Health Economics and Health Care Management, Hochschule Neubrandenburg, Neubrandenburg, Germany; Gesellschaft für empirische Beratung GmbH, Berlin, Germany.
Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, Allschwil, Switzerland.
Value Health. 2024 Feb;27(2):206-215. doi: 10.1016/j.jval.2023.10.012. Epub 2023 Nov 9.
Pulmonary arterial hypertension (PAH) is a chronic, progressive disease of the pulmonary circulation characterized by vascular remodeling that, if untreated, can lead to right heart dysfunction and death. This analysis measured heterogeneity in patient preferences for PAH-specific treatment regimens.
Adult patients with PAH with slight to marked limitations during physical activity were recruited through a patient organization in Germany. Participants completed an online best-worst scaling case 3 survey. Patients chose among 3 hypothetical treatment profiles defined by 6 benefits and risks at varying levels. Participants completed 12 choice tasks. Preference heterogeneity was assessed using latent class analysis.
A total of 83 participants (76% female) completed the survey. Best-fit model revealed 4 classes. Class 1 (19% of participants) assigned importance to multiple attributes particularly side effects, class 2 (34%) to physical activity limitations, class 3 (30%) to survival and physical activity limitations, and class 4 (17%) to survival. No differences in sociodemographic characteristics were observed across classes. Compared with other classes, class 4 was most likely to report having marked physical activity limitations (79%) and needing daily help (100%), while considering higher daily activity levels to be ordinary (walking >1 km [71%] or climbing several flights of stairs [50%]).
This first patient preference study in a PAH population suggests that physical activity limitations in addition to survival matter most to patients; however, preference heterogeneity between groups of patients was observed. Patient preferences should be considered in treatment decision making to better balance patient's expectations regarding the known risk-benefit ratio of treatment.
肺动脉高压(PAH)是一种慢性进行性肺循环疾病,其特征为血管重构,如果不治疗,可导致右心功能障碍和死亡。本分析衡量了患者对 PAH 特定治疗方案的偏好差异。
通过德国的一个患者组织招募了在体力活动期间有轻微至明显活动受限的成年 PAH 患者。参与者完成了一项在线最佳最差分级病例 3 调查。患者在 3 种假设的治疗方案中进行选择,这些方案由不同水平的 6 种获益和风险定义。参与者完成了 12 项选择任务。使用潜在类别分析评估偏好异质性。
共有 83 名参与者(76%为女性)完成了调查。最佳拟合模型显示有 4 个类别。第 1 类(19%的参与者)特别重视多种属性,如副作用;第 2 类(34%)重视体力活动受限;第 3 类(30%)重视生存和体力活动受限;第 4 类(17%)重视生存。各类别之间在社会人口学特征方面没有差异。与其他类别相比,第 4 类最有可能报告有明显的体力活动受限(79%)和需要日常帮助(100%),同时认为更高的日常活动水平是正常的(步行>1 公里[71%]或爬几段楼梯[50%])。
这是 PAH 人群中的第一项患者偏好研究,表明除了生存外,体力活动受限对患者最重要;然而,观察到了患者群体之间的偏好异质性。在治疗决策中应考虑患者的偏好,以更好地平衡患者对治疗已知风险-获益比的预期。