10. Preliminary study of composite clinical worsening definitions in chinese people with pulmonary arterial hypertension associated with congenital heart disease – A single center experience

Zhuoyuan Xu, Qiangqiang Li, Chen Zhang, Hongsheng Zhang, Hong Gu
2021 International Journal of Cardiology Congenital Heart Disease  
Composite clinical worsening (cCW) outcomes might allow measurement of disease progression in patients with pulmonary arterial hypertension (PAH). The use of composite CW outcomes is increasingly encouraged in PAH clinical research. This study investigated the relevant factors affecting the prognosis and their predictive strength for lung transplantation/death. Methods: Patients with CHD-PAH referred to Beijing Anzhen Hospital between Jan 2007 and Jul 2018 were included. Patients from at least
more » ... 7 regions and hundreds of medical centers in China were referred to our center. CW was defined as PAH-related hospitalization, NYHA cardiac function deterioration (cCW1, 2 and 3), syncope (cCW2 and 3), and the occurrence/worsening of 2 PAH symptoms at diagnosis and during follow-up (cCW3). The primary endpoint events were defined as all-cause death and lung transplantation. Results: 525 consecutive patients (children and adults, with or without Down syndrome) with CHD-PAH were included. All patients met the clinical definition of PAH with mean pulmonary arterial pressure (mPAP)25 mmHg and a pulmonary artery wedge pressure 15 mmHg measured by right heart catheterization (RHC) and/or invasive PAP monitoring. The median age at diagnosis was 20.7 (11.2, 30.3) years. Children (<18 years) accounted for 43.8%, women accounted for 68.8%. 249 patients with Eisenmenger syndrome, 43 patients with PAH associated with prevalent systemic-to-pulmonary shunts, 48 with PAH with small/coincidental defect, and 185 with PAH after defect correction were included. The median follow-up time was 4.5 years. 47 patients (9.0%) died, and heart failure was the most common cause of death (27.7%). Survival rates of endpoint-free events at 1, 3, 5 and 10 years after diagnosis were 98.0%, 95.4%, 89.9%, and 84.4%, respectively (Log-rank p¼0.016). Cox multivariate analysis showed that NYHA cardiac function deterioration, occurrence/worsening of 2 PAH symptoms, PAH-related hospitalization and syncope has a high predictive value for lung transplantation or death. Composite clinical worsening was associated with significantly increased risk of lung transplantation or death (p<0.0001), with cCW2 having the highest predictive value (HR:15.476, 95%CI: 4.346-37.576, p<0.0001). Conclusion: These data support the use of cCW outcomes in CHD-PAH research. Composite clinical worsening events are initially considered as clinical endpoints in clinical trials. NYHA cardiac function deterioration, occurrence/worsening of 2 PAH symptoms, PAH-related hospitalization and syncope may be important
doi:10.1016/j.ijcchd.2020.100038 fatcat:hxloiju5t5dknp3y5o6efqnuru