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羟基脲治疗镰状细胞病患者的治疗模式和经济负担:一项基于回顾性索赔的研究。

Treatment patterns and economic burden of sickle-cell disease patients prescribed hydroxyurea: a retrospective claims-based study.

机构信息

Duke University, Durham, NC, USA.

Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.

出版信息

Health Qual Life Outcomes. 2019 Oct 16;17(1):155. doi: 10.1186/s12955-019-1225-7.

Abstract

BACKGROUND

This study aimed to evaluate sickle-cell disease (SCD) treatment patterns and economic burden among patients prescribed hydroxyurea (HU) in the US, through claims data.

METHODS

SCD patients with pharmacy claims for HU were selected from the Medicaid Analytic Extracts (MAX) from January 1, 2009 - December 31, 2013. The first HU prescription during the identification period was defined as the index date and patients were required to have had continuous medical and pharmacy benefits for ≥6 months baseline and 12 months follow-up periods. Patient demographics, clinical characteristics, treatment patterns, health care utilization, and costs were examined, and variables were analyzed descriptively.

RESULTS

A total of 3999 SCD patients prescribed HU were included; the mean age was 19.24 years, most patients were African American (73.3%), and the mean Charlson comorbidity index (CCI) score was 0.6. Asthma (20.3%), acute chest syndrome (15.6%), and infectious and parasitic diseases (20%) were the most prevalent comorbidities. During the 12-month follow-up period, 58.9% (N = 2357) of patients discontinued HU medication. The mean medication possession ratio (MPR) was 0.52, and 22.3% of patients had MPR ≥80%. The average length of stay (LOS) for SCD-related hospitalization was 13.35 days; 64% of patients had ≥1 SCD-related hospitalization. The mean annual total SCD-related costs per patient were $27,779, mostly inpatient costs ($20,128).

CONCLUSIONS

Overall, the study showed the patients had significant unmet needs manifest as poor medication adherence, high treatment discontinuation rates, and high economic burden.

摘要

背景

本研究旨在通过索赔数据评估美国接受羟基脲(HU)治疗的镰状细胞病(SCD)患者的治疗模式和经济负担。

方法

从 2009 年 1 月 1 日至 2013 年 12 月 31 日的 Medicaid Analytic Extracts(MAX)中选择有 HU 药房索赔的 SCD 患者。鉴定期内的首次 HU 处方被定义为索引日期,患者需要在基线和 12 个月随访期间有连续的医疗和药房福利≥6 个月。检查了患者的人口统计学、临床特征、治疗模式、医疗保健利用情况和成本,并对变量进行了描述性分析。

结果

共纳入 3999 例接受 HU 治疗的 SCD 患者;平均年龄为 19.24 岁,大多数患者为非裔美国人(73.3%),平均 Charlson 合并症指数(CCI)评分为 0.6。哮喘(20.3%)、急性胸部综合征(15.6%)和传染性和寄生虫病(20%)是最常见的合并症。在 12 个月的随访期间,58.9%(N=2357)的患者停止使用 HU 药物。平均用药比例(MPR)为 0.52,22.3%的患者 MPR≥80%。SCD 相关住院的平均住院时间(LOS)为 13.35 天;64%的患者有≥1 次 SCD 相关住院。每位患者的 SCD 相关年度总费用为 27779 美元,主要为住院费用(20128 美元)。

结论

总体而言,研究表明患者存在显著的未满足需求,表现为药物依从性差、治疗中断率高和经济负担高。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/46ea/6794855/8d048d93693f/12955_2019_1225_Fig1_HTML.jpg

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