New trial designs and potential therapies for pulmonary artery hypertension.
New trial designs and potential therapies for pulmonary artery hypertension.
复制标题
DOI：
10.1016/j.jacc.2013.10.026
复制
发表时间：
2013-12-24
影响因子：
24
通讯作者：
Rubin, Lewis J.
中科院分区：
文献类型：
--
作者：
Gomberg-Maitland, Mardi;Bull, Todd M.;Saggar, Rajeev;Barst, Robyn J.;Elgazayerly, Amany;Fleming, Thomas R.;Grimminger, Friedrich;Rainisio, Maurizio;Stewart, Duncan J.;Stockbridge, Norman;Ventura, Carlo;Ghofrani, Ardeschir H.;Rubin, Lewis J.
对肺动脉高压（PAH）的流行病学、发病机制和病理生理学的深入了解已经取得了重大进展，但这种疾病仍然是致命的。治疗方案既不是普遍可用的，也不是总是有效的，这强调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有效性的证据，但在评估治疗效果的范围和持续时间方面受到限制。除了熟悉疗法的新用途外，在药物开发的不同阶段存在许多潜在的靶点。对基因和细胞治疗的追求仍在继续，用于帮助急性恶化和慢性治疗的设备正在出现。这种药物开发的快速激增导致了多中心关键临床试验，并导致了新的伦理和全球临床试验问题。本文将概述多环芳烃治疗新方法的发展所面临的机遇和挑战。
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.
登录
查看更多内容
影响因子：
4.2
作者：
Conrad, SA;Rycus, PT;Dalton, H
通讯作者：
Dalton, H
DOI：
10.1177/1740774511410994
发表时间：
2011-08
期刊：
Clinical trials (London, England)
影响因子：
--
作者：
Fleming TR;Odem-Davis K;Rothmann MD;Li Shen Y
通讯作者：
Li Shen Y
DOI：
10.1152/ajplung.1999.277.3.l472
发表时间：
1999-09-01
影响因子：
4.9
作者：
Fagan, KA;Tyler, RC;Rodman, DR
通讯作者：
Rodman, DR
影响因子：
24
作者：
Brandt, Mathias C.;Reda, Sara;Hoppe, Uta C.
通讯作者：
Hoppe, Uta C.
影响因子：
37.8
作者：
Cooper, CJ;Landzberg, MJ;Selwyn, AP
通讯作者：
Selwyn, AP