慢性淋巴细胞白血病一线治疗的偏好:来自患者和医生视角的多国研究结果。

IF 2 3区 医学 Q2 MEDICINE, GENERAL & INTERNAL
Patient preference and adherence Pub Date : 2025-03-29 eCollection Date: 2025-01-01 DOI:10.2147/PPA.S510401
Bersabeh Sile, Dipen Patel, Dahbia Horchi, Bleuenn Rault, Emily Mulvihill, Kathleen Beusterien, Katherine Stewart, Paulo Miranda, Xavier Guillaume
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引用次数: 0

摘要

背景:很少有研究探讨医生和患者对治疗慢性淋巴细胞白血病(CLL)的疗效、不良事件(AEs)和疗程的偏好。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:在美国、英国、德国、法国和澳大利亚五个国家的 192 名患者和 259 名医生中进行了在线离散选择实验,考察了结果和治疗属性的重要性:对患者和医生而言,提高 5 年无进展生存期(5 年 PFS)最为重要,患者和医生的相对重要性分别为 30.3% 和 37.8%,其次是降低常见副作用风险(患者的相对重要性为 21.6%,医生的相对重要性为 22.9%)和导致治疗中止的不良事件(AEs)(患者的相对重要性为 22.1%,医生的相对重要性为 20.6%)。患者强烈倾向于有时间限制的治疗方案,而不是治疗至病情进展(TTP)。具体来说,患者和医生需要分别将5年PFS提高6.4% vs 2.3%,将所有等级的常见副作用风险降低19.4% vs 8.9%,将因AE而中断治疗的风险降低7.5% vs 3.7%,才能补偿每日口服药物无限期服用与每日口服药物服用24个月:总体而言,患者和医生更倾向于有时间限制的治疗方案,而不是TTP,他们更看重PFS收益更大、副作用更小的治疗方案。患者和医生都愿意权衡从限时治疗转为 TTP 治疗,以获得更好的 5 年 PFS、减少副作用和因 AE 而中断治疗的风险。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Preferences for First-Line Chronic Lymphocytic Leukemia Treatments: Results From a Multinational Study on the Perspectives of Patients and Physicians.

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.

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来源期刊
Patient preference and adherence
Patient preference and adherence MEDICINE, GENERAL & INTERNAL-
CiteScore
3.60
自引率
4.50%
发文量
354
审稿时长
6-12 weeks
期刊介绍: Patient Preference and Adherence is an international, peer reviewed, open access journal that focuses on the growing importance of patient preference and adherence throughout the therapeutic continuum. The journal is characterized by the rapid reporting of reviews, original research, modeling and clinical studies across all therapeutic areas. Patient satisfaction, acceptability, quality of life, compliance, persistence and their role in developing new therapeutic modalities and compounds to optimize clinical outcomes for existing disease states are major areas of interest for the journal. As of 1st April 2019, Patient Preference and Adherence will no longer consider meta-analyses for publication.
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