Department of Pulmonary Vascular Disease, National Center for Cardiovascular Disease, Chinese Academy of Medical Sciences and Peking Union Medical College, Fuwai Hospital, Beijing, China.
Department of Epidemiology and Biostatistics, Institute of Basic Medical Sciences Chinese Academy of Medical Sciences, School of Basic Medicine Peking Union Medical College, Beijing, China.
Chest. 2023 Jun;163(6):1531-1542. doi: 10.1016/j.chest.2022.11.038. Epub 2022 Dec 5.
Registry-based studies of pediatric pulmonary arterial hypertension (PPAH) are scarce in developing countries, including China. The PPAH risk assessment tool needs further evaluation and improvement.
What are the characteristics and long-term survival of PPAH in China and what is the performance of the PPAH risk model in Chinese patients?
Patients with PAH were enrolled in the national prospective multicenter registry from August 2009 through December 2019. Children 3 months to 18 years of age at the time of PAH diagnosis were analyzed.
A total of 247 children with PAH were enrolled. The median patient age was 14.8 years, and 58.3% of patients were female. Most patients had a diagnosis of PAH associated with congenital heart disease (CHD; 61.5%) and idiopathic or heritable PAH (37.7%). The median time from symptom onset to PAH diagnosis was 24 months. The mean pulmonary artery pressure and pulmonary vascular resistance index were 70.78 ± 19.80 mm Hg and 21.82 ± 11.18 Wood Units·m, respectively. Patients with CHD-associated PAH experienced a longer diagnostic delay and demonstrated higher pulmonary artery pressure, but better cardiac performance, than those with idiopathic or heritable PAH. An increased number of patients received targeted therapy at the last follow-up compared with baseline. The 5- and 10-year survival rates of the entire cohort were 74.9% and 55.7%, respectively, with better survival in patients with CHD-associated PAH than in those with idiopathic or heritable PAH. Patients with low risk had better survival than those with high risk according to the simplified noninvasive risk score model with weight, function class, and echocardiographic right ventricular size, both at baseline and follow-up.
Patients with PPAH in China showed severely compromised hemodynamics with marked diagnostic delay. The long-term survival of PPAH is poor despite the increased usefulness of targeted drugs. The simplified noninvasive risk model demonstrated good performance for predicting survival in Chinese children with PAH.
ClinicalTrials.gov; No.: NCT01417338; URL: www.
gov.
儿科肺动脉高压（PPAH）的基于登记的研究在包括中国在内的发展中国家较为少见。PPAH 风险评估工具需要进一步评估和改进。
中国 PPAH 的特征和长期生存率如何，以及 PPAH 风险模型在中国人患者中的表现如何？
2009 年 8 月至 2019 年 12 月，全国前瞻性多中心登记处纳入肺动脉高压患者。分析诊断时年龄为 3 个月至 18 岁的儿童患者。
共纳入 247 例儿童肺动脉高压患者。中位患者年龄为 14.8 岁，58.3%为女性。大多数患者为先天性心脏病（CHD）相关肺动脉高压（61.5%）和特发性或遗传性 PAH（37.7%）。从症状出现到肺动脉高压诊断的中位时间为 24 个月。平均肺动脉压和肺血管阻力指数分别为 70.78±19.80mmHg 和 21.82±11.18Wood 单位·m。CHD 相关 PAH 患者的诊断延迟时间更长，肺动脉压更高，但心功能优于特发性或遗传性 PAH 患者。与基线相比，最后一次随访时接受靶向治疗的患者数量增加。整个队列的 5 年和 10 年生存率分别为 74.9%和 55.7%，CHD 相关 PAH 患者的生存率优于特发性或遗传性 PAH 患者。根据简化的无创风险评分模型（包括体重、功能分级和超声心动图右心室大小），基线和随访时低危患者的生存率均优于高危患者。
中国 PPAH 患者的血流动力学严重受损，诊断延迟明显。尽管靶向药物的使用更加广泛，但 PPAH 的长期生存率仍然较差。简化的无创风险模型在预测中国儿童 PAH 患者的生存率方面表现良好。
ClinicalTrials.gov；编号：NCT01417338；网址：www.clinicaltrials.gov。
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