Outcome of adults with Eisenmenger syndrome treated with drugs specific to pulmonary arterial hypertension: A French multicentre study

• DOI: 10.1016/j.acvd.2017.01.006
• Publication Date: 2017-03-09T12:15:30Z
• Authors: Sebastien Hascoet; Emmanuelle Fournier; Xavier Jaïs; Lauriane Le Gloan; Claire Dauphin; Ali Houeijeh; Francois Godart; Xavier Iriart; Adelaïde Richard; Jelena Radojevic; Pascal Amedro; Gilles Bosser; Nathalie Souletie; Yvette Bernard; Pamela Moceri; Hélène Bouvaist; Pierre Mauran; Elise Barre; Adeline Basquin; Clement Karsenty; Damien Bonnet; Laurence Iserin; Olivier Sitbon; Jérôme Petit; Elie Fadel; Marc Humbert; Magalie Ladouceur
• Keywords: Adult; Male; Adolescent; Hypertension, Pulmonary; Kaplan-Meier Estimate; Pulmonary arterial hypertension; Congenital heart diseases; Disease-Free Survival; 03 medical and health sciences; 0302 clinical medicine; [SDV.MHEP.CSC]Life Sciences [q-bio]/Human health and pathology/Cardiology and cardiovascular system; Cause of Death; Pronostic; Humans; Arterial Pressure; Longitudinal Studies; Child; Antihypertensive Agents; Outcome; Chi-Square Distribution; Cardiopathies congénitales; Age Factors; Eisenmenger syndrome; Hypertension artérielle pulmonaire; [SDV.SP]Life Sciences [q-bio]/Pharmaceutical sciences; Eisenmenger Complex; Middle Aged; Syndrome d’Eisenmenger; [SDV.MHEP.CSC] Life Sciences [q-bio]/Human health and pathology/Cardiology and cardiovascular system; [SDV.SP] Life Sciences [q-bio]/Pharmaceutical sciences; [SDV.SPEE] Life Sciences [q-bio]/Santé publique et épidémiologie; Multivariate Analysis; Disease Progression; [SDV.SPEE]Life Sciences [q-bio]/Santé publique et épidémiologie; Female; Drug therapy; France; Médicament

The relationship between pulmonary arterial hypertension-specific drug therapy (PAH-SDT) and mortality in Eisenmenger syndrome (ES) is controversial.To investigate outcomes in patients with ES, and their relationship with PAH-SDT.Retrospective, observational, nationwide, multicentre cohort study.We included 340 patients with ES: genetic syndrome (n=119; 35.3%); pretricuspid defect (n=75; 22.1%). Overall, 276 (81.2%) patients received PAH-SDT: monotherapy (endothelin receptor antagonist [ERA] or phosphodiesterase 5 inhibitor [PDE5I]) 46.7%; dual therapy (ERA+PDE5I) 40.9%; triple therapy (ERA+PDE5I+prostanoid) 9.1%. Median PAH-SDT duration was 5.5 years [3.0-9.1 years]. Events (death, lung or heart-lung transplantation) occurred in 95 (27.9%) patients at a median age of 40.5 years [29.4-47.6]. The cumulative occurrence of events was 16.7% [95% confidence interval 12.8-21.6%] and 46.4% [95% confidence interval 38.2-55.4%] at age 40 and 60 years, respectively. With age at evaluation or time since PAH diagnosis as time scales, cumulative occurrence of events was lower in patients taking one or two PAH-SDTs (P=0.0001 and P=0.004, respectively), with the largest differences in the post-tricuspid defect subgroup (P<0.001 and P<0.02, respectively) versus patients without PAH-SDT. By multivariable Cox analysis, with time since PAH diagnosis as time scale, New York Heart Association/World Health Organization functional class III/IV, lower peripheral arterial oxygen saturation and pretricuspid defect were associated with a higher risk of events (P=0.002, P=0.01 and P=0.04, respectively), and one or two PAH-SDTs with a lower risk of events (P=0.009).Outcomes are poor in ES, but seem better with PAH-SDT. ES with pretricuspid defects has worse outcomes despite the delayed disease onset.