Am J Respir Crit Care Med. 2014 Feb 1;189(3):345-55. doi: 10.1164/rccm.201311-1954ST.
Current classification of pulmonary hypertension (PH) is based on a relatively simple combination of patient characteristics and hemodynamics. This limits customization of treatment, and lacks the clarity of a more granular identification based on individual patient phenotypes. Rapid advances in mechanistic understanding of the disease, improved imaging methods, and innovative biomarkers now provide an opportunity to define PH phenotypes on the basis of biomarkers, advanced imaging, and pathobiology. This document organizes our current understanding of PH phenotypes and identifies gaps in our knowledge.
A multidisciplinary committee with expertise in clinical care (pulmonary, cardiology, pediatrics, and pathology), clinical research, and/or basic science in the areas of PH identified important questions and reviewed and synthesized the literature.
This document describes selected PH phenotypes and serves as an initial platform to define additional relevant phenotypes as new knowledge is generated. The biggest gaps in our knowledge stem from the fact that our present understanding of PH phenotypes has not come from any particularly organized effort to identify such phenotypes, but rather from reinterpreting studies and reports that were designed and performed for other purposes.
Accurate phenotyping of PH can be used in research studies to increase the homogeneity of study cohorts. Once the ability of the phenotypes to predict outcomes has been validated, phenotyping may also be useful for determining prognosis and guiding treatment. This important next step in PH patient care can optimally be addressed through a consortium of study sites with well-defined goals, tasks, and structure. Planning and support for this could include the National Institutes of Health and the U.S. Food and Drug Administration, with industry and foundation partnerships.
目前的肺动脉高压(PH)分类是基于患者特征和血液动力学的相对简单的组合。这限制了治疗的定制化,并且缺乏基于个体患者表型的更精细识别的清晰度。对疾病机制理解的快速进步、改进的成像方法和创新的生物标志物现在为基于生物标志物、先进的成像和病理生物学来定义 PH 表型提供了机会。本文档组织了我们对 PH 表型的现有理解,并确定了我们知识中的空白。
一个在 PH 领域具有临床护理(肺、心脏病学、儿科和病理学)、临床研究和/或基础科学专业知识的多学科委员会确定了重要问题,并审查和综合了文献。
本文档描述了选定的 PH 表型,并作为定义其他相关表型的初始平台,随着新知识的产生。我们知识中的最大空白源于这样一个事实,即我们目前对 PH 表型的理解并非来自任何特别有组织的识别此类表型的努力,而是来自重新解释为其他目的而设计和执行的研究和报告。
PH 的准确表型分析可用于研究研究,以提高研究队列的同质性。一旦表型预测结果的能力得到验证,表型分析也可能有助于确定预后和指导治疗。PH 患者护理的这一重要下一步可以通过具有明确目标、任务和结构的研究站点联盟来最佳解决。美国国立卫生研究院和美国食品和药物管理局可以为规划和支持提供资金,包括工业和基金会的合作。