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复发/难治性多发性骨髓瘤治疗的真实世界数据:来自 ASH 2023 的关键数据——一档播客

英文原题:Real-World Data on Therapies for Relapsed or Refractory Multiple Myeloma: Key Data from ASH 2023-A Podcast.

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Real-World Data on Therapies for Relapsed or Refractory Multiple Myeloma: Key Data from ASH 2023-A Podcast.

PubMed 2024/04/20(内容时间) Adv Ther Q1 · IF 4.7(JCR 2025)

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中文摘要

免疫疗法显著改善了多发性骨髓瘤患者的治疗结局,但对于既往接受多线治疗的患者,维持持久应答仍具挑战。靶向 B 细胞成熟抗原(BCMA)的疗法为早期治疗线中已对多类药物耐药的患者提供了更多选择。来自特定患者群体和受控条件下的临床试验数据,可由真实世界数据(RWD)加以补充。本播客中,作者回顾并讨论了第 65 届美国血液学会年会上发表的七篇摘要,聚焦 BCMA 靶向疗法,强调 RWD 在治疗决策中的价值,并探讨 RWD 如何推动多发性骨髓瘤研究。这些摘要包括:三类药物均暴露或耐药的复发/难治性多发性骨髓瘤患者真实世界结局研究(摘要 542、3358 和 6727);延迟诊断相关疾病负担分析(摘要 3771);真实世界结局与临床试验数据的可比性(摘要 91 和 545);以及初始四联治疗后早期治疗失败患者的结局(摘要 1989)。本文附有播客。多发性骨髓瘤(MM)是一种累及骨髓浆细胞的血液系统恶性肿瘤。目前 MM 尚无法治愈,治疗进展已改善患者生存,但许多患者最终仍会复发,并对一种或多种早期治疗药物产生耐药。随着三联和四联疗法应用增加,部分患者在二线或三线治疗时即会对三类药物均耐药。

因此,需要开发作用机制不同于免疫调节剂、蛋白酶体抑制剂和抗 CD38 抗体的其他治疗选择。纳入三类药物耐药 MM 患者的多项临床试验取得令人鼓舞的结果,促成近期靶向 BCMA 药物获批,包括嵌合抗原受体(CAR)T 细胞疗法和 BCMA×CD3 双特异性抗体。对临床实践中接受 BCMA 靶向疗法患者进行的真实世界疗效、安全性和用药情况研究,可为临床试验结果提供重要补充。2023 年 12 月在加利福尼亚州圣迭戈举行的第 65 届美国血液学会年会上,研究人员报告了 BCMA 靶向药物用于复发/难治性 MM(RRMM)患者的真实世界研究结果。在本播客中,两位血液学专家讨论这些药物对患者报告结局的影响、其有效性和安全性与临床试验数据的比较,以及真实世界研究将如何影响未来 RRMM 患者的治疗。补充文件 1(MP4,51,557 KB)。

展开英文摘要原文

Immunotherapies have significantly improved outcomes in patients with multiple myeloma yet maintaining a durable response in heavily pretreated patients remains challenging. Therapies that target B cell maturation antigen (BCMA) provide additional treatment options in patients whose disease becomes refractory to several drug classes in early lines of therapy. Clinical trial data from selected patient populations and controlled settings are complemented by real-world data (RWD) from actual clinical practice. In this podcast, the authors reviewed and discussed seven abstracts presented at the 65th Annual Meeting of the American Society of Hematology, focusing on BCMA-directed therapies, emphasizing the value of RWD in treatment decision-making, and suggesting how RWD can help advance multiple myeloma research.

These abstracts include real-world outcome studies in patients with relapsed or refractory multiple myeloma with triple-class exposed or refractory disease (abstracts 542, 3358, and 6727); an analysis on disease burden associated with delayed diagnosis (abstract 3771); comparability of real-world outcomes vs clinical trial data (abstracts 91 and 545); and outcomes in patients with multiple myeloma who experienced early treatment failure after upfront quadruplet therapy (abstract 1989).

Podcast available for this article. Multiple myeloma (MM) is a hematologic cancer that affects plasma cells in the bone marrow. Although there is no cure for MM, advances in treatment have improved survival outcomes in patients with MM.

However, for many patients the disease will eventually relapse and become refractory to one or more early-line agents. Indeed, with increased use of triplet and quadruplet therapies, the myeloma of some patients will become triple-class refractory as early as their second or third line of therapy.

Thus, additional treatment options that are mechanistically distinct from immunomodulatory drugs, proteasome inhibitors, and anti-CD38 antibodies are needed. Encouraging outcomes from several clinical trials enrolling patients with triple-class refractory MM have led to the recent regulatory approval of drugs that target B cell maturation antigen (BCMA), such as chimeric antigen receptor (CAR)-T cell therapies and BCMA CD3 bispecific antibodies. Real-world evidence on the efficacy, safety, and usage of these BCMA-directed therapies from patients treated in clinical practice provides valuable evidence that complements the findings from clinical trials.

At the 65th Annual Meeting of the American Society of Hematology held in December 2023 in San Diego, CA, researchers presented results from real-world studies of BCMA-targeting drugs in patients with relapsed or refractory MM (RRMM). In this podcast, two leading hematologists discuss how these drugs affect patient-reported outcomes, how the effectiveness and safety of these drugs compare with data from clinical trials, and how real-world studies shape how patients with RRMM may be treated in the future. Supplementary file1 (MP4 51,557 KB).

论文信息

作者
Costa LJ、Rodriguez-Otero P
第一作者单位
University of Alabama at Birmingham, Birmingham, AL, USA.United Kingdom
通讯作者单位
Clinica Universidad de Navarra, Pamplona, Spain. paurodriguez@unav.es.Spain
文献类型
综述 · 非美国政府资助研究
期刊
Advances in therapy2024 Aug
原文标识
PubMed 38642197 · DOI 10.1007/s12325-024-02842-9