Univ. Paris-Sud, Faculté de Médecine, Le Kremlin-Bicêtre APHP, Centre de Référence de l'Hypertension Pulmonaire Sévère, Service de Pneumologie et Soins Intensifs, DHU Thorax Innovation, Hôpital de Bicêtre, Le Kremlin-Bicêtre INSERM U999, LabEx LERMIT, Centre Chirurgical Marie-Lannelongue, Le Plessis-Robinson
Univ. Paris-Sud, Faculté de Médecine, Le Kremlin-Bicêtre APHP, Centre de Référence de l'Hypertension Pulmonaire Sévère, Service de Pneumologie et Soins Intensifs, DHU Thorax Innovation, Hôpital de Bicêtre, Le Kremlin-Bicêtre INSERM U999, LabEx LERMIT, Centre Chirurgical Marie-Lannelongue, Le Plessis-Robinson.
Eur Respir J. 2014 Jun;43(6):1691-7. doi: 10.1183/09031936.00116313. Epub 2014 Mar 13.
Patients with severe pulmonary arterial hypertension (PAH) in New York Heart Association (NYHA) functional class (FC) III/IV have a poor prognosis, despite survival benefits being demonstrated with intravenous epoprostenol. In this pilot study, the efficacy and safety of a triple combination therapy regimen in patients with severe PAH was investigated. Data from newly diagnosed NYHA FC III/IV PAH patients (n=19) initiated on upfront triple combination therapy (intravenous epoprostenol, bosentan and sildenafil) were collected retrospectively from a prospective registry. Significant improvements in 6-min walk distance and haemodynamics were observed after 4 months' triple combination therapy in 18 patients (p<0.01); 17 patients had improved to NYHA FC I or II. One patient was not included in the month 4 assessment (due to an emergency lung transplant in month 3). At the final evaluation (mean ± sd 32 ± 19 months), all 18 patients had sustained clinical and haemodynamic improvement. Overall survival estimates for the triple combination cohort were 100% at 1, 2 and 3 years. Expected survival calculated from the French equation was 75% (95% CI 68-82%), 60% (95% CI 50-70%) and 49% (95% CI 38-60%) at 1, 2 and 3 years, respectively. This pilot study provides preliminary evidence of the long-term benefits of upfront triple combination therapy in patients with severe PAH.
患有纽约心脏协会（NYHA）功能分级（FC）III/IV 级严重肺动脉高压（PAH）的患者预后较差，尽管静脉注射依前列醇已证明可提高生存率。在这项初步研究中，研究了严重 PAH 患者三联疗法的疗效和安全性。回顾性收集了 19 例新诊断为 NYHA FC III/IV PAH 患者（n=19）直接开始三联疗法（静脉注射依前列醇、波生坦和西地那非）的数据，这些患者来自前瞻性登记。18 例患者在开始三联疗法后 4 个月时，6 分钟步行距离和血液动力学显著改善（p<0.01）；17 例患者改善到 NYHA FC I 或 II 级。1 例患者未纳入第 4 个月的评估（由于第 3 个月紧急进行了肺移植）。在最终评估时（平均±标准差 32±19 个月），18 例患者的临床和血液动力学均持续改善。三联治疗组的总生存率估计在 1、2 和 3 年时分别为 100%。根据法国方程计算的预期生存率分别为 75%（95%CI 68-82%）、60%（95%CI 50-70%）和 49%（95%CI 38-60%）。这项初步研究为严重 PAH 患者直接三联疗法的长期获益提供了初步证据。
