Section of Cardiology, Department of Medicine, University of Chicago, Chicago, Illinois.
Section of Pulmonary and Critical Care, Department of Medicine, University of Colorado, Aurora, Colorado.
J Am Coll Cardiol. 2013 Dec 24;62(25 Suppl):D82-91. doi: 10.1016/j.jacc.2013.10.026.
A greater understanding of the epidemiology, pathogenesis, and pathophysiology of pulmonary artery hypertension (PAH) has led to significant advances, but the disease remains fatal. Treatment options are neither universally available nor always effective, underscoring the need for development of novel therapies and therapeutic strategies. Clinical trials to date have provided evidence of efficacy, but were limited in evaluating the scope and duration of treatment effects. Numerous potential targets in varied stages of drug development exist, in addition to novel uses of familiar therapies. The pursuit of gene and cell-based therapy continues, and device use to help acute deterioration and chronic management is emerging. This rapid surge of drug development has led to multicenter pivotal clinical trials and has resulted in novel ethical and global clinical trial concerns. This paper will provide an overview of the opportunities and challenges that await the development of novel treatments for PAH.
对肺动脉高压（PAH）的流行病学、发病机制和病理生理学有了更深入的了解，这使得该领域取得了重大进展，但该病仍然致命。治疗选择不仅不是普遍可用的，而且并不总是有效的，这突显了开发新疗法和治疗策略的必要性。迄今为止的临床试验提供了疗效的证据，但在评估治疗效果的范围和持续时间方面存在局限性。除了对熟悉疗法的新用途外，在药物开发的不同阶段还存在许多潜在的靶点。对基因和细胞治疗的探索仍在继续，并且使用设备来帮助急性恶化和慢性管理也正在出现。这种药物开发的快速增长导致了多中心关键临床试验的出现，并产生了新的伦理和全球临床试验关注点。本文将概述 PAH 新型治疗方法的发展所面临的机遇和挑战。
