Ruilin Quan1, Yuanhua Yang2, Zhenwen Yang3, Hongyan Tian4, Shengqing Li5, Jieyan Shen6, Yingqun Ji7, Gangcheng Zhang8, Caojin Zhang9, Guangyi Wang10, Yuhao Liu11, Zhaozhong Cheng12, Zaixin Yu13, Zhiyuan Song14, Zeqi Zheng15, Wei Cui16, Yucheng Chen17, Shuang Liu18, Xiaoxi Chen1, Yuling Qian1, Changming Xiong1, Guangliang Shan19, Jianguo He20. 1. Department of Pulmonary Vascular Disease, State Key Laboratory of Cardiovascular Disease, Fuwai Hospital, National Center for Cardiovascular Disease, Chinese Academy of Medical Sciences and Peking Union Medical College, No. 167, Beilishi Road, Xicheng District, Beijing, 100037, China. 2. Respiratory and Critical Care Medicine, Beijing Institute of Respiratory Medicine, Beijing Chao-Yang Hospital, Capital Medical University, Beijing, 100043, China. 3. Cardiovascular, Tianjin Medical University General Hospital, Tianjin, 300052, China. 4. Peripheral Vascular Department of First Affiliated Hospital, Medical College of Xi'an Jiaotong University, Xi'an, 710061, China. 5. Department of Respiratory Medicine, Xijing Hospital, Fourth Military Medical University, Xi'an, 710032, China. 6. Renji Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, 200240, China. 7. Department of Respiratory, First Affiliated Hospital of Dalian Medical University, Dalian, 116011, China. 8. Department of Cardiology, Wuhan Asia Heart Hospital, Wuhan, 430022, China. 9. Department of Cardiology, Guangdong Provincial People's Hospital, Guangdong Academy of Medical Sciences, Guangzhou, 510080, China. 10. Chinese PLA General Hospital, Medical School of Chinese PLA, Beijing, 100039, China. 11. Heart Center of Henan Provincial People's Hospital, Central China Fuwai Hospital, Central China Fuwai Hospital of Zhengzhou University, Zhengzhou, 450003, China. 12. Respiratory Department, The Affiliated Hospital of Qingdao University, Qingdao, 266000, China. 13. Department of Cardiology, Xiangya Hospital, Central South University, Changsha, 410008, China. 14. Department of Cardiology, Southwest Hospital, Third Military Medical University (Army Medical University), Chongqing, 400038, China. 15. Department of Cardiology, The First Affiliated Hospital of Nanchang University, Nanchang, 330006, China. 16. Department of Cardiology, Second Hospital of Hebei Medical University, Shijiazhuang, 050000, China. 17. Department of Cardiology, West China Hospital, Sichuan University, Chengdu, 610041, China. 18. Beijing Anzhen Hospital, Capital Medical University, Beijing, 100029, China. 19. Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences, Beijing, 100037, China. 20. Department of Pulmonary Vascular Disease, State Key Laboratory of Cardiovascular Disease, Fuwai Hospital, National Center for Cardiovascular Disease, Chinese Academy of Medical Sciences and Peking Union Medical College, No. 167, Beilishi Road, Xicheng District, Beijing, 100037, China. hejianguofw@163.com.
Abstract
BACKGROUND: At present, there is no generally accepted comprehensive prognostic risk prediction model for medically treated chronic thromboembolic pulmonary hypertension (CTEPH) patients. METHODS: Consecutive medically treated CTEPH patients were enrolled in a national multicenter prospective registry study from August 2009 to July 2018. A multivariable Cox proportional hazards model was utilized to derive the prognostic model, and a simplified risk score was created thereafter. Model performance was evaluated in terms of discrimination and calibration, and compared to the Swedish/COMPERA risk stratification method. Internal and external validation were conducted to validate the model performance. RESULTS: A total of 432 patients were enrolled. During a median follow-up time of 38.73 months (IQR: 20.79, 66.10), 94 patients (21.8%) died. The 1-, 3-, and 5-year survival estimates were 95.5%, 83.7%, and 70.9%, respectively. The final model included the following variables: the Swedish/COMPERA risk stratum (low-, intermediate- or high-risk stratum), pulmonary vascular resistance (PVR, ≤ or > 1600 dyn·s/cm5), total bilirubin (TBIL, ≤ or > 38 µmol/L) and chronic kidney disease (CKD, no or yes). Compared with the Swedish/COMPERA risk stratification method alone, both the derived model [C-index: 0.715; net reclassification improvement (NRI): 0.300; integrated discriminatory index (IDI): 0.095] and the risk score (C-index: 0.713; NRI: 0.300; IDI: 0.093) showed improved discriminatory power. The performance was validated in a validation cohort of 84 patients (C-index = 0.707 for the model and 0.721 for the risk score). CONCLUSIONS: A novel risk stratification strategy can serve as a useful tool for determining prognosis and guide management for medically treated CTEPH patients. TRIAL REGISTRATION: ClinicalTrials.gov (Identifier: NCT01417338).
BACKGROUND: At present, there is no generally accepted comprehensive prognostic risk prediction model for medically treated chronic thromboembolic pulmonary hypertension (CTEPH) patients. METHODS: Consecutive medically treated CTEPHpatients were enrolled in a national multicenter prospective registry study from August 2009 to July 2018. A multivariable Cox proportional hazards model was utilized to derive the prognostic model, and a simplified risk score was created thereafter. Model performance was evaluated in terms of discrimination and calibration, and compared to the Swedish/COMPERA risk stratification method. Internal and external validation were conducted to validate the model performance. RESULTS: A total of 432 patients were enrolled. During a median follow-up time of 38.73 months (IQR: 20.79, 66.10), 94 patients (21.8%) died. The 1-, 3-, and 5-year survival estimates were 95.5%, 83.7%, and 70.9%, respectively. The final model included the following variables: the Swedish/COMPERA risk stratum (low-, intermediate- or high-risk stratum), pulmonary vascular resistance (PVR, ≤ or > 1600 dyn·s/cm5), total bilirubin (TBIL, ≤ or > 38 µmol/L) and chronic kidney disease (CKD, no or yes). Compared with the Swedish/COMPERA risk stratification method alone, both the derived model [C-index: 0.715; net reclassification improvement (NRI): 0.300; integrated discriminatory index (IDI): 0.095] and the risk score (C-index: 0.713; NRI: 0.300; IDI: 0.093) showed improved discriminatory power. The performance was validated in a validation cohort of 84 patients (C-index = 0.707 for the model and 0.721 for the risk score). CONCLUSIONS: A novel risk stratification strategy can serve as a useful tool for determining prognosis and guide management for medically treated CTEPHpatients. TRIAL REGISTRATION: ClinicalTrials.gov (Identifier: NCT01417338).