Moor Catharina C, Heukels Peter, Kool Mirjam, Wijsenbeek Marlies S
Department of Respiratory Medicine, Erasmus Medical Center, University Hospital Rotterdam, Rotterdam, Netherlands.
Front Med (Lausanne). 2017 Dec 20;4:226. doi: 10.3389/fmed.2017.00226. eCollection 2017.
Idiopathic pulmonary fibrosis (IPF) is a progressive and ultimately fatal disease which has a major impact on patients' quality of life (QOL). Except for lung transplantation, there is no curative treatment option. Fortunately, two disease-modifying drugs that slow down disease decline were recently approved. Though this is a major step forward, these drugs do not halt or reverse the disease, nor convincingly improve health-related QOL. In daily practice, disease behavior and response to therapy greatly vary among patients. It is assumed that this is related to the multiple biological pathways and complex interactions between genetic, molecular, and environmental factors that are involved in the pathogenesis of IPF. Recently, research in IPF has therefore started to focus on developing targeted therapy through identifying genetic risk factors and biomarkers. In this rapidly evolving field of personalized medicine, patient factors such as lifestyle, comorbidities, preferences, and experiences with medication should not be overlooked. This review describes recent insights and methods on how to integrate patient perspectives into personalized medicine. Furthermore, it provides an overview of the most used patient-reported outcome measures in IPF, to facilitate choices for both researchers and clinicians when incorporating the patient voice in their research and care. To enhance truly personalized treatment in IPF, biology should be combined with patient perspectives.
特发性肺纤维化（IPF）是一种进行性且最终致命的疾病，对患者的生活质量（QOL）有重大影响。除了肺移植外，没有治愈性的治疗选择。幸运的是，最近有两种减缓疾病进展的疾病修饰药物获批。尽管这是向前迈出的重要一步，但这些药物并不能阻止或逆转疾病，也不能令人信服地改善与健康相关的生活质量。在日常实践中，患者之间的疾病行为和对治疗的反应差异很大。据推测，这与IPF发病机制中涉及的多种生物学途径以及遗传、分子和环境因素之间的复杂相互作用有关。因此，最近IPF的研究开始聚焦于通过识别遗传风险因素和生物标志物来开发靶向治疗。在这个快速发展的个性化医学领域，生活方式、合并症、偏好以及用药体验等患者因素不应被忽视。这篇综述描述了关于如何将患者观点纳入个性化医学的最新见解和方法。此外，它概述了IPF中最常用的患者报告结局指标，以便在研究人员和临床医生将患者声音纳入其研究和护理时为他们提供选择便利。为了在IPF中加强真正的个性化治疗，生物学应与患者观点相结合。
