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日本多发性骨髓瘤患者的治疗现状与疾病负担:一项真实世界调查

Treatment landscape and disease burden of patients with multiple myeloma in Japan: a real-world survey.

作者信息

Yasutomi Yusuke, Ribbands Amanda, Luke Emily, McNamara Simon

机构信息

GSK, Tokyo, Japan.

Adelphi Real World, Bollington, UK.

出版信息

Future Oncol. 2025 Mar;21(6):681-690. doi: 10.1080/14796694.2025.2460419. Epub 2025 Feb 4.

DOI:10.1080/14796694.2025.2460419
PMID:39902484
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11881850/
Abstract

AIM

Multiple myeloma (MM) is a hematological malignancy associated with poor health-related quality of life (HRQoL). Safe and effective treatments for MM are limited. There is a need for real-world data to improve understanding of treatment patterns and sequencing in routine clinical practice in Japan. This study evaluated disease burden, treatment patterns, treatment sequencing, and reasons for treatment selection in patients with MM in Japan.

METHODS

This analysis used survey data of hematologists or hemato-oncologists and their adult patients with MM who received active treatment in a real-world setting in Japan between September 2022 and May 2023. Treatment and retreatment patterns and data from several validated patient reported outcome tools were analyzed. Formal sample size calculations were not applicable.

RESULTS

Fifty-one physicians provided data for 309 patients, of whom 52 completed a quality-of-life survey (median [interquartile range] overall health status by EQ-5D-3L questionnaire: 0.7 [0.6-1.0]). Of 309 patients, most (77%) of the first-line cohort received a lenalidomide-based therapy. Lenalidomide retreatment was common in patients with relapsed/refractory MM (80%).

CONCLUSION

Poor HRQoL and high retreatment rates indicate a need for new therapy options in patients with MM in Japan. These findings may guide healthcare policies and clinical practice in Japan.

摘要

目的

多发性骨髓瘤(MM)是一种血液系统恶性肿瘤,与健康相关生活质量(HRQoL)较差有关。MM的安全有效治疗方法有限。需要真实世界的数据来增进对日本常规临床实践中治疗模式和治疗顺序的了解。本研究评估了日本MM患者的疾病负担、治疗模式、治疗顺序以及治疗选择的原因。

方法

本分析使用了血液科医生或血液肿瘤学家及其成年MM患者的调查数据,这些患者于2022年9月至2023年5月在日本的真实环境中接受了积极治疗。分析了治疗和再治疗模式以及来自几种经过验证的患者报告结局工具的数据。未进行正式的样本量计算。

结果

51名医生提供了309名患者的数据,其中52名完成了生活质量调查(EQ-5D-3L问卷的总体健康状况中位数[四分位间距]:0.7[0.6-1.0])。在309名患者中,大多数(77%)一线队列患者接受了基于来那度胺的治疗。来那度胺再治疗在复发/难治性MM患者中很常见(80%)。

结论

HRQoL较差和再治疗率较高表明日本MM患者需要新的治疗选择。这些发现可能会指导日本的医疗政策和临床实践。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/88db7c0e4347/IFON_A_2460419_F0004_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/1da59af612f7/IFON_A_2460419_F0001_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/a82d51fd181e/IFON_A_2460419_F0002_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/8d0c67aa1cf4/IFON_A_2460419_F0003_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/88db7c0e4347/IFON_A_2460419_F0004_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/1da59af612f7/IFON_A_2460419_F0001_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/a82d51fd181e/IFON_A_2460419_F0002_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/8d0c67aa1cf4/IFON_A_2460419_F0003_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2497/11881850/88db7c0e4347/IFON_A_2460419_F0004_OC.jpg

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本文引用的文献

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Br J Haematol. 2024 Sep;205(3):780-797. doi: 10.1111/bjh.19627. Epub 2024 Jul 19.
2
Considerations for next therapy after anti-CD38 monoclonal antibodies used as first line.考虑将抗 CD38 单克隆抗体作为一线治疗后,下一步的治疗方案。
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):443-449. doi: 10.1182/hematology.2023000444.
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Real-world evidence generation from patients, their caregivers and physicians supporting clinical, regulatory and guideline decisions: an update on Disease Specific Programmes.
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Curr Med Res Opin. 2023 Dec;39(12):1707-1715. doi: 10.1080/03007995.2023.2279679. Epub 2023 Dec 15.
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Drivers of physician decision-making and patient perspectives across lines of therapy in multiple myeloma in the USA.美国多发性骨髓瘤治疗线间的医生决策驱动因素和患者观点。
Future Oncol. 2023 Jul;19(22):1549-1562. doi: 10.2217/fon-2023-0020. Epub 2023 Jun 7.
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Real-world patient-reported outcomes and concordance between patient and physician reporting of side effects across lines of therapy in multiple myeloma within the USA.美国多发性骨髓瘤中,跨线治疗的真实世界患者报告结局与患者和医生报告的副作用之间的一致性。
Support Care Cancer. 2023 Jun 2;31(6):371. doi: 10.1007/s00520-023-07836-x.
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Survival outcomes among patients with multiple myeloma in the era of novel agents: exploratory assessment using an electronic medical record database in Japan.新型药物时代多发性骨髓瘤患者的生存结局:来自日本电子病历数据库的探索性评估。
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