Advanced Lung Disease and Lung Transplant Program, Inova Heart and Vascular Institute, Inova Fairfax Hospital, Falls Church, Virginia, USA
Pulmonary and Critical Care Medicine, Pulmonary Associates of Richmond Inc, Richmond, Virginia, USA.
Thorax. 2024 Mar 15;79(4):301-306. doi: 10.1136/thorax-2023-220821.
A post-hoc analysis of the INCREASE trial and its open-label extension (OLE) was performed to evaluate whether inhaled treprostinil has a long-term survival benefit in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD).
Two different models of survival were employed; the inverse probability of censoring weighting (IPCW) and the rank-preserving structural failure time (RPSFT) models both allow construction of a pseudo-placebo group, thereby allowing for long-term survival evaluation of patients with PH-ILD receiving inhaled treprostinil. Time-varying stabilised weights were calculated by fitting Cox proportional hazards models based on the baseline and time-varying prognostic factors to generate weighted Cox regression models with associated adjusted HRs.
In the INCREASE trial, there were 10 and 12 deaths in the inhaled treprostinil and placebo arms, respectively, during the 16-week randomised trial. During the OLE, all patients received inhaled treprostinil and there were 29 and 33 deaths in the prior inhaled treprostinil arm and prior placebo arm, respectively. With a conventional analysis, the HR for death was 0.71 (95% CI 0.46 to 1.10; p=0.1227). Both models demonstrated significant reductions in death associated with inhaled treprostinil treatment with HRs of 0.62 (95% CI 0.39 to 0.99; p=0.0483) and 0.26 (95% CI 0.07 to 0.98; p=0.0473) for the IPCW and RPSFT methods, respectively.
Two independent modelling techniques that have been employed in the oncology literature both suggest a long-term survival benefit associated with inhaled treprostinil treatment in patients with PH-ILD.
对 INCREASE 试验及其开放标签扩展(OLE)进行了事后分析,以评估吸入曲前列尼尔是否对特发性肺纤维化相关肺动脉高压(PH-ILD)患者具有长期生存获益。
采用了两种不同的生存模型;逆概率 censoring 加权(IPCW)和秩保持结构失效时间(RPSFT)模型均允许构建伪安慰剂组,从而可以对接受吸入曲前列尼尔治疗的 PH-ILD 患者进行长期生存评估。通过拟合基于基线和时变预后因素的 Cox 比例风险模型来计算时变稳定权重,以生成带有相关调整后的 HR 的加权 Cox 回归模型。
在 INCREASE 试验中,在 16 周随机试验中,吸入曲前列尼尔组和安慰剂组分别有 10 例和 12 例死亡。在 OLE 期间,所有患者均接受吸入曲前列尼尔治疗,先前吸入曲前列尼尔组和先前安慰剂组分别有 29 例和 33 例死亡。常规分析显示,死亡的 HR 为 0.71(95%CI 0.46 至 1.10;p=0.1227)。两种模型均显示吸入曲前列尼尔治疗与死亡显著相关,HR 分别为 0.62(95%CI 0.39 至 0.99;p=0.0483)和 0.26(95%CI 0.07 至 0.98;p=0.0473)。
两种在肿瘤学文献中使用的独立建模技术均表明,吸入曲前列尼尔治疗 PH-ILD 患者具有长期生存获益。