Department of Cardiovascular Medicine, The Second Affiliated Hospital of Nanchang University, Nanchang, Jiangxi, China.
Department of Pulmonary Vascular Disease, National Center for Cardiovascular Disease, Fuwai Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, State Key Laboratory of Cardiovascular Disease, Beijing, China.
J Heart Lung Transplant. 2023 Jul;42(7):974-984. doi: 10.1016/j.healun.2023.02.1494. Epub 2023 Feb 25.
The purpose of this registry was to provide insights into the characteristics, treatments and survival of patients with PAH-CHD in China.
Patients diagnosed with PAH-CHD were enrolled in this national multicenter prospective registry. Baseline and follow-up data on clinical characteristics, PAH-targeted treatments and survival were collected.
A total of 1060 PAH-CHD patients (mean age 31 years; 67.9% females) were included, with Eisenmenger syndrome (51.5%) being the most common form and atrial septal defects (37.3%) comprising the most frequent underlying defect. Approximately 33.0% of the patients were in World Health Organization functional class III to IV. The overall mean pulmonary arterial pressure and pulmonary vascular resistance were 67.1 (20.1) mm Hg and 1112.4 (705.9) dyn/s/cm, respectively. PAH-targeted therapy was utilized in 826 patients (77.9%), and 203 patients (19.1%) received combination therapy. The estimated 1-, 3-, 5-, and 10-year survival rates of the overall cohort were 96.9%, 92.9%, 87.6% and 73.0%, respectively. Patients received combination therapy had significantly better survival than those with monotherapy (p = 0.016). NT-proBNP >1400 pg/ml, SvO ≤ 65% and Borg dyspnea index ≥ 3 and PAH-targeted therapy were independent predictors of mortality. Hemoglobin > 160g/L was a unique predictor for mortality in Eisenmenger syndrome.
Chinese PAH-CHD patients predominantly exhibit Eisenmenger syndrome and have significantly impaired exercise tolerance and right ventricular function at diagnosis, which are closely associated with long-term survival. PAH-targeted therapy including combination therapy showed a favorable effect on survival in PAH-CHD. The long-term survival of Chinese CHD-PAH patients remains to be improved.
本研究旨在为中国先心病相关性肺动脉高压（PAH-CHD）患者的特征、治疗和生存提供见解。
本研究为全国多中心前瞻性注册研究，共纳入 1060 例 PAH-CHD 患者，收集患者的临床特征、PAH 靶向治疗及生存随访资料。
1060 例 PAH-CHD 患者中，女性占比 67.9%（67.9%），平均年龄为 31 岁，最常见的类型为艾森曼格综合征（51.5%），最常见的基础心脏畸形为房间隔缺损（37.3%）。约 33.0%的患者处于世界卫生组织（WHO）功能分级Ⅲ-Ⅳ级。平均肺动脉压和肺血管阻力分别为 67.1（20.1）mmHg 和 1112.4（705.9）dyn·s·cm-5。826 例（77.9%）患者接受了 PAH 靶向治疗，203 例（19.1%）患者接受了联合治疗。全组患者的 1、3、5 和 10 年生存率分别为 96.9%、92.9%、87.6%和 73.0%。联合治疗患者的生存明显优于单药治疗患者（p=0.016）。NT-proBNP＞1400 pg/ml、SvO2≤65%、Borg 呼吸困难指数≥3 分和 PAH 靶向治疗是死亡的独立预测因素。血红蛋白＞160 g/L 是艾森曼格综合征患者死亡的独特预测因素。
中国 PAH-CHD 患者以艾森曼格综合征为主，诊断时运动耐量和右心室功能显著受损，与长期生存密切相关。包括联合治疗在内的 PAH 靶向治疗对 PAH-CHD 患者的生存有良好的效果。中国 CHD-PAH 患者的长期生存状况仍有待改善。
