{"title":"标签外靶向药物对慢性血栓栓塞性肺动脉高压患者长期生存的影响:全国多中心前瞻性登记的启示。","authors":"Wanying Xia, Yuling Qian, Yangyi Lin, Ruilin Quan, Yuanhua Yang, Zhenwen Yang, Hongyan Tian, Shengqing Li, Jieyan Shen, Yingqun Ji, Qing Gu, Huijun Han, Changming Xiong, Jianguo He","doi":"10.1111/resp.14700","DOIUrl":null,"url":null,"abstract":"<p><strong>Background and objective: </strong>Off-label pulmonary arterial hypertension (PAH)-targeted drugs are commonly prescribed for non-operated chronic thromboembolic pulmonary hypertension (CTEPH), but their effect on the long-term prognosis of CTEPH remains unknown. This study investigated the effect of off-label PAH-targeted drugs on the long-term survival of CTEPH patients.</p><p><strong>Methods: </strong>CTEPH patients were enrolled from a prospective multicentre national registry. Except for licensed riociguat and treprostinil, other PAH-targeted drugs were off-label. In the original and propensity score-matched (PSM) samples, five-year survival was compared in two groups: (a) patients not receiving off-label PAH-targeted drugs (control) versus (b) patients receiving off-label PAH-targeted drugs (treatment). The latter group was investigated for the effect of started off-label PAH-targeted drugs at baselines (initial) or during follow-up (subsequent).</p><p><strong>Results: </strong>Of 347 enrolled patients, 212 were treated with off-label PAH-targeted drugs initially (n = 173) or subsequently (n = 39), and 135 were untreated. The 1-, 2-, 3- and 5-year survival of the treatment group was significantly higher than that of the control group (97.1% vs. 89.4%, 92.3% vs. 82.1%, 83.2% vs. 75.1% and 71.1% vs. 55.3%, respectively, log-rank test, p = 0.005). Initial treatment was correlated with better 5-year survival after excluding patients with subsequent treatment to reduce the immortal-time bias (hazard ratio: 0.611; 95% CI: 0.397-0.940; p = 0.025). In PSM samples, patients given initial treatment showed significantly better 5-year survival than untreated patients (68.9% vs. 49.3%, log-rank test, p = 0.008).</p><p><strong>Conclusion: </strong>Off-label targeted drugs contributed to improved long-term survival in CTEPH patients receiving pharmacotherapies.</p>","PeriodicalId":21129,"journal":{"name":"Respirology","volume":null,"pages":null},"PeriodicalIF":6.6000,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Effect of off-label targeted drugs on long-term survival in chronic thromboembolic pulmonary hypertension: Insights from a national multicentre prospective registry.\",\"authors\":\"Wanying Xia, Yuling Qian, Yangyi Lin, Ruilin Quan, Yuanhua Yang, Zhenwen Yang, Hongyan Tian, Shengqing Li, Jieyan Shen, Yingqun Ji, Qing Gu, Huijun Han, Changming Xiong, Jianguo He\",\"doi\":\"10.1111/resp.14700\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background and objective: </strong>Off-label pulmonary arterial hypertension (PAH)-targeted drugs are commonly prescribed for non-operated chronic thromboembolic pulmonary hypertension (CTEPH), but their effect on the long-term prognosis of CTEPH remains unknown. This study investigated the effect of off-label PAH-targeted drugs on the long-term survival of CTEPH patients.</p><p><strong>Methods: </strong>CTEPH patients were enrolled from a prospective multicentre national registry. Except for licensed riociguat and treprostinil, other PAH-targeted drugs were off-label. In the original and propensity score-matched (PSM) samples, five-year survival was compared in two groups: (a) patients not receiving off-label PAH-targeted drugs (control) versus (b) patients receiving off-label PAH-targeted drugs (treatment). The latter group was investigated for the effect of started off-label PAH-targeted drugs at baselines (initial) or during follow-up (subsequent).</p><p><strong>Results: </strong>Of 347 enrolled patients, 212 were treated with off-label PAH-targeted drugs initially (n = 173) or subsequently (n = 39), and 135 were untreated. The 1-, 2-, 3- and 5-year survival of the treatment group was significantly higher than that of the control group (97.1% vs. 89.4%, 92.3% vs. 82.1%, 83.2% vs. 75.1% and 71.1% vs. 55.3%, respectively, log-rank test, p = 0.005). Initial treatment was correlated with better 5-year survival after excluding patients with subsequent treatment to reduce the immortal-time bias (hazard ratio: 0.611; 95% CI: 0.397-0.940; p = 0.025). In PSM samples, patients given initial treatment showed significantly better 5-year survival than untreated patients (68.9% vs. 49.3%, log-rank test, p = 0.008).</p><p><strong>Conclusion: </strong>Off-label targeted drugs contributed to improved long-term survival in CTEPH patients receiving pharmacotherapies.</p>\",\"PeriodicalId\":21129,\"journal\":{\"name\":\"Respirology\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":6.6000,\"publicationDate\":\"2024-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Respirology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1111/resp.14700\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/3/17 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q1\",\"JCRName\":\"RESPIRATORY SYSTEM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Respirology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/resp.14700","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/3/17 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
引用次数: 0
摘要
背景和目的:标签外肺动脉高压(PAH)靶向药物是治疗非手术慢性血栓栓塞性肺动脉高压(CTEPH)的常用药物,但它们对CTEPH长期预后的影响仍然未知。本研究调查了标签外PAH靶向药物对CTEPH患者长期生存的影响:从一个前瞻性多中心国家登记处招募 CTEPH 患者。除了已获许可的利奥吉曲和曲普瑞替尼外,其他PAH靶向药物均为非标签药物。在原始样本和倾向评分匹配(PSM)样本中,对两组患者的五年生存率进行了比较:(a) 未接受标签外 PAH 靶向药物治疗的患者(对照组)与 (b) 接受标签外 PAH 靶向药物治疗的患者(治疗组)。对后一组患者在基线(初始)或随访(后续)期间开始使用标示外 PAH 靶向药物的效果进行了调查:结果:在347名入选患者中,212人最初(173人)或随后(39人)接受了标签外PAH靶向药物治疗,135人未接受治疗。治疗组的1年、2年、3年和5年生存率明显高于对照组(分别为97.1% vs. 89.4%、92.3% vs. 82.1%、83.2% vs. 75.1%和71.1% vs. 55.3%,log-rank检验,P = 0.005)。为减少不死时间偏差,在排除接受后续治疗的患者后,初始治疗与较好的 5 年生存率相关(危险比:0.611;95% CI:0.397-0.940;P = 0.025)。在PSM样本中,接受初始治疗的患者的5年生存率明显高于未接受治疗的患者(68.9%对49.3%,对数秩检验,p = 0.008):结论:标签外靶向药物有助于提高接受药物治疗的CTEPH患者的长期生存率。
Effect of off-label targeted drugs on long-term survival in chronic thromboembolic pulmonary hypertension: Insights from a national multicentre prospective registry.
Background and objective: Off-label pulmonary arterial hypertension (PAH)-targeted drugs are commonly prescribed for non-operated chronic thromboembolic pulmonary hypertension (CTEPH), but their effect on the long-term prognosis of CTEPH remains unknown. This study investigated the effect of off-label PAH-targeted drugs on the long-term survival of CTEPH patients.
Methods: CTEPH patients were enrolled from a prospective multicentre national registry. Except for licensed riociguat and treprostinil, other PAH-targeted drugs were off-label. In the original and propensity score-matched (PSM) samples, five-year survival was compared in two groups: (a) patients not receiving off-label PAH-targeted drugs (control) versus (b) patients receiving off-label PAH-targeted drugs (treatment). The latter group was investigated for the effect of started off-label PAH-targeted drugs at baselines (initial) or during follow-up (subsequent).
Results: Of 347 enrolled patients, 212 were treated with off-label PAH-targeted drugs initially (n = 173) or subsequently (n = 39), and 135 were untreated. The 1-, 2-, 3- and 5-year survival of the treatment group was significantly higher than that of the control group (97.1% vs. 89.4%, 92.3% vs. 82.1%, 83.2% vs. 75.1% and 71.1% vs. 55.3%, respectively, log-rank test, p = 0.005). Initial treatment was correlated with better 5-year survival after excluding patients with subsequent treatment to reduce the immortal-time bias (hazard ratio: 0.611; 95% CI: 0.397-0.940; p = 0.025). In PSM samples, patients given initial treatment showed significantly better 5-year survival than untreated patients (68.9% vs. 49.3%, log-rank test, p = 0.008).
Conclusion: Off-label targeted drugs contributed to improved long-term survival in CTEPH patients receiving pharmacotherapies.
期刊介绍:
Respirology is a journal of international standing, publishing peer-reviewed articles of scientific excellence in clinical and clinically-relevant experimental respiratory biology and disease. Fields of research include immunology, intensive and critical care, epidemiology, cell and molecular biology, pathology, pharmacology, physiology, paediatric respiratory medicine, clinical trials, interventional pulmonology and thoracic surgery.
The Journal aims to encourage the international exchange of results and publishes papers in the following categories: Original Articles, Editorials, Reviews, and Correspondences.
Respirology is the preferred journal of the Thoracic Society of Australia and New Zealand, has been adopted as the preferred English journal of the Japanese Respiratory Society and the Taiwan Society of Pulmonary and Critical Care Medicine and is an official journal of the World Association for Bronchology and Interventional Pulmonology.