Pragmatic and Observational Research最新文献

Background: There is heightened interest in how real-world data (RWD) can be used to supplement or replace traditional mechanisms for collecting clinical information. A critical component in evaluating utility of RWD is assessing the validity and reliability of event measurement. Only two studies have validated Medicare claims with physician-adjudicated data collected in a clinical study and none in the pacemaker patient population. This study compares events identified in physician-adjudicated clinical registry data collected in the Micra Post-Approval Registry (PAR) with events identified via Medicare administrative claims in the Micra Coverage with Evidence (CED) Study.

Methods: Patients who were dually enrolled in the Micra CED and the Micra PAR between March 9, 2017 and December 1, 2017 were included in the validation analysis. All patients intended to be implanted with a Micra device were eligible for participation in the Micra PAR. All Medicare fee-for-service beneficiaries implanted with a Micra device who met the 12-month continuous enrollment criteria were included in the Micra CED. We compared the count of acute (30-day) complications identified in the Medicare claims and the physician-adjudicated PAR data to assess agreement between data sources.

Results: There were 230 patients dually enrolled in the Micra CED and Micra PAR studies during the study period. Overall, there were 17 acute events reported in either the Micra CED or the Micra PAR, with 95% agreement in the identification of events and absence of events between studies. Study disagreement between events reported in either study varied: arteriovenous fistula (50%), pulmonary embolism (67%), hemorrhage/hematoma (75%), and deep vein thrombosis (100%). Among physician-adjudicated events, there was no disagreement between the Micra CED and Micra PAR studies in any event type.

Conclusion: Findings from this study demonstrate high agreement in event identification between Medicare claims data and registries for patients implanted with Micra leadless pacemakers.

Background and objectives: To determine the rate of significant endometrial abnormalities in premenopausal women at low risk for endometrial hyperplasia and cancer presenting with abnormal uterine bleeding (AUB).

Patients and methods: This descriptive study was conducted from January 1, 2016 to March 31, 2019. The inclusion criteria were premenopausal women, 35-50 years, presenting with AUB, low risk for endometrial hyperplasia or endometrial cancer, and having undergone endometrial sampling or uterine curettage. Nulliparous, obesity, diabetes mellitus, polycystic ovary syndrome, chronic anovulation, infertility, tamoxifen therapy and/or a family history of uterine, ovarian, breast and colon cancer were excluded. Data regarding baseline characteristics were collected, and histopathology reports were reviewed.

Results: During the study period, 644 subjects were recruited, 557 of whom had adequate endometrial tissue for histopathology study. The pathology demonstrated benign in most cases (96%). The rate of significant abnormal endometrial pathology was 4% (23 cases) including 19 cases of endometrial hyperplasia without atypia (3.3%), and 4 cases of endometrial cancer (0.7%).

Conclusion: The rate of significant abnormal endometrial pathology in premenopausal women at low risk for endometrial hyperplasia or endometrial cancer presenting with AUB was very low. This information should be incorporated into the counseling process regarding the risks and benefits of endometrial sampling.

Purpose: The objective of this qualitative study was to explore patient, rheumatologist, and extended role practitioner (ERP) perspectives on the integration of an allied health rheumatology triage (AHRT) intervention in Ontario rheumatology clinics. Triage is the process of identifying the urgency of a patient's condition to ensure they receive specialist care within an appropriate length of time. This research explores the clinical/logistical impact of triage by occupational and physical therapists with advanced arthritis training (ERPs), including facilitators and barriers of success, and recommendations for future application.

Participants and methods: Semi-structured telephone interviews were held with participating rheumatologists, ERPs, and a sample of patients from each clinical site (4 community, 3 hospital) in five Ontario cities. Interviews were audio-recorded and transcribed verbatim. Transcripts were analyzed using basic qualitative description. Two independent researchers compared coding and achieved consensus.

Results: Patients (n=10), rheumatologists (n=6), and ERPs (n=5) participated in the study and reported reduced wait-times to rheumatology care, diagnosis, and treatment for those with inflammatory arthritis (IA). Rheumatologists and ERPs perceived that the intervention improved clinical efficiency and quality of care. Patients reported high satisfaction with ERP assessments, valuing early joint examination/laboratory tests, urgent referral if needed, and the provision of information, support, and management strategies. Facilitators of success included: supportive clinical staff, regular communication and collaboration between rheumatologist and ERP, and sufficient clinical space. Recommendations included extending ERP roles to include stable patient follow-up, and ERP care between scheduled rheumatology appointments.

Conclusion: Findings support the integration of ERPs in a triage role in the community and hospital-based rheumatology models of care. Future research is needed to explore the impact of utilizing ERPs for stable patient follow-up in rheumatology settings.

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.