Stefan Söderberg, Pilar Escribano-Subias, Ciara Archey, Audrey Muller, Martina Fontana, Tobias J Lange, Sean Gaine
{"title":"临床应用Selexipag治疗肺动脉高压患者的生存比较分析（暴露研究）。","authors":"Stefan Söderberg, Pilar Escribano-Subias, Ciara Archey, Audrey Muller, Martina Fontana, Tobias J Lange, Sean Gaine","doi":"10.1007/s40801-025-00488-9","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>In pulmonary arterial hypertension (PAH), comparative assessment of treatment effect on survival in randomized controlled settings of contemporary patients has not been feasible.</p><p><strong>Objective: </strong>The aim of this study was to use EXPOSURE, the ongoing, real-world, post-authorization safety study, and commitment to the European Medicines Agency to perform pre-specified comparative survival analyses between patients that newly initiated selexipag versus other PAH-specific therapies by applying statistical methods to account for population differences.</p><p><strong>Methods: </strong>EXPOSURE (EUPAS19085) is an observational study comprising patients with PAH who initiated selexipag or other PAH-specific therapy. To balance characteristics of patients in the other PAH therapy cohort with the selexipag cohort at PAH therapy initiation (baseline), propensity score weighting was applied. Mortality rate ratios (MRRs) were calculated.</p><p><strong>Results: </strong>2014 patients were available for analysis. Prior to applying propensity score weighting, patients in the selexipag cohort were more likely to have longer time from diagnosis, less functional impairment, and treatment with combination background therapy versus the other PAH therapy cohort. Following weighting, baseline variables for both cohorts were well balanced. Weighted treatment exposure was 827.9 and 840.5 person-years for the selexipag and modelled other PAH therapy cohort, respectively. Weighted proportion of deaths was lower in the selexipag versus modelled other PAH therapy cohort; MRR showed a higher survival rate for selexipag-treated patients (MRR [95% confidence interval]: 0.55 [0.31-0.99]).</p><p><strong>Conclusions: </strong>Survival analyses in EXPOSURE suggest a reduced risk of mortality among the cohort of patients newly initiated on selexipag compared with the modelled cohort newly initiated with other PAH-specific therapies. Further research is needed to confirm this observation.</p><p><strong>Trial registry: </strong>Trial registration: EUPAS19085.</p>","PeriodicalId":11282,"journal":{"name":"Drugs - Real World Outcomes","volume":" ","pages":""},"PeriodicalIF":1.9000,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study).\",\"authors\":\"Stefan Söderberg, Pilar Escribano-Subias, Ciara Archey, Audrey Muller, Martina Fontana, Tobias J Lange, Sean Gaine\",\"doi\":\"10.1007/s40801-025-00488-9\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>In pulmonary arterial hypertension (PAH), comparative assessment of treatment effect on survival in randomized controlled settings of contemporary patients has not been feasible.</p><p><strong>Objective: </strong>The aim of this study was to use EXPOSURE, the ongoing, real-world, post-authorization safety study, and commitment to the European Medicines Agency to perform pre-specified comparative survival analyses between patients that newly initiated selexipag versus other PAH-specific therapies by applying statistical methods to account for population differences.</p><p><strong>Methods: </strong>EXPOSURE (EUPAS19085) is an observational study comprising patients with PAH who initiated selexipag or other PAH-specific therapy. To balance characteristics of patients in the other PAH therapy cohort with the selexipag cohort at PAH therapy initiation (baseline), propensity score weighting was applied. Mortality rate ratios (MRRs) were calculated.</p><p><strong>Results: </strong>2014 patients were available for analysis. Prior to applying propensity score weighting, patients in the selexipag cohort were more likely to have longer time from diagnosis, less functional impairment, and treatment with combination background therapy versus the other PAH therapy cohort. Following weighting, baseline variables for both cohorts were well balanced. Weighted treatment exposure was 827.9 and 840.5 person-years for the selexipag and modelled other PAH therapy cohort, respectively. Weighted proportion of deaths was lower in the selexipag versus modelled other PAH therapy cohort; MRR showed a higher survival rate for selexipag-treated patients (MRR [95% confidence interval]: 0.55 [0.31-0.99]).</p><p><strong>Conclusions: </strong>Survival analyses in EXPOSURE suggest a reduced risk of mortality among the cohort of patients newly initiated on selexipag compared with the modelled cohort newly initiated with other PAH-specific therapies. Further research is needed to confirm this observation.</p><p><strong>Trial registry: </strong>Trial registration: EUPAS19085.</p>\",\"PeriodicalId\":11282,\"journal\":{\"name\":\"Drugs - Real World Outcomes\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":1.9000,\"publicationDate\":\"2025-04-30\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Drugs - Real World Outcomes\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1007/s40801-025-00488-9\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Drugs - Real World Outcomes","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1007/s40801-025-00488-9","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study).
Background: In pulmonary arterial hypertension (PAH), comparative assessment of treatment effect on survival in randomized controlled settings of contemporary patients has not been feasible.
Objective: The aim of this study was to use EXPOSURE, the ongoing, real-world, post-authorization safety study, and commitment to the European Medicines Agency to perform pre-specified comparative survival analyses between patients that newly initiated selexipag versus other PAH-specific therapies by applying statistical methods to account for population differences.
Methods: EXPOSURE (EUPAS19085) is an observational study comprising patients with PAH who initiated selexipag or other PAH-specific therapy. To balance characteristics of patients in the other PAH therapy cohort with the selexipag cohort at PAH therapy initiation (baseline), propensity score weighting was applied. Mortality rate ratios (MRRs) were calculated.
Results: 2014 patients were available for analysis. Prior to applying propensity score weighting, patients in the selexipag cohort were more likely to have longer time from diagnosis, less functional impairment, and treatment with combination background therapy versus the other PAH therapy cohort. Following weighting, baseline variables for both cohorts were well balanced. Weighted treatment exposure was 827.9 and 840.5 person-years for the selexipag and modelled other PAH therapy cohort, respectively. Weighted proportion of deaths was lower in the selexipag versus modelled other PAH therapy cohort; MRR showed a higher survival rate for selexipag-treated patients (MRR [95% confidence interval]: 0.55 [0.31-0.99]).
Conclusions: Survival analyses in EXPOSURE suggest a reduced risk of mortality among the cohort of patients newly initiated on selexipag compared with the modelled cohort newly initiated with other PAH-specific therapies. Further research is needed to confirm this observation.
期刊介绍：
Drugs - Real World Outcomes targets original research and definitive reviews regarding the use of real-world data to evaluate health outcomes and inform healthcare decision-making on drugs, devices and other interventions in clinical practice. The journal includes, but is not limited to, the following research areas: Using registries/databases/health records and other non-selected observational datasets to investigate: drug use and treatment outcomes prescription patterns drug safety signals adherence to treatment guidelines benefit : risk profiles comparative effectiveness economic analyses including cost-of-illness Data-driven research methodologies, including the capture, curation, search, sharing, analysis and interpretation of ‘big data’ Techniques and approaches to optimise real-world modelling.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
