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美国高危骨髓增生异常综合征患者中低甲基化药物的未充分使用:一项大型基于人群的分析。

Under-use of Hypomethylating Agents in Patients With Higher-risk Myelodysplastic Syndrome in the United States: A Large Population-based Analysis.

机构信息

Pharmerit International, LP, Bethesda, MD.

Pharmerit International, LP, Bethesda, MD.

出版信息

Clin Lymphoma Myeloma Leuk. 2021 Feb;21(2):e206-e211. doi: 10.1016/j.clml.2020.10.013. Epub 2020 Oct 26.

Abstract

BACKGROUND

Recent data suggest significant underutilization of hypomethylating agents (HMAs) that are recommended treatments for patients with myelodysplastic syndromes (MDS) with refractory anemia with excess blasts (RAEB). The study objective was to assess the degree of HMA use and predictors of HMA underuse in this population.

PATIENTS AND METHODS

This was a retrospective study including patients diagnosed with the RAEB form of MDS between January 2011 and December 2015 using the Surveillance, Epidemiology, and End Results-Medicare linked database. Patients were excluded if they had < 1 year of continuous enrollment before diagnosis or received stem cell transplant or lenalidomide during the follow-up period. HMA non-peristence was defined as use of < 4 cycles (3-10 HMA days/28 days) of HMAs or a gap of ≥ 90 days between consecutive cycles. Patients were characterized as HMA never-users, HMA-persistent users, and HMA-non-persistent users. Descriptive statistics were used to summarize patient characteristics. Multivariable logistic regression was used to assess predictors of HMA underuse and persistence.

RESULTS

Of the 1190 patients, 526 (44%) were never-users, 295 (25%) were non-persistent users, and 369 (31%) were persistent users. Age at diagnosis (eg, 66-70 years vs. ≥ 80 years; odds ratio [OR], 2.36; 95% confidence interval [CI], 1.56-3.56), marital status (single vs. married; OR, 0.67; 95% CI, 0.51-0.89), National Cancer Institute comorbidity index (≥ 3 vs. 0-1; OR, 0.62; 95% CI, 0.46-0.83), and performance status (poor vs. good; OR, 0.67; 95% CI, 0.51-0.87) were significantly associated with HMA underuse.

CONCLUSION

Several demographic and clinical factors were associated with underuse of HMAs. There is need for a better understanding of suboptimal HMA use and its relationship with clinical response.

摘要

背景

最近的数据表明,对于骨髓增生异常综合征(MDS)伴难治性贫血伴原始细胞过多(RAEB)的患者,低甲基化药物(HMA)的应用明显不足,HMA 是这些患者的推荐治疗药物。本研究的目的是评估该人群中 HMA 的应用程度和 HMA 应用不足的预测因素。

患者和方法

这是一项回顾性研究,纳入了 2011 年 1 月至 2015 年 12 月期间使用监测、流行病学和最终结果-医疗保险链接数据库诊断为 RAEB 型 MDS 的患者。如果患者在诊断前连续登记时间<1 年,或在随访期间接受过干细胞移植或来那度胺,则排除这些患者。HMA 无持续性定义为接受<4 个周期(3-10 HMA 天/28 天)的 HMA 治疗或连续周期之间的间隔>90 天。患者被分为 HMA 从未使用者、HMA 持续使用者和 HMA 非持续使用者。采用描述性统计方法总结患者特征。采用多变量逻辑回归评估 HMA 应用不足和持续性的预测因素。

结果

在 1190 例患者中,526 例(44%)从未使用者、295 例(25%)为非持续性使用者、369 例(31%)为持续性使用者。诊断时的年龄(如,66-70 岁比≥80 岁;比值比[OR],2.36;95%置信区间[CI],1.56-3.56)、婚姻状况(单身比已婚;OR,0.67;95%CI,0.51-0.89)、国家癌症研究所合并症指数(≥3 比 0-1;OR,0.62;95%CI,0.46-0.83)和体能状态(差比好;OR,0.67;95%CI,0.51-0.87)与 HMA 应用不足显著相关。

结论

一些人口统计学和临床因素与 HMA 的应用不足有关。需要更好地了解 HMA 应用不足及其与临床反应的关系。

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