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1.
A Real-World Observational Cohort of Patients with Hepatocellular Carcinoma: Design and Rationale for TARGET-HCC.
Cabrera, R, Singal, AG, Colombo, M, Kelley, RK, Lee, H, Mospan, AR, Meyer, T, Newell, P, Parikh, ND, Sangro, B, et al
Hepatology communications. 2021;(3):538-547
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Abstract
This study describes the design of the TARGET-hepatocellular carcinoma (HCC) cohort and descriptive characteristics of the patient population at diagnosis among those who were enrolled in the cohort across academic and community clinical centers. TARGET-HCC is a 5-year, longitudinal, observational cohort of patients with HCC receiving care in usual clinical practice. Redacted clinical information, obtained from medical records, captures the natural history and management of the disease, including the safety and efficacy of treatment interventions used in usual clinical practice. Patients can complete patient-reported outcome measures and provide biological specimens for future translational studies. The TARGET-HCC study includes adults with histologic, cytologic, or radiologic diagnosis of HCC from academic and community centers in both the United States and Europe. A total of 1,841 participants were enrolled between January 9, 2017, and July 23, 2019, at 67 sites in the United States and Europe. To date, the most common liver disease etiology in the cohort continues to be hepatitis C, although nearly half had a nonviral etiology, including alcohol-related liver disease or nonalcoholic steatohepatitis. Most included patients were diagnosed at an early stage (Barcelona Clinic Liver Cancer Stage [BCLC] 0/A), but only approximately one third underwent curative treatment. Systemic therapy has been used in 7.3% of enrolled patients, including 45.7% of those with BCLC stage C tumors. Conclusion: Overall, the TARGET-HCC cohort allows for the assessment of patient characteristics and investigation of new treatment paradigms and sequencing with existing agents as well as novel regimens for HCC.
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Short Physical Performance Battery: Response to Pulmonary Rehabilitation and Minimal Important Difference Estimates in Patients With Chronic Obstructive Pulmonary Disease.
Stoffels, AA, De Brandt, J, Meys, R, van Hees, HW, Vaes, AW, Klijn, P, Burtin, C, Franssen, FM, van den Borst, B, Sillen, MJ, et al
Archives of physical medicine and rehabilitation. 2021;(12):2377-2384.e5
Abstract
OBJECTIVE To determine the response to a pulmonary rehabilitation (PR) program and minimal important differences (MIDs) for the Short Physical Performance Battery (SPPB) subtests and SPPB summary score in patients with chronic obstructive pulmonary disease (COPD). DESIGN Retrospective analysis using distribution- and anchor-based methods. SETTING PR center in the Netherlands including a comprehensive 40-session 8-week inpatient or 14-week outpatient program. PARTICIPANTS A total of 632 patients with COPD (age, 65±8y; 50% male; forced expiratory volume in the first second=43% [interquartile range, 30%-60%] predicted). INTERVENTIONS Not applicable. MAIN OUTCOME MEASURE Baseline and post-PR results of the SPPB, consisting of 3 balance standing tests, 4-meter gait speed (4MGS), and 5-repetition sit-to-stand (5STS). The chosen anchors were the 6-Minute Walk Test and COPD Assessment Test. Patients were stratified according to their SPPB summary scores into low-performance, moderate-performance, and high-performance groups. RESULTS 5STS (∆=-1.14 [-4.20 to -0.93]s) and SPPB summary score (∆=1 [0-2] points) improved after PR in patients with COPD. In patients with a low performance at baseline, balance tandem and 4MGS significantly increased as well. Based on distribution-based calculations, the MID estimates ranged between 2.19 and 6.33 seconds for 5STS and 0.83 to 0.96 points for SPPB summary score. CONCLUSIONS The 5STS and SPPB summary score are both responsive to PR in patients with COPD. The balance tandem test and 4MGS are only responsive to PR in patients with COPD with a low performance at baseline. Based on distribution-based calculations, an MID estimate of 1 point for the SPPB summary score is recommended in patients with COPD. Future research is needed to confirm MID estimates for SPPB in different centers.
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Impact of discordance between patient's and evaluator's global assessment on treatment outcomes in 14 868 patients with spondyloarthritis.
Michelsen, B, Ørnbjerg, LM, Kvien, TK, Pavelka, K, Nissen, MJ, Nordström, D, Santos, MJ, Koca, SS, Askling, J, Rotar, Z, et al
Rheumatology (Oxford, England). 2020;(9):2455-2461
Abstract
OBJECTIVES To assess the impact of 'patient's minus evaluator's global assessment of disease activity' (ΔPEG) at treatment initiation on retention and remission rates of TNF inhibitors (TNFi) in psoriatic arthritis (PsA) and axial spondyloarthritis (axSpA) patients across Europe. METHODS Real-life data from PsA and axSpA patients starting their first TNFi from 11 countries in the European Spondyloarthritis Research Collaboration Network were pooled. Retention rates were compared by Kaplan-Meier analyses with log-rank test and by Cox regression, and remission rates by χ2 test and by logistic regression across quartiles of baseline ΔPEG, separately in female and male PsA and axSpA patients. RESULTS We included 14 868 spondyloarthritis (5855 PsA, 9013 axSpA) patients. Baseline ΔPEG was negatively associated with 6/12/24-months' TNFi retention rates in female and male PsA and axSpA patients (P <0.001), with 6/12/24-months' BASDAI < 2 (P ≤0.002) and ASDAS < 1.3 (P ≤0.005) in axSpA patients, and with DAS28CRP(4)<2.6 (P ≤0.04) and DAPSA28 ≤ 4 (P ≤0.01), but not DAS28CRP(3)<2.6 (P ≥0.13) in PsA patients, with few exceptions on remission rates. Retention and remission rates were overall lower in female than male patients. CONCLUSION High baseline patient's compared with evaluator's global assessment was associated with lower 6/12/24-months' remission as well as retention rates of first TNFi in both PsA and axSpA patients. These results highlight the importance of discordance between patient's and evaluator's perspective on disease outcomes.
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Identifying Common Predictors of Multiple Adverse Outcomes Among Elderly Adults With Type-2 Diabetes.
Kabue, S, Liu, V, Dyer, W, Raebel, M, Nichols, G, Schmittdiel, J
Medical care. 2019;(9):702-709
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Abstract
OBJECTIVE As part of a multidisciplinary team managing patients with type-2 diabetes, pharmacists need a consistent approach of identifying and prioritizing patients at highest risk of adverse outcomes. Our objective was to identify which predictors of adverse outcomes among type-2 diabetes patients were significant and common across 7 outcomes and whether these predictors improved the performance of risk prediction models. Identifying such predictors would allow pharmacists and other health care providers to prioritize their patient panels. RESEARCH DESIGN AND METHODS Our study population included 120,256 adults aged 65 years or older with type-2 diabetes from a large integrated health system. Through an observational retrospective cohort study design, we assessed which risk factors were associated with 7 adverse outcomes (hypoglycemia, hip fractures, syncope, emergency department visit or hospital admission, death, and 2 combined outcomes). We split (50:50) our study cohort into a test and training set. We used logistic regression to model outcomes in the test set and performed k-fold validation (k=5) of the combined outcome (without death) within the validation set. RESULTS The most significant predictors across the 7 outcomes were: age, number of medicines, prior history of outcome within the past 2 years, chronic kidney disease, depression, and retinopathy. Experiencing an adverse outcome within the prior 2 years was the strongest predictor of future adverse outcomes (odds ratio range: 4.15-7.42). The best performing models across all outcomes included: prior history of outcome, physiological characteristics, comorbidities and pharmacy-specific factors (c-statistic range: 0.71-0.80). CONCLUSIONS Pharmacists and other health care providers can use models with prior history of adverse event, number of medicines, chronic kidney disease, depression and retinopathy to prioritize interventions for elderly patients with type-2 diabetes.
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Impact of Financial Incentives on Health Outcomes and Costs of Care among Medicaid Beneficiaries with Diabetes in Hawai'i.
Fernandes, R, Chinn, CC, Li, D, Halliday, T, Frankland, T, Ozaki, RR
Hawai'i journal of medicine & public health : a journal of Asia Pacific Medicine & Public Health. 2019;(1):19-25
Abstract
The Hawai'i Patient Reward And Incentives to Support Empowerment (HI-PRAISE) project, part of the Medicaid Incentives for Prevention of Chronic Diseases program of the Affordable Care Act, examined the impact of financial incentives on Medicaid beneficiaries with diabetes. It included an observational pre-post study which was conducted at nine Federally Qualified Health Centers (FQHCs) between 2013 to 2015. The observational study enrolled 2,003 participants. Participants could earn up to $320/year in financial incentives. Primary outcomes were change in hemoglobin A1c, blood pressure, and cholesterol; secondary outcomes included compliance with American Diabetes Association (ADA) standards of diabetes care and cost effectiveness. Generalized estimating equation models were used to assess differences in clinical outcomes and general linear models were utilized to estimate the medical costs per patient/day. Changes in clinical outcomes in the observational study were statistically significant: mean hemoglobin A1c decreased from 8.56% to 8.24% (P < .0001); mean systolic blood pressure decreased from 125.16 to 124.18 mm Hg (P = .0137); mean diastolic blood pressure decreased from 75.54 to 74.78 mm Hg (P = .0005); total cholesterol decreased from 180.77 to 174.21 mg/dl (P < .0001); and low-density lipoprotein decreased from 106.17 to 98.55 mg/dl (P < .0001). Improved ADA compliance was also observed. A key limitation was a reduced sample size due to participant's fluctuating Medicaid eligibility status. HI-PRAISE showed no reduction in total health cost during the project period.
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Outcome of acute respiratory distress syndrome in university and non-university hospitals in Germany.
Raymondos, K, Dirks, T, Quintel, M, Molitoris, U, Ahrens, J, Dieck, T, Johanning, K, Henzler, D, Rossaint, R, Putensen, C, et al
Critical care (London, England). 2017;(1):122
Abstract
BACKGROUND This study investigates differences in treatment and outcome of ventilated patients with acute respiratory distress syndrome (ARDS) between university and non-university hospitals in Germany. METHODS This subanalysis of a prospective, observational cohort study was performed to identify independent risk factors for mortality by examining: baseline factors, ventilator settings (e.g., driving pressure), complications, and care settings-for example, case volume of ventilated patients, size/type of intensive care unit (ICU), and type of hospital (university/non-university hospital). To control for potentially confounding factors at ARDS onset and to verify differences in mortality, ARDS patients in university vs non-university hospitals were compared using additional multivariable analysis. RESULTS Of the 7540 patients admitted to 95 ICUs from 18 university and 62 non-university hospitals in May 2004, 1028 received mechanical ventilation and 198 developed ARDS. Although the characteristics of ARDS patients were very similar, hospital mortality was considerably lower in university compared with non-university hospitals (39.3% vs 57.5%; p = 0.012). Treatment in non-university hospitals was independently associated with increased mortality (OR (95% CI): 2.89 (1.31-6.38); p = 0.008). This was confirmed by additional independent comparisons between the two patient groups when controlling for confounding factors at ARDS onset. Higher driving pressures (OR 1.10; 1 cmH2O increments) were also independently associated with higher mortality. Compared with non-university hospitals, higher positive end-expiratory pressure (PEEP) (mean ± SD: 11.7 ± 4.7 vs 9.7 ± 3.7 cmH2O; p = 0.005) and lower driving pressures (15.1 ± 4.4 vs 17.0 ± 5.0 cmH2O; p = 0.02) were applied during therapeutic ventilation in university hospitals, and ventilation lasted twice as long (median (IQR): 16 (9-29) vs 8 (3-16) days; p < 0.001). CONCLUSIONS Mortality risk of ARDS patients was considerably higher in non-university compared with university hospitals. Differences in ventilatory care between hospitals might explain this finding and may at least partially imply regionalization of care and the export of ventilatory strategies to non-university hospitals.
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Diabetes self-care behaviours and clinical outcomes among Taiwanese patients with type 2 diabetes.
Ouyang, CM, Dwyer, JT, Jacques, PF, Chuang, LM, Haas, CF, Weinger, K
Asia Pacific journal of clinical nutrition. 2015;(3):438-43
Abstract
We examined the influences of patients' background characteristics on the frequency of performing five diabetes self-care behaviours that 185 Taiwanese outpatients reported. All patients had type 2 diabetes diagnosed for more than a year and attended an outpatient clinic at a large university hospital where they had received at least one dietitian-led individual nutrition education session and one nurse-led diabetes education session during the course of their care. Seventy nine percent of the patients regularly (defined as responses often or always on the questionnaire) took their medications and over half followed recommended meal plans and exercised, but fewer performed foot care (38%) or checked their blood glucose levels (20%) regularly. The associations between patients' demographics and disease-related characteristics and their performance of self-care behaviours were assessed with logistic regression. Although checking blood glucose levels and performing diabetes foot care were unrelated to any clinical outcome examined, patients who took their diabetes medications had lower hemoglobin A1c levels and fewer chronic complications than those who did not. Furthermore, patients who followed a diabetes meal plan also had lower hemoglobin A1c levels, and those who exercised regularly had healthier body mass indices (BMI) than those who did not.
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Effectiveness, tolerability and acceptance of a low-dosed estradiol/dienogest formulation (Lafamme 1 mg/2 mg) for the treatment of menopausal complaints: a non-interventional observational study over 6 cycles of 28 days.
Rouskova, D, Mittmann, K, Schumacher, U, Dietrich, H, Zimmermann, T
Gynecological endocrinology : the official journal of the International Society of Gynecological Endocrinology. 2015;(7):560-4
Abstract
BACKGROUND Concern and controversy characterize nowadays the use of hormone therapy for management of patients with menopausal complaints. This observational non-interventional study examined the use of a marketed oral formulation containing 1 mg estradiol valerate and 2 mg dienogest for treatment of menopausal symptoms in 1292 women visiting 243 gynecological practices in Germany. METHODS Score changes in the Menopausal Rating Scale (MRS) after three and six 28-day cycles were primary endpoints. Subjective reports on skin- and hair-related complaints and satisfaction with treatment effects were assessed. The incidence of adverse drug reactions (ADRs), adverse events (AEs) and vaginal bleeding was evaluated. RESULTS MRS total score decreased substantially and stronger than the clinically relevant change of 5 points (p < 0.0001) as compared with baseline. Subjective skin- and hair-related complaints declined. No unexpected ADRs were reported. AEs (including ADRs) were registered in 8.8% of the participants; most frequent AEs/ADRs were postmenopausal hemorrhage (2.9%) and drug ineffective (1.4%). Nearly 76% of the subjects remained amenorrheic. Approximately 90% of the patients rated the medication's effectiveness/tolerability as good/very good; 84% intended to continue the treatment. CONCLUSION This low-dose estradiol/dienogest formulation proved efficient and well-tolerated option for the alleviation of menopausal symptoms associated with estrogen deficiency.
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Cranial neurosurgical 30-day readmissions by clinical indication.
Moghavem, N, Morrison, D, Ratliff, JK, Hernandez-Boussard, T
Journal of neurosurgery. 2015;(1):189-97
Abstract
OBJECT Postsurgical readmissions are common and vary by procedure. They are significant drivers of increased expenditures in the health care system. Reducing readmissions is a national priority that has summoned significant effort and resources. Before the impact of quality improvement efforts can be measured, baseline procedure-related 30-day all-cause readmission rates are needed. The objects of this study were to determine population-level, 30-day, all-cause readmission rates for cranial neurosurgery and identify factors associated with readmission. METHODS The authors identified patient discharge records for cranial neurosurgery and their 30-day all-cause readmissions using the Agency for Healthcare Research and Quality (AHRQ) State Inpatient Databases for California, Florida, and New York. Patients were categorized into 4 groups representing procedure indication based on ICD-9-CM diagnosis codes. Logistic regression models were developed to identify patient characteristics associated with readmissions. The main outcome measure was unplanned inpatient admission within 30 days of discharge. RESULTS A total of 43,356 patients underwent cranial neurosurgery for neoplasm (44.23%), seizure (2.80%), vascular conditions (26.04%), and trauma (26.93%). Inpatient mortality was highest for vascular admissions (19.30%) and lowest for neoplasm admissions (1.87%; p < 0.001). Thirty-day readmissions were 17.27% for the neoplasm group, 13.89% for the seizure group, 23.89% for the vascular group, and 19.82% for the trauma group (p < 0.001). Significant predictors of 30-day readmission for neoplasm were Medicaid payer (OR 1.33, 95% CI 1.15-1.54) and fluid/electrolyte disorder (OR 1.44, 95% CI 1.29-1.62); for seizure, male sex (OR 1.74, 95% CI 1.17-2.60) and index admission through the emergency department (OR 2.22, 95% CI 1.45-3.43); for vascular, Medicare payer (OR 1.21, 95% CI 1.05-1.39) and renal failure (OR 1.52, 95% CI 1.29-1.80); and for trauma, congestive heart failure (OR 1.44, 95% CI 1.16-1.80) and coagulopathy (OR 1.51, 95% CI 1.25-1.84). Many readmissions had primary diagnoses identified by the AHRQ as potentially preventable. CONCLUSIONS The frequency of 30-day readmission rates for patients undergoing cranial neurosurgery varied by diagnosis between 14% and 24%. Important patient characteristics and comorbidities that were associated with an increased readmission risk were identified. Some hospital-level characteristics appeared to be associated with a decreased readmission risk. These baseline readmission rates can be used to inform future efforts in quality improvement and readmission reduction.
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Correlates and responsiveness to change of measures of skin and musculoskeletal disease in early diffuse systemic sclerosis.
Wiese, AB, Berrocal, VJ, Furst, DE, Seibold, JR, Merkel, PA, Mayes, MD, Khanna, D
Arthritis care & research. 2014;(11):1731-9
Abstract
OBJECTIVE Skin and musculoskeletal involvement are frequently present early in diffuse cutaneous systemic sclerosis (dcSSc). The current study examined the correlates for skin and musculoskeletal measures in a 1-year longitudinal observational study. METHODS Patients with dcSSc were recruited at 4 US centers and enrolled in a 1-year study. Prespecified and standardized measures included physician and patient assessments of skin involvement, modified Rodnan skin score (MRSS), durometer score, Health Assessment Questionnaire disability index, serum creatine phosphokinase, tender joint counts, and presence/absence of tendon friction rubs, small joint contractures, and large joint contractures. Additionally, physician and patient global health assessments and health-related quality of life assessments were recorded. Correlations were computed among the baseline global assessments, skin variables, and musculoskeletal variables. Using the followup physician and patient anchors, effect sizes were calculated. RESULTS A total of 200 patients were studied: 75% were women, mean ± SD age was 50.0 ± 11.9 years, and mean ± SD disease duration from first non-Raynaud's phenomenon symptom was 1.6 ± 1.4 years. Physician global health assessment had large correlations with MRSS (r = 0.60) and physician-reported skin involvement visual analog scale in the last month (r = 0.74), whereas patient global assessment had large correlations with MRSS, the Short Form 36 health survey physical component scale, skin interference, and skin involvement in the last month (r = 0.37-0.72). Four of 9 skin variables had moderate to large effect sizes (0.51-1.09). CONCLUSION Physician and patient global assessments have larger correlations with skin measures compared to musculoskeletal measures. From a clinical trial perspective, skin variables were more responsive to change than musculoskeletal variables over a 1-year period, although both provide complementary information.