UNC Lineberger Comprehensive Cancer Center, Division of Hematology/Oncology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
The Cecil G. Sheps Center for Health Services Research, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
Cancer Epidemiol Biomarkers Prev. 2020 May;29(5):942-948. doi: 10.1158/1055-9965.EPI-19-1277. Epub 2020 Mar 4.
The recent expansion of treatment options in acute myeloid leukemia (AML) has necessitated a greater understanding of patient preferences for treatment benefits, about which little is known.
We sought to quantify and assess heterogeneity of the preferences of AML patients for treatment outcomes. An AML-specific discrete choice experiment (DCE) was developed involving multiple stakeholders. Attributes included in the DCE were event-free survival (EFS), complete remission (CR), time in the hospital, short-term side effects, and long-term side effects. Continuously coded conditional, stratified, and latent-class logistic regressions were used to model preferences of 294 patients with AML.
Most patients were white (89.4%) and in remission (95.0%). A 10% improvement in the chance of CR was the most meaningful offered benefit ( < 0.001). Patients were willing to trade up to 22 months of EFS or endure 8.7 months in the hospital or a two-step increase in long-term side effects to gain a 10% increase in chance of CR. Patients diagnosed at 60 years or older (21.6%) more strongly preferred to avoid short-term side effects ( = 0.03). Latent class analysis showed significant differences of preferences across gender and insurance status.
In this national sample of mostly AML survivors, patients preferred treatments that maximized chance at remission; however, significant preference heterogeneity for outcomes was identified. Age and gender may affect patients' preferences.
Survivor preferences for outcomes can inform patient-focused drug development and shared decision-making. Further studies are necessary to investigate the use of DCEs to guide treatment for individual patients.
急性髓细胞白血病 (AML) 的治疗选择最近有所扩大，这就需要更深入地了解患者对治疗益处的偏好，而目前对此知之甚少。
我们旨在量化和评估 AML 患者对治疗结果的偏好的异质性。我们开发了一项 AML 特定的离散选择实验 (DCE)，涉及多个利益相关者。DCE 中包含的属性包括无事件生存 (EFS)、完全缓解 (CR)、住院时间、短期副作用和长期副作用。连续编码的条件、分层和潜在类别逻辑回归用于对 294 名 AML 患者的偏好进行建模。
大多数患者为白人 (89.4%) 且处于缓解期 (95.0%)。CR 几率提高 10% 是最有意义的获益 ( < 0.001)。患者愿意用 22 个月的 EFS 或 8.7 个月的住院时间或长期副作用增加两步来换取 CR 几率提高 10%。60 岁或以上诊断的患者 (21.6%) 更强烈地希望避免短期副作用 ( = 0.03)。潜在类别分析显示，性别和保险状况对偏好存在显著差异。
在这个以 AML 幸存者为主的全国性样本中，患者更倾向于选择能最大限度提高缓解几率的治疗方案；然而，对结果的偏好存在显著异质性。年龄和性别可能会影响患者的偏好。
幸存者对结果的偏好可以为以患者为中心的药物开发和共同决策提供信息。需要进一步研究使用 DCE 来指导个别患者的治疗。
