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BACKGROUND: Patients with chronic thromboembolic pulmonary hypertension (CTEPH) in countries with limited resources have, to date, been poorly represented in registries. OBJECTIVE: This work assesses the epidemiology, diagnosis, hemodynamic and functional parameters, and treatment of CTEPH in Russia, Kazakhstan, Turkey, Lebanon, and Saudi Arabia. METHODS: A prospective, cohort, phase IV, observational registry with 3-year follow-up (n = 212) in patients aged ≥ 18 years diagnosed with CTEPH was created. Clinical, hemodynamic, and functional parameters were obtained at an initial visit, follow-up visits, and a final visit at the end of 3 years' observation or end of follow-up. Data were recorded on electronic case report forms. Parameters evaluated included 6-minute walking distance (6MWD), use of pulmonary endarterectomy (PEA), balloon pulmonary angioplasty (BPA), pulmonary hypertension (PH)-targeted therapy, and survival. All statistical analyses were exploratory and descriptive, and were performed in the overall population. RESULTS: The most common symptoms were typical of those expected for CTEPH. Almost 90% of patients underwent right heart catheterization at diagnosis or initial study visit. In total, 66 patients (31%) underwent PEA before the initial visit; 95 patients (45%) were considered operable, 115 (54%) were inoperable, and two (1%) had no operability data. Only 26 patients (12%) had been assessed for BPA at their initial visit. PH-targeted therapy was documented at diagnosis for 77 patients (36%), most commonly a phosphodiesterase type 5 inhibitor (23%). Use of PH-targeted therapy increased to 142 patients (67%) at the initial visit, remaining similar after 3 years. Use of riociguat increased from 6% of patients at diagnosis to 38% at 3 years. Between baseline and end of observation, results for patients with paired data showed an increase in 6MWD. Survival at the end of observation was 88%. CONCLUSIONS: These data highlight the current diagnosis and management of CTEPH in the participating countries. They show that early CTEPH diagnosis remains challenging, and use of off-label PH-targeted therapy is common. CLINICALTRIALS: gov: NCT02637050; registered December 2015.
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BACKGROUND: The use of digital technology to assess patients remotely can reduce clinical study costs. In the European Union, the 2D matrix code on prescription drug packaging serves as a unique identifier of a given package of medication, and thus, also of the patient receiving that medication. Scanning of the 2D matrix code may therefore allow remote patient authentication in clinical studies. OBJECTIVE: The aim of the DePRO study was to assess the feasibility of a fully digital data-capture workflow, the authentication of participants via drug packaging 2D matrix codes, in patients with type 2 diabetes mellitus (T2DM) who use metformin. The primary objective was to describe the self-care activities of these patients. Secondary objectives were to evaluate (1) the self-reported health status of these patients, (2) the association of self-care activities with demographics and disease characteristics, and (3) the usability of the my ePRO app. METHODS: DePRO was an observational, multicenter, cross-sectional, digital, and patient-driven study conducted in Germany from June to December 2020. Adult patients prescribed metformin were invited to participate via their pharmacist or a medication tracker app. Participants downloaded the my ePRO app onto their own mobile device, scanned the 2D matrix code on their metformin package for registration and authentication, and provided informed consent via an electronic form. They were then able to complete a study-specific questionnaire on demographics and clinical characteristics, the German version of the Summary of Diabetes Self-Care Activities measure (SDSCA-G), the Diabetes Treatment Satisfaction Questionnaire (DTSQ), and the EQ-5D-5L. The patients conducted the study without support from a health care professional. Statistical analyses were exploratory and descriptive. RESULTS: In total, 3219 patients were invited to participate. The proportion of patients giving consent was greater among those invited by pharmacists (19/217, 8.8%) than among those invited via the medication tracker app (13/3002, 0.4%). Of the 29 patients eligible for analysis, 28 (97%) completed all study questionnaires. Most of the patients (23/29, 79%) were aged <60 years, and 59% (17/29) were male. The patients spent a mean total of 3.5 (SD 1.3) days out of 7 days on self-care activities (SDSCA-G). Most patients (24/29, 83%) were satisfied to extremely satisfied with their current treatment (DTSQ). Events of perceived hyperglycemia or hypoglycemia were reported by 20 of 29 (69%) patients. The best possible health status (EQ-5D-5L) was reported by 18 of 28 (64%) patients. Age was positively correlated with time spent on general and specific diet (Spearman coefficient 0.390 and 0.434, respectively). CONCLUSIONS: The DePRO study demonstrates the feasibility of fully digital authentication (via 2D matrix codes on drug packaging) and data capture in patients with T2DM. Personal invitations yielded higher recruitment rates than remote invitations via the medication tracker app. A high questionnaire completion rate was realized, based on completion by 28 out of 29 patients. TRIAL REGISTRATION: ClinicalTrials.gov NCT04383041; https://clinicaltrials.gov/ct2/show/NCT04383041. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): RR2-10.2196/21727.
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BACKGROUND: Diabetes mellitus (DM) is one of the most common noncommunicable diseases. DM has a substantial negative impact on patients' quality of life, which is measured using a variety of diabetes-specific measures covering multiple aspects of patients' psychological state, behavior, and treatment satisfaction. A fully digital data collection system, including patient identification, would represent a substantial advance in how these patient-reported outcome (PRO) data are measured. Within the European Union, one way to identify patients without the involvement of health care professionals is to use the unique 2D matrix codes on the packaging of prescription medication-for example, metformin, the recommended initial treatment for patients with type 2 DM (T2DM). OBJECTIVE: In the DePRO study we aim to (1) describe the self-care activities of patients with T2DM using metformin-containing medication; (2) describe the self-reported health status (eg, presence of diabetes complications and quality of life) of these patients; (3) describe associations between self-care activities and demographics and disease characteristics; and (4) assess the usability of the my ePRO app. METHODS: DePRO is an observational, multicenter, cross-sectional, digital, patient-driven study conducted in Germany. Patients with a prescription for a metformin-containing medication will be given a postcard by their pharmacist, which will include a download link for the my ePRO app. In total, 12 diabetes-focused pharmacies, selected to represent urban and rural areas, will be recruited. Participants will use their own mobile device (bring your own device) to download the my ePRO app and access the DePRO study, for which they can register using the 2D matrix code on their medication. An electronic informed consent form will be displayed to the patients and only after giving consent will patients be able to complete the study questionnaires. The PRO instruments used in the study are the Summary of Diabetes Self-Care Activities Scale, the Diabetes Treatment Satisfaction Questionnaire, and the 5 level, 5-dimension EuroQol Questionnaire. Patients will also be asked to complete a questionnaire with items addressing demographics, patient characteristics, disease history, complications, and concomitant medications. Data will be transferred to the study database by the app upon completion of each questionnaire. Statistical analyses of primary and secondary endpoints will be exploratory and descriptive. RESULTS: Enrollment began in June 2020. The estimated study completion date is December 31, 2020, and the planned sample size is 300 patients. CONCLUSIONS: The DePRO study uses completely digital data collection, including authentication of eligible patients and completion of the study questionnaires. Therefore, the design of the DePRO study represents a substantial advance in the evaluation of the digital capturing of PRO data. TRIAL REGISTRATION: ClinicalTrials.gov NCT04383041; https://clinicaltrials.gov/ct2/show/NCT04383041. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/21727.
