Zannad Faiez, Alikhaani Jacqueline, Alikhaani Sadegh, Butler Javed, Gordon Jason, Jensen Klaus, Khatib Rani, Mantovani Lorenzo, Martinez Robin, Moore Wanda F, Murakami Masahiro, Roessig Lothar, Stockbridge Norman, Van Spall Harriette G C, Yancy Clyde, Spertus John A
Université de Lorraine, Inserm Clinical Investigation Center at Institut Lorrain du Coeur et des Vaisseaux, University Hospital of Nancy, Nancy, France.
Los Angeles, CA, USA.
Eur J Heart Fail. 2023 Apr;25(4):478-487. doi: 10.1002/ejhf.2828. Epub 2023 Mar 22.
There are many consequences of heart failure (HF), including symptoms, impaired health-related quality of life (HRQoL), and physical and social limitations (functional status). These have a substantial impact on patients' lives, yet are not routinely captured in clinical trials. Patient-reported outcomes (PROs) can quantify patients' experiences of their disease and its treatment. Steps can be taken to improve the use of PROs in HF trials, in regulatory and payer decisions, and in patient care. Importantly, PRO measures (PROMs) must be developed with involvement of patients, family members, and caregivers from diverse demographic groups and communities. PRO data collection should become more routine not only in clinical trials but also in clinical practice. This may be facilitated by the use of digital tools and interdisciplinary patient advocacy efforts. There is a need for standardization, not only of the PROM instruments, but also in procedures for analysis, interpretation and reporting PRO data. More work needs to be done to determine the degree of change that is important to patients and that is associated with increased risks of clinical events. This 'minimal clinically important difference' requires further research to determine thresholds for different PROMs, to assess consistency across trial populations, and to define standards for improvement that warrant regulatory and reimbursement approvals. PROs are a vital part of patient care and drug development, and more work should be done to ensure that these measures are both reflective of the patient experience and that they are more widely employed.
心力衰竭（HF）有许多后果，包括症状、健康相关生活质量（HRQoL）受损以及身体和社会限制（功能状态）。这些对患者的生活有重大影响，但在临床试验中却未被常规记录。患者报告结局（PROs）可以量化患者对其疾病及其治疗的体验。可以采取措施改善PROs在心力衰竭试验、监管和支付方决策以及患者护理中的应用。重要的是，PRO测量工具（PROMs）的开发必须有来自不同人口群体和社区的患者、家庭成员及护理人员的参与。PRO数据收集不仅在临床试验中，而且在临床实践中都应变得更加常规。使用数字工具和跨学科患者宣传工作可能会促进这一点。不仅需要对PROM工具进行标准化，还需要对PRO数据分析、解释和报告的程序进行标准化。需要做更多工作来确定对患者重要且与临床事件风险增加相关的变化程度。这种“最小临床重要差异”需要进一步研究，以确定不同PROMs的阈值，评估不同试验人群的一致性，并确定值得监管和报销批准的改善标准。PROs是患者护理和药物开发的重要组成部分，应该做更多工作以确保这些测量工具既能反映患者体验，又能得到更广泛应用。
