Registry Participants Research Articles

Experience of a systematic approach to care and prevention of fragility fractures in New Zealand.

SummaryThis narrative review describes efforts to improve the care and prevention of fragility fractures in New Zealand from 2012 to 2022. This includes development of clinical standards and registries to benchmark provision of care, and public awareness campaigns to promote a life-course approach to bone health.PurposeThis review describes the development and implementation of a systematic approach to care and prevention for New Zealanders with fragility fractures, and those at high risk of first fracture. Progression of existing initiatives and introduction of new initiatives are proposed for the period 2022 to 2030.MethodsIn 2012, Osteoporosis New Zealand developed and published a strategy with objectives relating to people who sustain hip and other fragility fractures, those at high risk of first fragility fracture or falls and all older people. The strategy also advocated formation of a national fragility fracture alliance to expedite change.ResultsIn 2017, a previously informal national alliance was formalised under the Live Stronger for Longer programme, which includes stakeholder organisations from relevant sectors, including government, healthcare professionals, charities and the health system. Outputs of this alliance include development of Australian and New Zealand clinical guidelines, clinical standards and quality indicators and a bi-national registry that underpins efforts to improve hip fracture care. All 22 hospitals in New Zealand that operate on hip fracture patients currently submit data to the registry. An analogous approach is ongoing to improve secondary fracture prevention for people who sustain fragility fractures at other sites through nationwide access to Fracture Liaison Services.ConclusionWidespread participation in national registries is enabling benchmarking against clinical standards as a means to improve the care of hip and other fragility fractures in New Zealand. An ongoing quality improvement programme is focused on eliminating unwarranted variation in delivery of secondary fracture prevention.

Association of age and disease duration with comorbidities and disability: A study of the Swiss Multiple Sclerosis Registry

BackgroundWhile comorbidities increase with age, duration of multiple sclerosis (MS) leads to disability accumulation in persons with MS. The influence of ageing vis-a-vis MS duration remains largely unexplored. We studied the independent associations of ageing and MS duration with disability and comorbidities in the Swiss MS Registry participants. MethodsSelf-reported data was cross-sectionally analyzed using confounder-adjusted logistic regression models for 6 outcomes: cancer, type 2 diabetes (T2D), hypertension, cardiac diseases, depression, and having at least moderate or severe gait disability. Using cubic splines, we explored non-linear changes in risk shapes. ResultsAmong 1615 participants age was associated with cardiac diseases (OR 1.05, 95% CI [1.02, 2.08]), hypertension (OR 1.08, 95% CI [1.06, 2.10]), T2D (OR 1.10, 95%CI [1.05, 1.16]) and cancer (OR 1.04, 95% CI [1.01, 1.07]). MS duration was not associated with comorbidities, except for cardiac diseases (OR 1.03, 95% CI [1.00, 1.06]). MS duration and age were independently associated with having at least moderate gait disability (OR 1.06, 95% CI [1.04, 1.07]; OR 1.04, 95% CI [1.02, 1.05], respectively), and MS duration was associated with severe gait disability (OR 1.05, 95% CI [1.03, 1.08]). The spline analysis suggested a non-linear increase of having at least moderate gait disability with age. ConclusionsPresence of comorbidities was largely associated with age only. Having at least moderate gait disability was associated with both age and MS duration, while having severe gait disabity was associated with MS duration only.

Quantitative computed tomography predicts outcomes in idiopathic pulmonary fibrosis.

Prediction of disease course in patients with progressive pulmonary fibrosis remains challenging. The purpose of this study was to assess the prognostic value of lung fibrosis extent quantified at computed tomography (CT) using data-driven texture analysis (DTA) in a large cohort of well-characterized patients with idiopathic pulmonary fibrosis (IPF) enrolled in a national registry. This retrospective analysis included participants in the Australian IPF Registry with available CT between 2007 and 2016. CT scans were analysed using the DTA method to quantify the extent of lung fibrosis. Demographics, longitudinal pulmonary function and quantitative CT metrics were compared using descriptive statistics. Linear mixed models, and Cox analyses adjusted for age, gender, BMI, smoking history and treatment with anti-fibrotics were performed to assess the relationships between baseline DTA, pulmonary function metrics and outcomes. CT scans of 393 participants were analysed, 221 of which had available pulmonary function testing obtained within 90 days of CT. Linear mixed-effect modelling showed that baseline DTA score was significantly associated with annual rate of decline in forced vital capacity and diffusing capacity of carbon monoxide. In multivariable Cox proportional hazard models, greater extent of lung fibrosis was associated with poorer transplant-free survival (hazard ratio [HR] 1.20, p< 0.0001) and progression-free survival (HR 1.14, p< 0.0001). In a multi-centre observational registry of patients with IPF, the extent of fibrotic abnormality on baseline CT quantified using DTA is associated with outcomes independent of pulmonary function.

Milestones of progression in myotonic dystrophy type 1 and type 2.

Disease progression in myotonic dystrophy (DM) is marked by milestone events when functional thresholds are crossed. DM type 2 (DM2) is considered less severe than DM type 1 (DM1), but it is unknown whether this applies uniformly to all features. We compared the age-dependent risk for milestone events in DM1 and DM2 and tested for associations with age of onset and sex. We studied a large cohort of adult participants in a national registry of DM1 and DM2. Using annual surveys from participants, we ascertained milestone events for motor involvement (use of cane, walker, ankle brace, wheelchair, or ventilatory device), systemic involvement (diabetes, pacemaker, cancer), loss of employment due to DM, and death. Mean follow-up of registry participants (929 DM1 and 222 DM2patients) was 7 years. Disability and motor milestones occurred at earlier ages in DM1 than in DM2. In contrast, the risk of diabetes was higher and tended to occur earlier in DM2 (hazard ratio [HR],0.56; P≤ .001). In DM1, the milestone events tended to occur earlier, and life expectancy was reduced, when symptoms began at younger ages. In DM1, men were at greater risk for disability (HR,1.34; P≤ .01), use of ankle braces (HR,1.41; P= .02), and diabetes (HR,2.2; P≤ .0001), whereas women were at greater risk for needing walkers (HR,0.68; P= .001) or malignancy (HR, 0.66; P≤ .01). Milestone events recorded through registries can be used to assess long-term impact of DM in large cohorts. Except for diabetes, the age-related risk of milestone events is greater in DM1 than in DM2.

Circulating cardiac biomarkers improve risk stratification for incident cardiovascular disease in community dwelling populations.

SummaryBackgroundPlasma cardiac markers may assist in prediction of incident cardiovascular disease.MethodsThe incremental value of cardiac Troponins (T and I) and NT-proBNP added to risk factors in the PREDICT score for incident cardiovascular disease (CVD) in primary care, was assessed in 4102 asymptomatic participants in a randomised controlled trial of Vitamin D (ViDA). Findings were corroborated in 2528 participants in a separate community-based observational registry of CVD-free volunteers (HVOLS).FindingsHazard ratios for first cardiovascular events adjusted for PREDICT risk factors, comparing fifth to first quintiles of marker plasma concentrations, were 2.57 (95% CI 1.47-4.49); 3.01 (1.66-5.48) and 3.38 (2.04-5.60) for hs-cTnI, hs-cTnT and NT-proBNP respectively. The C statistic for discrimination of the primary endpoint increased from 0.755 to 0.771 (+0.016, p = 0.01). Cardiac marker data correctly reclassified risk upwards in 6.7% of patients and downwards in 3.3%. These findings were corroborated by results from HVOLS.InterpretationIncrements in plasma cardiac biomarkers robustly and reproducibly predicted increased hazard of incident CVD, independent of established risk factors, in two community-dwelling populations. Cardiac markers may augment risk assessment for onset of CVD in primary care.FundingViDA was funded by the Health Research Council of New Zealand (grant 10/400) and the Accident Compensation Corporation. HVOLS was funded by the Health Research Council of NZ Programme Grants (grants 02/152 and 08/070) and by grants from the Heart Foundation of NZ and the Christchurch Heart Institute Trust. Roche Diagnostics provided in-kind support for NT-proBNP and hs-cTnT assays and Abbott Laboratories for hs-cTnI assays.

Cardiometabolic multimorbidity is common among patients with psoriasis and is associated with poorer outcomes compared to those without comorbidity

Background Associations between cardiometabolic multimorbidity and response to therapy in psoriasis are unknown. Objective Determine the associations of multimorbidity with response to biologic treatment in psoriasis patients. Methods CorEvitas Psoriasis Registry participants who initiated biologic therapy and had 6-month follow-up were stratified by 0, 1, 2+ comorbidities (diabetes, hypertension, hyperlipidemia). Adjusted odds ratios (95% CIs) were calculated overall and separately by biologic class (TNFi, IL-17i, IL-12/23i + IL-23i), to assess the likelihood of achieving response for the 1 and 2+ groups vs. 0. Results Of 2,923 patients, 49.5%, 24.7% and 25.8% reported 0, 1 and 2+ comorbidities, respectively. Overall, likelihood of PASI75 was 18% (OR = 0.82; 95%CI: 0.67, 1.00) and 23% (OR = 0.77; 95%CI: 0.63, 0.96) lower in those with 1 and 2+ comorbidities, respectively, vs. 0. In those who initiated IL-17i, odds of PASI75 and PAS90 were 34% (OR = 0.66; 95%CI: 0.48-0.91) and 35% (OR = 0.65; 95%CI: 0.47-0.91) lower in the 2+ multimorbidity cohort. No significant associations were found among users of TNFi or IL-12/23i + IL-23i groups in the multimorbidity group. Limitations Patients may not be representative of all psoriasis patients. Conclusion Multimorbidity in psoriasis may decrease the likelihood of achieving treatment response to biologic therapy and should be considered when discussing treatment expectations with patients.

Rates and patterns of physical activity among Hispanics with multiple sclerosis in the NARCOMS Registry

BackgroundMultiple sclerosis (MS) is the most common cause of non-traumatic neurological disability among adults with an estimated incidence of 2.9 cases per 100,000 Hispanic adults. Hispanics with MS experience disproportionate rates of mobility disability compared to non-Hispanic Whites with MS. The current study aimed to examine the rates and patterns of physical activity (PA) behavior among Hispanics with MS and compare physical activity behavior to non-Hispanic Whites with MS in the North American Research Committee on Multiple Sclerosis Registry (NARCOMS). MethodsNARCOMS registry participants who completed the physical activity questions in the Spring 2015 semi-annual survey were included in the analyses. Self-identified Hispanic and non-Hispanic Whites were compared. Rates and patterns of physical activity were based on the International Physical Activity Questionnaire. We performed a 2:1 propensity score matching for comparing non-Hispanic White and Hispanic participants. ResultsOf the 6,352 NARCOMS participants in Spring 2015, we included 136 Hispanic and 6,216 non-Hispanic White participants. Among the Hispanic sample, median number of days engaged in vigorous PA was 0 and moderate PA was 1 day. Mean number of minutes sitting per day was 531±266 (approximately 9 hours). Further, a minority of Hispanic participants classified themselves as moderate or heavy active for work (11%), home (24%), and leisure (33%) physical activity. There were no significant differences between participants who identified as Hispanic (n=119) and propensity-matched non-Hispanics White participants (n=238) in demographics, clinical characteristics, or physical activity variables. ConclusionRates of physical activity were low among Hispanics with MS. This highlights an opportunity for examining the impact of physical activity interventions for improving mobility disability.

One-year follow-up of disease burden and medication changes in patients with myasthenia gravis: From the MG Patient Registry.

We studied the progression of myasthenia gravis (MG) disease burden and medication adjustment among MG Patient Registry participants. Participants diagnosed with MG (age ≥18 years), registered between July 1, 2013 and July 31, 2018 and completing both 6- and 12-month follow-up surveys, were included in this investigation. Participants were grouped into high-burden (Myasthenia Gravis Activity of Daily Living scale [MG-ADL] score ≥6) and low-burden (MG-ADL <6) groups based on MG-ADL scores at enrollment. Demographics and disease history were compared between groups. MG-ADL score change and medication changes (escalation, no change, de-escalation) between enrollment and 12-month follow-up were compared between groups. Minimal symptom expression (MSE, MG-ADL <2) at 12 months was compared between groups. Logistic regression analysis was performed to study factors associated with MSE at 12 months. In total, 520 participants (56% female) were included in high-burden (n=248) and low-burden (n=272) groups. Those in the high-burden group were more likely to be younger, female, and have shorter disease duration. At 12 months, MSE was achieved in 6% of the high-burden group and newly achieved (42 of 201, 21%) or maintained (52 of 71, 73%) in the low-burden group. In the multivariable analysis, being in the high-burden group and use of pyridostigmine were associated with less likelihood of MSE, whereas MG-ADL score improvement (>2 or >20%) at 6months significantly increased the likelihood of achieving MSE at 12 months (P= .0004). In both groups, but more so in the high-burden group, patients infrequently achieved MSE after 1year of MG treatment. Baseline low disease burden, improvement at 6months and no pyridostigmine use were associated with a higher likelihood of MSE at 12 months.

Abstract LB173: AACR President's initiative - 2020 by 2020: Democratizing precision cancer medicine and advancing health equity in the black belt

Abstract African Americans (AA) have higher incidence and mortality rates for several cancer types in comparison to their European American (EA) counterparts. Increasing participation in clinical research and patient registries, related to precision cancer medicine, will significantly improve cancer health equity. Many AA cancer patients are treated in community oncology clinics. Unfortunately, these health systems have limited access to Clinical Laboratory Improvement Amendments (CLIA) next generation sequence (NGS) germline and somatic DNA and RNA testing that are used to inform oncologists on the best treatment and/or clinical trial options for cancer patients. Indeed, AA CLIA NGS sample sets are poorly represented, which could presumably result in incomplete knowledge of genomic variants that could affect their treatment and overall outcomes. Hence, it is crucial to implement CLIA NGS efforts for all cancer patients. To address these disparities, Morehouse School of Medicine has formed the Comprehensive Approach to Reimagine health Equity Solutions (CARhES) consortium with Tuskegee University that has engaged community oncology practices in Alabama and Georgia - two of five Black Belt states. The CARhES consortium aims to implement precision cancer medicine to underserved and underrepresented communities that will improve the standard of cancer care by providing access to CLIA NGS testing, clinical trials, and personalized cancer care. Here we describe the first proof of concept of this approach with community oncology partners, i.e., Grady Health System, Wellstar Health System, Georgia Urology, Midtown Urology, and Maui Memorial Medical Center. At the time of consent, saliva, buccal, and tumor samples were collected from participants. Germline and somatic CLIA NGS was performed, and medical reports were returned to practitioners within 14 days. Prior to the COVID pandemic, the study enrolled over 880 patients with a 88% consent rate (n = 1000) in the first 11months of the program. At the start of the COVID pandemic, recruitment efforts were suspended for four months with a slow restart by June 2020. A decrease in the number of staff, office visits (67% reduction), and increase in COVID cases significantly limited recruitment efforts. During this slowdown, we established and improved eConsenting capabilities, which exist today. Community anxiety, due to the pandemic and SARS-CoV-19 vaccine efforts, resulted in a significant reduction in consent rates (88% to 60%). Nevertheless, this study began in April of 2019 and consented 1,750 participants in less than 2 years. Taken together, our study shows that a community-focused precision medicine approach requires meeting people where they are and providing them with access and understanding the benefit of clinical trial participation. The approximate 2,000 clinically annotated genomic AA datasets will greatly contribute to our understanding of cancer health disparities and among the first steps to democratize precision medicine. This study was funded by the American Association for Cancer Research, The Pelotonia Foundation, the M2Gen Foundation, and the National Cancer Institute (U54CA118638). Citation Format: Latrisha L. Horne, Gabriella M. Oprea-Ilies, Eddie R. Stanley, Carla M. Holloway, Margaret P. Hooker, Amina Isom, Derrick J. Beech, Ana Gaonkar, Shavette Harden, Jeffrey F. Hines, LaTonya Randolph, James K. Bennett, Daniel Canter, Darrell J. Carmen, Pooja Mishra, Giuseppe Del Priore, Roland Matthews, Brian M. Rivers, James W. Lillard. AACR President's initiative - 2020 by 2020: Democratizing precision cancer medicine and advancing health equity in the black belt [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2022; 2022 Apr 8-13. Philadelphia (PA): AACR; Cancer Res 2022;82(12_Suppl):Abstract nr LB173.

Patient-reported outcomes among males undergoing prostate cancer germline testing: Interim results from the Prostate Cancer Genetic Risk, Experience, and Support Study (PROGRESS) Registry.

10595 Background: Indications for prostate cancer (PCA) germline testing (GT) have greatly expanded, necessitating thousands of men to seek genetic evaluation through traditional genetic counseling and alternate delivery approaches. Therefore, there is an important need for patient-reported outcomes among men undergoing genetic testing which has been understudied. PROGRESS (Prostate Cancer Genetic Risk, Experience, and Support Study) was therefore developed to garner men’s experience with PCA germline testing. Here we provide an interim update on recruitment and results from registry participants. Methods: PROGRESS is a patient-driven registry of clinical, genetic, and patient-experience information among men undergoing PCA genetic testing. Survey covers demographic information, PCA history, mode of genetic counseling, satisfaction, decisional conflict, intention to share genetic results with family and physicians, attitude towards genetic testing, knowledge of cancer genetics, medical literacy, and numeracy. Descriptive statistics summarized results with counts and percentages for categorical variables and mean and standard deviation for continuous variables. All analyses were performed with SAS 9.4 (Cary, NC). Results: Current enrollment is 217 participants. Among 140 men with complete demographics, 93.57% are White, 98.6% non-Hispanic/Latino, 85% with bachelor’s degree or higher. Among 143 respondents, 60% reported a diagnosis of PCA. Among 85 men who reported on Gleason score, 24.7% had Gleason &gt; = 8. Regarding family history, 23.5% reported a 1st/2nd degree relative with PCA and 58.1% reported a 1st/2nd degree relative with any cancer. Among 150 men who reported genetic results, 40.7% had a genetic mutation, 18% reported having a VUS, and 10.7% reported not knowing their results. Among 185 men who reported on mode of genetic evaluation, 58.4% met with a genetics professional and 21.7% reported their doctor had discussed genetic testing. Satisfaction was high (n = 121) (highest score 30; mean score 27.92 + 2.73) and decisional conflict was low (n = 138) (mean score 26.07 + 10.18; range 16-80). Majority of 140 respondents discussed their results with their family (93.6%). Knowledge of cancer genetics was modest, with correct responses among 140 respondents of 50.1% + 28.1. Conclusions: Current participants of the PROGRESS Registry report high satisfaction with their genetic evaluation process. However, resources are needed to increase knowledge and understanding of genetic results. Greater engagement of African American males is critically needed to garner diverse perspectives and develop resources for men and their families applicable across populations. Target goal of the registry is 500 males; men can be referred to participate in the PROGRESS Registry at www.progressregistry.com .

Capturing uveal melanoma (UM) global practice patterns and clinical outcomes in the collaborative ocular melanoma natural history (OMNi) study (NCT04588662).

TPS9610 Background: Geographical differences in the management of primary UM, surveillance for recurrence, and care of metastatic disease have emerged based upon local expertise, treatment availability and insurance coverage. We have initiated accrual to OMNi (NCT04588662), an ambispective database developed to provide contemporary real-world data of UM, capturing its natural history and serving as a virtual biospecimen repository. The overall objectives of OMNi are to characterize regional/international UM management practice patterns and associated clinical outcomes in an effort to inform best practice recommendations. Methods: OMNi utilizes the Pulse Infoframe Healthie platform, a globally compliant platform which enables the structured collection of data mapped to Observational Medical Outcomes Partnership. The data fields created permit longitudinal capture of data including baseline patient and tumor characteristics, treatment of primary lesion and outcomes, surveillance patterns, time to disease recurrence, treatment of recurrent disease with outcomes, and survival. Inclusion criteria include a diagnosis of uveal melanoma and the ability to provide written informed consent for participation in the prospective registry or an institutional waiver by the IRB/ethics committee for retrospective data collection without written informed consent. We have initiated data collection at 3 US and 3 Australian centers, with 184 patients enrolled to date. Based upon feasibility assessment, we anticipate retrospective data entry for ̃2,000 patients and annual recruitment of ̃700 patients once all centers are active. Data collected in this OMNi collaboration, which will include additional US, UK and Australian sites, will facilitate new insights, hypothesis testing, as well as clinical trial development and conduct, and through a governance structure, will be made accessible for research. The OMNi dataset can serve and aid in interpretation of clinical trial outcomes in the real-world, facilitate cutting-edge research, and accelerate the development of diagnostics and therapeutics. Clinical trial information: NCT04588662.

Historical Redlining Impacts Contemporary Environmental and Asthma-related Outcomes in Black Adults.

Rationale: Environmental threats and poorly controlled asthma disproportionately burden Black people. Some have attributed this to socioeconomic or biologic factors; however, racism, specifically historical redlining, a U.S. discriminatory mortgage lending practice in existence between the 1930s and the 1970s, may have actuated and then perpetuated poor asthma-related outcomes. Objectives: To link historical redlining (institutional racism) to contemporary environmental quality- and lung health-related racial inequity. Methods: Leveraging a broadly recruited asthma registry, we geocoded 1,034 registry participants from Pittsburgh/Allegheny County, Pennsylvania, to neighborhoods subjected to historical redlining, as defined by a 1930s Home Owners' Loan Corporation (HOLC) map. Individual-level clinical/physiologic data, residential air pollution, demographics, and socioeconomic factors provided detailed characterization. We determined the prevalence of uncontrolled and/or severe asthma and other asthma-related outcomes by HOLC (neighborhood) grade (A-D). We performed a stratified analysis by self-identified race to assess the distribution of environmental and asthma risk within each HOLC grade. Measurements and Main Results: The registry sampling overall reflected Allegheny County neighborhood populations. The emissions of carbon monoxide, filterable particulate matter <2.5 μm, sulfur dioxide, and volatile organic compounds increased across HOLC grades (all P ⩽ 0.004), with grade D neighborhoods encumbered by the highest levels. The persistent, dispersive socioenvironmental burden peripherally extending from grade D neighborhoods, including racialized access to healthy environments (structural racism), supported a long-term impact of historical/HOLC redlining. The worst asthma-related outcomes, including uncontrolled and/or severe asthma (P < 0.001; Z = 3.81), and evidence for delivery of suboptimal asthma care occurred among registry participants from grade D neighborhoods. Furthermore, elevated exposure to filterable particulate matter <2.5 μm, sulfur dioxide, and volatile organic compound emissions (all P < 0.050) and risk of uncontrolled and/or severe asthma (relative risk [95% confidence interval], 2.30 [1.19, 4.43]; P = 0.009) demonstrated inequitable distributions within grade D neighborhood boundaries, disproportionately burdening Black registry participants. Conclusions: The racist practice of historical/HOLC redlining profoundly contributes to long-term environmental and asthma-related inequities in Black adults. Acknowledging the role racism has in these outcomes should empower more specific and novel interventions targeted at reversing these structural issues.

Cross-sectional survey of parental barriers to participation in pediatric participant research registries.

Research registries are a powerful tool for boosting recruitment into clinical trials. However, little is known about how parents approach the decision to enroll their child in a pediatric participant research registry (PPRR). We conducted in-person, written, or telephone surveys with parents/guardians of children hospitalized at Children’s Hospital of Omaha, Nebraska to identify attitudes towards and barriers to enrollment in PPRRs. Overall, our population (N = 36) had positive attitudes toward PPRRs, with 77.8% (CI: 61.6, 88.4) of participants stating they were “somewhat” or “very” likely to enroll their child. Likelihood to enroll differed between various recruitment and enrollment methods, with participants stating they would be more likely to enroll their child in a PPRR if they were recruited by their child’s primary care provider or a nurse in clinic (p = 0.02) and less likely to enroll if they were recruited through social media (p<0.001). Additionally, over 90% of participants who were likely to enroll their child in a PPRR (N = 28) were also willing to provide demographic, medical, and lifestyle information. However, these participants remained concerned about inappropriate sharing of their information with insurance or for-profit companies (53.6%, CI: 35.8, 70.4) and about receiving unwanted telephone calls from the registry (78.6%, CI: 60.0, 90.0). Parents are generally willing to enroll their child in a PPRR. However, to optimize enrollment, investigators must understand parental preferences for and concerns surrounding enrollment in a PPRR.

True Efficacy of LAA Closure: Patient Outcomes on Long-term Single-Antiplatelet or No Therapy: Insights From the EWOLUTION Registry.

Left atrial appendage occlusion (LAAO) is recommended for patients with atrial fibrillation at increased stroke risk, where effective long-term oral anticoagulation (OAC) is not feasible. In order to assess long-term safety of LAAO with aspirin monotherapy or no therapy, we aimed to report on patients with the Watchman LAAO device (Boston Scientific) once postimplantation intensified antiplatelet or anticoagulation therapy is discontinued. A total of 1025 patients scheduled for elective LAAO therapy prospectively consented for participation in the EWOLUTION registry; 1005 patients received a successful implant and were followed for 2 years. We identified 766 patients in EWOLUTION on single-antiplatelet therapy (SAPT; n = 639) or no therapy (n = 127) for ≥1 year following LAAO. Three to 6 months after LAAO, 766 patients were switched to SAPT or no therapy and were followed for at least 1 year until the study's conclusion or with events while on SAPT/ no therapy; mean time on SAPT/no therapy was 536.56 ± 177.59 days. Patients experienced 1.4 ischemic strokes per 100 patient years (PY) despite a CHA2DS2-VASC score of 4.3 ± 1.6. Major nonprocedural bleeding rates were low, with 1.3 major bleeds per 100 PY with a mean HAS-BLED score of 2.2 ± 1.2. Furthermore, the ischemic stroke rate in the SAPT/no-therapy subgroup was similar to the whole EWOLUTION collective and high-risk subgroups; the bleeding rate was even lower. When analyzed separately, strokes (2.1/100 PY) and bleedings (1.4/100 PY) of the no-therapy subgroup were similar to patients on SAPT (strokes 0.7/100 PY [P=.70]; bleedings 1.4/100 PY [P=.90]). Outcome data of patients on SAPT/no therapy for ≥1 year following Watchman implantation in the EWOLUTION registry suggest the efficacy and safety of LAAO.

Current Best Practice in the Management of Varicose Veins.

This article outlines the current best practice in the management of varicose veins. “Varicose veins” traditionally means bulging veins, usually seen on the legs, when standing. It is now a general term used to describe these bulging veins, and also underlying incompetent veins that reflux and cause the surface varicose veins. Importantly, “varicose veins” is often used for superficial venous reflux even in the absence of visible bulging veins. These can be simply called “hidden varicose veins”. Varicose veins usually deteriorate, progressing to discomfort, swollen ankles, skin damage, leg ulcers, superficial venous thrombosis and venous bleeds. Patients with varicose veins and symptoms or signs have a significant advantage in having treatment over conservative treatment with compression stockings or venotropic drugs. Small varicose veins or telangiectasia without symptoms or signs can be treated for cosmetic reasons. However, most have underlying venous reflux from saphenous, perforator or local “feeding veins” and so investigation with venous duplex should be mandatory before treatment. Best practice for investigating leg varicose veins is venous duplex ultrasound in the erect position, performed by a specialist trained in ultrasonography optimally not the doctor who performs the treatment. Pelvic vein reflux is best investigated with transvaginal duplex ultrasound (TVS), performed using the Holdstock-Harrison protocol. In men or women unable to have TVS, venography or cross-sectional imaging is needed. Best practice for treating truncal vein incompetence is endovenous thermal ablation. Increasing evidence suggests that significant incompetent perforating veins should be found and treated by thermal ablation using the transluminal occlusion of perforator (TRLOP) approach, and that incompetent pelvic veins refluxing into symptomatic varicose veins in the genital region or leg should be treated by coil embolisation. Bulging varicosities should be treated by phlebectomy at the time of truncal vein ablation. Monitoring and reporting outcomes is essential for doctors and patients; hence, participation in a venous registry should probably be mandatory.

139 Engaging the public in bidirectional communications to increase research literacy and improve the health of Indiana residents through All IN for Health

OBJECTIVES/GOALS: All IN for Health has initiated social media engagement strategies to create bi-directional communication around improved health and research literacy with the public. In addition, the All IN for Healths community-led advisory board, comprised of diverse individuals and organizations, will provide insight on research design and dissemination. METHODS/STUDY POPULATION: The Indiana Clinical and Translational Sciences Institute (CTSI) has committed to enhancing the publics engagement efforts in the All IN for Health program. This program was established in 2017 to improve the health of Indiana residents through the promotion of health resources and opportunities to participate in research. In 2021, the Indiana CTSI decided to integrate public engagement more formally. An official collaboration with the Indiana CTSIs Community Health Partnerships program was created, a digital engagement specialist was hired, and several new community and population health advisory board members were welcomed for quarterly meetings representing the rural and urban areas of the state, faith-based communities, health departments, and underrepresented groups in research. RESULTS/ANTICIPATED RESULTS: A social media campaign, informed by the All IN for Health advisory board was launched in November 2021, and the advisory board has been meeting on a quarterly basis. All IN for Healths established priority area is currently COVID-19 vaccination uptake, and our prioritized audiences are underserved and under resourced in urban and rural communities, and minoritized populations. All IN for Health has posted approximately 20 times per month on topics including COVID-19, Alzeheimers Disease, Diabetes, fitness, mental health, and research studies with a goal to increase followers and engagements by 20%. The hope is that this engagement will also result in an increase in the monthly e-newsletter subscribers and study volunteer registry participants. DISCUSSION/SIGNIFICANCE: All IN for Health has initiated new public engagement efforts to increase the value of the services offered to Indiana residents. Through consultation and co-design with members of the public and dissemination of research opportunities All IN for Health will be better suited to improve health and research literacy.

Protocol for the unclassified primary antibody deficiency (unPAD) study: Characterization and classification of patients using the ESID online Registry.

BackgroundPrimary antibody deficiencies (PADs) without an identified monogenetic origin form the largest and most heterogeneous group of primary immunodeficiencies. These patients often remain undiagnosed for years and many present to medical attention in adulthood after several infections risking structural complications. Not much is known about their treatment, comorbidities, or prognosis, nor whether the various immunological forms (decreased total IgG, IgG subclass(es), IgM, IgA, specific antibody responses, alone or in combination(s)) should be considered as separate, clearly definable subgroups. The unclassified primary antibody deficiency (unPAD) study aims to describe in detail all PAD patients without an identified specific monogenetic defect regarding their demographical, clinical, and immunological characteristics at presentation and during follow-up. In constructing these patterns, the unPAD study aims to reduce the number of missed and unidentified PAD patients in the future. In addition, this study will focus on subclassifying unPAD to support the identification of patients at higher risk for infection or immune dysregulation related complications, enabling the development of personalized follow-up and treatment plans.Methods and analysisWe present a protocol for a multicenter observational cohort study using the ESID online Registry. Patients of all ages who have given informed consent for participation in the ESID online Registry and fulfill the ESID Clinical Working Definitions for ‘unclassified antibody deficiency’, ‘deficiency of specific IgG’, ‘IgA with IgG subclass deficiency’, ‘isolated IgG subclass deficiency’, ‘selective IgM deficiency’, ‘selective IgA deficiency’ or ‘common variable immunodeficiency’ will be included. For all patients, basic characteristics can be registered at first registration and yearly thereafter in level 1 forms. Detailed characteristics of the patients can be registered in level 2 forms. Consecutive follow-up forms can be added indefinitely. To ensure the quality of the collected data, all data will be fully monitored before they are exported from the ESID online Registry for analysis. Outcomes will be the clinical and immunological characteristics of unPAD at presentation and during follow-up. Subgroup analyses will be made based on demographical, clinical and immunological characteristics.

Coronavirus disease 2019 and vaccination in patients with Shwachman-Diamond syndrome.

Because they can experience neutropenia due to bone marrow failure, patients with Shwachman‐Diamond syndrome (SDS) carry increased risk for serious infections compared with the general population; however, there has been a paucity of data on the incidence and severity of coronavirus disease 2019 (COVID‐19) in patients with SDS. We compiled results from a survey distributed to participants in the SDS Registry in May‐June 2021. In this report, we describe the characteristics and outcomes of patients with SDS who had COVID‐19. Patients reported a short clinical course without significant complications or cytopenias. Additionally, COVID‐19 vaccines were well tolerated with minor side effects.

Patient Research Interest Differences by Gender and Race/Ethnicity: A North Texas Primary Care Practice-Based Research Network (NorTex) Study.

Recruiting and increasing participation of women and racial/ethnic groups remains an ongoing struggle despite the National Institutes of Health Revitalization Act mandating the inclusion of these populations. This study examined gender and racial/ethnic differences in research interest in participating in Practice-Based Research Network studies focused on cardiovascular disease (CVD), diabetes, cancer, and mental health research. A total of 1348 participants and 18 NorTex clinics from the North Texas Primary Care Registry Project (NRP) database were included in this cross-sectional study. Participants who signed up through the registry to participate in future research projects and self-reported as non-Hispanic White, Hispanic, or non-Hispanic Black were included. Research interest in heart disease, high cholesterol, high blood pressure and heart failure were categorized as CVD; depression and anxiety were categorized as mental health; diabetes and cancer research were coded as single item dependent variables. Of registry participants, 72% were female, 34.5% were Black, and 24.4% were Hispanic. Of participants, 70% (n = 942) were interested in CVD research, the leading area of interest. Mental health research (56.3%, n = 755) was the second highest area of interest, while cancer had the least interest (38.4%, n = 515). After controlling for age, smoking, and having a diagnosis of the medical condition, gender did not predict interest in CVD, diabetes, cancer, or mental health research. However, race/ethnicity significantly predicted interest in diabetes and cancer research. Results indicate there are racial/ethnic differences in interest in specific research topics among our registry participants. This information may be helpful to develop successful recruitment strategies.

Remote blood collection from older adults in the Brain Health Registry for plasma biomarker and genetic analysis.

Use of online registries to efficiently identify older adults with cognitive decline and Alzheimer's disease (AD) is an approach with growing evidence for feasibility and validity. Linked biomarker and registry data can facilitate AD clinical research. We collected blood for plasma biomarker and genetic analysis from older adult Brain Health Registry (BHR) participants, evaluated feasibility, and estimated associations between demographic variables and study participation. Of 7150 participants invited to the study, 864 (12%) enrolled and 629 (73%) completed remote blood draws. Participants reported high study acceptability. Those from underrepresented ethnocultural and educational groups were less likely to participate. This study demonstrates the challenges of remote blood collection from a large representative sample of older adults. Remote blood collection from>600 participants within a short timeframe demonstrates the feasibility of our approach, which can be expanded for efficient collection of plasma AD biomarker and genetic data.