Université Paris-Saclay, School of Medicine, Le Kremlin-Bicêtre, France.
Dept of Respiratory and Intensive Care Medicine, AP-HP, Hôpital Bicêtre, Le Kremlin-Bicêtre, France.
Eur Respir J. 2022 Jun 30;59(6). doi: 10.1183/13993003.02419-2021. Print 2022 Jun.
Contemporary risk assessment tools categorise patients with pulmonary arterial hypertension (PAH) as low, intermediate or high risk. A minority of patients achieve low risk status with most remaining intermediate risk. Our aim was to validate a four-stratum risk assessment approach categorising patients as low, intermediate-low, intermediate-high or high risk, as proposed by the Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA) investigators.
We evaluated incident patients from the French PAH Registry and applied a four-stratum risk method at baseline and at first reassessment. We applied refined cut-points for three variables: World Health Organization functional class, 6-min walk distance and N-terminal pro-brain natriuretic peptide. We used Kaplan-Meier survival analyses and Cox proportional hazards regression to assess survival according to three-stratum and four-stratum risk approaches.
At baseline (n=2879), the four-stratum approach identified four distinct risk groups and performed slightly better than a three-stratum method for predicting mortality. Four-stratum model discrimination was significantly higher than the three-stratum method when applied during follow-up and refined risk categories among subgroups with idiopathic PAH, connective tissue disease-associated PAH, congenital heart disease and portopulmonary hypertension. Using the four-stratum approach, 53% of patients changed risk category from baseline compared to 39% of patients when applying the three-stratum approach. Those who achieved or maintained a low risk status had the best survival, whereas there were more nuanced differences in survival for patients who were intermediate-low and intermediate-high risk.
The four-stratum risk assessment method refined risk prediction, especially within the intermediate risk category of patients, performed better at predicting survival and was more sensitive to change than the three-stratum approach.
当代风险评估工具将肺动脉高压(PAH)患者分为低危、中危或高危。少数患者达到低危状态,大多数仍为中危。我们的目的是验证 COMPERA 研究者提出的四层次风险评估方法,将患者分为低危、中低危、中高危或高危。
我们评估了法国 PAH 登记处的新发病例,并在基线和首次重新评估时应用了四层次风险方法。我们对三个变量应用了更精细的截断值:WHO 功能分级、6 分钟步行距离和 N 末端脑钠肽前体。我们使用 Kaplan-Meier 生存分析和 Cox 比例风险回归来评估根据三层次和四层次风险方法的生存情况。
在基线时(n=2879),四层次方法确定了四个不同的风险组,在预测死亡率方面略优于三层次方法。四层次模型的判别能力明显高于三层次方法,当应用于特发性 PAH、结缔组织病相关 PAH、先天性心脏病和门脉高压性肺高压亚组的随访和细化风险分类时。使用四层次方法,与三层次方法相比,有 53%的患者在基线时改变了风险类别,而 39%的患者改变了风险类别。那些达到或维持低危状态的患者具有最佳的生存,而对于中低危和中高危患者的生存则存在更多细微的差异。
四层次风险评估方法细化了风险预测,特别是在患者的中危风险类别中,在预测生存方面表现更好,并且比三层次方法更敏感于变化。