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.
Respiratory and Critical Care Medicine, Beijing Institute of Respiratory Medicine, Beijing Chao-Yang Hospital, Capital Medical University, Beijing, 100043, China.
BMC Pulm Med. 2021 Apr 20;21(1):128. doi: 10.1186/s12890-021-01495-6.
At present, there is no generally accepted comprehensive prognostic risk prediction model for medically treated chronic thromboembolic pulmonary hypertension (CTEPH) patients.
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.
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/cm), 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).
A novel risk stratification strategy can serve as a useful tool for determining prognosis and guide management for medically treated CTEPH patients.
ClinicalTrials.gov (Identifier: NCT01417338).
目前，对于接受药物治疗的慢性血栓栓塞性肺动脉高压（CTEPH）患者，尚无普遍接受的综合预后风险预测模型。
连续纳入 2009 年 8 月至 2018 年 7 月期间参加全国多中心前瞻性登记研究的接受药物治疗的 CTEPH 患者。利用多变量 Cox 比例风险模型推导预后模型，随后创建简化风险评分。通过区分度和校准度评估模型性能，并与瑞典/COMPERA 风险分层方法进行比较。进行内部和外部验证以验证模型性能。
共纳入 432 例患者。中位随访时间为 38.73 个月（IQR：20.79，66.10），94 例患者（21.8%）死亡。1、3 和 5 年生存率分别为 95.5%、83.7%和 70.9%。最终模型纳入以下变量：瑞典/COMPERA 风险分层（低危、中危或高危分层）、肺血管阻力（PVR，≤或>1600 dyn·s/cm）、总胆红素（TBIL，≤或>38 μmol/L）和慢性肾脏病（CKD，无或有）。与瑞典/COMPERA 风险分层方法相比，推导的模型[C 指数：0.715；净重新分类改善（NRI）：0.300；综合判别指数（IDI）：0.095]和风险评分（C 指数：0.713；NRI：0.300；IDI：0.093）均显示出更好的区分度。在 84 例患者的验证队列中验证了该模型的性能（模型的 C 指数为 0.707，风险评分的 C 指数为 0.721）。
一种新的风险分层策略可作为确定接受药物治疗的 CTEPH 患者预后和指导管理的有用工具。
ClinicalTrials.gov（标识符：NCT01417338）。
