• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验

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.

DOI:10.1016/j.clml.2020.10.013
PMID:33293239
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。

相似文献

1
Under-use of Hypomethylating Agents in Patients With Higher-risk Myelodysplastic Syndrome in the United States: A Large Population-based Analysis.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。
Clin Lymphoma Myeloma Leuk. 2021 Feb;21(2):e206-e211. doi: 10.1016/j.clml.2020.10.013. Epub 2020 Oct 26.
2
Impact of Hypomethylating Agent Use on Hospital and Emergency Room Visits, and Predictors of Early Discontinuation in Patients With Higher-Risk Myelodysplastic Syndromes.低甲基化药物的使用对高危骨髓增生异常综合征患者的住院和急诊就诊的影响,以及早期停药的预测因素。
Clin Lymphoma Myeloma Leuk. 2022 Sep;22(9):670-679. doi: 10.1016/j.clml.2022.04.016. Epub 2022 Apr 22.
3
Comparative clinical effectiveness of azacitidine versus decitabine in older patients with myelodysplastic syndromes.阿扎胞苷与地西他滨治疗老年骨髓增生异常综合征患者的临床疗效比较
Br J Haematol. 2016 Dec;175(5):829-840. doi: 10.1111/bjh.14305. Epub 2016 Sep 21.
4
Hypomethylating agent (HMA) therapy use and survival in older adults with Refractory Anemia with Excess Blasts (RAEB) in the United States (USA): a large propensity score-matched population-based study.美国老年难治性贫血伴原始细胞过多型(RAEB)患者中低甲基化药物(HMA)治疗的应用与生存:一项大型倾向评分匹配的基于人群的研究。
Leuk Lymphoma. 2020 May;61(5):1178-1187. doi: 10.1080/10428194.2019.1703970. Epub 2019 Dec 26.
5
Direct Medical Costs Associated With Treatment Nonpersistence in Patients With Higher-Risk Myelodysplastic Syndromes Receiving Hypomethylating Agents: A Large Retrospective Cohort Analysis.接受低甲基化药物治疗的高危骨髓增生异常综合征患者治疗不持续相关的直接医疗费用:一项大型回顾性队列分析。
Clin Lymphoma Myeloma Leuk. 2021 Mar;21(3):e248-e254. doi: 10.1016/j.clml.2020.12.002. Epub 2020 Dec 9.
6
Hypomethylating agent therapy use and survival in older patients with chronic myelomonocytic leukemia in the United States: A large population-based study.美国老年慢性粒单核细胞白血病患者的低甲基化药物治疗与生存情况:一项基于大规模人群的研究
Cancer. 2017 Oct 1;123(19):3754-3762. doi: 10.1002/cncr.30814. Epub 2017 Jun 16.
7
RBC transfusion independence among lower risk MDS patients receiving hypomethylating agents: a population-level analysis.接受低甲基化药物治疗的低风险骨髓增生异常综合征患者实现红细胞输注独立:一项基于人群水平的分析。
Leuk Lymphoma. 2019 Dec;60(13):3181-3187. doi: 10.1080/10428194.2019.1622700. Epub 2019 Jun 7.
8
Clinical Outcomes in Patients With Refractory Anemia With Excess Blasts (RAEB) Who Receive Hypomethylating Agents (HMAs).接受低甲基化药物治疗的难治性贫血伴原始细胞过多(RAEB)患者的临床结局。
Clin Lymphoma Myeloma Leuk. 2024 Mar;24(3):177-186. doi: 10.1016/j.clml.2023.10.010. Epub 2023 Oct 30.
9
Persistence to hypomethylating agents and clinical and economic outcomes among patients with myelodysplastic syndromes.骨髓增生异常综合征患者持续接受低甲基化药物治疗与临床和经济结局的关系。
Hematology. 2021 Dec;26(1):261-270. doi: 10.1080/16078454.2021.1889161.
10
Real-world use and outcomes of hypomethylating agent therapy in higher-risk myelodysplastic syndromes: why are we not achieving the promise of clinical trials?高危骨髓增生异常综合征中低甲基化剂治疗的真实世界应用和结局:我们为何未能实现临床试验的承诺?
Future Oncol. 2021 Dec;17(36):5163-5175. doi: 10.2217/fon-2021-0936. Epub 2021 Oct 12.

引用本文的文献

1
Real-World Treatment Patterns, Clinical Outcomes, and Costs in Patients with Higher-Risk Myelodysplastic Syndromes Across France, Germany, and the United Kingdom.法国、德国和英国高危骨髓增生异常综合征患者的真实世界治疗模式、临床结局及成本
J Blood Med. 2025 Jun 25;16:307-319. doi: 10.2147/JBM.S516558. eCollection 2025.
2
Advances and challenges in the treatment of myelodysplastic syndromes.骨髓增生异常综合征治疗的进展与挑战
Exp Hematol Oncol. 2025 Jun 18;14(1):87. doi: 10.1186/s40164-025-00678-9.
3
Role of Population Based Studies in Advancing our Knowledge of Myelodysplastic Syndromes.
基于人群的研究在增进我们对骨髓增生异常综合征认识方面的作用。
Curr Hematol Malig Rep. 2025 Jan 17;20(1):6. doi: 10.1007/s11899-025-00750-5.
4
Patients' perspectives on oral decitabine/cedazuridine for the treatment of myelodysplastic syndromes/neoplasms.患者对口服地西他滨/西扎珠利治疗骨髓增生异常综合征/肿瘤的看法。
Ther Adv Hematol. 2024 Jul 30;15:20406207241257313. doi: 10.1177/20406207241257313. eCollection 2024.
5
Development of the treatment preference in myelodysplasia questionnaire for clinicians, carers, and patients.面向临床医生、护理人员和患者的骨髓增生异常综合征治疗偏好问卷的编制
EJHaem. 2024 May 28;5(3):535-540. doi: 10.1002/jha2.930. eCollection 2024 Jun.
6
Clinical activity, pharmacokinetics, and pharmacodynamics of oral hypomethylating agents for myelodysplastic syndromes/neoplasms and acute myeloid leukemia: A multidisciplinary review.口服低甲基化药物治疗骨髓增生异常综合征/肿瘤和急性髓系白血病的临床活性、药代动力学和药效学:多学科综述。
J Oncol Pharm Pract. 2024 Jun;30(4):721-736. doi: 10.1177/10781552241238979. Epub 2024 Mar 21.
7
Impact of preexisting autoimmune disease on myelodysplastic syndromes outcomes: a population analysis.预先存在的自身免疫性疾病对骨髓增生异常综合征结局的影响:一项人群分析。
Blood Adv. 2023 Nov 28;7(22):6913-6922. doi: 10.1182/bloodadvances.2023011050.
8
Considerations for Drug Development in Myelodysplastic Syndromes.骨髓增生异常综合征药物研发的考量因素。
Clin Cancer Res. 2023 Jul 14;29(14):2573-2579. doi: 10.1158/1078-0432.CCR-22-3348.
9
Finding consistency in classifications of myeloid neoplasms: a perspective on behalf of the International Workshop for Myelodysplastic Syndromes.寻找髓系肿瘤分类的一致性:代表国际骨髓增生异常综合征研讨会的观点
Leukemia. 2022 Dec;36(12):2939-2946. doi: 10.1038/s41375-022-01724-9. Epub 2022 Oct 20.
10
A randomized phase 2 trial of azacitidine with or without durvalumab as first-line therapy for higher-risk myelodysplastic syndromes.一项阿扎胞苷联合或不联合度伐利尤单抗作为高危骨髓增生异常综合征一线治疗的随机 2 期临床试验。
Blood Adv. 2022 Apr 12;6(7):2207-2218. doi: 10.1182/bloodadvances.2021005487.