P4673Macitentan in chronic thromboembolic pulmonary hypertension (CTEPH): combined data from OPUS and OrPHeUS real-world data sets

2019 ◽  
Vol 40 (Supplement_1) ◽  
Author(s):  
R Channick ◽  
K Chin ◽  
N Kim ◽  
J McConnell ◽  
D Poch ◽  
...  
Keyword(s):  
Pulmonary Hypertension ◽  
Incidence Rate ◽  
Real World ◽  
Chronic Thromboembolic Pulmonary Hypertension ◽  
Liver Function Tests ◽  
Functional Class ◽  
Real World Data ◽  
Data Set ◽  
World Data

Abstract Introduction The OPsumit® USers Registry (OPUS) provides real-world data in patients (pts) with pulmonary hypertension newly started on macitentan. The OPsumit® Historical USers cohort (OrPHeUS) study was conducted to supplement OPUS to fulfil the FDA request to characterise the safety of macitentan in clinical practice. Both studies included pts with CTEPH. Purpose To describe the characteristics and safety outcomes of CTEPH pts newly treated with macitentan. Methods OPUS is a prospective, US, multicentre, long-term, observational drug registry (NCT02126943) ongoing since Apr 2014. OrPHeUS was a retrospective, US, multicentre medical chart review (NCT03197688); data collected Oct 2013-Mar 2017. This subgroup analysis describes CTEPH patient characteristics at macitentan initiation, hospitalisations, survival and liver function tests (LFTs) in the combined OPUS/OrPHeUS data set. LFTs are also provided for the OPUS/OrPHeUS pulmonary arterial hypertension (PAH) population. Results As of Oct 2018, OPUS/OrPHeUS included 144 CTEPH pts with follow-up data. At macitentan initiation, median (Q1, Q3) age was 66 (56, 73) years; of the 53 pts with a WHO functional class (FC) assessment, 35.8%, 49.1% and 15.1% were in FC II, III and IV, respectively. Median (Q1, Q3) time from CTEPH diagnosis was 18.6 (7.4, 56.4) months (n=141). At macitentan initiation, 32.6% of pts had no PAH therapy, 63.9% were receiving one and 3.5% were receiving two PAH therapies. Median (Q1, Q3) exposure to macitentan was 15.9 (4.3, 30.4) months. There were 43 (29.9%) CTEPH pts who discontinued treatment; 23 due to an adverse event (AE), 0 due to a hepatic AE (HAE) and 20 not due to an AE/HAE. There were 58 (40.3%) CTEPH pts who experienced ≥1 hospitalisation and 9 (6.3%) pts died. LFTs are presented in the table. LFTs CTEPH follow-up pts PAH follow-up pts (N=144) (N=4072) ALT or AST ≥3x ULN   Pts with ≥1 event, n (%) 1 (0.7) 132 (3.2)   Incidence rate per 1 person-year (95% CI) 0.005 (0.001, 0.035) 0.024 (0.020, 0.029) ALT or AST ≥3x ULN and total bilirubin ≥2x ULN   Pts with ≥1 event, n (%) 1 (0.7) 27 (0.7)   Incidence rate per 1 person-year (95% CI) 0.005 (0.001, 0.035) 0.005 (0.003, 0.007) ALT: alanine aminotransferase; AST: aspartate aminotransferase; CI: confidence interval; ULN: upper limit of normal. Conclusions Analysis of OPUS/OrPHeUS data provides further insight into real-world use of macitentan in CTEPH pts. The observed hepatic safety profile in CTEPH pts is in line with that of PAH pts. Acknowledgement/Funding Actelion Pharmaceuticals Ltd

Combination therapy with macitentan and phosphodiesterase type-5 inhibitor (PDE5i) in pulmonary arterial hypertension (PAH): real-world data from OPUS and OrPHeUS

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
V McLaughlin ◽  
R Channick ◽  
N.H Kim ◽  
M Flynn ◽  
S Leroy ◽  
...  
Keyword(s):  
Combination Therapy ◽  
Real World ◽  
Patient Characteristics ◽  
Functional Class ◽  
Funding Source ◽  
Real World Data ◽  
World Data ◽  
Start Date ◽  
Phosphodiesterase Type 5 Inhibitor

Abstract Introduction Guidelines for the management of PAH recommend combination therapy for most patients, yet real-world data on treatment patterns in PAH are limited. Purpose To describe the characteristics, safety and clinical outcomes of PAH patients newly treated with double combination therapy with macitentan and PDE5i in the OPsumit® USers (OPUS) Registry and the OPsumit® Historical USers cohort study (OrPHeUS) combined dataset. Methods OPUS is a prospective, US, multicentre, observational drug registry (NCT02126943) ongoing since April 2014. OrPHeUS was a retrospective, US, multicentre chart review (NCT03197688); Oct 2013–Mar 2017. This cohort included patients initiating combination therapy with macitentan and a PDE5i (in any order) ≤60 days apart. Baseline was defined as the start date of the second therapy (i.e., start of combination therapy). Patient characteristics at baseline, changes in 6-minute walk distance (6MWD) and WHO functional class (FC) from baseline to follow-up, safety and outcomes are described. Results Of the 4428 OPUS/OrPHeUS PAH patients initiating macitentan, 2490 received this in combination with a PDE5i; of these patients, 740 (29.7%) initiated macitentan and a PDE5i concurrently (≤60 days apart). Data on disease duration was recorded in 729 patients at baseline; of these, 588 (80.7%) patients were incident (≤6 months since diagnosis) and 141 (19.3%) were prevalent (>6 months since diagnosis); median time from diagnosis to start of combination therapy of was 1.4 (Q1=0.6, Q3=3.6) months. At baseline, median age was 60 (Q1=49, Q3=70) years and 73.6% of patients were female. Mean baseline 6MWD was 264.5 (SD=119.8) m, recorded in 240 (32.4%) patients. WHO FC was recorded at baseline for 347 (46.9%) patients; 263 (75.8%) were in FC III/IV. Median combination therapy exposure was 10.2 (Q1=3.4, Q3=21.8) months, with 58.8% of patients ongoing at data cut. Changes from baseline to follow up in FC and 6MWD are shown in the figure. There was ≥1 adverse event (AE) reported in 455 (61.5%) patients and ≥1 hepatic AE (HAE) in 76 (10.3%) patients. In total, 232 (31.4%) patients discontinued macitentan; 122 (16.5%) due to AEs, 4 (0.5%) due to HAEs, 98 (13.2%) not due to an AE/HAE, and 8 (1.1%) for unknown reasons. Of the 305 patients who discontinued combination therapy, 137 (18.5%) discontinued macitentan only, 73 (9.9%) discontinued PDE5i only, and 95 (12.8%) discontinued both drugs at the same time. Kaplan-Meier estimates (95% CI) showed that 60.7% (56.4, 64.8) of patients were free from hospitalisation and 88.7% (85.7, 91.1) were alive at 12 months. Conclusions In this real-world setting, less than one third of patients treated with macitentan received initial oral combination therapy, despite current expert consensus favouring such therapeutic approaches. Patients initiating macitentan+PDE5i ≤60 days apart had improved 6MWD and WHO FC from baseline to follow-up. Funding Acknowledgement Type of funding source: Other. Main funding source(s): Actelion Pharmaceuticals Ltd

928-P: Dulaglutide Has Higher Adherence and Persistence than Semaglutide and Exenatide QW: 6-Month Follow-Up from U.S. Real-World Data

Diabetes ◽  
2020 ◽  
Vol 69 (Supplement 1) ◽  
pp. 928-P
Author(s):  
REEMA MODY ◽  
MARIA YU ◽  
BAL K. NEPAL ◽  
MANIGE KONIG ◽  
MICHAEL GRABNER
Keyword(s):  
Real World ◽  
Real World Data ◽  
World Data ◽  
Exenatide Qw

scholarly journals Application of Health Technology Assessment (HTA) to Evaluate New Laboratory Tests in a Health System: A Case Study of Bladder Cancer Testing

2020 ◽  
Vol 7 ◽  
pp. 237428952096822
Author(s):  
Erik J. Landaas ◽  
Ashley M. Eckel ◽  
Jonathan L. Wright ◽  
Geoffrey S. Baird ◽  
Ryan N. Hansen ◽  
...  
Keyword(s):  
Bladder Cancer ◽  
Pilot Study ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Test System ◽  
Real World Data ◽  
World Data

We describe the methods and decision from a health technology assessment of a new molecular test for bladder cancer (Cxbladder), which was proposed for adoption to our send-out test menu by urology providers. The Cxbladder health technology assessment report contained mixed evidence; predominant concerns were related to the test’s low specificity and high cost. The low specificity indicated a high false-positive rate, which our laboratory formulary committee concluded would result in unnecessary confirmatory testing and follow-up. Our committee voted unanimously to not adopt the test system-wide for use for the initial diagnosis of bladder cancer but supported a pilot study for bladder cancer recurrence surveillance. The pilot study used real-world data from patient management in the scenario in which a patient is evaluated for possible recurrent bladder cancer after a finding of atypical cytopathology in the urine. We evaluated the type and number of follow-up tests conducted including urine cytopathology, imaging studies, repeat cystoscopy evaluation, biopsy, and repeat Cxbladder and their test results. The pilot identified ordering challenges and suggested potential use cases in which the results of Cxbladder affected a change in management. Our health technology assessment provided an objective process to efficiently review test performance and guide new test adoption. Based on our pilot, there were real-world data indicating improved clinician decision-making among select patients who underwent Cxbladder testing.

Continuous Glucose Monitoring in Adults with Type 1 Diabetes: Real-World Data from the German/Austrian Prospective Diabetes Follow-Up Registry

2020 ◽  
Vol 22 (8) ◽  
pp. 602-612
Author(s):  
Dirk Sandig ◽  
Julia Grimsmann ◽  
Christina Reinauer ◽  
Andreas Melmer ◽  
Stefan Zimny ◽  
...  
Keyword(s):  
Type 1 Diabetes ◽  
Continuous Glucose Monitoring ◽  
Real World ◽  
Glucose Monitoring ◽  
Real World Data ◽  
World Data

scholarly journals P335 Long-term follow-up of switching from original infliximab to infliximab biosimilar: real-world data

2019 ◽  
Vol 13 (Supplement_1) ◽  
pp. S268-S269
Author(s):  
M Guerra Veloz ◽  
M Belvis Jimenez ◽  
T Valdes Delgado ◽  
L Castro Laria ◽  
B Maldonado Pérez ◽  
...  
Keyword(s):  
Real World ◽  
Real World Data ◽  
World Data ◽  
Long Term Follow Up

Safety and efficacy analysis of long-term follow up real-world data with ibrutinib monotherapy in 58 patients with CLL treated in a single-center in Greece

2019 ◽  
Vol 60 (12) ◽  
pp. 2939-2945 ◽  
Author(s):  
Maria Dimou ◽  
Theodoros Iliakis ◽  
Vasileios Pardalis ◽  
Catherin Bitsani ◽  
Theodoros P. Vassilakopoulos ◽  
...  
Keyword(s):  
Real World ◽  
Single Center ◽  
Real World Data ◽  
Safety And Efficacy ◽  
World Data ◽  
Efficacy Analysis ◽  
Long Term Follow Up

scholarly journals P705 Switching from originator to biosimilar infliximab—real-world data from 18 months prospective follow-up of a single-centre IBD population

2018 ◽  
Vol 12 (supplement_1) ◽  
pp. S467-S468
Author(s):  
M L Hoivik ◽  
L C Buer ◽  
N Bolstad ◽  
B Moum ◽  
A W Medhus
Keyword(s):  
Real World ◽  
Single Centre ◽  
Real World Data ◽  
World Data

scholarly journals Consensus Development of a Modern Ontology of Emergency Department Presenting Problems—The Hierarchical Presenting Problem Ontology (HaPPy)

2019 ◽  
Vol 10 (03) ◽  
pp. 409-420 ◽  
Author(s):  
Steven Horng ◽  
Nathaniel R. Greenbaum ◽  
Larry A. Nathanson ◽  
James C. McClay ◽  
Foster R. Goss ◽  
...  
Keyword(s):  
Emergency Department ◽  
Real World ◽  
Emergency Department Patient ◽  
Snomed Ct ◽  
Presenting Problems ◽  
Real World Data ◽  
Validation Data ◽  
Data Set ◽  
World Data ◽  
Academic Level

Objective Numerous attempts have been made to create a standardized “presenting problem” or “chief complaint” list to characterize the nature of an emergency department visit. Previous attempts have failed to gain widespread adoption as they were not freely shareable or did not contain the right level of specificity, structure, and clinical relevance to gain acceptance by the larger emergency medicine community. Using real-world data, we constructed a presenting problem list that addresses these challenges. Materials and Methods We prospectively captured the presenting problems for 180,424 consecutive emergency department patient visits at an urban, academic, Level I trauma center in the Boston metro area. No patients were excluded. We used a consensus process to iteratively derive our system using real-world data. We used the first 70% of consecutive visits to derive our ontology, followed by a 6-month washout period, and the remaining 30% for validation. All concepts were mapped to Systematized Nomenclature of Medicine–Clinical Terms (SNOMED CT). Results Our system consists of a polyhierarchical ontology containing 692 unique concepts, 2,118 synonyms, and 30,613 nonvisible descriptions to correct misspellings and nonstandard terminology. Our ontology successfully captured structured data for 95.9% of visits in our validation data set. Discussion and Conclusion We present the HierArchical Presenting Problem ontologY (HaPPy). This ontology was empirically derived and then iteratively validated by an expert consensus panel. HaPPy contains 692 presenting problem concepts, each concept being mapped to SNOMED CT. This freely sharable ontology can help to facilitate presenting problem-based quality metrics, research, and patient care.

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