University of Miami, 5665 Ponce De Leon Blvd, Coral Gables, FL 33146, USA.
Paediatr Respir Rev. 2013 Sep;14(3):146-51. doi: 10.1016/j.prrv.2013.04.003. Epub 2013 May 27.
Patient-reported outcomes (PROs) have been successfully developed for a variety of chronic respiratory diseases, such as asthma and cystic fibrosis (CF). They have recently been used to evaluate the efficacy of new medications and assess current patient functioning. Although regulatory bodies have favored PROs that measures symptoms, other domains of functioning, such as treatment burden, should be considered. This review examines current guidelines for the development and application of PROs in clinical trials, describes methods for selecting appropriate measures for paediatric populations, and presents a model incorporating PROs into clinical practice. Guidance on interpretation of these measures and graphic presentation of results are illustrated. PROs can serve as the link between the health care provider and patient to foster collaborative and personalized medicine. This model promotes greater patient responsibility, facilitates communication with providers, encourages shared decision-making, and enhances adherence.
患者报告结局(PROs)已经成功地应用于各种慢性呼吸系统疾病,如哮喘和囊性纤维化(CF)。它们最近被用于评估新药的疗效和评估当前患者的功能。尽管监管机构青睐于能够测量症状的 PROs,但其他功能领域,如治疗负担,也应该被考虑。这篇综述检查了目前用于临床试验的 PRO 开发和应用的指南,描述了为儿科人群选择适当措施的方法,并提出了将 PRO 纳入临床实践的模型。对这些措施的解释和结果的图形呈现的指导都有所说明。PROs 可以作为医疗保健提供者和患者之间的联系,以促进协作和个性化的医疗。该模型增强了患者的责任感,促进了与提供者的沟通,鼓励了共同决策,并提高了患者的依从性。