• 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, and Magalie Ladouceur.
• Department of congenital heart diseases, centre de référence malformations cardiaques congénitales complexes M3C, hôpital Marie-Lannelongue, 133, avenue de la Résistance, 92350 Le Plessis-Robinson, France; Faculté de médecine Paris-Sud, université Paris-Sud, université Paris-Saclay, Paris, France. Electronic address: s.hascoet@ccml.fr.
• Arch Cardiovasc Dis. 2017 May 1; 110 (5): 303-316.

BackgroundThe relationship between pulmonary arterial hypertension-specific drug therapy (PAH-SDT) and mortality in Eisenmenger syndrome (ES) is controversial.AimsTo investigate outcomes in patients with ES, and their relationship with PAH-SDT.MethodsRetrospective, observational, nationwide, multicentre cohort study.ResultsWe 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).ConclusionsOutcomes are poor in ES, but seem better with PAH-SDT. ES with pretricuspid defects has worse outcomes despite the delayed disease onset.Copyright © 2017 Elsevier Masson SAS. All rights reserved.

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