Prospective Registry Data Research Articles

Small renal mass: which criteria are decisive for a tumor board?

Small renal masses (SRM) are aheterogeneous group of tumors with varying metastatic potential. The increasing use and improvement in the quality of abdominal imaging have led to an increasingly earlier diagnosis of incidental SRM, which are asymptomatic and confined to the organ. Despite these advances in imaging and the growing use of renal tumor biopsies, preoperative diagnosis of malignancy remains difficult. The treatment of SRM has shifted away from radical nephrectomy and now primarily includes organ-sparing surgery or active surveillance. The optimal strategy for treating SRM is continuously evolving as studies from prospective data registries can identify factors that influence both short- and long-term patient outcomes. Recent research on biomarkers, imaging techniques, and machine learning offer promising approaches to adeeper understanding of tumor biology and treatment options for this patient population.

Impact of Impella5.0/5.5 use on short-term prognosis in patients with AMI-related cardiogenic shock receiving Impella-incorporated MCS in Japan

Abstract Background The short-term prognosis in patients with acute myocardial infarction-related cardiogenic shock (AMI-CS) remains unsatisfactory even in the era of Impella. The survival benefit of Impella 5.0/5.5 use remains uncertain. Purpose This study examined the clinical impact of Impella device type on clinical outcomes among patients with AMI-CS receiving Impella support using the prospective multi-center J-PVAD registry data. Methods We included patients with AMI-CS who received Impella-incorporated temporary MCS and were enrolled in the J-PVAD registry between Feb 2022 and Dec 2023. The primary outcome was 30-day survival. Results A total of 2405 patients (average 71 y; LVEF 30%; Lac 5.6 mmol/L) were included. 1409 (58.6%) patients received Impella alone and the others (41.4%) received ECPELLA support. Patients who received ECPELLA had a lower 30-day survival compared to those receiving Impella alone (46.1% vs 62.3%, p < 0.0001). Of note, patients who received ECMO with Impella5.0/5.5 had a greater survival compared to those who received ECMO with Impella2.5/CP (64.4% vs 43.0%, Figure 1). Conclusions Patients who received ECPELLA had a lower 30-day survival compared to Impella alone. Impella 5.0/5.5 use may improve prognosis for those with ECMO support in AMI-CS.

ID: 342538 SCS for Painful Diabetic Peripheral Neuropathy: Consistent Outcomes from Multiple RCTs and Prospective Registry Data

ID: 342538 SCS for Painful Diabetic Peripheral Neuropathy: Consistent Outcomes from Multiple RCTs and Prospective Registry Data

262. EOSINOPHILIC ESOPHAGITIS: A SEVEN YEAR ANALYSIS OF EPIDEMIOLOGY IN A UNITED KINGDOM COUNTY

Abstract Background Eosinophilic Esophagitis (EoE) is growing in incidence internationally. It is an immune-mediated disease that is characterised by esophageal mucosal eosinophilia and esophageal dysfunction. Awareness of EoE is also crucial for Esophagogastric surgeons as symptoms may mimic reflux disease or neoplasia, key considerations for an accurate differential diagnosis. Presently, there are no incidence estimates of EoE in the adult population in the UK. This study seeks to establish a cohort of EoE patients in Norfolk, a county in the East of England region, and to describe the incidence, characteristics, and diagnostic features of affected patients in both the adult and paediatric populations. Methods A retrospective analysis was performed of a prospectively maintained pathology database servicing a population of 761,000 persons in Norfolk. All newly diagnosed cases of EoE across a seven-year period between January 2017 and December 2023 were identified. Patients included for analysis had a histological diagnosis of EoE with >15 eosinophils/hpf and symptoms of oesophageal dysfunction. Patient electronic health care records were reviewed for histology reports, endoscopic findings, and clinical history. The primary outcome was incidence of EoE within the county. Secondary outcomes assessed included: patient characteristics; allergy and atopy history; endoscopic features, biopsy protocol and referral indication. Results 187 (median age 43 (4-85) years, Male: Female (126:61)) new cases of EoE were identified, of whom eight were in the paediatric age group of ≤16 years. The average annual incidence of EoE was 4.29 [95% CI 2.92, 5.66] and 0.89 [95% CI 0, 1.39] per 100,000 person/years in adult and paediatric groups respectively, with a significantly higher incidence in adults (RR 4.86 (95% CI [2.39 – 9.85] P<0.005) (Figure 1). The most common referral indications for endoscopy were dysphagia (80.7%) and food bolus obstruction episodes (25.1%). The most common endoscopic feature of EoE was circular rings (49.2%). Conclusion This is the first study reporting incidence of EoE in the UK adult population. There was a significantly greater incidence of EoE in adults compared to the paediatric population. However, there was no significant change in incidence of EoE in either group over the study period. Incidence estimates identified are similar to those reported in other European populations. Importantly 18.2% of patients had normal appearance of oesophagus on endoscopy, which reinforces the need to take oesophageal biopsies where there is clinical suspicion. Ideally, prospective registry data is needed for a comprehensive characterisation of incidence and prevalence of EoE.

One year analysis of Prospective Memory Clinics Registry in Qatar: A Critical Tool for Dementia Research and Policy Planning.

The Global Dementia Observatory (GDO) is a monitoring and accountability tool for the Global Action Plan on Public Response to Dementia 2017-25. Evidence from dementia registries may be utilized to better address WHO efforts in member countries, as well as to improve clinical practice and public health policy. The goal of this study was to analyze one-year data from a prospective memory clinic registry. This study was a baseline analysis of prospective memory clinics registry data of Qatar from January 1, 2023, through December 31, 2023. This study investigated the demographic, clinical, and lifestyle characteristics of 464 participants who were enrolled in memory clinics. Mild neurocognitive disorders were the most prevalent diagnoses in both sexes, affecting 61.5% of male patients and 63.7% of female patients. Dementia was slightly more common in men (19.8% vs. 18.9%), although delirium was more common in women (1.9% vs. 0%). In terms of risk factors, the analysis revealed that females were more likely to be obese (36.8% vs. 16.7% in males), while males had higher rates of diabetes (61.1% vs. 51.9% in females), hypertension (69.4% vs. 62.7% in females), and smoking (17.1% vs. 3.8% in females). The findings of this study highlight the differences in dementia risk factors between genders and races, highlighting the need for customized interventions. Furthermore, the registry is a great resource for policymakers and healthcare professionals, providing evidence-based suggestions to improve dementia care, increase the well-being of patients and caregivers, and maximize resource allocation.

Pain with orgasm in endometriosis: potential etiologic factors and clinical correlates.

Pelvic pain worsened by orgasm is a poorly understood symptom in patients with endometriosis. To assess the prevalence of pelvic pain worsened by orgasm in patients with endometriosis and explore its association with potential etiologic factors, including pelvic floor myalgia, uterine tenderness and adenomyosis, and central nervous system sensitization. An analysis was done of a prospective data registry based at a tertiary referral center for endometriosis. Eligible participants were patients aged 18 to 50 years who were referred between January 1, 2018, and December 31, 2019, diagnosed with endometriosis, and subsequently underwent surgery at the center. Clinical features were compared between participants reporting worsening pelvic pain with orgasm and those without worsening pain with orgasm, including patient-reported variables, physical examination findings, and anatomic phenotyping at the time of surgery. Pelvic floor myalgia and uterine tenderness were assessed by palpation on pelvic examination, adenomyosis by ultrasound, and central nervous system sensitization via the Central Sensitization Inventory (range, 0-100). Outcomes included pelvic or lower abdominal pain in the last 3months that worsened with orgasm (yes/no). Among 358 participants with endometriosis, 14% (49/358) reported pain worsened by orgasm while 86% (309/358) did not. Pain with orgasm was significantly associated with pelvic floor myalgia (55% [27/49] vs 35% [109/309]; Cohen's h = 0.40, P = .01) and higher scores on the Central Sensitization Inventory (mean ± SD, 53.3 ± 17.0 vs 42.7 ± 18.2; Cohen's d = 0.60, P < .001) but not with uterine tenderness or adenomyosis. Other clinical features associated with pain with orgasm were poorer sexual health (higher scores: deep dyspareunia, Cohen's h = 0.60; superficial dyspareunia, Cohen's h = 0.34; and Female Sexual Distress Scale-Revised, Cohen's d = 0.68; all P < .05) and poorer mental health (higher scores: Patient Health Questionnaire-9, 12.9 ± 6.7 vs 9.1 ± 6.3, Cohen's d = 0.59, P < .001; Generalized Anxiety Disorder-7, 9.4 ± 5.6 vs 6.8 ± 5.5, Cohen's d = 0.48, P = .002). Anatomic findings at the time of surgery did not significantly differ between the groups. Interventions targeting pelvic floor myalgia and central nervous system sensitization may help alleviate pain worsened by orgasm in patients with endometriosis. A strength is that pain worsened by orgasm was differentiated from dyspareunia. However, pain with orgasm was assessed by only a binary question (yes/no). Also, the study is limited to a single center, and there were limited data on sexual function. Pelvic pain exacerbated by orgasm in people with endometriosis may be related to concurrent pelvic floor myalgia and central sensitization.

The multiple roles of nerve biopsy in the diagnosis and prognosis of suspected immune neuropathies

IntroductionThe value of a sural nerve biopsy for the diagnosis of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is controversial. Evidence-based recommendations for its implementation are lacking. We investigated factors leading to biopsy and analyzed biopsy outcomes and consequences, assessed the predictability of biopsy outcomes through clinical parameters to avoid unnecessary biopsies, and compared results with electrophysiological and clinical severity to determine their prognostic value.Methods190 sural nerve biopsies were analyzed in two cohorts. One consisted of 163 biopsies and the second of 72 biopsies from the prospective Immune-mediated Neuropathies Biomaterial and Data registry (INHIBIT). Both have an intersection of 45 patients. 75 data sets from patients without biopsy were used. Analysis of nerve conduction studies, treatment, overall disability sum score (ODSS), biopsy outcomes, and diagnosis was performed.Results51% of biopsied patients received the diagnosis CIDP (77% fulfilled EFNS/PNS criteria), 21% were not CIDP typical, and 27% were unspecific. Biopsied patients responded less frequently to immunotherapies at time of biopsy than non-biopsied patients (p = 0.003). Immunotherapy was initiated more frequently after biopsy (p < 0.001) and more often with intravenous immunoglobulins (p < 0.0001). 76% of all biopsied patients met the electrophysiological criteria for CIDP. Sensory nerve action potential amplitudes of 0 µV still provide 73% of histological diagnostic value. Histologic signs of degeneration predicted ODSS worsening after 1 year (p = 0.028) but disease severity did not correlate with histological damage severity.DiscussionThe main indication for nerve biopsy was the treatment of refractory cases of autoimmune neuropathies with the therapeutic consequence of treatment initiation or escalation. Sural biopsy also provided prognostic information. Even with extinguished sural SNAP, the biopsy can still have diagnostic value.

Characterization of Biologic Discontinuation Among Pediatric Patients With Crohn’s Disease

Biologic therapies may effectively treat Crohn's disease (CD), and pediatric patients who discontinue multiple biologics risk exhausting treatment options. The frequency and context of biologic discontinuation have not been well-characterized. We aimed to determine patterns of biologic use, discontinuation, and evaluation in pediatric patients with CD. Pediatric patients with CD at 7 U.S. centers (2010-2020) were identified. Prospective ImproveCareNow registry data were supplemented with medical record abstraction. Biologics included monoclonal antibody and small molecule medications. Therapeutic drug monitoring (TDM) was considered induction if <14 weeks after biologic start, proactive if later during quiescent disease, and reactive during active disease. Of 823 patients included (median age, 13.0 years; 40% female), 86% started biologics (78% infliximab, 21% adalimumab, <1% others). Twenty-six percent used concomitant immunomodulators for ≥12 months. Most (85%) measured TDM including 47% induction, 69% proactive, and 24% reactive. Twenty-nine percent discontinued their first biologic after median 793 days because of inefficacy (34%), anti-drug antibodies (8%), adverse events (8%), or non-adherence (12%). If inefficacy, 86% underwent pre-discontinuation evaluation. If infliximab or adalimumab inefficacy and TDM was done, 62% had levels <10 μg/mL. Proactive TDM and concomitant immunomodulators were associated with 60% and 32% reduced biologic discontinuation. Most children with CD are treated with biologics; 25%-37% discontinue biologics, resulting in 1 in 12 using >2 biologics during pediatric care. Half of patients discontinued biologics without trial of high-dose therapy and 14% without any evaluation. Concomitant immunomodulator use and proactive TDM decreased risk of biologic discontinuation. Strategies are needed to preserve biologic efficacy and prevent biologic discontinuation.

Serious bacterial infection risk in recently immunized febrile infants in the emergency department

Study objectivesFever following immunizations is a common presenting chiefcomplaint among infants. The 2021 American Academy of Pediatrics (AAP) febrile infant clinical practice guidelines exclude recently immunized (RI) infants. This is a challenge for clinicians in the management of the febrile RI young infant. The objective of this study was to assess the prevalence of SBI in RI febrile young infants between 6 and 12 weeks of age. MethodsThis was a retrospective chart review of infants 6–12 weeks who presented with a fever ≥38 °C to two U.S. military academic Emergency Departments over a four-year period. Infants were considered recently immunized (RI) if they had received immunizations in the preceding 72 h prior to evaluation and not recently immunized (NRI) if they had not received immunizations during this time period. The primary outcome was prevalence of serious bacterial infection (SBI) further delineated into invasive-bacterial infection (IBI) and non-invasive bacterial infection (non-IBI) based on culture and/or radiograph reports. ResultsOf the 508 febrile infants identified, 114 had received recent immunizations in the preceding 72 h. The overall prevalence of SBI was 11.4% (95% CI = 8.9–14.6) in our study population. The prevalence of SBI in NRI infants was 13.7% (95% CI = 10.6–17.6) compared to 3.5% (95% CI = 1.1–9.3) in RI infants. The relative risk of SBI in the setting of recent immunizations was 0.3 (95% CI = 0.1–0.7). There were no cases of invasive-bacterial infections (IBI) in the RI group with all but one of the SBI being urinary tract infections (UTI). The single non-UTI was a case of pneumonia in an infant who presented with respiratory symptoms within 24 h of immunizations. ConclusionThe risk of IBI (meningitis or bacteremia) in RI infants aged 6 to 12 weeks is low. Non-IBI within the first 24 h following immunization was significantly lower than in febrile NRI infants. UTIs remain a risk in the RI population and investigation with urinalysis and urine culture should be encouraged. Shared decision making with families guide a less invasive approach to the care of these children. Future research utilizing a large prospective multi-center data registry would aid in further defining the risk of both IBI and non-IBI among RI infants.

Abstract 4852: High brain relapse rates in stage III triple negative inflammatory breast cancer are independent of systemic therapy

Abstract Background - Inflammatory breast cancer (IBC) is an aggressive subtype of invasive breast carcinoma that confers a comparatively poorer prognosis owing to consistently observed inferior responses to systemic therapies as compared to non-IBC cases. Triple negative (TN) breast cancer which is characterized by lack of estrogen receptor (ER) and progesterone receptor (PR) expression, as well as by lack of HER2 amplification portends particularly poor outcomes including high rates of brain failure. Here we reviewed our prospective IBC registry data for contemporary TNIBC patients to assess current outcomes and brain failure risk in the context of standard neoadjuvant immunotherapy. Methods - Given the change to the inclusion of pembrolizumab in addition to chemotherapy in the neoadjuvant setting in 2020, we reviewed records from 2016-2023 for Stage III TNIBC patients who presented to MD Anderson Cancer Center (MDACC) prior to receiving other therapy. TNIBC was defined as ER &amp;lt;1%, PR &amp;lt;1%, HER2 IHC 0/1+ or ISH-. N = 38. We examined PCR and brain mets as first failure by regimen (KN522, ACT, AC/TC) and compared using descriptive statistics. Log rank statistic was used to compare overall survival (OS) by PCR. Patients with stage III TNIBC who started the KN522 regimen achieved a pCR rate of 35% (N= 20) and pCR was less frequent in postmenopausal KN522 patients (1/7 PCRs. P = 0.016). Among this cohort, 20% of patients experienced relapse in the CNS as a site of first failure including two patients who achieved a PCR, and 25% had any brain failure at a median follow up post-surgery of 8M. Among 9 patients treated with AC/T 22% achieved pCR with 22% brain metastasis relapse rate as site of first failure and 33% overall was observed at a median follow up of 14M post-surgery. In 9 patients receiving AC/TC, the PCR was 44% and any brain relapse rate was 22%, 1/2 was a site of first failure, median follow up 38M. Despite similarities in brain relapse rates, achieving pCR nonetheless correlated with improved overall survival, P &amp;lt; 0.05. For comparison, among 11 stage IV TNIBC patients treated with neoadjuvant systemic therapy pre-KN522, median follow up 22M from initial diagnosis, 6/11 patients developed brain involvement, 5/6 as a site of first failure. Given small numbers it is difficult to compare PCR by regimen, however we note the PCR in KN522 is lower than expected, and the rate of brain metastases particularly as a site of first failure is consistently high across regimens regardless of PCR. It is critical to consider brain penetrance of neoadjuvant systemic therapies, brain staging and even prophylaxis of brain metastasis in patients with non-metastatic and metastatic TNIBC. Current efforts are ongoing to determine molecular signatures which may aid in prognostication and potentially study design to incorporate prophylactic therapies with the overarching goal of improving outcomes in patients with triple negative IBC. Citation Format: Azadeh Nasrazadani, Megumi Kai, Rachel Layman, Vicente Valero, Sadia Saleem, Bisrat G. Debeb, Anthony Lucci, Susie X. Sun, Swetha Bopparaju, Hope E. Murphy, Angela Alexander, Angela N. Marx, Michael C. Stauder, Bora Lim, Wendy A. Woodward. High brain relapse rates in stage III triple negative inflammatory breast cancer are independent of systemic therapy [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 4852.

The evolving management of small renal masses.

Small renal masses (SRMs) are a heterogeneous group of tumours with varying metastatic potential. The increasing use and improving quality of abdominal imaging have led to increasingly early diagnosis of incidental SRMs that are asymptomatic and organ confined. Despite improvements in imaging and the growing use of renal mass biopsy, diagnosis of malignancy before treatment remains challenging. Management of SRMs has shifted away from radical nephrectomy, with active surveillance and nephron-sparing surgery taking over as the primary modalities of treatment. The optimal treatment strategy for SRMs continues to evolve as factors affecting short-term and long-term outcomes in this patient cohort are elucidated through studies from prospective data registries. Evidence from rapidly evolving research in biomarkers, imaging modalities, and machine learning shows promise in improving understanding of the biology and management of this patient cohort.

Increasing Prevalence of Cerebral Palsy Among Two-Year-Old Children Born at

ObjectiveTo evaluate changes in prevalence and severity of cerebral palsy (CP) among surviving children born at <27 weeks of gestation over time and to determine associations between CP and other developmental domains, functional impairment, medical morbidities, and resource utilization among two-year old children who were born extremely preterm. Study DesignRetrospective cohort study using prospective registry data, conducted at 25 centers of the Eunice Kennedy Shriver NICHD Neonatal Research Network. Participants were children born at <27 weeks of gestation and followed at 18 through 26 months corrected age (CA) from 2008 through 2019. Outcomes of interest were changes in prevalence of any CP and severity of CP over time, and associations between CP and other neurodevelopmental outcomes, functional impairment, and medical comorbidities. Adjusted logistic, linear, multinomial logistic, and robust Poisson regression evaluated the relationships between child characteristics, CP severity, and outcomes. ResultsAmong 6,927 surviving children with complete follow-up data, 3,717 (53.7%) had normal neurologic examinations, 1,303 (18.8%) had CP, and the remainder had abnormal neurologic examinations not classified as CP. Adjusted rates of any CP increased each year of the study period (aOR 1.11 per year, 95% CI 1.08-1.14). Cognitive development was significantly associated with severity of CP. Children with CP were more likely to have multiple medical comorbidities, neurosensory problems, and poor growth at follow-up. ConclusionsThe rate of CP among surviving children who were born extremely preterm increased from 2008 through 2019. At 18 to 26 months CA, neurodevelopmental and medical comorbidities are strongly associated with all severity levels of CP.

Demographic, Clinical, Management, and Outcome Characteristics of 8,004 Young Children With Type 1 Diabetes.

To compare demographic, clinical, and therapeutic characteristics of children with type 1 diabetes age <6 years across three international registries: Diabetes Prospective Follow-Up Registry (DPV; Europe), T1D Exchange Quality Improvement Network (T1DX-QI; U.S.), and Australasian Diabetes Data Network (ADDN; Australasia). An analysis was conducted comparing 2019-2021 prospective registry data from 8,004 children. Mean ± SD ages at diabetes diagnosis were 3.2 ± 1.4 (DPV and ADDN) and 3.7 ± 1.8 years (T1DX-QI). Mean ± SD diabetes durations were 1.4 ± 1.3 (DPV), 1.4 ± 1.6 (T1DX-QI), and 1.5 ± 1.3 years (ADDN). BMI z scores were in the overweight range in 36.2% (DPV), 41.8% (T1DX-QI), and 50.0% (ADDN) of participants. Mean ± SD HbA1c varied among registries: DPV 7.3 ± 0.9% (56 ± 10 mmol/mol), T1DX-QI 8.0 ± 1.4% (64 ± 16 mmol/mol), and ADDN 7.7 ± 1.2% (61 ± 13 mmol/mol). Overall, 37.5% of children achieved the target HbA1c of <7.0% (53 mmol/mol): 43.6% in DPV, 25.5% in T1DX-QI, and 27.5% in ADDN. Use of diabetes technologies such as insulin pump (DPV 86.6%, T1DX 46.6%, and ADDN 39.2%) and continuous glucose monitoring (CGM; DPV 85.1%, T1DX-QI 57.6%, and ADDN 70.5%) varied among registries. Use of hybrid closed-loop (HCL) systems was uncommon (from 0.5% [ADDN] to 6.9% [DPV]). Across three major registries, more than half of children age <6 years did not achieve the target HbA1c of <7.0% (53 mmol/mol). CGM was used by most participants, whereas insulin pump use varied across registries, and HCL system use was rare. The differences seen in glycemia and use of diabetes technologies among registries require further investigation to determine potential contributing factors and areas to target to improve the care of this vulnerable group.

Development and Validation of the Early-Stage Hodgkin Lymphoma (HL) International Prognostication Index (E-HIPI): A Report from the Hodgkin Lymphoma International Study for Individual Care (HoLISTIC) Consortium

Background: There are a lack of clinical prediction tools in early-stage Hodgkin lymphoma (E-HL). We developed and validated a modern-day model, known as E-HIPI, to predict progression-free survival (PFS) within the first 5 years (y) incorporating detailed individual patient (pt) data from international clinical trials and prospective registry data that were standardized, normalized &amp; harmonized as part of the HoLISTIC Consortium (www.hodgkinconsortium.org). Methods: Model development utilized Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis (TRIPOD) guidelines (Moons, Ann Intern Med 2015) on a cohort of 3,080 newly-diagnosed E-HL pts treated on 4 seminal phase 3 clinical trials (NCIC: Meyer, NEJM 2012; RAPID: Radford, NEJM 2015; EORTC-GELA H9U: Ferme, E ur J Cancer 2017; EORTC/LYSA/FIL H10: Andre, JCO 2017). External validation was done in an independent cohort of 462 E-HL pts from a large registry (Princess Margaret, Toronto). Pts with classic HL, stage I or II disease, and ages 18-65 y were included. The primary outcome was PFS. Cox models were utilized with follow-up truncated at 5 years. Baseline predictors were: sex, stage, B symptoms, histology, number &amp; location of nodal sites, and continuous data values of age, maximum tumor diameter (MTD), white blood cell count (WBC), lymphocyte count, hemoglobin, albumin &amp; erythrocyte sedimentation rate (ESR). Multiple imputation was used for missing data. To consider possible non-linear relationships for continuous variables we examined plots &amp; cubic splines. We used backward elimination ( P&amp;lt;0.1) to develop the models &amp; internal validation with 200 bootstrap samples was conducted to estimate optimism &amp; correct for overfitting. The final prediction equations applied optimism corrections to beta coefficients &amp; hazard ratios (HR). Sensitivity analyses included model results stratified by PET staging (vs CT) and each HL trial. The models were evaluated in the external validation cohort &amp; compared using c-statistics. Model scores were also grouped by tertiles for visualization in Kaplan-Meier (KM) curves. Results: Median age in the development cohort was 35y; 51% were female; 81% had nodular sclerosis &amp; 77% stage II disease; 26% had B symptoms; 32% had mediastinal bulk (≥10cm or &amp;gt;1/3 diameter) and the mean MTD was 6.4 cm (interquartile range (IQR), 3.9-8.2). Median follow-up was 60 months (IQR 45-75). KM estimates at 5y was 90.7% (264 events) for PFS, and 97.1% (78 events) for overall survival (OS). After backward elimination, significant variables retained were age, sex, nodal location (cervical region), MTD &amp; albumin. Stage, ESR, or the number of nodal sites were not significant. Age was modeled with piecewise linear splines due to its non-linear relationship. See the Table for the final prediction model and optimism-corrected HRs. Optimism-corrected c-statistics in the development model was 0.63. Sensitivity analyses of stratification by type of imaging at staging or by each clinical trial demonstrated no change in model parameters or c-statistics. Most baseline characteristics and outcomes of the external validation cohort were similar besides median follow-up time (74 months, IQR 37-129). C-statistics for the E-HIPI model in external validation was 0.63. In addition, observed 5y outcomes stratified by tertile of predicted risk in the external cohort separated pts into a low &amp; higher-risk group, which comprised 1/3 &amp; 2/3 of pts, respectively ( Figure). Finally, model diagnostics revealed the potential for time dependency in the retained covariates. Further assessment of differential prediction between early (&amp;lt;2y) vs. later (2-5y) PFS events is ongoing. Conclusion: Following TRIPOD methodology, we rigorously developed and externally validated the E-HIPI among &amp;gt;3,500 E-HL pts.Discrete risk groups were delineated, and we identified several novel factors, including nodal location, sex &amp; key continuous variables that predict 5y PFS. Additional model refinement is underway, including formal calibration &amp; external validation via other HL registries (Stanford, Mayo/Iowa SPORE). A web-based calculator to simplify application of the E-HIPI will also be presented at ASH. Future planned modeling includes integration of differing treatments &amp; response-adapted imaging via multistate modeling to enrich individualized survival estimates and simulation modeling to predict late consequences.

Socioeconomic and demographic factors predictive of missed appointments in outpatient radiation oncology: an evaluation of access

PurposeWhile missed patient appointments reduce clinic efficiency and limit effective resource allocation, factors predictive of “no shows” are poorly understood in radiation oncology.Methods and materialsA prospective data registry of consecutive patients referred for initial consultation from October 2,018 to April 2022 was reviewed. Demographic characteristics recorded included age, gender, race, language preference, living situation, and insurance status. Zip code data linked to a patient’s residential address was used to determine socioeconomic status (SES) based on publicly available data on median household income. No show encounters were defined as all encounters where the patient failed to cancel their visit and did not sign-in to their scheduled appointment. Descriptive statistics were presented to identify factors predictive of missed appointments.ResultsA total of 9,241 consecutive patients were referred and logged into the database during the 4-year period, of which 5,755 were successfully scheduled and registered. A total of 523 patients (9%) failed to show for their appointments. Missed appointments were associated with low-income status, homeless living situation, and Black or Latino race (p &amp;lt; 0.05, for all). The proportion of White, Latino, Asian, and Black patients who missed appointments was 6%, 14%, 9%, and 12%, respectively (p &amp;lt; 0.001). Patient characteristics independently associated with higher odds of appointment non-adherence included low-income status ((OR) = 2.90, 95% CI (1.44–5.89) and Black or Latino race [(OR) = 3.31, 95% CI: 1.22–7.65].ConclusionsOur results highlight the influence of demographic, financial, and racial disparities on proper health care utilization among patients with cancer. Future interventions aimed at reducing appointment no shows could channel resources to the at risk-populations identified in this analysis, improving access to care, and optimize clinic efficiency.

Association between excess catecholamine synthesis and polymorphic premature ventricular contraction

BackgroundThe reasons for the etiology of premature ventricular contractions (PVCs) are not specifically known. Many patients are resistant to medical treatment, and a factor that would predict response to medical treatment cannot be identified. This study aims to investigate if a high catecholamine level results in polymorphic PVC. MethodsThis study was obtained by prospective data registry analysis. A total of 100 patients, 50 from the PVC group, and 50 from the control group have been evaluated. The participants who were included in the patient group had a polymorphic PVC of 5% or more in their 24-h Holter evaluations. Metanephrine showing the level of adrenaline and normetanephrine, showing the level of noradrenaline levels have been measured from these urine samples. ResultThere was no difference between the two groups in terms of biochemical and essential characteristics. Normetanephrine level has been significantly higher in the PVC group compared to the control group (323.9 ± 208.9 μg to 129.25 ± 67.88 μg; p < 0.001). Similarly, metanephrine level has also been higher in the PVC group (124.75 ± 82.43 μg to 52.615 ± 36,54 μg; p < 0.001). A positive and moderate correlation has been identified between the number and ratio of PVC and the metanephrine and normetanephrine levels. ConclusionIn this study, we found that the catecholamine levels were higher in the polymorphic PVC group than in the healthy volunteers. Also, an increase in the number and rate of PVC has been observed as the catecholamine levels increased. Clinical Trial RegistrationUrine Levels of Metanephrine and Normetanephrine in Patients With Frequent PVC; ClinicalTrials.gov number NCT03447002

Sex-related differences on short-term prognosis in patients with acute myocardial infarction related cardiogenic shock receiving Impella support in Japan

Abstract Background Sex-specific outcomes in patients with acute myocardial infarction related cardiogenic shock (AMI-CS) receiving temporary MCS remains controversial in the United States. Survival benefit of Impella support in female patients in Japan also remains uncertain. Purpose This study examined sex-related differences in clinical outcomes among patients with AMI-CS receiving Impella support using the prospective multi-center J-PVAD registry data. Methods We included patients with AMI-CS who received Impella-incorporated temporary MCS and were enrolled in the J-PVAD registry between Jan 2021 and Dec 2022. Patients with out-of-hospital cardiac arrest were excluded. The primary outcome was 30-day survival. Results A total of 979 patients (average 71 y; female 21%; LVEF 30%) were included. All patients received revascularization and 69% of them received during Impella support. Female patients had a lower 30-day survival compared to male patients (60.2% vs 67.9%, p = 0.0089). Of note, female patients who received Impella support before revascularization had a greater survival compared to those who received Impella support after revascularization (69.2% vs 41.8%, Figure 1). Conclusions Female patients in AMI-CS had a lower short-term prognosis compared to male. Early initiation of Impella support before revascularization is highly recommended for those with AMI-CS.

High ApoB/ApoA-I Ratio Predicts Post-Stroke Cognitive Impairment in Acute Ischemic Stroke Patients with Large Artery Atherosclerosis.

We aimed to investigate the association between the ApoB/ApoA-I ratio and post-stroke cognitive impairment (PSCI) in patients with acute stroke of large artery atherosclerosis etiology. Prospective stroke registry data were used to consecutively enroll patients with acute ischemic stroke due to large artery atherosclerosis. Cognitive function assessments were conducted 3 to 6 months after stroke. PSCI was defined as a z-score of less than -2 standard deviations from age, sex, and education-adjusted means in at least one cognitive domain. The ApoB/ApoA-I ratio was calculated, and patients were categorized into five groups according to quintiles of the ratio. Logistic regression analyses were performed to assess the association between quintiles of the ApoB/ApoA-I ratio and PSCI. A total of 263 patients were included, with a mean age of 65.9 ± 11.6 years. The median NIHSS score and ApoB/ApoA-I ratio upon admission were 2 (IQR, 1-5) and 0.81 (IQR, 0.76-0.88), respectively. PSCI was observed in 91 (34.6%) patients. The highest quintile (Q5) of the ApoB/ApoA-I ratio was a significant predictor of PSCI compared to the lowest quintile (Q1) (adjusted OR, 3.16; 95% CI, 1.19-8.41; p-value = 0.021) after adjusting for relevant confounders. Patients in the Q5 group exhibited significantly worse performance in the frontal domain. The ApoB/ApoA-I ratio in the acute stage of stroke independently predicted the development of PSCI at 3-6 months after stroke due to large artery atherosclerosis. Further, a high ApoB/ApoA-I ratio was specifically associated with frontal domain dysfunction.

Big data and RCT's in surgical oncology: Impact on improving hepatopancreatobiliary cancer surgical care on the global stage.

Randomized controlled clinical trials (RCTs) are at the heart of "evidence-based" medicine. Conducting well-designed RCTs for surgical procedures is often challenged by inadequate recruitment accrual, blinding, or standardization of the surgical procedure, as well as lack of funding and evolution of the treatment strategy during the many years over which such trials are conducted. In addition, most clinical trials are performed in academic high-volume centers with highly selected patients, which may not necessarily reflect a "real-world" practice setting. Large databases provide easy and inexpensive access to data on a large and diverse patient population at a variety of treatment centers. Furthermore, large database studies provide the opportunity to answer questions that would be impossible or very arduous to answer using RCTs, including questions regarding health policy efficacy, trends in surgical practice, access to health care, the impact of hospital volume, and adherence to practice guidelines, as well as research questions regarding rare disease, infrequent surgical outcomes, and specific subpopulations. Prospective data registries may also allow for quality benchmarking and auditing. There are several high-quality RCTs providing evidence to support current practices in hepatopancreatobiliary (HPB) oncology. Evidence from big data bridges the gap in several instances where RCTs are lacking. In this article, we review the evidence from RCTs and big data in HPB oncology identify the existing lacunae, and discuss the future directions of research in HPB oncology.

Proton Beam Therapy for Hepatocellular Carcinoma: Multicenter Prospective Registry Study in Japan

A prospective multicenter registry study was started May 2016 in Japan to evaluate the efficacy and safety of proton beam therapy (PBT) for hepatocellular carcinoma (HCC). Patients who received PBT for HCC from May 2016 to June 2018 were registered in the database of the Particle Beam Therapy Committee and Subcommittee of the Japanese Society for Radiation Oncology. Overall survival (OS), progression-free survival (PFS), and local recurrence were evaluated. Of the 755 registered patients, 576 with initial PBT and no duplicate cancer were evaluated. At final follow-up, 322 patients were alive and 254 had died. The median follow-up period for survivors was 39 months (0-58 months). The median OS time of the 576 patients was 48.8 months (95% CI, 42.0-55.6 months) and the 1-, 2-, 3-, and 4-year OS rates were 83.8% (95% CI, 80.5%-86.6%), 68.5% (64.5%-72.2%), 58.2% (53.9%-62.2%), and 50.1% (44.9%-55.0%), respectively. Recurrence was observed in 332 patients, including local recurrence in 45 patients. The median PFS time was 14.7 months (95% CI, 12.4-17.0 months) and the 1-, 2-, 3-, and 4-year PFS rates were 55.2% (95% CI, 51.0%-59.2%), 37.5% (33.5%-41.5%), 30.2% (26.3%-34.2%), and 22.8% (18.5%-27.4%), respectively. The 1-, 2-, 3-, and 4-year OS rates were significantly higher for tumor size <5 versus 5 to 10 cm (P < .001) and <5 versus ≥10 cm (P < .001); Child-Pugh score A/B versus C (P < .001); and distance of the tumor from the gastrointestinal tract <1 versus 1 to 2 cm (P < .008) and <1 versus >2 cm (P < .001). At final follow-up, 27 patients (4.7%) had late adverse events of grade 3 or higher, with liver failure (n=7), and dermatitis (n=7) being most common. This multicenter prospective data registry indicated that PBT for HCC gives good therapeutic effects (3-year local control rate of 90%) with a low risk of severe late adverse events.