex and dementia severity at diagnosis. A multivariate analysis stratified by sex evaluated the risk of having a moderate/severe dementia diagnosis.Of 9614 new dementia cases, 6040 (62.8%) were women, and 3574 (37.2%) were men. Women and men aged 75–85 years had 27.9% (p = .003) and 43.1% (p < .001) less risk of moderate/severe diagnosis, respectively, than those >85. Being institutionalized increased the risk of moderate/severe diagnosis more in women (159.9%; p < .001) than in men (114.8%; p = .030). Being single and having a higher education increased the risk of moderate/severe diagnosis by 76.2% (p = .039) and 69.8% (p = .021), respectively, only in women.Age, education level, marital status, and place of residence were differentially associated with moderate/severe dementia at the time of diagnosis in women and men, indicating sex/gender differences in dementia severity at diagnosis, with an increased impact on women. [ABSTRACT FROM AUTHOR]

Survival analysis (also referred to as time-to-event analysis) is the study of the time elapsed from a starting date to some event of interest. In practice, these analyses can be challenging and, if methodological errors are to be avoided, require the application of appropriate techniques. By using simulations and real-life data based on the French national registry of patients with primary immunodeficiencies (CEREDIH), we sought to highlight the basic elements that need to be handled correctly when performing the initial steps in a survival analysis. We focused on non-parametric methods to deal with right censoring, left truncation, competing risks, and recurrent events. Our simulations show that ignoring these aspects induces a bias in the results; we then explain how to analyze the data correctly in these situations using non-parametric methods. Rare disease registries are extremely valuable in medical research. We discuss the application of appropriate methods for the analysis of time-to-event from the CEREDIH registry. The objective of this tutorial article is to provide clinicians and healthcare professionals with better knowledge of the issues facing them when analyzing time-to-event data. [ABSTRACT FROM AUTHOR]

Background: Metabolic syndrome (MS) has emerged as a significant global health concern. The relationship between MS and the risk of cancer doesn't seem clear, whether examining by components or in combination. The objective of this study is to examine the relationship between MS, its components, and the overall risk of cancer, including the risk of 13 specific cancer types. Methods: We included 3,918,781 individuals aged 40 years or older sourced from the SIDIAP database between 2008 and 2017. Cox models were employed with MS components and their combinations. A subsample was created using a matched cohort (by age and sex). Incidence curves were computed to determine the time elapsed between the date of having 1–5 MS components and cancer incidence, compared to matched participants with no MS components, which showed that individuals who had one MS component experienced a greater incidence of cancer over 5 and 10 years than individuals with no MS, and the incidence rose with an increase in the number of MS components. Results: Individuals exposed to MS components were diagnosed with cancer earlier than those who were not exposed to them. In the Cox model, HDL (HR 1.46, 95% CI: 1.41–1.52) and Glycemia (HR 1.40, 95% CI: 1.37–1.44) were the individual combinations with the highest risk of overall cancer. In combinations with two components, the highest HR was HDL+Glycemia (HR 1.52, 95% CI: 1.45–1.59) and Glycemia+HBP (HR 1.48, 95% CI: 1.45–1.50). In combinations with three components, the highest HR was HDL+Glycemia+HBP (HR 1.58, 95% CI: 1.55–1.62). Conclusion: In summary, having one or more MS components raises the risk of developing at least 11 cancer types and these risk differ according to type of component included. Some sex differences are also observed. Our findings suggest that implementing prevention measures aimed at specific MS components may lower the risk of various cancer types. [ABSTRACT FROM AUTHOR]

Background: Multiple myeloma often presents with vague and non-specific symptoms. Many patients are diagnosed in unplanned rather than elective (planned) diagnostic pathways. This study investigates the diagnosis of multiple myeloma in unplanned pathways and the association with patient characteristics, disease profile, and survival. Methods: We conducted a nationwide register-based study, including all patients diagnosed with multiple myeloma in Denmark in 2014–2018. Patients were categorised as diagnosed in an unplanned pathway if registered with an acute admission within 30 days prior to the multiple myeloma diagnosis and no other previously registered pathway to this diagnosis. Unplanned pathways were compared to all other pathways combined. Results: We included 2,213 patients diagnosed with multiple myeloma, hereof 32% diagnosed in an unplanned pathway. Comorbidity, no prior cancer diagnosis, a history of few visits to the general practitioner (GP), multiple myeloma complications at diagnosis, high-risk cytogenetics, and advanced cancer stage were associated with a higher probability of being diagnosed in an unplanned pathway. For example, 24.4% (95% confidence interval (CI): 21.8–27.0) of patients with low comorbidity (Charlson Comorbidity Index (CCI) score 0) were diagnosed in an unplanned pathway as were 50.9% (95% CI: 45.6–56.1) of patients with high comorbidity (CCI score 3+). For patients with dialysis need at the time of diagnosis the probability was 66.0% (95% CI 54.2–77.8) and 30.9% (95% CI: 28.9–32.9) for patients with no dialysis need. Patients diagnosed in an unplanned pathway had inferior survival (hazard ratio 1.44 (95% CI: 1.26–1.64)). However, this association was not seen in analyses restricted to patients surviving for more than three years. Conclusions: High comorbidity level, few usual GP visits, advanced disease status at diagnosis, and complications were associated with diagnosis in an unplanned pathway. Further, patients diagnosed in an unplanned pathway had inferior survival. Promoting earlier diagnosis and preventing unplanned pathways may help improve survival in multiple myeloma. [ABSTRACT FROM AUTHOR]

Background Objectives Methods Results Conclusions Biologics are among the most effective therapies for psoriasis. However, many patients are only introduced to them at advanced stages of the disease course.Our aim was to identify predictors of initiating biologic therapy in patients with psoriasis and compare patients initiating biologics early versus late in their disease course.Kaplan–Meier curves visualized time to biologic initiation, while Cox regression models further explored variables as predictors of biologic initiation. Mann–Whitney U and chi‐squared tests compared patients who started biologics early with those who began biologics later in the disease course.Our primary analysis included 233 psoriasis patients. Cox regression showed that age at diagnosis (P = 0.007), general physical well‐being (P = 0.02), and nail psoriasis severity (P = 0.02) were significantly associated with time to biologic initiation. Our secondary analysis, the comparisons between patients starting biologics early versus later in the disease course, included a total of 378 patients. The median (interquartile range [IQR]) age at diagnosis was 34.5 (25.0–51.2) years for patients initiating biologics within 5 years, compared to 22.0 (15.0–32.8) years for patients initiating biologics later (P < 0.0001). The median (IQR) age at initiation was 37.0 (27.0–53.2) and 45.0 (36.0–55.0) years for patients initiating biologics earlier versus later than 5 years (P = 0.04).Age at diagnosis, general well‐being, and severity of nail psoriasis significantly predicted future initiation of biologic treatment. Patients initiating biologics early in their disease course were generally older at diagnosis but younger at the time of biologic initiation compared to patients initiating biologics later in their disease course. [ABSTRACT FROM AUTHOR]

Background Empirical evaluation of inverse probability weighting (IPW) for self-selection bias correction is inaccessible without the full source population. We aimed to: (i) investigate how self-selection biases frequency and association measures and (ii) assess self-selection bias correction using IPW in a cohort with register linkage. Methods The source population included 17 936 individuals invited to the Copenhagen Aging and Midlife Biobank during 2009–11 (ages 49–63 years). Participants counted 7185 (40.1%). Register data were obtained for every invited person from 7 years before invitation to the end of 2020. The association between education and mortality was estimated using Cox regression models among participants, IPW participants and the source population. Results Participants had higher socioeconomic position and fewer hospital contacts before baseline than the source population. Frequency measures of participants approached those of the source population after IPW. Compared with primary/lower secondary education, upper secondary, short tertiary, bachelor and master/doctoral were associated with reduced risk of death among participants (adjusted hazard ratio [95% CI]: 0.60 [0.46; 0.77], 0.68 [0.42; 1.11], 0.37 [0.25; 0.54], 0.28 [0.18; 0.46], respectively). IPW changed the estimates marginally (0.59 [0.45; 0.77], 0.57 [0.34; 0.93], 0.34 [0.23; 0.50], 0.24 [0.15; 0.39]) but not only towards those of the source population (0.57 [0.51; 0.64], 0.43 [0.32; 0.60], 0.38 [0.32; 0.47], 0.22 [0.16; 0.29]). Conclusions Frequency measures of study participants may not reflect the source population in the presence of self-selection, but the impact on association measures can be limited. IPW may be useful for (self-)selection bias correction, but the returned results can still reflect residual or other biases and random errors. [ABSTRACT FROM AUTHOR]

To describe the epidemiology, incidence, mortality and survival of ocular cancer in Cali between 1962 and 2019. Ecological population-based study analyzing data of incidence, mortality, and 5-years survival of malignant ocular tumors from the Populational Cancer Registry of Cali between 1962 and 2019. Between 1962 and 2019, 586 ocular tumors were found, 50.5% occurred in females, the mean age at diagnosis was 45 years (standard deviation = 25), 70.3% of ocular malignancies occurred in >14 years. The average annual incidence rate was 7.8 per million for male and 6.9 per million for females. Retinoblastoma (21%), squamous cell carcinoma (20%), melanoma (16%) and lymphoma (8%) were the most common neoplasm. In those <15 years, the most frequent malignant tumors were retinoblastomas (85.7%), followed by non-specified malignant neoplasm (NOS, 7.9%), and rhabdomyosarcoma (3.6%). In those >14 years, there were NOS (30%), followed by squamous cell carcinomas (28%), melanomas (23%), and lymphomas (9.7%). Conjunctiva (38.2%), retina (21%) and orbit (10%) constituted the majority of anatomical sites of ocular tumors. The survival rate was about 83.2% and mortality did not show a decreasing trend over time (p >.05). The incidence of ocular cancer in Cali has a slightly increasing trend, with stable behavior in the last decades. Squamous cell carcinoma, retinoblastoma, melanoma and lymphoma are the most frequent ocular cancers, with being retinoblastoma more frequent than melanoma. In general, ocular cancer had good survival rates in Cali. [ABSTRACT FROM AUTHOR]

Thrombosis in antiphospholipid syndrome (APS) involves in most cases the venous circulation. Why in some patients thrombotic APS affects the arterial circulation and in particular cerebral circulation is unknown. In previous studies, both patient characteristics and antiphospholipid antibody types and titers have been associated with arterial thrombosis. Aim of this study was to compare the clinical characteristics and laboratory findings of venous and arterial thrombotic APS from a large series of patients. Data were retrieved from the Start 2 antiphospholipid, a multicenter prospective register of long-term collected data from Thrombosis Centers in Italy. Of 167 patients with thrombotic APS, 114 (68 %) had a venous and 53 (32 %) had an arterial event as first clinical manifestation. Several clinical characteristics and risk factors were different among groups in univariate analysis. Using logistic regression analysis, reduced creatinine clearance and hyperlipidemia were independent variable for the occurrence of arterial APS. Notably, no difference in antiphospholipid antibody profiles and aβ2-Glycoprotein I levels were found between groups. A higher adjusted global antiphospholipid syndrome score (aGAPSS) was found in arterial group indicating a possible high recurrence rate in arterial APS. These data have pathophysiological and clinical implication since associated conditions might predispose patients to arterial rather than venous events and call to a close monitoring and treatment of arterial APS due to their increased tendency to recurrence. [ABSTRACT FROM AUTHOR]

Vascular surgery registries report on procedures and outcomes to promote patient safety and drive quality improvement. International registries have contributed significantly to the VASCUNET collaborative abdominal aortic aneurysm (AAA) outcome projects. This scoping review aimed to outline the national registries in vascular surgery that currently participate in the VASCUNET collaborative AAA projects. A scoping review of all published VASCUNET AAA studies and validation reports between 1997 and 2024 was undertaken. A survey was conducted among representatives of the international vascular registries contributing to VASCUNET collaborative AAA projects. Currently, vascular registries from 10 countries (Australia, Denmark, Finland, Hungary, Iceland, New Zealand, Norway, Sweden, Switzerland, and the UK) contribute to the current VASCUNET collaborative AAA project, of which eight have national coverage. In the past, three countries (Germany, Malta, and Italy) have participated in previous VASCUNET AAA projects, and a further three countries (Serbia, Greece, and Portugal) have planned participation in future projects. External validity is high for all current registries, with most reporting rates of > 90%. The majority have internal validation processes to assess data accuracy. VASCUNET mediated validation has also been performed by the consortium for five countries to date (Hungary, Sweden, Denmark, Malta, and Switzerland), for which a high degree of external and internal validity was identified. Most registries have established mechanisms for data linkage with national administrative datasets or insurance claims datasets and contribute to quality improvement through regular reporting to participating centres. National vascular registries from nations participating in the VASCUNET collaborative AAA projects are largely comprehensive, with high case ascertainment rates and good quality data with internal quality assurance. This provides a template for new registries wishing to join the VASCUNET collaboration and a benchmark for future research. [ABSTRACT FROM AUTHOR]

Despite improvements over recent years, morbidity and mortality associated with heart failure (HF) are higher in countries in the Central and Eastern Europe and Baltic region than in Western Europe. With the goal of improving the standard of HF care and patient outcomes in the Central and Eastern Europe and Baltic region, this review aimed to identify the main barriers to optimal HF care and potential areas for improvement. This information was used to suggest methods to improve HF management and decrease the burden of HF in the region that can be implemented at the national and regional levels. We performed a literature search to collect information about HF epidemiology in 11 countries in the region (Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, Serbia, and Slovenia). The prevalence of HF in the region was 1.6–4.7%, and incidence was 3.1–6.0 per 1000 person‐years. Owing to the scarcity of published data on HF management in these countries, we also collected insights on local HF care and management practices via two surveys of 11 HF experts representing the 11 countries. Based on the combined results of the literature review and surveys, we created national HF care and management profiles for each country and developed a common patient pathway for HF for the region. We identified five main barriers to optimal HF care: (i) lack of epidemiological data, (ii) low awareness of HF, (iii) lack of national HF strategies, (iv) infrastructure and system gaps, and (v) poor access to novel HF treatments. To overcome these barriers, we propose the following routes to improvement: (i) establish regional and national prospective HF registries for the systematic collection of epidemiological data; (ii) establish education campaigns for the public, patients, caregivers, and healthcare professionals; (iii) establish formal HF strategies to set clear and measurable policy goals and support budget planning; (iv) improve access to quality‐of‐care centres, multidisciplinary care teams, diagnostic tests, and telemedicine/telemonitoring; and (v) establish national treatment monitoring programmes to develop policies that ensure that adequate proportions of healthcare budgets are reserved for novel therapies. These routes to improvement represent a first step towards improving outcomes in patients with HF in the Central and Eastern Europe and Baltic region by decreasing disparities in HF care within the region and between the region and Western Europe. [ABSTRACT FROM AUTHOR]

Background/Aims: Identifying patients at the end of life can facilitate easier access to support and reduce unnecessary emergency care admissions. This study aimed to increase the number of adults on the palliative and end-of-life care register by at least 10% in 20% of primary care practices in an English integrated care system. Methods: A quality improvement project was carried out, using the Model for Improvement, with plan-do-study-act cycles. A hospice within a UK integrated care system added standard wording and system codes to letters sent to patients' primary care provider. The number of patients on the palliative and end-of-life care register was monitored monthly between September 2022 and April 2023. Results: By the end of the study period, the number of patients on the palliative and end-of-life care register had increased by >10% in 20% of practices. Conclusions: Adding a recommendation to add a patient to palliative and end-of-life care registers on letters sent to primary care providers can lead to an increase in the number of patients being identified to be in the last year of their life. [ABSTRACT FROM AUTHOR]

Introduction: Prescription of secondary prevention medications (SPMs) and effective control of cardiovascular risk factors (RFs) are crucial to reduce the risk of recurrent cardiovascular events, particularly in high-risk individuals including those with diabetes mellitus (DM). This study aimed to analyse the trends in SPM prescription and identify the factors associated with RF control among patients with DM and cardiovascular diseases in Perak health clinics. Methods: Data of patients with ischaemic heart disease (IHD) and cerebrovascular diseases (CeVDs) audited from 2018 to 2022, excluding those lost to follow-up, were extracted from the National Diabetes Registry. Descriptive and trend analyses were conducted. Multivariable logistic regression was utilised to identify the factors associated with RF control. Results: Most patients (76.7%) were aged ≥60 years and were Malays (62.3%). The majority had IHD (60.8%) and CeVDs (54.7%) for ≥5 years. SPM prescription increased significantly over the past 5 years. However, blood pressure (BP) and lipid control remained static. Good BP control was associated with a DM duration of ≥10 years and poor control with Malay ethnicity and prescription of two or three antihypertensives. Good DM control was associated with an age of ≥60 years and age at DM diagnosis of ≥60 years and poor control with Malay and Indian ethnicities, DM duration of ≥10 years and prescription of two or three and more glucose-lowering drugs. Poor lipid control was associated only with Malay and Indian ethnicities. Conclusion: SPM prescription has increased over time, but the achievement of treatment targets, particularly for lipid control, has remained poor and unchanged. Statin use is not associated with lipid control. The accessibility and availability of alternative lipid-lowering drugs must be improved to enhance overall RF control, especially lipid control, in patients with DM and cardiovascular diseases. [ABSTRACT FROM AUTHOR]

People with rare lysosomal storage diseases face challenges in their care that arise from disease complexity and heterogeneity, compounded by many healthcare professionals being unfamiliar with these diseases. These challenges can result in long diagnostic journeys and inadequate care. Over 30 years ago, the Rare Disease Registries for Gaucher, Fabry, Mucopolysaccharidosis type I and Pompe diseases were established to address knowledge gaps in disease natural history, clinical manifestations of disease and treatment outcomes. Evidence generated from the real-world data collected in these registries supports multiple stakeholders, including patients, healthcare providers, drug developers, researchers and regulators. To maximise the impact of real-world evidence from these registries, engagement and collaboration with the patient communities is essential. To this end, the Rare Disease Registries Patient Council was established in 2019 as a partnership between the Rare Disease Registries and global and local patient advocacy groups to share perspectives on how registry data are used and disseminated. The Patient Council has resulted in a number of patient initiatives including patient representation at Rare Disease Registries advisory boards; development of plain language summaries of registry publications to increase availability of real-world evidence to patient communities; and implementation of digital innovations such as electronic patient-reported outcomes, and patient-facing registry reports and electronic consent (in development), all to enhance patient engagement. The Patient Council is building on the foundations of industry–patient advocacy group collaboration to fully integrate patient communities in decision-making and co-create solutions for the rare disease community. [ABSTRACT FROM AUTHOR]

BACKGROUND: Ménière's disease is a disabling condition causing vertigo and hearing loss yet remains incompletely understood. Registry studies have the potential to answer important questions about phenotypes and natural history of clinical conditions. The aim of this study was to explore the feasibility of a patient-centered national Ménière's disease registry. METHODS: This was an observational study carried out at 4 state-funded hospitals and 4 independent clinics, within 3 distinct urban and rural regions within the UK. Adults with Ménière's disease were eligible to participate. A range of patient reported data, questionnaire data and clinical data (audiometric, radiological, and specialist balance testing data) was inputted into a bespoke database. RESULTS: The study recruited 411 participants. The majority of participants chose online recruitment (73%) and 27% chose via paper-based methods for participation. A small majority (57%) of participants were female. 96% of participants were of white ethnicity. Data completeness from online or postal data collection was similar. Around 20% of participants had audiological evidence of bilateral Ménière's disease. CONCLUSION: This feasibility study has successfully piloted methods for recruitment of hundreds of participants diagnosed with Ménière's disease. Participants actively contributed their data to a robust and extensive data collection platform. The positive outcomes from this initial feasibility study are anticipated to serve as a foundation for the future expansion of the registry. This expansion holds the potential to address a broad spectrum of request, encompassing all aspects of the nature of Ménière's disease. [ABSTRACT FROM AUTHOR]

Background and Aim: The REgistry of Selective Internal radiation therapy in AsiaNs (RESIN) was a multicenter, single‐arm, prospective, observational study of 90Y resin microspheres in patients with hepatocellular carcinoma (HCC) or metastatic colorectal cancer (mCRC) from Taiwan. RESIN is the first real‐life clinical study of this therapy in an Asian cohort. Study objectives were to evaluate the safety and efficacy of 90Y resin microspheres. Methods: Adults with HCC or mCRC scheduled to receive SIRT with 90Y resin microspheres were included. Primary endpoints were best overall response rate (ORR), adverse events, and changes from baseline in liver function. Secondary efficacy endpoints included overall survival (OS). Results: Of 107 enrolled patients, 83 had HCC, and 24 had mCRC. ORR was 55.41% (HCC) and 33.33% (mCRC). Of 58 HCC patients with 6‐month post‐SIRT data, 13.79% (n = 8) had resection, transplantation, transarterial chemoembolization, or radiofrequency ablation as the result of down‐staging or down‐sizing of their lesions. One hundred and ten treatment emergent adverse events (TEAEs) were reported in 51 patients, and five serious adverse events (SAEs) were reported in five patients. The most frequent TEAEs were abdominal pain, nausea and decreased appetite (HCC), and abdominal pain, decreased appetite, fatigue, and vomiting (mCRC). Two deaths due to SAEs (probably related to SIRT) were reported, both in patients with extensive HCC, active hepatitis infection, and other comorbidities. Median OS was 24.07 (HCC) and 12.66 (mCRC) months. Conclusions: Safety and efficacy outcomes with the routine use of SIRT with 90Y resin microspheres in Taiwan are consistent with published data. [ABSTRACT FROM AUTHOR]

Background and Objective: Off‐label pulmonary arterial hypertension (PAH)‐targeted drugs are commonly prescribed for non‐operated chronic thromboembolic pulmonary hypertension (CTEPH), but their effect on the long‐term prognosis of CTEPH remains unknown. This study investigated the effect of off‐label PAH‐targeted drugs on the long‐term survival of CTEPH patients. Methods: CTEPH patients were enrolled from a prospective multicentre national registry. Except for licensed riociguat and treprostinil, other PAH‐targeted drugs were off‐label. In the original and propensity score–matched (PSM) samples, five‐year survival was compared in two groups: (a) patients not receiving off‐label PAH‐targeted drugs (control) versus (b) patients receiving off‐label PAH‐targeted drugs (treatment). The latter group was investigated for the effect of started off‐label PAH‐targeted drugs at baselines (initial) or during follow‐up (subsequent). Results: Of 347 enrolled patients, 212 were treated with off‐label PAH‐targeted drugs initially (n = 173) or subsequently (n = 39), and 135 were untreated. The 1‐, 2‐, 3‐ and 5‐year survival of the treatment group was significantly higher than that of the control group (97.1% vs. 89.4%, 92.3% vs. 82.1%, 83.2% vs. 75.1% and 71.1% vs. 55.3%, respectively, log‐rank test, p = 0.005). Initial treatment was correlated with better 5‐year survival after excluding patients with subsequent treatment to reduce the immortal‐time bias (hazard ratio: 0.611; 95% CI: 0.397–0.940; p = 0.025). In PSM samples, patients given initial treatment showed significantly better 5‐year survival than untreated patients (68.9% vs. 49.3%, log‐rank test, p = 0.008). Conclusion: Off‐label targeted drugs contributed to improved long‐term survival in CTEPH patients receiving pharmacotherapies. [ABSTRACT FROM AUTHOR]

Population‐based studies have demonstrated a high risk of second cancers, especially of the skin, among patients with chronic lymphocytic leukaemia (CLL). We describe age‐standardised incidence ratios (SIRs) of second primary malignancies (SPM) in Australian patients with relapsed/refractory CLL treated with at least two lines of therapy, including ibrutinib. From December 2014 to November 2017, 156 patients were identified from 13 sites enrolled in the Australasian Lymphoma and Related Diseases Registry, and 111 had follow‐up data on rates of SPM. At 38.4 months from ibrutinib therapy commencement, 25% experienced any SPM. SIR for melanoma and all cancers (excluding nonmelanomatous skin cancers) were 15.8 (95% confidence interval (CI): 7.0–35.3) and 4.6 (95% CI: 3.1–6.9) respectively. These data highlight the importance of primary preventive interventions and surveillance, particularly as survival from CLL continues to improve. [ABSTRACT FROM AUTHOR]

Aims: To describe trends in risk factor control and serious hypoglycaemia in people with type 1 diabetes and to assess the effect of starting continuous glucose monitoring (CGM) in the real‐world setting. Methods: Two cross‐sectional surveys including 5746 individuals in 2012 and 18,984 individuals in 2020 based on data recorded in the Norwegian Diabetes Register for Adults (NDR‐A) and an analysis of a longitudinal cohort of 2057 individuals where data on CGM and HbA1c were available in the NDR‐A in 2012 and 2020. Results: In the cross‐sectional surveys mean HbA1c decreased from 66 mmol/mol (99% CI 65, 66) (8.2%) in 2012 to 61 mmol/mol (99% CI 61, 61) (7.7%) in 2020 (p < 0.0001). The proportion reporting serious hypoglycaemia decreased from 16.9 to 6.2% in 2020 (p < 0.0001). Mean LDL‐cholesterol decreased from 2.80 (99% CI 2.78, 2.83) to 2.63 (99% CI 2.61, 2.65) mmol/l in 2020 (p < 0.0001). Mean blood pressure increased slightly. In the CGM cohort, we found a 3 mmol/mol (0.3%) greater improvement in mean HbA1c and a greater reduction in serious hypoglycaemia (−12.3% vs. −6.2%) among individuals that had started using CGM between 2013 and 2020 when compared with individuals that had not started using CGM. Conclusions: Between 2012 and 2020, we found marked improvements in glycaemic control and a considerable decrease in the proportion of individuals reporting serious hypoglycaemia. The proportion of individuals using CGM increased substantially and individuals that had started using CGM by 2020 showed greater improvement in glycaemic control and less serious hypoglycaemia. [ABSTRACT FROM AUTHOR]

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Background: India is a vast and diverse country with existing variations in the frequency and distribution of cancers across its various parts. In regions lacking population-based cancer registries (PBCRs) in a vast country like India, hospital-based cancer registry (HBCR) data become an important source of information on the trends and patterns of a region. To determine the numerical trends of cases of the top ten cancer sites reporting to HBCR of a tertiary care cancer center in Bihar from 2014 to 2021. Materials and Methods: The details of all histopathologically confirmed cancer cases registered in the HBCR department of radiation oncology, State Cancer Institute, Indira Gandhi Institute of Medical Sciences, Patna, India between January 2014 and December 2021 were included in this retrospective observational study. All these cases were grouped site-wise and listed in descending order of the total number of cases reported in each group. Cross-tabulation with age and sex distribution was done. The frequency distribution of the top ten leading cancers for every consecutive calendar year was plotted in line diagrams for time trend analysis. Statistical Package for the Social Sciences (IBM SPSS Statistics for Windows, Version 20.0. Armonk, NY: IBM Corp.). was used for analysis. Annual percent change (APC) was determined for the number of cases of all ten cancer sites using joinpoint regression analysis (Joinpoint Regression Software, Version 4.0.4—May 2013; Statistical Methodology and Applications Branch, Surveillance Research Program of the US National Cancer Institute; Bethesda, MD, USA). Results: Out of 32,057 total cancer cases registered between Jan 2014 and Dec 2021, 21,848 patients (68.2%) cases constituted the top ten cancers. The top ten cancers among both sexes were cancer gallbladder (n = 4204, 13.1%), head and neck (n = 3395, 10.6%), breast (n = 3392, 10.6%), lung (n = 2069, 6.5%), cervix (n = 2039, 6.4%), hematolymphoid (n = 1930, 6.0%), liver (n = 1572, 4.9%), stomach (n = 1116, 3.5%), ovary (n = 1103, 3.4%), and colon-rectum (n = 1028, 3.2%). Except for cervical and hematolymphoid cancers, the rest all showed a rising trend over consecutive years. Conclusion: Cancer of the gallbladder continues to be among the most common cancers in the region. Focused research in all aspects of this deadly disease is needed. Strengthening of prevention and screening programs for common cancers and upliftment of the existing infrastructure for diagnosis and treatment of cancer in the region are necessitated.

Abstract Despite improvements over recent years, morbidity and mortality associated with heart failure (HF) are higher in countries in the Central and Eastern Europe and Baltic region than in Western Europe. With the goal of improving the standard of HF care and patient outcomes in the Central and Eastern Europe and Baltic region, this review aimed to identify the main barriers to optimal HF care and potential areas for improvement. This information was used to suggest methods to improve HF management and decrease the burden of HF in the region that can be implemented at the national and regional levels. We performed a literature search to collect information about HF epidemiology in 11 countries in the region (Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, Serbia, and Slovenia). The prevalence of HF in the region was 1.6–4.7%, and incidence was 3.1–6.0 per 1000 person‐years. Owing to the scarcity of published data on HF management in these countries, we also collected insights on local HF care and management practices via two surveys of 11 HF experts representing the 11 countries. Based on the combined results of the literature review and surveys, we created national HF care and management profiles for each country and developed a common patient pathway for HF for the region. We identified five main barriers to optimal HF care: (i) lack of epidemiological data, (ii) low awareness of HF, (iii) lack of national HF strategies, (iv) infrastructure and system gaps, and (v) poor access to novel HF treatments. To overcome these barriers, we propose the following routes to improvement: (i) establish regional and national prospective HF registries for the systematic collection of epidemiological data; (ii) establish education campaigns for the public, patients, caregivers, and healthcare professionals; (iii) establish formal HF strategies to set clear and measurable policy goals and support budget planning; (iv) improve access to quality‐of‐care centres, multidisciplinary care teams, diagnostic tests, and telemedicine/telemonitoring; and (v) establish national treatment monitoring programmes to develop policies that ensure that adequate proportions of healthcare budgets are reserved for novel therapies. These routes to improvement represent a first step towards improving outcomes in patients with HF in the Central and Eastern Europe and Baltic region by decreasing disparities in HF care within the region and between the region and Western Europe.

Abstract Background The PI*S variant is one of the most prevalent mutations within alpha-1 antitrypsin deficiency (AATD). The risk of developing AATD-related lung disease in individuals with the PI*SS genotype is poorly defined despite its substantial prevalence. Our study aimed to characterize this genotype and its risk for lung disease and compare it with the PI*ZZ and PI*SZ genotypes using data from the European Alpha-1 antitrypsin Deficiency Research Collaboration international registry. Method Demographic, clinical, functional, and quality of life (QoL) parameters were assessed to compare the PI*SS characteristics with the PI*SZ and PI*ZZ controls. A propensity score with 1:3 nearest-neighbour matching was performed for the most important confounding variables. Results The study included 1007 individuals, with PI*SS (n = 56; 5.6%), PI*ZZ (n = 578; 57.4%) and PI*SZ (n = 373; 37.0%). The PI*SS population consisted of 58.9% men, with a mean age of 59.2 years and a mean FEV1(% predicted) of 83.4%. Compared to PI*ZZ individuals they had less frequent lung disease (71.4% vs. 82.2%, p = 0.037), COPD (41.4% vs. 60%, p = 0.002), and emphysema (23.2% vs. 51.9%, p

Lotte Sahin Levison,1 Peter Jepsen,2,3 Henning Andersen1 1Department of Neurology, Aarhus University Hospital, Aarhus, Denmark; 2Department of Hepatology and Gastroenterology, Aarhus University Hospital, Aarhus, Denmark; 3Department of Clinical Epidemiology, Aarhus University Hospital, Aarhus, DenmarkCorrespondence: Lotte Sahin Levison, Department of Neurology, Aarhus University Hospital, Palle Juul–Jensens Boulevard 165, Aarhus, 8200, Denmark, Tel +45 26807160, Fax +4578463300, Email lotlev@rm.dkPurpose: Health care databases are a valuable source for epidemiological research on amyotrophic lateral sclerosis (ALS) if diagnosis codes are valid. We evaluated the validity of the diagnostic codes for ALS in the Danish National Patient Registry (DNPR).Patients and Methods: We obtained data from the DNPR for all adult (> 17 years) patients registered with ALS in Denmark between 1987 and 2022 (median population of 4.2 million during the study period). We randomly selected adult patients living in the North Denmark Region and Central Denmark Region (median population 1.4 million), with a primary discharge diagnosis code of ALS, diagnosed at three departments of neurology. We retrieved and reviewed medical records and estimated the positive predictive value (PPV) of the ALS diagnosis.Results: Over 36 years, we identified 5679 patients. From the validation cohort of 300 patients, we were able to retrieve 240 (80%) medical records, and 215 ALS diagnoses were confirmed. The overall positive predictive value was 89.6% (95% confidence interval (CI): 85.1– 92.8). The highest PPV was achieved for diagnoses registered for patients aged ≥ 70 years (93.8; 95% CI: 86.2– 97.3) compared to patients < 60 years (83.4; 95% CI: 73.3– 90.7).Conclusion: We found a high PPV of primary diagnostic codes for ALS from Danish departments of neurology, demonstrating high validity. Thus, the DNPR is a well–suited data source for large-scale epidemiological research on ALS.Keywords: registries, positive predictive value, international classification of disease codes, epidemiology, amyotrophic lateral sclerosis

Abstract Background Disease registries are comprehensive databases that record detailed information on patients diagnosed with specific conditions, providing valuable insights into their diagnosis, treatment, and outcomes. This study aims to describe the pilot phase of the national pediatric Immune Thrombocytopenia(ITP) registry (NPITP) in Iran, serving as the inaugural interpretive report. Methods This patient-centered software system was implemented as a national program across multiple pediatric centers in Iran. Several focus groups were conducted to establish a minimum data set (MDS) comprising six main classes, 14 sub-classes, and 187 data elements. Following expert consensus on the final data set, a web-based software tool was developed by the dedicated IT team, accessible online and offline via https://disreg.sbmu.ac.ir/q/ITP.html . The registry included children aged between two months and 18 years with a platelet count below 100 × 109/L, based on predefined inclusion criteria. Results Within a four-month period, a total of 60 ITP patients were registered, including 41 (68.3%) newly diagnosed cases, 68 (13.6%) persistent cases, and 14 (23.3%) with chronic ITP. The mean age of the registered patients was 55.93 ± 9.72 months. The most frequently observed bleeding symptoms were petechiae (68.3%), purpura (51.6%), and ecchymosis (13.3%). Among the newly diagnosed patients, 20 (33.3%) received intravenous immunoglobulin (IVIG), 17 (28.3%) were treated with prednisolone, and 17 (28.3%) received combined IVIG and steroid therapy. Of all patients, 40 (66.7%) demonstrated a complete response to treatment, while 16 (26.7%) exhibited a partial response. Four patients (6.7%) remained unresponsive to therapy. Treatment-related complications, such as Cushing’s syndrome, edema, weight gain, hirsutism, and mood disorders, were reported in 10 patients (16.6%). However, the majority of patients (81.7%) did not experience therapy-related complications. Conclusion The pilot phase of the NPITP registry successfully implemented a web-based software tool for data collection, aiming to enhance the quality of care, facilitate clinical research, and support health service planning in the future.

Abstract Background Rare disease registries (RDRs) are valuable tools for improving clinical care and advancing research. However, they often vary qualitatively, structurally, and operationally in ways that can determine their potential utility as a source of evidence to support decision-making regarding the approval and funding of new treatments for rare diseases. Objectives The goal of this research project was to review the literature on rare disease registries and identify best practices to improve the quality of RDRs. Methods In this scoping review, we searched MEDLINE and EMBASE as well as the websites of regulatory bodies and health technology assessment agencies from 2010 to April 2023 for literature offering guidance or recommendations to ensure, improve, or maintain quality RDRs. Results The search yielded 1,175 unique references, of which 64 met the inclusion criteria. The characteristics of RDRs deemed to be relevant to their quality align with three main domains and several sub-domains considered to be best practices for quality RDRs: (1) governance (registry purpose and description; governance structure; stakeholder engagement; sustainability; ethics/legal/privacy; data governance; documentation; and training and support); (2) data (standardized disease classification; common data elements; data dictionary; data collection; data quality and assurance; and data analysis and reporting); and (3) information technology (IT) infrastructure (physical and virtual infrastructure; and software infrastructure guided by FAIR principles (Findability; Accessibility; Interoperability; and Reusability). Conclusions Although RDRs face numerous challenges due to their small and dispersed populations, RDRs can generate quality data to support healthcare decision-making through the use of standards and principles on strong governance, quality data practices, and IT infrastructure.

Abstract Background Patient-Reported Outcome Measures (PROM) provide important information, however, missing PROM data threaten the interpretability and generalizability of findings by introducing potential bias. This study aims to provide insight into missingness mechanisms and inform future researchers on generalizability and possible methodological solutions to overcome missing PROM data problems during data collection and statistical analyses. Methods We identified 10,236 colorectal cancer survivors (CRCs) above 18y, diagnosed between 2014 and 2018 through the Danish Clinical Registries. We invited a random 20% (2,097) to participate in a national survey in May 2023. We distributed reminder e-mails at day 10 and day 20, and compared Initial Responders (response day 0–9), Subsequent Responders (response day 10–28) and Non-responders (no response after 28 days) in demographic and cancer-related characteristics and PROM-scores using linear regression. Results Of the 2,097 CRCs, 1,188 responded (57%). Of these, 142 (7%) were excluded leaving 1,955 eligible CRCs. 628 (32%) were categorized as initial responders, 418 (21%) as subsequent responders, and 909 (47%) as non-responders. Differences in demographic and cancer-related characteristics between the three groups were minor and PROM-scores only marginally differed between initial and subsequent responders. Conclusion In this study of long-term colorectal cancer survivors, we showed that initial responders, subsequent responders, and non-responders exhibit comparable demographic and cancer-related characteristics. Among respondents, Patient-Reported Outcome Measures were also similar, indicating generalizability. Assuming Patient-Reported Outcome Measures of subsequent responders represent answers by the non-responders (would they be available), it may be reasonable to judge the missingness mechanism as Missing Completely At Random.