Pragmatic and Observational Research最新文献

Objective: The objective of this study was to determine whether the effectiveness of budesonide comparator is non-inferior to budesonide reference in the prevention of asthma exacerbations. Asthma-related hospitalizations and safety were also examined.

Methods: This study used a matched, historic cohort design. Data were drawn from the Clinformatics™ Data Mart US claims database and included a 1-year baseline, starting 1 year before the index prescription date, and a 1-year outcome period. Patients received budesonide comparator or reference treatment. The primary outcome was the rate of asthma exacerbations. Non-inferiority for budesonide comparator vs budesonide reference was established if the 95% confidence interval (CI) upper limit of mean difference in proportions between treatments was <15%. Secondary outcomes examined rate of asthma-related hospitalizations and adverse events (AEs).

Results: The budesonide comparator and reference-matched cohorts each included 3109 patients. The adjusted upper 95% CI for the difference in proportions of patients experiencing asthma exacerbations was 0.035 (3.5%), demonstrating non-inferiority. Cohorts did not significantly differ in the rate of asthma exacerbations (adjusted rate ratio [RR]=1.04, 95% CI: 0.95-1.14) or rate of asthma-related hospitalizations (adjusted RR=1.10, 95% CI: 0.99-1.24) after adjusting for baseline confounders. No asthma exacerbations occurred during the outcome period in 72.9% of budesonide comparator patients and 71.8% of budesonide reference patients. No asthma-related hospitalizations occurred in 77.9% of patients in the budesonide comparator cohort and 79.0% of patients in the budesonide reference cohort. The most frequent AEs were throat irritation (≤0.4% of patients) and hoarseness/dysphonia (0.02% of patients). AEs did not significantly differ between treatment cohorts.

Conclusion: In this real-life study, non-inferiority of the budesonide comparator vs reference was met for the primary end point of asthma exacerbation rates. Asthma-related hospitalization and AE rates did not differ between the two treatment cohorts. The budesonide comparator is an effective and safe treatment alternative for asthma exacerbations.

Background: Differentiated thyroid cancer (DTC) occurs in relatively young patients and is associated with a good prognosis and long survival. The management of this disease involves thyroidectomy, radioiodine therapy, and long-term thyroid-stimulating hormone suppression therapy (THST). The long-term effects of the treatment and the interaction between subclinical hyperthyroidism and long-term hypoparathyroidism are poorly understood. This review sought to examine the available evidence.

Methods: A PubMed search was carried out using the search terms "Thyroid Neoplasms" AND ("Thyroxine" OR "Hypocalcemia" OR "Thyrotropin"). Original English language articles published in the last 30 years studying the morbidity from thyroid-stimulating hormone (TSH) suppression and hypoparathyroidism following a surgery for DTC were retrieved and reviewed by 2 authors.

Results: Of the 3,000 results, 66 papers including 4,517 patients were selected for the present study. Studies reported on a range of skeletal (included in 34 studies, 1,647 patients), cardiovascular (17 studies, 957 patients), psychological (10 studies, 663 patients), and other outcomes (10 studies, 1,348 patients). Nine of 26 studies on patients who underwent THST showed a reduction in bone density, and 13 of 23 studies showed an increase in bone turnover markers. Skeletal effects were more marked in postmenopausal women. There was no evidence of increased fracture risk, and only little data were available on hypoparathyroidism. Four of five studies showed an increased left ventricular mass index on echocardiography, and one study showed a higher prevalence of atrial fibrillation (AF). There was little difference in basic physiological parameters and limited literature regarding symptoms or significant events. Six studies showed associations between long-term TSH suppression and impaired quality of life. Impaired glucose metabolism and prothrombotic states were also found in DTC patients.

Conclusion: There is limited literature regarding long-term DTC treatment-related morbidity, particularly regarding the effects of long-term hypocalcemia. Most studies have focused on surrogate markers and not on clinical outcomes. A large prospective study on defined clinical outcomes would help characterize the morbidity of treatment and stimulate research on tailoring treatment strategies.

Evidence-based practice is an important component of health care service delivery. However, there is a tendency, embodied in tools such as Grades of Recommendation, Assessment, Development, and Evaluation, to focus principally on the classification of study design, at the expense of a detailed assessment of the strengths and limitations of the individual study. Randomized controlled trials (RCTs), and in particular the classical "explanatory" RCT, have a privileged place in the hierarchy of evidence. However, classical RCTs have substantial limitations, most notably a lack of generalizability, which limit their direct applicability to clinical practice implementation. Pragmatic and observational studies can provide an invaluable perspective into real-world applicability. This evidence could be used more widely to complement ideal-condition results from classical RCTs, following the principle of triangulation. In this review article, we discuss several types of pragmatic and observational studies that could be used in this capacity. We discuss their particular strengths and how their limitations may be overcome and provide real-life examples by means of illustration.

Background: Motor vehicle collisions (MVCs) are a leading cause of injury in the US. While the probability of collision with a pedestrian (PMVC) has declined in recent years, the probability of a pedestrian fatality has risen. Our objective was to determine whether older age impacts potential outcomes in patients involved in low-velocity PMVCs.

Materials and methods: We performed a retrospective-cohort study of adult patients aged >14 years involved in low-velocity pedestrian-MVCs (<15 miles per hour [24.14 km/h]), presenting to an urban level I trauma center from January to November 2013. Subjects were identified via trauma registry and stratified: ages 15-49 years and ≥50 years. Electronic medical records were reviewed for demographics, vital signs, and laboratory results on initial presentation, presence or absence of systemic inflammatory response syndrome (SIRS), shock index (SI), injury-severity score (ISS), length of stay (LOS), and survival to discharge. For statistical analysis, χ² or Student's t-tests were utilized.

Results: Our study included 145 patients (77 female) with a mean age of 41.9±3 years; 95 patients were aged 15-49 years (mean 31.9±2.2 years), and 50 patients were aged ≥50 years or older (mean 62.44±2.9 years). Mean ISS was 10.05±1.95, mean SI was 0.68±0.03, and mean LOS was 3.67±0.57 days. A total of 41 patients met SIRS criteria on arrival, and nine patients expired (6.2%). Mean ISS (15.64±4.42 vs 7.1±1.64, P<0.001) and mean SI (0.75±0.07 vs 0.65±0.03, P=0.002) were higher in patients aged ≥50 years. Mean LOS was longer in older patients (5.22±1.14 vs 2.85±0.58 days, P<0.001). Older age was associated with SIRS on arrival (P=0.023) and associated with mortality (P=0.004).

Conclusion: Age ≥50 years is associated with greater severity of injury and poor outcomes for patients involved in low-velocity PMVCs. Increased clinical attention and resource allocation should be directed toward older patients after low-velocity PMVCs.

Background: Adherence to asthma and chronic obstructive pulmonary disease (COPD) treatment has been shown to depend on patient-level factors, such as disease severity, and medication-level factors, such as complexity. However, little is known about the impact of prescription charges - a factor at the health care system level. This study used real-life data to investigate whether co-payment affects adherence (implementation and persistence) and disease outcomes in patients with asthma or COPD.

Methods: A matched, historical cohort study was carried out using two UK primary care databases. The exposure was co-payment for prescriptions, which is required for most patients in England but not in Scotland. Two comparison cohorts were formed: one comprising patients registered at general practices in England and the other comprising patients registered in Scotland. Patients aged 20-59 years with asthma, or 40-59 years with COPD, who were initiated on fluticasone propionate/salmeterol xinafoate, were included, matched to patients in the opposite cohort, and followed up for 1 year following fluticasone propionate/salmeterol xinafoate initiation. The primary outcome was good adherence, defined as medication possession ratio ≥80%, and was analyzed using conditional logistic regression. Secondary outcomes included exacerbation rate.

Results: There were 1,640 patients in the payment cohort, ie, England (1,378 patients with asthma and 262 patients with COPD) and 619 patients in the no-payment cohort, ie, Scotland (512 patients with asthma and 107 patients with COPD). The proportion of patients with good adherence was 34.3% and 34.9% in the payment and no-payment cohorts, respectively, across both disease groups. In a multivariable model, no difference in odds of good adherence was found between the cohorts (odds ratio, 1.04; 95% confidence interval, 0.85-1.27). There was also no difference in exacerbation rate.

Conclusion: There was no difference in adherence between matched patients registered in England and Scotland, suggesting that prescription charges do not have an impact on adherence to treatment.

Background: Cohort matching and regression modeling are used in observational studies to control for confounding factors when estimating treatment effects. Our objective was to evaluate exact matching and propensity score methods by applying them in a 1-year pre-post historical database study to investigate asthma-related outcomes by treatment.

Methods: We drew on longitudinal medical record data in the PHARMO database for asthma patients prescribed the treatments to be compared (ciclesonide and fine-particle inhaled corticosteroid [ICS]). Propensity score methods that we evaluated were propensity score matching (PSM) using two different algorithms, the inverse probability of treatment weighting (IPTW), covariate adjustment using the propensity score, and propensity score stratification. We defined balance, using standardized differences, as differences of <10% between cohorts.

Results: Of 4064 eligible patients, 1382 (34%) were prescribed ciclesonide and 2682 (66%) fine-particle ICS. The IPTW and propensity score-based methods retained more patients (96%-100%) than exact matching (90%); exact matching selected less severe patients. Standardized differences were >10% for four variables in the exact-matched dataset and <10% for both PSM algorithms and the weighted pseudo-dataset used in the IPTW method. With all methods, ciclesonide was associated with better 1-year asthma-related outcomes, at one-third the prescribed dose, than fine-particle ICS; results varied slightly by method, but direction and statistical significance remained the same.

Conclusion: We found that each method has its particular strengths, and we recommend at least two methods be applied for each matched cohort study to evaluate the robustness of the findings. Balance diagnostics should be applied with all methods to check the balance of confounders between treatment cohorts. If exact matching is used, the calculation of a propensity score could be useful to identify variables that require balancing, thereby informing the choice of matching criteria together with clinical considerations.

Purpose: The purpose of the present study was to investigate the association between the perioperative neutrophil to lymphocyte ratio (NLR) and cardiac surgery patient outcomes.

Patients and methods: A retrospective cohort study of 145 patients who underwent cardiac surgery in a tertiary hospital of Athens, Greece, from January to March 2015, was conducted. By using a structured short questionnaire, this study reviewed the electronic hospital database and the medical and nursing patient records for data collection purposes. The statistical significance was two-tailed, and p-values <0.05 were considered significant. The statistical analysis was performed with Mann-Whitney U test and Spearman's correlation coefficient, by using the Statistical Package for Social Sciences software (IBM SPSS 21.0 for Windows).

Results: The increased preoperative levels of NLR were associated with significantly higher mortality, both in-hospital (p=0.001) and 30-day (p=0.002), prolonged postoperative hospital length of stay (LOS), both in the cardiac intensive care unit (ICU) (p=0.002), and in-hospital (p=0.018), and likewise with delayed tracheal extubation (p≤0.001). Furthermore, patients with elevated NLR during the second postoperative day had significantly higher in-hospital mortality (p=0.018), increased incidence of pneumonia (p=0.022), higher probability of readmission to the ICU (p=0.002), prolonged ICU LOS (p≤0.001), and delayed tracheal extubation (p≤0.001).

Conclusion: Increased perioperative NLR seems to be associated with significantly higher mortality and morbidity in cardiac surgery patients. At the same time, NLR is a significant and inexpensive biomarker for the early identification of patients at high risk for complications. In addition, NLR levels could lead clinicians to perform measures for the optimal therapeutic patient approach.

Atrial fibrillation (AF) is the most common arrhythmia worldwide, leading to an extensive public health and economic burden. The increasing incidence and prevalence of AF is due to the advancing age of the population, structural heart disease, hypertension, diabetes, and thyroid disease. The majority of costs associated with AF have been attributed to the cost of hospitalization. In order to minimize costs and decrease hospitalizations, counseling on modifiable risk factors contributing to AF has been strongly emphasized. With the release of novel oral anticoagulants bypassing the need for anticoagulant bridging or laboratory monitoring, post-discharge nurse-led home intervention, and novel methods of heart rate monitoring, home-based AF management has reached a new level of ease and sophistication. In this review, we aimed to review modifiable risk factors for AF and various methods of home-based management of AF, along with their benefits.