Department of Medicine, Section of Rheumatology, Yale University School of Medicine, New Haven, Connecticut, United States of America.
Department of Medicine, Section of Rheumatology, Berkshire Health Systems, Pittsfield, Massachusetts, United States of America.
PLoS One. 2021 Jan 29;16(1):e0245598. doi: 10.1371/journal.pone.0245598. eCollection 2021.
A measure that encompasses both benefits and harms at the individual patient level may facilitate comparisons between treatment options and improve shared decision-making. The objective of this study was to develop a patient reported measure to capture overall experience (including both benefits and harms) of treatment using rheumatoid arthritis (RA) as a case example.
Hierarchies for treatment benefits are known. Therefore, we developed a hierarchy of adverse events (AEs) using a series of trajectory mapping and paired comparison surveys. We subsequently used these data to construct a paired comparison survey, asking patients to compare options including both a specified level of benefit and an AE. These data were used to generate a hierarchy of overall experience on treatment.
782 participants completed a series of three surveys. The trajectory mapping procedure and a paired comparison survey led to the generation of a hierarchy of AEs with nine levels ranging from No AEs to irreversible serious complications. In a third survey, in which AEs were paired with benefits, participants' ratings generated a 6-level hierarchy of overall experiences ranging from Major improvement + No, mild or manageable AEs (Level 1) to No improvement + Irreversible AEs (Level 6).
Using a trajectory mapping approach, we developed a patient reported measure representing the distribution of patients' overall experiences on treatment. The intent of this measure is to enable patients and their physicians to compare the percentage of patients experiencing each level of outcome, from most to least desirable, across treatments.
在个体患者层面上涵盖获益和危害的衡量指标可能有助于比较治疗选择，并改善共同决策。本研究的目的是开发一种患者报告的衡量指标，以捕捉使用类风湿关节炎（RA）作为案例的治疗的总体体验（包括获益和危害）。
已知治疗获益的层次结构。因此，我们使用一系列轨迹映射和配对比较调查来构建不良事件（AE）的层次结构。随后，我们使用这些数据构建了配对比较调查，要求患者比较包括特定获益水平和 AE 的选项。这些数据用于生成治疗总体体验的层次结构。
782 名参与者完成了一系列三项调查。轨迹映射程序和配对比较调查导致产生了一个 9 级的 AE 层次结构，范围从无 AE 到不可逆转的严重并发症。在第三次调查中，将 AE 与获益配对，参与者的评分生成了一个 6 级的总体体验层次结构，从获益大且无、轻度或可管理的 AE（第 1 级）到获益小或无且不可逆转的 AE（第 6 级）。
使用轨迹映射方法，我们开发了一种患者报告的衡量指标，代表了患者治疗总体体验的分布。该衡量指标的目的是使患者及其医生能够比较不同治疗方法中每个体验水平的患者比例，从最理想到最不理想。
