The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care
Abstract Background The EMPA‐REG OUTCOME trial showed that empagliflozin reduced the risk of cardiovascular death and hospitalization for heart failure (HHF) in diabetic patients with cardiovascular disease. EMPRISE is a study programme on the effectiveness, safety and healthcare utilization of empa...
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| Format: | Article |
| Language: | English |
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Wiley
2020-01-01
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| Series: | Endocrinology, Diabetes & Metabolism |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/edm2.103 |
| _version_ | 1818306190282588160 |
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| author | Elisabetta Patorno Mehdi Najafzadeh Ajinkya Pawar Jessica M. Franklin Anouk Déruaz‐Luyet Kimberly G. Brodovicz Adrian J. Santiago Ortiz Lily G. Bessette Martin Kulldorff Sebastian Schneeweiss |
| author_facet | Elisabetta Patorno Mehdi Najafzadeh Ajinkya Pawar Jessica M. Franklin Anouk Déruaz‐Luyet Kimberly G. Brodovicz Adrian J. Santiago Ortiz Lily G. Bessette Martin Kulldorff Sebastian Schneeweiss |
| author_sort | Elisabetta Patorno |
| collection | DOAJ |
| description | Abstract Background The EMPA‐REG OUTCOME trial showed that empagliflozin reduced the risk of cardiovascular death and hospitalization for heart failure (HHF) in diabetic patients with cardiovascular disease. EMPRISE is a study programme on the effectiveness, safety and healthcare utilization of empagliflozin in routine care, leveraging real‐world data from two commercial and one federal US data sources from 2014 to 2019. Objectives To describe rationale and design of EMPRISE, assess ability to minimize confounding and evaluate the time to reach sufficient statistical power for a key study outcome, HHF, using baseline information from the first year of EMPRISE. Methods In 3 claims data sets, we identified a 1:1 propensity score (PS)‐matched cohort of diabetic patients ≥18 years initiating empagliflozin or a dipeptidyl peptidase‐4 inhibitor (DPP4i), resulting in 6643 total pairs. The PS model included >140 baseline covariates. We measured covariate balance via standardized differences (SD) and postmatching c‐statistic. We computed the incidence rate (IR) of HHF, predicted exposure accrual over time and calculated expected power. Results After PS matching, patient characteristics were balanced with SD <0.1 and c‐statistic between 0.54 and 0.59. The population IR of HHF was 4.4 per 1000 person‐years using a specific HHF definition and 14.8 using a broader HHF definition. In our projection, 80%‐powered analyses would require a minimum of 169 HHF events, expected to accumulate by year 3 (specific definition) or year 2 (broader definition). Conclusion Baseline information from EMPRISE provided evidence of solid confounding control and adequate exposure accrual with expected powered analyses for the primary outcomes. |
| first_indexed | 2024-12-13T06:38:33Z |
| format | Article |
| id | doaj.art-3b1d4ccae1a34e5da8d6f66e7b2e3401 |
| institution | Directory Open Access Journal |
| issn | 2398-9238 |
| language | English |
| last_indexed | 2024-12-13T06:38:33Z |
| publishDate | 2020-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Endocrinology, Diabetes & Metabolism |
| spelling | doaj.art-3b1d4ccae1a34e5da8d6f66e7b2e34012022-12-21T23:56:29ZengWileyEndocrinology, Diabetes & Metabolism2398-92382020-01-0131n/an/a10.1002/edm2.103The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical careElisabetta Patorno0Mehdi Najafzadeh1Ajinkya Pawar2Jessica M. Franklin3Anouk Déruaz‐Luyet4Kimberly G. Brodovicz5Adrian J. Santiago Ortiz6Lily G. Bessette7Martin Kulldorff8Sebastian Schneeweiss9Division of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USADivision of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USADivision of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USADivision of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USABoehringer Ingelheim GmbH Ingelheim GermanyBoehringer Ingelheim Pharmaceuticals, Inc. Ridgefield CT USADivision of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USADivision of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USADivision of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USADivision of Pharmacoepidemiology and Pharmacoeconomics Department of Medicine Brigham and Women’s Hospital and Harvard Medical School Boston MA USAAbstract Background The EMPA‐REG OUTCOME trial showed that empagliflozin reduced the risk of cardiovascular death and hospitalization for heart failure (HHF) in diabetic patients with cardiovascular disease. EMPRISE is a study programme on the effectiveness, safety and healthcare utilization of empagliflozin in routine care, leveraging real‐world data from two commercial and one federal US data sources from 2014 to 2019. Objectives To describe rationale and design of EMPRISE, assess ability to minimize confounding and evaluate the time to reach sufficient statistical power for a key study outcome, HHF, using baseline information from the first year of EMPRISE. Methods In 3 claims data sets, we identified a 1:1 propensity score (PS)‐matched cohort of diabetic patients ≥18 years initiating empagliflozin or a dipeptidyl peptidase‐4 inhibitor (DPP4i), resulting in 6643 total pairs. The PS model included >140 baseline covariates. We measured covariate balance via standardized differences (SD) and postmatching c‐statistic. We computed the incidence rate (IR) of HHF, predicted exposure accrual over time and calculated expected power. Results After PS matching, patient characteristics were balanced with SD <0.1 and c‐statistic between 0.54 and 0.59. The population IR of HHF was 4.4 per 1000 person‐years using a specific HHF definition and 14.8 using a broader HHF definition. In our projection, 80%‐powered analyses would require a minimum of 169 HHF events, expected to accumulate by year 3 (specific definition) or year 2 (broader definition). Conclusion Baseline information from EMPRISE provided evidence of solid confounding control and adequate exposure accrual with expected powered analyses for the primary outcomes.https://doi.org/10.1002/edm2.103comparative effectivenessconfounding (epidemiology)empagliflozinheart failurereal‐world datastudy validity |
| spellingShingle | Elisabetta Patorno Mehdi Najafzadeh Ajinkya Pawar Jessica M. Franklin Anouk Déruaz‐Luyet Kimberly G. Brodovicz Adrian J. Santiago Ortiz Lily G. Bessette Martin Kulldorff Sebastian Schneeweiss The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care Endocrinology, Diabetes & Metabolism comparative effectiveness confounding (epidemiology) empagliflozin heart failure real‐world data study validity |
| title | The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care |
| title_full | The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care |
| title_fullStr | The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care |
| title_full_unstemmed | The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care |
| title_short | The EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) study programme: Design and exposure accrual for an evaluation of empagliflozin in routine clinical care |
| title_sort | empagliflozin comparative effectiveness and safety emprise study programme design and exposure accrual for an evaluation of empagliflozin in routine clinical care |
| topic | comparative effectiveness confounding (epidemiology) empagliflozin heart failure real‐world data study validity |
| url | https://doi.org/10.1002/edm2.103 |
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