RT Journal Article
SR Electronic
T1 Clinical trial design and new therapies for pulmonary arterial hypertension
JF European Respiratory Journal
JO Eur Respir J
FD European Respiratory Society
SP 1801908
DO 10.1183/13993003.01908-2018
VO 53
IS 1
A1 Olivier Sitbon
A1 Mardi Gomberg-Maitland
A1 John Granton
A1 Michael I. Lewis
A1 Stephen C. Mathai
A1 Maurizio Rainisio
A1 Norman L. Stockbridge
A1 Martin R. Wilkins
A1 Roham T. Zamanian
A1 Lewis J. Rubin
YR 2019
UL http://erj.ersjournals.com/content/53/1/1801908.abstract
AB Until 20 years ago the treatment of pulmonary arterial hypertension (PAH) was based on case reports and small series, and was largely ineffectual. As a deeper understanding of the pathogenesis and pathophysiology of PAH evolved over the subsequent two decades, coupled with epidemiological studies defining the clinical and demographic characteristics of the condition, a renewed interest in treatment development emerged through collaborations between international experts, industry and regulatory agencies. These efforts led to the performance of robust, high-quality clinical trials of novel therapies that targeted putative pathogenic pathways, leading to the approval of more than 10 novel therapies that have beneficially impacted both the quality and duration of life. However, our understanding of PAH remains incomplete and there is no cure. Accordingly, efforts are now focused on identifying novel pathogenic pathways that may be targeted, and applying more rigorous clinical trial designs to better define the efficacy of these new potential treatments and their role in the management scheme. This article, prepared by a Task Force comprised of expert clinicians, trialists and regulators, summarises the current state of the art, and provides insight into the opportunities and challenges for identifying and assessing the efficacy and safety of new treatments for this challenging condition.State of the art and research perspectives in clinical trial design and new therapies for pulmonary arterial hypertension http://ow.ly/VHQ030mfRxc