Pragmatic and Observational Research最新文献

Introduction: There are many options available to patients who are placed on constant positive airway pressure (CPAP) for obstructive sleep apnea. Despite the success of CPAP in correcting apnea, a significant number of patients have difficulty with the therapy. A large number of those patients who have difficulty stop therapy and are often labeled as "CPAP Failure". Non-sleep specialists may view CPAP therapy as a singular course of treatment, but there are many ways CPAP may be ordered for a patient. Each patient experiences a unique set of options that constitute a unique order set.

Methods: In order to demonstrate the magnitude of the possible options, estimates of the number of unique order sets were calculated. The author chose individual order options and the number of selections possible within each option. The calculated sets included a "Generous, Limited and Minimal" number of selections for each option. Calculations were done separately for standard CPAP and for auto-adjusting CPAP. Additional calculations were performed using the number of commercially available masks in the United States.

Results: The maximum number of unique order sets was seen using a standard CPAP combined with commercially available masks: 49,152 unique order sets. The fewest number of unique order sets were seen with the auto-adjusting CPAP and the "Minimal" selections: 288 unique order sets.

Discussion: There are a large number of unique CPAP orders that a patient may experience. CPAP treatment is not a singular or simple therapy. When evaluating obstructive sleep apnea patients with histories of CPAP failure or prior difficulty with CPAP, paying close attention to the patient's treatment experiences may help explain a significant number of those patients' CPAP therapy problems.

Purpose: Chronic obstructive pulmonary disease (COPD) and heart failure (HF) often occur concomitantly, presenting diagnostic and therapeutic challenges for clinicians. We examined the characteristics of patients prescribed adequate versus inadequate therapy within 3 months after newly diagnosed comorbid COPD or HF.

Patients and methods: Eligible patients in longitudinal UK electronic medical record databases had pre-existing HF and newly diagnosed COPD (2017 GOLD groups B/C/D) or pre-existing COPD and newly diagnosed HF. Adequate COPD therapy was defined as long-acting bronchodilator(s) with/without inhaled corticosteroid; adequate HF therapy was defined as beta-blocker plus angiotensin-converting enzyme inhibitor and/or angiotensin receptor blocker.

Results: Of 2439 patients with HF and newly diagnosed COPD (mean 75 years, 61% men), adequate COPD therapy was prescribed for 726 (30%) and inadequate for 1031 (42%); 682 (28%) remained untreated for COPD. Adequate (vs inadequate) COPD therapy was less likely for women (35%) than men (45%), smokers (36%) than ex-/non-smokers (45%), and non-obese (41%) than obese (47%); spirometry was recorded for 57% prescribed adequate versus 35% inadequate COPD therapy. Of 12,587 patients with COPD and newly diagnosed HF (mean 75 years, 60% men), adequate HF therapy was prescribed for 2251 (18%) and inadequate for 5332 (42%); 5004 (40%) remained untreated for HF. Adequate (vs inadequate) HF therapy was less likely for smokers (27%) than ex-/non-smokers (32%) and non-obese (30%) than obese (35%); spirometry was recorded for 65% prescribed adequate versus 39% inadequate HF therapy.

Conclusion: Many patients with comorbid COPD/HF receive inadequate therapy after new diagnosis. Improved equity of access to integrated care is needed for all patient subgroups.

Objective: To evaluate the long-term safety and effectiveness of tadalafil in Japanese men with lower urinary tract symptoms secondary to benign prostatic hyperplasia in real-world clinical practice; and to investigate the safety profile in patients aged ≥75 years.

Patients and methods: This was a prospective, non-interventional, multicenter, post-marketing surveillance study in which Japanese patients with lower urinary tract symptoms secondary to benign prostatic hyperplasia were observed for up to 18 months after initiating tadalafil treatment. The real-world safety and effectiveness outcomes were assessed at baseline and at 1, 3, 6, 12, and 18 months post-treatment or the last day of treatment.

Results: Most patients received tadalafil 5 mg per day throughout the observation period. Among 1393 patients analyzed for safety, the overall incidence of adverse drug reactions was 8.3%. These adverse drug reactions were generally consistent with the known safety profile of tadalafil and no new safety risks were identified in long-term use. There was no statistical difference in the frequency of adverse drug reactions between patients aged <75 and ≥75 years. The mean change in total International Prostate Symptom Score (IPSS) and IPSS-quality of life subscore was significantly improved at each timepoint. At 18 months, IPSS had improved by 5.0 points (P < 0.001) and IPSS-quality of life subscore had improved by 1.5 points (P < 0.001). The mean change in post-voiding residual urine volume from baseline was significant at each time point and was -9.8 mL at 18 months (P < 0.001); there were no significant differences from baseline in maximum urinary flow rate.

Conclusion: This surveillance demonstrated that tadalafil has favorable safety and effectiveness profiles for long-term use in Japanese men with lower urinary tract symptoms secondary to benign prostatic hyperplasia. In addition, safety profiles in patients aged ≥75 years were similar to patients aged <75 years.

Background: As real-world data resources expand and improve, there will increasingly be opportunities to study the effectiveness of interventions. There is a need to ensure that study designs explore potential sources of bias and either acknowledge or mitigate them, in order to improve the accuracy of findings. The objective of this study was to understand newly approved drug utilization patterns in real-world clinical settings over time.

Methods: This retrospective study included three sources of real-world data (claims, electronic health records, and recoded data from a quality care program) collected from patients diagnosed with gastric cancer who initiated therapy with either trastuzumab or ramucirumab. Linear regression was used to investigate trends in the use of these drugs for the care of patients with gastric cancer over time from Food and Drug Administration (FDA) approval.

Results: Eligible patients (n=1700) had consistent demographic and clinical characteristics over time. After regulatory approval, trastuzumab was used in later lines of therapy and then shifted to earlier lines (p=0.002), while ramucirumab utilization remained consistent over time after FDA approval (p=0.49). Ramucirumab augmentation, defined as the addition of the drug after initiation of a line of therapy, decreased over time (p=0.03), and trastuzumab augmentation remained consistent over time (p=0.58).

Conclusion: Since treatment effectiveness may change across lines of treatment, bias may arise if there are changes in the use of the drug (such as line migration) during the time period of analysis using real-world data.

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.