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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。

Direct Medical Costs Associated With Treatment Nonpersistence in Patients With Higher-Risk Myelodysplastic Syndromes Receiving Hypomethylating Agents: A Large Retrospective Cohort Analysis.

机构信息

Real-world Evidence/Data Analytics Center of Excellence, Pharmerit International LP, Bethesda, MD.

Real-world Evidence/Data Analytics Center of Excellence, Pharmerit International LP, Bethesda, MD.

出版信息

Clin Lymphoma Myeloma Leuk. 2021 Mar;21(3):e248-e254. doi: 10.1016/j.clml.2020.12.002. Epub 2020 Dec 9.

Abstract

BACKGROUND

Suboptimal use of hypomethylating agents (HMAs) among higher-risk myelodysplastic syndrome (HR-MDS) patients can translate into worse health outcomes and economic burden. We estimated the direct medical costs associated with HMA treatment nonpersistence among HR-MDS patients.

PATIENTS AND METHODS

Using the Surveillance, Epidemiology, and End Results-Medicare linked database, a retrospective cohort of patients diagnosed with refractory anemia with excess blasts (RAEB), a diagnosis that substantially overlaps with HR-MDS, between January 2011 and December 2015 was analyzed. Patients who had ≥ 1 year of continuous Medicare enrollment before diagnosis and who did not receive stem cell transplant or lenalidomide in the follow-up period were included. Patients receiving HMAs were stratified into HMA persistent (≥4 HMA cycles) and HMA nonpersistent (<4 cycles or a gap of ≥ 90 days between cycles) groups. Healthcare resource use and costs during the follow-up period were reported descriptively as total and per patient per month (PPPM). Weighted generalized linear models (GLM) were used to compare estimated healthcare resource use and costs between HMA groups.

RESULTS

Among the 664 patients with RAEB, 295 (44.4%) were HMA nonpersistent and 369 (55.6%) HMA persistent. On the basis of weighted GLM analysis, the HMA nonpersistent group incurred significantly (P < .05) higher total PPPM costs compared to the HMA persistent group ($18,039 vs. $13,893), particularly for hospitalization ($3,375 vs. $2,131), and emergency room ($5,517 vs. $2,867) costs.

CONCLUSION

There is a substantial economic burden associated with early discontinuation of guideline-recommended HMA therapy in RAEB patients. The study findings necessitate closer care management in this population in order to improve outcomes and reduce healthcare spending.

摘要

背景

高危骨髓增生异常综合征(HR-MDS)患者中低甲基化药物(HMAs)的使用不当可能导致更差的健康结果和经济负担。我们估计了 HR-MDS 患者中 HMAs 治疗不持续相关的直接医疗成本。

患者和方法

利用监测、流行病学和最终结果-医疗保险链接数据库,分析了 2011 年 1 月至 2015 年 12 月期间被诊断为难治性贫血伴过多原始细胞(RAEB)的患者的回顾性队列,该诊断与 HR-MDS 有很大的重叠。患者在诊断前至少有 1 年的连续医疗保险登记,且在随访期间未接受干细胞移植或来那度胺。接受 HMAs 的患者分为 HMAs 持续(≥4 个 HMA 周期)和 HMAs 不持续(<4 个周期或周期之间的间隔≥90 天)组。报告了随访期间的医疗资源使用和成本,总费用和每位患者每月(PPPM)。使用加权广义线性模型(GLM)比较了 HMA 组之间的估计医疗资源使用和成本。

结果

在 664 名 RAEB 患者中,295 名(44.4%)为 HMAs 不持续,369 名(55.6%)为 HMAs 持续。基于加权 GLM 分析,HMAs 不持续组的总 PPPM 成本明显高于 HMAs 持续组(18039 美元比 13893 美元),特别是住院费用(3375 美元比 2131 美元)和急诊室费用(5517 美元比 2867 美元)。

结论

在 RAEB 患者中,早期停止推荐的 HMAs 治疗与大量的经济负担有关。研究结果表明,需要对该人群进行更密切的护理管理,以改善结局和降低医疗支出。

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