Pragmatic and Observational Research最新文献

Background: The design of inhaler devices may potentially influence adherence/persistence and outcomes in asthma. Objective: The primary objective was to assess asthma control and any change in the quality of life in patients using an intuitive dry powder inhaler containing fluticasone propionate/salmeterol (AirFluSal^® Forspiro^®) for the treatment of asthma in everyday practice. Methods: ASSURE was a multicenter, noninterventional, open-label, prospective study in patients with asthma, aged ≥12 years and treated with the Forspiro device in Denmark, Sweden and Norway. Patients' opinions of their asthma control were assessed by the Asthma Control Test (ACT) questionnaire and asthma-related quality of life by the Mini Asthma Quality of Life Questionnaire (miniAQLQ) at baseline and at two follow-up visits (approximately 4-8-week intervals). Results: Of 321 patients enrolled in the study, 299 received at least one dose of fluticasone propionate/salmeterol via the Forspiro device and 204 had evaluable data at the baseline visit and at least one later visit. Patients showed improvements in asthma control and quality of life during the study. The mean sum score of ACT increased from 18.0 (SD 4.5) at visit 1 to 19.9 (4.2) at visit 2 and 20.5 (4.3) at visit 3. Overall, 38.2% of patients improved by the minimal clinically important difference (MCID) of ≥3 points (45.6% among those with a baseline score below 23 [ie, not already well controlled]). The mean score on the miniAQLQ increased from 5.16 (SD 1.24) at visit 1 to 5.58 (SD 1.20) at visit 2 and 5.82 (SD 1.04) at visit 3. Overall, 42.6% of patients improved by the MCID of ≥0.5. Conclusion: This real-life study suggests that treatment with fluticasone propionate/salmeterol via the Forspiro device can improve asthma symptom control and quality of life.

Background and objectives: Following the discovery of new drugs, physicians and pharmaceutical companies have become interested in examining patients' mortality and morbidity rates. In this respect, the effects of methotrexate (MTX)+etanercept/infliximab (ETA/INF) therapy on the survival of rheumatoid arthritis patients (RA) were evaluated in this study using marginal structural piecewise constant baseline hazard model. Patients and methods: According to the standard protocol, MTX is considered as the first-line treatment for RA patients. If there is no adequate response to MTX, biologic drugs will be added. To compare the survival rates of RA patients in MTX- and MTX+ETA/INF-treated groups, the piecewise constant baseline hazard model was fitted. Then, due to the existence of the time-dependent confounder (VAS) which was affected by previous treatment, the weight for each person-time was calculated via the inverse probability treatment weighting method. These weights were then used by marginal structural piecewise constant baseline hazard model. Finally, these models were compared. Results: The median (IQR) of the follow-up period in patients receiving MTX+ETN/INF and MTX was 11 (15.25) and 11 (31), respectively, and the 8-year survival rate was reported by 70% versus 68%, respectively. First, the piece-wise constant baseline hazard model was fitted. Fitting the given model showed that MTX+ETA/INF had a significant effect on patients' survival (HR=0.789, 95% CI [0.634, 0.983]). Second, marginal structural piecewise constant baseline hazard model was fitted. But, the results of this model revealed that MTX+ETA/INF did not have a significant impact on patients' survival (HR=0.968, 95% CI [0.860, 1.090]). Conclusion: Adjusting the pain score over time as a time-dependent confounder via marginal structural piecewise constant baseline hazard model, it has been demonstrated that MTX+ETA/INF does not have a significant effect on patients' survival rates. Therefore, a significant difference can be found between survival rates of these groups using longitudinal studies.

Background: Alternative payment models frequently require attribution of patients to individual physicians to assign cost and quality outcomes. Our objective was to examine the performance of three methods for attributing a patient with cancer to the likeliest physician prescriber of anticancer drugs for that patient using administrative claims data.

Methods: We used the HealthCore Integrated Research Environment to identify patients who had claims for anticancer medication along with diagnosis codes for breast, lung, or colorectal lung cancer between July 2013 and September 2017. The index date was the first date with a record for anticancer medication and cancer diagnosis code. Included patients had continuous medical coverage from 6 months before index to at least 7 days after index. Patients who received anticancer drugs during the 6 months prior to index were excluded. The three methods attributed each patient to the physician with whom the patient had the most evaluation and management (E&M) visits within a 90-day window around the index date (Method 1); the most E&M visits with no time window (Method 2); or the E&M visit nearest in time to the index date (Method 3). We assessed the performance of the methods using the percentage of the study cohort successfully attributed to a physician, and the positive predictive value (PPV) relative to available physician-reported data on patient(s) they treat.

Results: In total, 70,641 patients were available for attribution to physicians. Percentages of the study cohort attributed to a physician were: Method 1, 92.6%; Method 2, 96.9%; and Method 3, 96.9%. PPVs for each method were 84.4%, 80.6%, and 75.8%, respectively.

Conclusion: We found that a claims-based algorithm - specifically, a plurality method with a 90-day time window - correctly attributed nearly 85% of patients to a prescribing physician. Claims data can reliably identify prescribing physicians in oncology.

Purpose: To describe the efficacy of a stratified approach on automatic office blood pressure (BP), 24-hour ambulatory BP, and BP variability (BPV) in treatment-naïve patients with systolic hypertension using lercanidipine for stage 1 and lercanidipine/enalapril for stage 2.

Patients and methods: This was an open-label, prospective interventional study conducted in 22 general practices in South Africa. Treatment-naïve patients with stage 1 hypertension received lercanidipine 10 mg and patients with stage 2 received lercanidipine 10 mg/enalapril 10 mg. After 6 weeks, patients not reaching target (<140/90 mmHg) were up-titrated to lercanidipine 10 mg/enalapril 10 mg or lercanidipine 10 mg/enalapril 20 mg, respectively, for a further 6 weeks. Office BP was determined at each visit, and 24-hour ambulatory BP monitor (ABPM) at baseline and 12 weeks. The primary end point was changes in office BP, and secondary end points were changes in 24-hour ABPM and BPV.

Results: Of the 198 patients, 48% had stage 1 and 52% stage 2 hypertension. The mean age was 55 years, body mass index was 29.2 kg/m², 48.5% were female, and 15.1% were diabetic. The mean (SD) office SBP and DBP at baseline, 6 weeks, and 12 weeks was 158.2 (13.8), 141.6 (11.1), and 138.7 (16.7) mmHg (P<0.00001), and 92.2 (10.6), 84.6 (11.1), and 82 (13.3) mmHg (P<0.00001), respectively. The mean (SD) systolic and diastolic daytime ABPM at baseline and 12 weeks was 157 (16.63) and 142 (14.41) mmHg (P<0.0001) and 88 (12.34) and 81 (10.79) mmHg (P<0.0001), and the nighttime ABPM was 146 (15.68) and 133 (13.94) mmHg (P<0.0001) and 79.5 (11.64) and 72.5 (10.05) mmHg (P<0.009), respectively. There were few adverse events.

Conclusion: Lercanidipine and lercanidipine/enalapril for stage 1 or 2 hypertension highly improves office SBP and DBP, overall 24-hour BP, daytime BP, and nighttime BP, also reducing BPV with few adverse effects.

Background: For decades, the optimal timing of surgery for acute cholecystitis has been controversial. Recent meta-analyses and population-based studies favor early surgery. One recent large randomized trial has demonstrated that a delayed approach increases morbidity and cost compared to early surgery within 24 hours of hospital admission. Since cases of severe cholecystitis were excluded from this trial, we argue that these results do not reflect real-world clinical situations. From our point of view, these results were in contrast to the clinical experience with our patients; so, we decided to analyze critically all our patients with the null hypothesis that the patients treated with a delayed cholecystectomy after an acute cholecystitis have a similar or even better outcome than those treated with an early operative approach.

Patients and methods: We retrospectively analyzed clinical data from all patients with cholecystectomies in the period between January 2006 and September 2015. A total of 1,723 patients were categorized into four groups: early (n=138): urgent surgery of patients with acute cholecystitis within the first 72 hours of the onset of symptoms; intermediate (n=297): surgery of patients with acute cholecystitis within an average of 10 days after the onset of symptoms; delayed (n=427): initial non-surgical treatment of acute cholecystitis with surgery performed within 6-12 weeks of the onset of symptoms; and elective (n=868): cholecystectomy within a symptom-free interval of choice in patients with symptomatic cholecystolithiasis without signs of acute cholecystitis.

Results: In a real-world scenario, early/intermediate cholecystectomy in acute cholecystitis was associated with a significant increase in morbidity and mortality (Clavien-Dindo score) compared to a delayed approach with surgery performed 6-12 weeks after the onset of symptoms. The adjusted linear rank statistics showed a decrease in the complication score with values of 2.29 in the early group, 0.48 in the intermediate group, -0.26 in the delayed group and -2.12 in the elective group. The results translate into a continuous decrease of the complication score from early over intermediate and delayed to the elective group.

Conclusion: These results demonstrate that delayed cholecystectomy can be performed safely. In cases with severe cholecystitis, early and/or intermediate approaches still have a relatively high risk of morbidity and mortality.

Introduction: Dysphagia is frequently reported after thyroidectomy. Here, we investigated the incidence of postoperative dysphagia after uncomplicated thyroidectomy and parathyroidectomy. Further, we analyzed diagnosis and types of therapy to identify possible patients at risk.

Patients and methods: A questionnaire was sent to 372 consecutive patients whose thyroid or parathyroid glands were operated on between May 2013 and October 2014 at Ulm University Hospital. Patients were questioned at least 6 months postoperatively.

Results: In the evaluation, 219 questionnaires could be included. Fifty-three (21.3%) patients reported that the overall postoperative swallowing process was better or more trouble-free. In 110 (50.2%) patients, dysphagia was reported only immediately postoperative and disappeared later spontaneously. Sixteen patients (7.3%) stated that after a maximum of 3 months after surgery they suffered from dysphagia. One (0.5%) patient stated that up to 3 months postoperatively, swallowing problems had been successfully treated by logopedic therapy. In 39 (17.6%) patients, the complaints persisted for more than 3 months or still existed at the time of the interview. We found no correlation between dysphagia and patients' age or gender, the specimen volume, and patients' body mass index. The more invasive the operation was, the more patients suffered from dysphagia. Analyzing the frequency of dysphagia according to different diagnoses, we found a significant risk of postoperative dysphagia in patients with Graves' disease and carcinoma. Patients operated on for hyperparathyroidism were at significantly decreased risk of dysphagia.

Conclusion: Nearly 20% of patients reported postoperative dysphagia after uncomplicated thyroidectomy and parathyroidectomy, especially after major surgical intervention. We found a significant risk of postoperative dysphagia in patients with Graves' disease and carcinoma and a decreased risk for patients operated on for hyperparathyroidism.

Background: Acceptability of alcohol consumption varies wildly across cultures. Several factors such as religious beliefs and social desirability might influence reporting of such behaviors to researchers during relevant investigations.

Aims: This study aimed at assessing reporting of alcohol consumption during participation in research studies in Jordan, and identifying potential reasons and ethical challenges associated with reporting this behavior.

Subjects and methods: A sample of 400 Jordanians was anonymously surveyed regarding alcohol consumption reporting.

Results: The study showed a tendency of not reporting alcohol consumption in research (56.8%). Religious belief and trust issues regarding reporting sensitive information during participation in research were significantly the main reasons of not reporting alcohol drinking (P<0.05), while social shame effect was limited to rural areas (P<0.05).

Conclusion: Raising Jordanians' awareness of benefits of reporting alcohol consumption is highly recommended. Improving confidence in privacy and data confidentiality among Jordanians might help in improving the level of reporting during participation in research.

Background: The aim of the study was to explore rhinitis therapy purchases in different Australian regions for patients with and without additional respiratory disease, using both doctor's prescriptions and over-the-counter (OTC) medications.

Patients and methods: It was a historical cohort study of pharmacy-related claims that included prescription or OTC rhinitis therapy, with or without asthma/COPD therapy, from January 2013 to December 2014.

Results: Overall, 4,247,193 prescription and OTC rhinitis treatments were purchased from 909 pharmacies over a calendar year; the majority were single-therapy purchases for rhinitis only patients. More multiple-therapy was purchased for rhinitis and asthma/COPD patients (4.4%) than for rhinitis only patients (4.0%), with a greater proportion purchased in VIC, SA and TAS (4.7% of rhinitis only patients and 4.5% of rhinitis and asthma/COPD patients) than in other areas. Dual therapy of oral antihistamine (OAH) and intranasal corticosteroid (INS) were the most frequently purchased multiple-therapy, with higher purchasing rates for rhinitis and asthma/COPD patients (2.6%) than for rhinitis only patients (1.6%). The most frequently purchased single therapy was OAH (70.1% of rhinitis only patients and 57.3% of rhinitis and asthma/COPD patients). First-line INS therapy was more likely to be purchased for rhinitis and asthma/COPD patients (15.3% by prescription and 11.7% OTC) than for rhinitis only patients (5.0% by prescription and 9.2% OTC); however, geographical differences in the proportion of therapies purchased OTC were noted, with a lower proportion of OTC OAH and INS purchases in Queensland and the Northern Territory for patients with and without comorbid respiratory disease.

Conclusion: Purchases of first-line INS therapy are more likely for patients with comorbid respiratory disease if they have received prescriptions and information/advice from their general practitioner. The study results indicate a need for patient information/education at the point-of-sale of OTC OAHs to enable patients to assess their nasal symptoms and receive treatment support from pharmacists. Greater availability to INSs in pharmacies as well as guidance from current guidelines and instruction in correct intranasal technique may also lead to greater uptake of INSs.

Background: Observational research is essential to evaluate the real-life effectiveness of asthma treatments and can now make use of outcomes derived from electronic medical records.

Aim: The aim of this study was to investigate the utility of several database outcome measures in asthma.

Methods: This study identified cohorts of patients with active asthma from a UK primary care database - Optimum Patient Care Research Database - approximately 10% of which was prospectively supplemented with questionnaire data. The "Questionnaire cohort" included patients aged 18-60 years with valid questionnaire data and 1 year of continuous primary care data. Separate "ICS initiation" and "ICS step-up" cohorts included patients aged 5-60 years initiated on inhaled corticosteroids (ICSs), who had 1 year of continuous primary care data before, and after, this index visit. Database measures of asthma symptom control and exacerbations were identified in the Optimum Patient Care Research Database and cross-tabulated with corresponding patient-reported (questionnaire) data. Responsiveness of the database outcomes was analyzed, using McNemar's and Wilcoxon's signed rank tests, and Poisson regression was used to estimate the association between database outcomes and future risk of database exacerbations, in the ICS initiation cohort.

Results: The final study included 2,366 Questionnaire cohort patients and 51,404 ICS initiation patients. Agreement between patient-reported and database-recorded exacerbations was fair (kappa 0.35). Following the initiation of ICS, database risk domain asthma control (based on exacerbations) improved (proportion of patients with uncontrolled asthma decreased from 24.9% to 18.6%; P<0.001) and mean number of database exacerbations decreased from 0.09 to 0.08 per patient per year (P=0.001). However, another measure of asthma control which includes short-acting beta-agonist prescription as part of the definition did not show this improvement. Patients with prior exacerbations had a higher risk of future exacerbation (rate ratio [95% confidence interval], 3.23 [3.03-3.57]).

Conclusion: Asthma control and exacerbations derived from primary care databases were responsive, with the exception of short-acting beta-agonist prescriptions, and useful for risk prediction.