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新型疗法在美国问世后多发性骨髓瘤的死亡率趋势。

Mortality trends in multiple myeloma after the introduction of novel therapies in the United States.

机构信息

Division of Hematology, Mayo Clinic, Rochester, MN, USA.

出版信息

Leukemia. 2022 Mar;36(3):801-808. doi: 10.1038/s41375-021-01453-5. Epub 2021 Oct 26.

DOI:10.1038/s41375-021-01453-5
PMID:34702976
Abstract

Advances in the understanding of disease biology, drug development, and supportive care have led to improved outcomes in multiple myeloma. Given that these improvements have been reported in clinical trial and referral center populations, questions remain about the generalizability of this observation to patients treated in the community. Contrasting the overall survival experience of 3783 patients seen at Mayo Clinic and 57,654 patients followed in the Surveillance, Epidemiology, and End Results Program (SEER) between 2004 and 2018, we observed different mortality trends across patient populations and subgroups. Early mortality decreased and estimated 5-year overall survival increased over time in both patient populations. Excess mortality (compared to the general population) declined over time in Mayo Clinic patients and remained largely unchanged in SEER patients. Improvements over time were primarily observed in patients with favorable disease characteristics and older patients with multiple myeloma remain a vulnerable population with significant excess mortality compared to the United States general population. Patients with unfavorable disease characteristics have derived disproportionately less benefit from recent advances in the field. Future efforts need to focus on the development of safe and effective therapies for these patients and on increasing timely access to specialized care for patients in the community.

摘要

疾病生物学、药物开发和支持性护理方面的进展使得多发性骨髓瘤的治疗结果得到了改善。鉴于这些改进是在临床试验和转诊中心人群中报告的,因此仍存在关于观察结果是否可以推广到社区中接受治疗的患者的问题。通过对比梅奥诊所 3783 名患者和 2004 年至 2018 年期间监测、流行病学和最终结果计划(SEER)中 57654 名患者的总生存经验,我们观察到患者人群和亚组之间存在不同的死亡率趋势。在这两个患者群体中,早期死亡率下降,估计 5 年总生存率随时间推移而增加。与一般人群相比,梅奥诊所患者的超额死亡率随时间下降,而 SEER 患者的超额死亡率基本保持不变。随着时间的推移,改善主要发生在疾病特征良好的患者和多发性骨髓瘤的老年患者中,与美国一般人群相比,这些患者仍然是一个脆弱的群体,死亡率明显过高。疾病特征不佳的患者从该领域的最新进展中获得的益处不成比例地减少。未来的工作重点需要放在为这些患者开发安全有效的治疗方法上,并增加社区患者获得专业护理的及时性。

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