Hascoet Sebastien, Fournier Emmanuelle, Jaïs Xavier, Le Gloan Lauriane, Dauphin Claire, Houeijeh Ali, Godart Francois, Iriart Xavier, Richard Adelaïde, Radojevic Jelena, Amedro Pascal, Bosser Gilles, Souletie Nathalie, Bernard Yvette, Moceri Pamela, Bouvaist Hélène, Mauran Pierre, Barre Elise, Basquin Adeline, Karsenty Clement, Bonnet Damien, Iserin Laurence, Sitbon Olivier, Petit Jérôme, Fadel Elie, Humbert Marc, Ladouceur Magalie
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.
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; Department of congenital heart diseases, centre de compétence M3C, CHU de Bordeaux, Bordeaux, France.
Arch Cardiovasc Dis. 2017 May;110(5):303-316. doi: 10.1016/j.acvd.2017.01.006. Epub 2017 Mar 9.
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.
肺动脉高压特异性药物治疗（PAH-SDT）与艾森曼格综合征（ES）死亡率之间的关系存在争议。
研究ES患者的预后及其与PAH-SDT的关系。
回顾性、观察性、全国性、多中心队列研究。
我们纳入了340例ES患者：遗传综合征（n = 119；35.3%）；三尖瓣前缺损（n = 75；22.1%）。总体而言，276例（81.2%）患者接受了PAH-SDT：单药治疗（内皮素受体拮抗剂[ERA]或磷酸二酯酶5抑制剂[PDE5I]）46.7%；联合治疗（ERA+PDE5I）40.9%；三联治疗（ERA+PDE5I+前列环素）9.1%。PAH-SDT的中位疗程为5.5年[3.0 - 9.1年]。95例（27.9%）患者发生了事件（死亡、肺或心肺移植），中位年龄为40.5岁[29.4 - 47.6]。在40岁和60岁时，事件的累积发生率分别为16.7%[95%置信区间12.8 - 21.6%]和46.4%[95%置信区间38.2 - 55.4%]。以评估时的年龄或自PAH诊断以来的时间为时间尺度，接受一种或两种PAH-SDT的患者事件累积发生率较低（分别为P = 0.0001和P = 0.004），在三尖瓣后缺损亚组中差异最大（分别为P < 0.001和P < 0.02），与未接受PAH-SDT的患者相比。通过多变量Cox分析，以自PAH诊断以来的时间为时间尺度，纽约心脏协会/世界卫生组织功能分级III/IV、外周动脉血氧饱和度降低和三尖瓣前缺损与事件风险较高相关（分别为P = 0.002、P = 0.01和P = 0.04），而接受一种或两种PAH-SDT的患者事件风险较低（P = 0.009）。
ES患者预后较差，但PAH-SDT似乎可改善预后。尽管疾病发病延迟，但伴有三尖瓣前缺损的ES预后更差。
