离散选择实验分析,了解患者对多发性骨髓瘤治疗的偏好。

IF 3.5 3区 医学 Q2 ONCOLOGY
Frontiers in Oncology Pub Date : 2025-09-16 eCollection Date: 2025-01-01 DOI:10.3389/fonc.2025.1628121
Beth Faiman, Hoa H Le, Julie Laurent, Saurabh Patel, Agne Paner-Straseviciute, Xinke Zhang, Joseph Mikhael
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引用次数: 0

摘要

简介:多发性骨髓瘤(Multiple myeloma, MM)是一种以骨髓内异常浆细胞克隆扩增为特征的恶性浆细胞疾病。复发/难治性多发性骨髓瘤(RRMM)的管理是一项重大挑战,因为疾病复发或对先前的治疗变得难治性。最近的治疗进展扩大了RRMM的治疗选择。本研究旨在更深入地了解患者对现有治疗方案的治疗偏好。方法:本研究是一项基于在线离散选择实验(DCE)的非介入性描述性横断面研究,研究对象为2023年11月至2024年3月生活在美国的成年RRMM患者。该调查包括从广泛的文献综述和对MM患者进行的指导访谈中得出的属性和水平。使用条件逻辑(CL)回归模型对偏好数据进行分析,并计算相对属性重要性(RAI)得分。使用从CL模型估计的帕特沃斯效用来评估患者权衡总缓解率(ORR)的意愿。结果:149例MM患者完成调查;66%接受过1-2次治疗,15%接受过3次治疗,19%接受过4次或更多治疗。患者明显倾向于更长的无进展生存期(PFS)和总生存期(OS)以及更高的ORR (RAI分别为36.4%和22.1%)的治疗。对于本研究评估的不良事件,患者表达了对细胞因子释放综合征(CRS) (RAI: 15.2%)和感染(RAI: 11.9%)的担忧。相比之下,指甲/皮肤疾病、住院时间和味觉障碍对患者来说不太重要。患者愿意接受高风险的CRS(72%比无风险),以获得增加29%的ORR。结论:患者对治疗疗效(PFS/OS和ORR)有明显的偏好。该研究证实了患者对新治疗方案中治疗属性的评价,并强调了共享治疗决策对最佳临床结果的重要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A discrete choice experiment analysis to understand patient preferences for multiple myeloma treatments.

Introduction: Multiple myeloma (MM) is a malignant plasma cell disorder characterized by the clonal expansion of abnormal plasma cells within the bone marrow. The management of relapsed/refractory multiple myeloma (RRMM) represents a significant challenge as the disease relapses or becomes refractory to previous treatments. Recent advances in therapy have expanded RRMM treatment options. This study aimed to gain a deeper understanding of patients' treatment preferences regarding available therapeutic options.

Methods: This study was designed as a non-interventional descriptive cross-sectional study based on an online discrete choice experiment (DCE) among adult RRMM patients living in the between USA November 2023 and March 2024. The survey included attributes and levels derived from an extensive literature review and guided interviews conducted with MM patients. Preference data were analyzed using a conditional logistic (CL) regression model and relative attribute importance (RAI) scores were calculated. Patients' willingness to trade off overall response rate (ORR) was evaluated using the partworth utilities estimated from the CL model.

Results: 149 MM patients completed the survey; 66% had received 1-2 prior lines of therapy, 15% three prior lines, 19% four or more prior lines. Patients significantly preferred treatments with longer progression-free survival (PFS) and overall survival (OS) and higher ORR (RAI: 36.4% and 22.1%, respectively). With respect to adverse events assessed in this study, patients expressed concern for cytokine release syndrome (CRS) (RAI: 15.2%) and infections (RAI: 11.9%). In contrast, nail/skin disorders, duration of hospitalization, and taste disorder were less important to patients. Patients would be willing to accept a high risk of CRS (72% over no risk) to gain 29% increase in ORR.

Conclusions: Patients showed a clear preference for treatment efficacy (PFS/OS and ORR). This study confirmed patients' valuation on treatment attributes in the new treatment landscape and highlighted the importance of shared treatment decision-making for optimal clinical outcomes.

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来源期刊
Frontiers in Oncology
Frontiers in Oncology Biochemistry, Genetics and Molecular Biology-Cancer Research
CiteScore
6.20
自引率
10.60%
发文量
6641
审稿时长
14 weeks
期刊介绍: Cancer Imaging and Diagnosis is dedicated to the publication of results from clinical and research studies applied to cancer diagnosis and treatment. The section aims to publish studies from the entire field of cancer imaging: results from routine use of clinical imaging in both radiology and nuclear medicine, results from clinical trials, experimental molecular imaging in humans and small animals, research on new contrast agents in CT, MRI, ultrasound, publication of new technical applications and processing algorithms to improve the standardization of quantitative imaging and image guided interventions for the diagnosis and treatment of cancer.
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