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美国多形性胶质母细胞瘤治疗患者的成本和护理模式的真实世界索赔分析。

A Real-World Claims Analysis of Costs and Patterns of Care in Treated Patients with Glioblastoma Multiforme in the United States.

机构信息

1 Dana-Farber/Brigham and Women's Cancer Center, Boston, Massachusetts.

2 Bristol-Myers Squibb, Princeton, New Jersey.

出版信息

J Manag Care Spec Pharm. 2019 Apr;25(4):428-436. doi: 10.18553/jmcp.2019.25.4.428.

Abstract

BACKGROUND

Patients with glioblastoma multiforme (GBM) have a poor prognosis and high likelihood of recurrence. Routine care for incident cases in the United States involves surgical resection, followed by radiation therapy (RT) with concurrent and adjuvant temozolomide. Real-world data reporting the treatments and health care burden associated with GBM are limited.

OBJECTIVE

To assess patterns of care, health care resource utilization (HCRU), and costs associated with treatment of GBM in the United States.

METHODS

This study is a retrospective claims database analysis. Adult patients with a GBM diagnosis (index date) between January 1, 2010, and June 30, 2016, who had undergone brain surgery within 90 days of the index date, had received temozolomide and/or RT up to 90 days after index date, and had at least 6 months of continuous enrollment before the index date, were identified. Patients were excluded if they had (a) another primary cancer within 6 months pre-index, (b) secondary brain metastases, or (c) received temozolomide and/or RT pre-index. Baseline characteristics, treatments, HCRU, and costs were reported. First-line therapy began upon first receipt of RT and/or temozolomide after index date; second-line therapy began when a new drug was added > 28 days after initiation of first-line therapy or when there was a treatment gap > 90 days. Treatment regimens, duration of treatment (corrected group prognosis method), HCRU, and costs were reported descriptively in the 0- to 6-month and 7- to 12-month periods following initiation of first-line and second-line therapy.

RESULTS

Baseline characteristics were comparable between patients receiving temozolomide and/or RT. Patients receiving RT without chemotherapy tended to be older, be retired, and have more baseline comorbidities. Of the 4,071 patients receiving first-line therapy for GBM, most (73.0%) received temozolomide + RT; 24.4% received RT; and 2.5% received temozolomide monotherapy. Of those receiving first-line therapy, 1,283 (31.5%) patients subsequently received second-line therapy: 39.4% received bevacizumab monotherapy; 28.9% received bevacizumab combination therapy (temozolomide, 45.2% of patients; irinotecan, 24.3%; and temozolomide + lomustine, 15.4%); 15.5% received temozolomide monotherapy; and 13.7% received other systemic cancer therapies. The proportion of patients with hospitalizations increased from 2.9% (4-6 months pre-index) to 20.8% in the 3 months before the index date (likely due to diagnostic procedures) and 28.1% in the first 6 months after index (likely due to surgery) and then decreased to 13.3% in the 7- to 12-month period after index. Mean total per-patient costs at 6 and 12 months were $117,325 and $162,550 (first line) and $126,128 and $243,833 (second line). Costs in all time periods were largely driven by costs of RT/systemic cancer therapy.

CONCLUSIONS

Most patients with newly diagnosed GBM received treatment according to recommendations. However, relatively few patients received second-line therapy, and the HCRU burden and costs associated with both lines of therapy were substantial. Novel therapies for GBM are required to improve treatment options and outcomes in these patients.

DISCLOSURES

This study was funded by Bristol-Myers Squibb (Princeton Pike, NJ). Neither honoraria nor payments were provided for authorship. Norden received consultancy fees relating to this study from Bristol-Myers Squibb. Dastani, Korytowsky, Le, Singh, and You are employees of Bristol-Myers Squibb. Dastani and Korytowsky are shareholders of Bristol-Myers Squibb. Bobiak was an employee of Bristol-Myers Squibb at the time of this study. Preliminary data from this study were previously presented at the International Society for Pharmacoeconomics and Outcomes Research 22nd Annual International Meeting in Boston, MA, May 20-24, 2017.

摘要

背景

胶质母细胞瘤(GBM)患者预后较差,复发可能性高。美国对新发病例的常规治疗包括手术切除,随后进行放疗(RT)联合和/或辅助替莫唑胺治疗。目前,有关 GBM 治疗和相关医疗保健负担的真实世界数据十分有限。

目的

评估美国 GBM 患者的治疗模式、医疗保健资源利用(HCRU)和治疗相关成本。

方法

本研究是一项回顾性的理赔数据库分析。纳入 2010 年 1 月 1 日至 2016 年 6 月 30 日期间诊断为 GBM 的成年患者,其在索引日期前 90 天内接受过脑部手术,在索引日期后 90 天内接受过替莫唑胺和/或 RT 治疗,并且在索引日期前至少有 6 个月的连续参保记录。排除标准为:(a)索引日期前 6 个月内有其他原发性癌症;(b)继发脑转移;(c)在索引日期前接受过替莫唑胺和/或 RT 治疗。报告了基线特征、治疗、HCRU 和成本。一线治疗从首次接受 RT 和/或替莫唑胺治疗后开始;二线治疗从首次治疗开始后 28 天以上添加新药或治疗间隔超过 90 天后开始。描述性报告了一线和二线治疗开始后 0-6 个月和 7-12 个月的治疗方案、治疗持续时间(校正后的群体预后方法)、HCRU 和成本。

结果

接受替莫唑胺和/或 RT 治疗的患者的基线特征无显著差异。未接受化疗的 RT 患者年龄较大,更可能已退休且有更多基线合并症。在接受 GBM 一线治疗的 4071 例患者中,大多数(73.0%)接受替莫唑胺+RT 治疗;24.4%接受 RT 治疗;2.5%接受替莫唑胺单药治疗。在接受一线治疗的患者中,1283 例(31.5%)随后接受二线治疗:39.4%接受贝伐珠单抗单药治疗;28.9%接受贝伐珠单抗联合治疗(替莫唑胺,45.2%的患者;伊立替康,24.3%;替莫唑胺+洛莫司汀,15.4%);15.5%接受替莫唑胺单药治疗;13.7%接受其他全身癌症治疗。住院比例从索引日期前 4-6 个月的 2.9%增加到索引日期前 3 个月的 20.8%(可能由于诊断程序)和索引日期后 6 个月的 28.1%(可能由于手术),然后在索引日期后 7-12 个月降至 13.3%。6 个月和 12 个月的平均每位患者总费用分别为 117325 美元和 162550 美元(一线)和 126128 美元和 243833 美元(二线)。所有时间段的费用主要由 RT/全身癌症治疗费用驱动。

结论

大多数新诊断的 GBM 患者按照建议接受了治疗。然而,只有少数患者接受了二线治疗,并且两线治疗的 HCRU 负担和相关成本都很高。需要新的 GBM 治疗方法来改善这些患者的治疗选择和结局。

披露

本研究由百时美施贵宝公司(新泽西州普林斯顿派克)资助。作者没有因撰写文章而获得报酬。Norden 因这项研究从百时美施贵宝公司获得咨询费。Dastani、Korytowsky、Le、Singh 和 You 是百时美施贵宝公司的员工。Dastani 和 Korytowsky 是百时美施贵宝公司的股东。Bobiak 在进行这项研究时是百时美施贵宝公司的员工。这项研究的初步数据先前在 2017 年 5 月 20 日至 24 日在马萨诸塞州波士顿举行的第 22 届国际药物经济学与结果研究学会年度国际会议上进行了介绍。

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