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真实世界中大型全国健康计划中多发性骨髓瘤患者的流行病学、治疗模式和生存情况。

Real-world epidemiology, treatment patterns and survival of multiple myeloma patients in a large nationwide health plan.

机构信息

Maccabitech Institute for Research & Innovation, Maccabi Healthcare Services, Tel Aviv, Israel.

Janssen Israel, J-C Health Care Ltd., Kibbutz Shefayim, Israel.

出版信息

Leuk Res. 2019 Oct;85:106219. doi: 10.1016/j.leukres.2019.106219. Epub 2019 Aug 23.

DOI:10.1016/j.leukres.2019.106219
PMID:31473468
Abstract

BACKGROUND

Survival of patients with multiple myeloma (MM) has improved significantly with access to autologous stem cell transplant (SCT) and new treatments. This study aims to describe epidemiology, treatment patterns, and outcomes of MM in Israel.

METHODS

A retrospective observational study was conducted in Maccabi Healthcare Services, a 2-million-member nationwide health plan in Israel. MM was defined by cross-linking data on MM diagnoses, dispensed treatments, and serum free light-chain assays. Point prevalence (31/12/2016) and incidence (2012-2016) rates were age-standardized. Newly diagnosed and treated patients (2009-2015) were followed through 31/12/2016 for progression to second-line (L2), with death as a competing risk.

RESULTS

MM prevalence and incidence rates were 26.2 and 4.6 per 100,000 population, respectively. In the treatment cohort (N = 552), mean ± SD) age was 65.6 ± 11.3) years (60.1% male) and median (95% CI) OS in years was 5.2 (4.3-6.1) overall and 6.5 (4.9-8.1) for first-line (L1) bortezomib (N = 421). In a multivariable analysis, OS was significantly higher among patients starting L1 in 2012-2015 vs. 2009-2011. Within a year, 38.4% underwent SCT. Cumulative incidence of L2 was 38.2% and 51.4% within 1 and 2 years, respectively, and was associated with older age (≥65y; P < 0.001).

CONCLUSION

These results from a large heterogeneous population demonstrate MM incidence and survival rates that are in line with the literature, together with a significant improvement in overall survival over time. Approximately half of newly treated patients progressed to L2 within two years. These results will serve as a baseline for further research to evaluate the clinical impact of new interventions.

摘要

背景

随着自体干细胞移植(SCT)和新疗法的应用,多发性骨髓瘤(MM)患者的生存率得到了显著提高。本研究旨在描述以色列的 MM 流行病学、治疗模式和结局。

方法

本研究是在 Maccabi Healthcare Services 进行的一项回顾性观察性研究,该服务机构是以色列一个拥有 200 万成员的全国性健康计划。通过 MM 诊断、开出的治疗药物和血清游离轻链检测的数据交叉链接来定义 MM。点患病率(2016 年 12 月 31 日)和发病率(2012-2016 年)采用年龄标准化。2009-2015 年新诊断和治疗的患者(N=552)随访至 2016 年 12 月 31 日,以二线进展(L2)为竞争风险。

结果

MM 的患病率和发病率分别为 26.2 和 4.6/100,000 人。在治疗队列(N=552)中,患者的平均(±SD)年龄为 65.6±11.3 岁(60.1%为男性),中位(95%CI)OS 为 5.2 年(4.3-6.1),L1 硼替佐米治疗组(N=421)为 6.5 年(4.9-8.1)。多变量分析显示,2012-2015 年开始 L1 治疗的患者 OS 显著更高。一年内,38.4%的患者接受了 SCT。L2 的累积发生率分别为 38.2%和 51.4%,分别在 1 年和 2 年内,且与年龄较大(≥65 岁;P<0.001)相关。

结论

来自大型异质人群的这些结果表明,MM 的发病率和生存率与文献一致,并且总体生存率随着时间的推移显著提高。大约一半的新治疗患者在两年内进展到 L2。这些结果将作为进一步研究的基线,以评估新干预措施的临床影响。

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