Department of Medicine, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Am J Respir Crit Care Med. 2012 May 1;185(9):1015-20. doi: 10.1164/rccm.201201-0049WS. Epub 2012 Feb 14.
Recognizing the importance of improving lung health through lung disease research, the National Heart, Lung, and Blood Institute (NHLBI) convened a workshop of multidisciplinary experts for the following purpose: (1) to review the current scientific knowledge underlying the basis for treatment of adults and children with pulmonary vascular diseases (PVDs); (2) to identify gaps, barriers, and emerging scientific opportunities in translational PVD research and the means to capitalize on these opportunities; (3) to prioritize new research directions that would be expected to affect the clinical course of PVDs; and (4) to make recommendations to the NHLBI on how to fill identified gaps in adult and pediatric PVD clinical research. Workshop participants reviewed experiences from previous PVD clinical trials and ongoing clinical research networks with other lung disorders, including acute respiratory distress syndrome, chronic obstructive lung disease, and idiopathic pulmonary fibrosis, as well. Bioinformatics experts discussed strategies for applying cutting-edge health information technology to clinical studies. Participants in the workshop considered approaches in the following broad concept areas: (1) improved phenotyping to identify potential subjects for appropriate PVD clinical studies; (2) identification of potential new end points for assessing key outcomes and developing better-designed PVD clinical trials; and (3) the establishment of priorities for specific clinical research needed to advance care of patients with various subsets of PVDs from childhood through adulthood. This report provides a summary of the objectives and recommendations to the NHLBI concentrating on clinical research efforts that are needed to better diagnose and treat PVDs.
认识到通过肺病研究改善肺部健康的重要性,国家心肺血液研究所(NHLBI)召集了多学科专家研讨会,目的如下:(1)审查治疗成人和儿童肺血管疾病(PVD)的基础的当前科学知识;(2)确定转化 PVD 研究中的差距、障碍和新兴科学机会,以及利用这些机会的方法;(3)确定预计会影响 PVD 临床进程的新研究方向,并向 NHLBI 提出建议,如何填补成人和儿科 PVD 临床研究中的现有空白;(4)审查来自先前 PVD 临床试验和正在进行的其他肺部疾病临床研究网络的经验,包括急性呼吸窘迫综合征、慢性阻塞性肺疾病和特发性肺纤维化。生物信息学专家还讨论了将最先进的健康信息技术应用于临床研究的策略。研讨会参与者考虑了以下广泛概念领域的方法:(1)改进表型分析,以确定适合进行 PVD 临床研究的潜在受试者;(2)确定评估关键结果和设计更好的 PVD 临床试验的潜在新终点;(3)确定推进各年龄段 PVD 患者护理所需的特定临床研究的优先事项。本报告提供了对 NHLBI 的目标和建议的总结,重点是需要更好地诊断和治疗 PVD 的临床研究工作。