Current Medical Research and Opinion最新文献

Objective: Identifying patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in secondary data sets is important for assessing effectiveness and safety of treatments.

Methods: We searched bibliographic databases from inception through February 15, 2025. These were augmented by Google Scholar and hand searches of references from identified studies. Studies were included if they utilized billing code and/or pharmacy record algorithms to identify ATTR-CM (wild-type, variant or both) in secondary datasets. Study characteristics and methodological attributes were summarized.

Results: Twenty-five studies (26 analyses) were identified. Analyses were performed in the United States (US)(53.8%) and outside the US (46.2%). They assessed wild-type alone (46.2%), variant-type alone (11.5%) and mixed-type (42.3%). Sixteen analyses (61.5%) used International Classification of Diseases-10th-Revision (ICD-10) codes, 23.1% used ICD-9 and -10 codes, 11.5% analyses used country unique billing codes, and 11.5% relied on tafamidis prescriptions (with/without billing codes) to identify ATTR-CM. Of the 22 analyses (84.6%) using ICD codes, E85.82 alone was most used to identify wild-type (40.9%), and E85.0-E85.2 to identify variant-type. Fourteen studies (53.8%) required diagnosis codes for cardiac conditions. Exclusion criteria included codes for light chain amyloidosis (53.8%), blood cancers (38.5%) and cerebral amyloid angiopathy (30.8%). Five analyses (19.2%) used data from 2018 onward.

Limitations: While we utilized bibliographic databases, gray literature sources and backward citation tracking, it remains possible that not all studies were captured.

Conclusions: Methods for identifying ATTR-CM in studies of secondary datasets were heterogeneous. Future research should focus on optimizing ATTR-CM identification algorithms and performing validation studies.

Background: The Cardiometabolic Index (CMI) is a recognized metabolic marker, but its predictive efficacy for major adverse cardiovascular events (MACEs) in patients with ST-segment elevation myocardial infarction (STEMI) remains uncertain. The primary aim of this study is to investigate the predictive value of CMI for long-term outcomes in patients with STEMI, focusing on inflammation's mediating effects.

Methods: We analyzed data from 905 consecutive STEMI patients undergoing primary percutaneous coronary intervention, with 785 included in the final analysis. The primary endpoint was a composite of cardiovascular death, non-fatal myocardial infarction, revascularization for unstable angina, and rehospitalization for congestive heart failure. We used Cox regression models and subgroup analyses to explore the relationships between CMI and these outcomes, and employed mediation analysis to assess the role of inflammation, specifically using the lymphocyte to C-reactive protein ratio (LCR).

Results: Over an average follow-up of 11 months, 296 MACEs occurred. Multivariable Cox regression showed a significant positive association between CMI and the incidence of MACEs (Hazard Ratio [HR]: 4.584, 95% CI: 3.134-6.704), non-fatal myocardial infarction (HR: 2.142, 95% CI: 1.110-4.135), revascularization (HR: 7.911, 95% CI: 3.986-15.700), and rehospitalization for heart failure (HR: 6.243, 95% CI: 2.779-14.023), but not with cardiovascular death. Mediation analysis indicated that LCR mediated -13.7% and -19.5% of the associations of CMI with MACEs and heart failure rehospitalization, respectively.

Conclusion: Elevated CMI is positively associated with MACEs and rehospitalization for heart failure in STEMI patients, with partial mediation through inflammatory pathways. CMI may thus be a valuable prognostic tool for this patient group.

Objective: To evaluate the real-world effectiveness of once-weekly semaglutide for weight reduction and change in cardiometabolic risk factors at 24 months in patients with obesity or overweight.

Methods: This real-world retrospective, cohort study used the US Komodo Health database, which included adults with obesity or overweight with ≥1 obesity-related condition (ORC) who started semaglutide after June 15, 2021. Eligible patients escalated to and remained on the 1.7-mg or 2.4-mg maintenance dose for the duration of the 24-month follow-up. Change in weight (primary objective) and change in cardiometabolic risk factors (BMI, blood pressure, glycated hemoglobin, cholesterol, and triglycerides; secondary objective) were assessed from index date to the end of 24-month follow-up. Paired t tests were used to compare means at baseline and 24 months.

Results: Of 2592 eligible patients, 630 had 24-month follow-up data for weight, BMI, or cardiometabolic risk factors. The mean (SD) age was 48.6 (9.8) years, 77.8% of patients were female, and musculoskeletal pain and dyslipidemia were the most common baseline ORCs. At 24 months, mean (%) change in body weight was -17.9 kg (-16.6%; p < 0.0001; n = 175) and mean change in BMI was -6.0 kg/m² (p < 0.0001; n = 361). Statistically significant improvements in mean values for all cardiometabolic risk factors were observed at 24 months.

Conclusion: Real-world use of semaglutide was associated with reductions in weight and BMI and improvement in cardiometabolic risk factors at 24 months among patients with obesity or overweight. These findings support the use of semaglutide in clinical practice as an effective treatment for chronic weight management.

Objective: This study evaluated adherence to multitarget stool DNA (mt-sDNA) testing, positivity rate, and timeliness of follow-up colonoscopy (FU-CY) after a positive result in a large academic hospital, while examining patient- and provider-level predictors of adherence.

Methods: We conducted a retrospective cohort analysis of 14,256 patients aged 45-75 years who received an mt-sDNA order at Tufts Medical Center between 2016 and 2023. Adherence was defined as returning a completed kit within 1 year. Time to FU-CY was measured within 365 days of a positive mt-sDNA result. Logistic regression identified factors associated with adherence, adjusting for demographics, socioeconomic variables, insurance type, provider specialty, and patient return history.

Results: Adherence was 71.7%, with a positivity rate of 13.3%; FU-CY completion after a positive test was 75.8% within 12 months. Younger patients (45-49 years) demonstrated the highest adherence to mt-sDNA (75.5%). Adherence to mt-sDNA was significantly higher among Asian or Pacific Islander patients compared with other racial/ethnic groups, and among Medicare/Medicare Advantage patients compared with commercial insurance. Lower adherence was observed in patients with household incomes of $25K-$50K and in patients new to mt-sDNA testing compared with those with prior test experience.

Conclusions: High mt-sDNA adherence and timely FU-CY completion suggest mt-sDNA is an effective, patient-acceptable option for colorectal cancer screening. However, disparities by age, race/ethnicity, income, and patient return history highlight the need for targeted outreach strategies to improve screening equity and follow-up completion.

Background: In the phase 3 ESSENCE clinical trial, semaglutide demonstrated improved histological outcomes for patients with metabolic dysfunction-associated steatohepatitis (MASH) with moderate or advanced fibrosis. There is limited real-world evidence regarding the effect of semaglutide on liver enzymes and cardiometabolic markers in routine clinical practice.

Methods: This retrospective cohort analysis used linked administrative claims and laboratory data from December 2020 to November 2024 and included patients with MASH and either overweight or obesity. We compared patients initiating semaglutide and those not using anti-obesity medications (comparator) in terms of changes in liver enzymes (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and other laboratory markers, including body weight, Hemoglobin A1c (HbA1c), and lipids. Absolute and percent change from baseline was reported for each outcome at 6 months. Dependent within-group comparisons were computed using paired t-tests. To compare the change in ALT and AST between the comparison groups, difference in differences (DiD) estimates were calculated using generalized linear models adjusting for baseline characteristics.

Results: We identified 4,124 individuals initiating semaglutide and 168,284 individuals not using anti-obesity medications. Those receiving semaglutide were younger, more often female, and had fewer cardiometabolic comorbidities. Patients on semaglutide with available liver enzyme data experienced a 36% reduction in ALT (mean decrease: 19.4 U/L; p < 0.01; N = 27) and a 29% reduction in AST (mean decrease: 10.9 U/L; p = 0.01; N = 29). The comparator group experienced a 7% reduction in ALT (mean decrease: 2.8 U/L; p < 0.01; N = 2,680) and a 5% reduction in AST (mean decrease: 1.7 U/L; p < 0.01; N = 2,699). Comparing the changes in ALT and AST between the two comparison groups, the semaglutide group showed a greater reduction in ALT (DiD: -15.9; 95% CI: -27.5 to -4.2; p = 0.01) and trend towards greater reduction in AST (DiD: -8.7; 95% CI: -18.5 to 1.2; p = 0.08). The semaglutide groups demonstrated a 5.3% reduction in body weight and a 5.4% decrease in Hemoglobin A1c (both p < 0.01), while the comparator groups showed a 0.3% weight loss (p < 0.01) and no change in Hemoglobin A1c.

Conclusion: In patients with MASH and either overweight or obesity, semaglutide was associated with a clinically and statistically significant reduction in ALT after 6 months of continuous use. Reductions in AST, weight, and HbA1c were also observed.

Objective: Although blood pressure (BP) variability (BPV) derived from home BP monitoring (HBPM) is a recognized cardiovascular risk marker, limited data on its reproducibility hinder its clinical application. This study aimed to address this gap.

Methods: We compared HBPM-derived BPV at two time points using three metrics [standard deviation (SDVar), coefficient of variation (CoV), and variability independent of the mean (VIM)] among 495 individuals not using antihypertensive medications (No-AH) (median time-span between HBPM exams = 392 [308-519] days) and 588 individuals using antihypertensive medications (AH) (time-span between HBPM exams = 400 [319-510] days).

Results: No significant changes were observed across the time points in systolic HBPM-derived BPV metrics: SDVar (8.55 ± 3.14 vs. 8.71 ± 3.52 in No-AH; 9.67 ± 3.62 vs. 9.50 ± 3.47 in AH), CoV (7.01 ± 2.47 vs. 7.10 ± 2.65 in No-AH; 7.65 ± 2.77 vs. 7.57 ± 2.62 in AH), and VIM (5.76 ± 2.10 vs. 5.87 ± 2.35 in No-AH; 6.29 ± 2.34 vs. 6.18 ± 2.24 in AH) (all p >.05). Similarly, diastolic HBPM-derived BPV metrics remained stable between the time points: SDVar-DBP (5.65 ± 2.32 vs. 5.62 ± 2.33 in No-AH; 5.99 ± 2.50 vs. 5.90 ± 2.37 in AH), CoV-DBP (7.26 ± 3.03 vs. 7.24 ± 3.13 in No-AH; 7.66 ± 3.20 vs. 7.63 ± 3.06 in AH) and VIM-DBP (4.78 ± 1.96 vs. 4.75 ± 1.97 in No-AH; 4.64 ± 1.93 vs. 4.58 ± 1.83 in AH) (all p >.05). However, the test-re-test correlation of all HBPM-derived BPV metrics was only modest (r ≈ .27-.45), revealing substantial intra-individual variability. In addition, similar results were obtained in an alternative sample of 498 individuals (265 using AH and 233 not using AH) who underwent OBP and HBPM measurements at four different time points.

Conclusion: These findings demonstrate that BPV parameters derived from HBPM had high reproducibility at the population level, but limited reproducibility at the individual level.

Objective: Bowel urgency (BU) can place a considerable burden on patients with inflammatory bowel disease (IBD). However, data on its real-world impact on health-related quality of life (HRQoL) are limited. This cross-sectional study assessed the prevalence of BU and its impact on HRQoL in a real-world IBD population.

Methods: Adults with ulcerative colitis (UC) or Crohn's disease (CD) were recruited from the IBD Partners cohort, an internet-based cohort of individuals with IBD. BU was assessed using the Urgency Numeric Rating Scale (UNRS). Scores were classified into "no or minimal" BU (UNRS = 0-1) and BU (UNRS = 2-10). Remission was measured via the Simple Clinical Colitis Activity Index or the Short Crohn's Disease Activity Index. HRQoL was assessed via Patient-Reported Outcomes Measurement Information System (PROMIS) measures, reported via T-scores.

Results: The study included 2,417 patients (UC: 884; CD: 1533). BU was reported by 44% of patients with UC and 58% with CD. BU rates were higher among patients with active disease than in remission (UC: 77% vs 28%; CD: 87% vs 48%, p < 0.001). Patients experiencing BU reported significantly worse HRQoL than patients without BU across all assessed PROMIS measures, including depression, anxiety, pain interference, sleep disturbance, fatigue, and social satisfaction. BU correlated with disease activity strongly in the UC group (r = 0.62) and moderately in the CD group (r = 0.52).

Conclusion: In this real-world IBD population, BU significantly affected patients' HRQoL. BU was associated with disease activity and HRQoL measures. BU assessments should be included in clinical trials and routine clinical practice for UC and CD.

Aim: Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD), and its more severe form, Metabolic Dysfunction-Associated Steatohepatitis (MASH), pose significant global health challenges. Conventional cardiovascular risk models, such as the ASCVD Risk Estimator Plus, are limited in accurately predicting CVD risk in MASLD/MASH patients. This study aims to enhance the predictive accuracy of cardiovascular events in MASLD/MASH patients by developing a novel regression model, the LIVER-ASCVD+ model, which integrates traditional cardiovascular risk factors with liver biomarkers.

Methods: A retrospective cohort study was conducted using data from 9,185 biopsy-confirmed MASH patients within an integrated delivery network in the US. The study compared the performance of the LIVER-ASCVD+ model against the ASCVD Risk Estimator Plus. Kaplan-Meier survival analysis was conducted to assess outcomes, with comparisons made to two propensity-matched non-MASH control cohorts.

Results: The LIVER-ASCVD+ model demonstrated superior predictive accuracy for myocardial infarction (MI)/stroke events (AUC: 0.68) and mortality (AUC: 0.63) compared to the ASCVD Risk Estimator Plus (MI/stroke AUC: 0.63; mortality AUC: 0.54). The model stratified patients into high and low-risk categories, with significant differences observed in 10-year MI/stroke incidence and mortality rates. Kaplan-Meier analyses further validated the improved performance of the LIVER-ASCVD+ model in predicting cardiovascular outcomes.

Conclusion: The integration of liver-specific biomarkers into cardiovascular risk assessment models for MASLD/MASH patients significantly enhances predictive accuracy. The LIVER-ASCVD+ model represents a promising approach to improving clinical decision-making and patient outcomes in MASLD/MASH, warranting further validation.

Purpose: According to the current guidelines, glucagon-like peptide-1 receptor agonists (GLP-1 agonists) are recommended for the treatment of Type II diabetes mellitus (T2DM) in patients who are at risk for developing atherosclerotic cardiovascular disease (ASCVD). The black population is at a greater risk of developing ASCVD. This research intends to investigate the prescribing patterns of GLP-1 agonists amongst blacks compared to patients of other races and ethnicities.

Methods: This is a retrospective study of patients who were 18 years or older with a diagnosis of T2DM and were prescribed GLP-1 agonists and at Ochsner Health System from January 2019 to July 2023. The primary outcome was comparison of major adverse cardiovascular events (MACE): myocardial infarction, stroke, and inpatient mortality among the three study groups. Secondary outcomes included differences in clinical outcomes (A1C reduction, BMI reduction) in patients using GLP-1 agonists compared to patients not receiving these agents.

Results: Of 92,861 patients who met the inclusion criteria, the proportion of patients prescribed GLP-1 agonists by race included black 12,225 (33.2%), white 17,931 (34.5%), and others 2,834 (71.5%). For the primary outcomes among patients prescribed GLP-1 agonists, there was a significant difference with patients with black race having a lower proportion of inpatient mortality and composite outcome [1.8%, 2.8%, respectively] compared to patients of white race [2.6%, 4%, respectively], (p < .0001). For the secondary outcomes, from baseline to 48 months, patients prescribed GLP-1 agonists with black race had a greater reduction in A1C than patients with white race [-0.80 vs-0.56] while patients with white race had a greater reduction in BMI [-1.74 vs -1.40], (p < .0001).

Conclusion: The use of GLP-1 agonists has been shown to improve various clinical outcomes. Further research may reveal methods to increase the use of these agents, which may reduce disease-related complications especially for the minority population.

Introduction: Adult-type granulosa cell tumor of the testis is an extremely rare sex cord-stromal tumor. While most AGCTs are considered indolent with low metastatic potential, a small subset may exhibit aggressive behavior. Due to the rarity of this tumor, optimal management strategies and prognostic markers remain unclear. We present a rare case of adult-type granulosa cell tumor of the testis with pulmonary metastases and review the clinical features, treatments, and outcomes of previously reported metastatic cases.

Case report: A 33-year-old man presented with a four-month history of left testicular enlargement. Partial orchiectomy revealed AGCT with a low proliferative index (Ki-67 ∼ 10%) and a negative FOXL2 mutation. One month later, he developed a local recurrence with elevated Ki-67 (∼60%) and vascular invasion. PET/CT revealed multiple pulmonary metastases. The patient received four cycles of BEP chemotherapy and achieved a partial response. However, early disease progression followed. Second-line treatment with tislelizumab, nab-paclitaxel, carboplatin, and anlotinib led to temporary stabilization. Salvage chemotherapy with doxorubicin, ifosfamide, and dacarbazine failed, and the patient died two months later. A review of 11 additional metastatic AGCT cases showed that patients with resectable metastases had favorable outcomes, while those with distant metastases responded poorly to systemic therapy.

Conclusion: Although adult-type granulosa cell tumor of the testis is generally indolent, some cases may behave aggressively. Tumor size, vascular invasion, and proliferative index may correlate with metastatic potential. Patients who undergo complete resection of metastatic lesions tend to achieve better outcomes, whereas responses to systemic therapy are generally poor. Early identification of high-risk features and consideration of retroperitoneal lymph node dissection may improve prognosis in selected patients.