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一线慢性淋巴细胞白血病治疗的偏好:一项关于患者和医生观点的多国研究结果

Preferences for First-Line Chronic Lymphocytic Leukemia Treatments: Results From a Multinational Study on the Perspectives of Patients and Physicians.

作者信息

Sile Bersabeh, Patel Dipen, Horchi Dahbia, Rault Bleuenn, Mulvihill Emily, Beusterien Kathleen, Stewart Katherine, Miranda Paulo, Guillaume Xavier

机构信息

AstraZeneca, Cambridge, UK.

AstraZeneca, Gaithersburg, MD, USA.

出版信息

Patient Prefer Adherence. 2025 Mar 29;19:823-837. doi: 10.2147/PPA.S510401. eCollection 2025.

Abstract

BACKGROUND

Few studies have explored physician and patient preferences for the treatment of chronic lymphocytic leukemia (CLL) related to treatment efficacy, adverse events (AEs), and treatment duration. Thus, this observational, mixed-methods study investigated patients' and physicians' preferences for CLL first-line treatments.

MATERIALS AND METHODS

An online discrete choice experiment in five countries among 192 patients and 259 physicians in the US, the UK, Germany, France, and Australia examined the importance of outcomes and treatment attributes.

RESULTS

Increasing 5-year progression-free survival (5-year PFS) was most important to patients and physicians, with a relative importance (RI) of 30.3% among patients and 37.8% among physicians, followed by reducing the risks of common side effects (RI 21.6% among patients, 22.9% among physicians) and adverse events (AEs) leading to treatment discontinuation (RI 22.1% among patients, 20.6% among physicians). Patients strongly preferred time limited treatment regimen over treatment to progression (TTP). Specifically, patients and physicians would require a 6.4% vs 2.3% increase in 5-year PFS, a 19.4% vs 8.9% decrease in the risk of common all grades side effects, and a 7.5% vs 3.7% decrease in the risk of treatment discontinuation due to AEs, respectively, to compensate for a daily oral medication taken indefinitely vs daily oral medication taken for 24 months.

CONCLUSION

Overall, patients and physicians favor time-limited treatment regimens over TTP and value treatments with greater PFS benefits followed by lower side effects. Patients and physicians were both willing to trade-off switching from time-limited treatment to TTP for a better 5-year PFS, decrease side effects, and risk of treatment discontinuation due to AEs.

摘要

背景

很少有研究探讨医生和患者对于与治疗效果、不良事件(AE)及治疗持续时间相关的慢性淋巴细胞白血病(CLL)治疗的偏好。因此,这项观察性混合方法研究调查了患者和医生对CLL一线治疗的偏好。

材料与方法

在美国、英国、德国、法国和澳大利亚的五个国家对192名患者和259名医生进行了一项在线离散选择实验,以检验治疗结果和治疗属性的重要性。

结果

提高5年无进展生存期(5年PFS)对患者和医生最为重要,患者中的相对重要性(RI)为30.3%,医生中为37.8%,其次是降低常见副作用的风险(患者中RI为21.6%,医生中为22.9%)以及导致治疗中断的不良事件(AE)(患者中RI为22.1%,医生中为20.6%)。患者强烈倾向于时间有限的治疗方案而非疾病进展才停药(TTP)治疗。具体而言,患者和医生分别需要5年PFS提高6.4%而非2.3%、常见所有等级副作用风险降低19.4%而非8.9%、因AE导致治疗中断的风险降低7.5%而非3.7%,才能弥补无限期每日口服药物治疗与每日口服药物治疗24个月之间的差异。

结论

总体而言,患者和医生更倾向于时间有限的治疗方案而非TTP,并且重视具有更高PFS获益且副作用更低的治疗。患者和医生都愿意为了更好的5年PFS、降低副作用以及因AE导致治疗中断的风险而在从时间有限治疗转换为TTP之间进行权衡。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fd95/11963817/975b55ffa2a6/PPA-19-823-g0001.jpg

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