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多发性骨髓瘤的损耗率和治疗结果:40年期间的真实世界数据。

Attrition rates and treatment outcomes in multiple myeloma: real-world data over a 40-year period.

作者信息

Rodríguez-Lobato Luis Gerardo, de Daniel Anna, Pereira Arturo, Fernández de Larrea Carlos, Tovar Natalia, Cibeira M Teresa, Moreno David F, Mateos Jose Miguel, Llobet Noemí, Carcelero Esther, Munárriz Daniel, Bladé Joan, Rosiñol Laura

机构信息

Amyloidosis and Multiple Myeloma Unit, Department of Hematology, Hospital Clínic de Barcelona, Universitat de Barcelona, Barcelona, Spain.

Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Barcelona, Spain.

出版信息

Blood Cancer J. 2025 May 26;15(1):103. doi: 10.1038/s41408-025-01311-y.

DOI:10.1038/s41408-025-01311-y
PMID:40419498
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12106733/
Abstract

The treatment landscape of multiple myeloma (MM) has evolved significantly over four decades, driven by novel therapies and optimized supportive care. However, the attrition rate (AR), defined as the proportion of patients who die without advancing to the next line of therapy (LOT) after treatment failure, remains a major challenge. To assess how treatment patterns and outcomes have evolved, we analyzed 1,297 MM patients treated between 1980 and 2020, stratified by diagnosis period and age. ARs declined from 38-55% in the 1980s to 15-20% in 2010-2020, but remained high in older patients, with 46.9% of those over 80 unable to proceed beyond first LOT. While progression-free survival gains were primarily observed in the first LOT (15.8 to 24.1 months, p = 0.001), overall survival (OS) improved across all LOTs and age groups, likely due to more effective salvage therapies and supportive care. Achieving a complete response in first-line therapy was associated with a significant OS benefit (4.5 vs. 1.6 years, p < 0.001), underscoring its importance, as many patients, particularly older ones, are less likely to reach subsequent LOTs. Despite advances in MM treatment, patient loss to attrition remains a challenge, highlighting the need for more effective therapies early in the disease course.

摘要

在新型疗法和优化的支持性治疗的推动下,多发性骨髓瘤(MM)的治疗格局在四十多年间发生了显著演变。然而,损耗率(AR),即治疗失败后未进入下一线治疗(LOT)就死亡的患者比例,仍然是一个重大挑战。为了评估治疗模式和结果是如何演变的,我们分析了1980年至2020年间接受治疗的1297例MM患者,按诊断时期和年龄进行分层。损耗率从20世纪80年代的38%-55%下降到2010-2020年的15%-20%,但老年患者的损耗率仍然很高,80岁以上的患者中有46.9%无法进入一线治疗之后的阶段。虽然无进展生存期的延长主要出现在一线治疗中(从15.8个月延长至24.1个月,p = 0.001),但所有治疗阶段和年龄组的总生存期(OS)均有所改善,这可能得益于更有效的挽救疗法和支持性治疗。一线治疗中实现完全缓解与显著的总生存期获益相关(4.5年对1.6年,p < 0.001),这凸显了其重要性,因为许多患者,尤其是老年患者,进入后续治疗阶段的可能性较小。尽管MM治疗取得了进展,但患者损耗仍是一个挑战,这突出了在疾病早期阶段需要更有效疗法的必要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/358c/12106733/7999ed700ba4/41408_2025_1311_Fig5_HTML.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/358c/12106733/736471a4d115/41408_2025_1311_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/358c/12106733/07b679e9d9fa/41408_2025_1311_Fig3_HTML.jpg
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本文引用的文献

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Clin Lymphoma Myeloma Leuk. 2024 Mar;24(3):e104-e111.e1. doi: 10.1016/j.clml.2023.11.008. Epub 2023 Nov 27.
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Treatment attrition rates and relevant risk factors in multiple myeloma: A real-world study in China.多发性骨髓瘤的治疗损耗率及相关危险因素:一项中国的真实世界研究。
Front Pharmacol. 2023 Jan 12;14:979111. doi: 10.3389/fphar.2023.979111. eCollection 2023.
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Real-world assessment of treatment patterns and outcomes in patients with relapsed-refractory multiple myeloma in an Italian haematological tertiary care centre.意大利血液学三级护理中心复发性/难治性多发性骨髓瘤患者的治疗模式和结局的真实世界评估。
Br J Haematol. 2023 May;201(3):432-442. doi: 10.1111/bjh.18658. Epub 2023 Jan 17.
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Impact of achieving a complete response to initial therapy of multiple myeloma and predictors of subsequent outcome.多发性骨髓瘤初始治疗达到完全缓解的影响及其后续结果的预测因素。
Am J Hematol. 2022 Mar 1;97(3):267-273. doi: 10.1002/ajh.26439. Epub 2022 Jan 3.
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Frontline treatment patterns and attrition rates by subsequent lines of therapy in patients with newly diagnosed multiple myeloma.初诊多发性骨髓瘤患者的一线治疗模式和后续治疗线的淘汰率。
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Long-term Outcomes in Patients With Multiple Myeloma: A Retrospective Analysis of the Dutch Population-based HAematological Registry for Observational Studies (PHAROS).多发性骨髓瘤患者的长期预后:基于荷兰人群的血液学观察性研究登记系统(PHAROS)的回顾性分析
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