Effect of off-label targeted drugs on long-term survival in chronic thromboembolic pulmonary hypertension: Insights from a national multicentre prospective registry.
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引用次数: 0
Abstract
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.
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