RTI Health Solutions, Research Triangle Park, NC, USA.
AstraZeneca, Gaithersburg, MD, USA.
Cancer Med. 2021 Apr;10(8):2690-2702. doi: 10.1002/cam4.3855. Epub 2021 Mar 18.
Information on overall survival (OS) and adverse events (AEs) in patients with chronic lymphocytic leukemia (CLL) is mostly available from clinical trials. We therefore conducted a population-based retrospective cohort study to assess OS, incidence of AEs, and economic burden in real-world practice among Medicare patients treated for CLL.
Patients with CLL receiving ≥1 systemic therapy from 2013 to 2015 were selected from the Medicare claims database and followed from the start of first observed systemic therapy (index date) through December 2016 or death. OS for patients receiving each of the most commonly observed treatments was estimated by the Kaplan-Meier method. AEs were assessed among patients receiving these treatments across all observed lines of therapy. All-cause direct medical costs were assessed from the Medicare system perspective.
Among 7,965 eligible patients across all observed therapy lines, ibrutinib monotherapy (Ibr; n = 2,708), chlorambucil monotherapy (Clb; n = 1,620), and bendamustine/rituximab (BR; n = 1,485) were the most common treatments. For first observed therapy, 24-month OS estimates for Ibr, Clb, and BR recipients were 69% (95% CI = 68%-71%), 68% (95% CI = 65%-71%), and 79% (95% CI = 77%-81%) respectively. The most frequently recorded AEs in patients receiving these treatments in any observed line of therapy were neutropenia, hypertension, anemia, and infection. For all patients, the mean monthly all-cause cost during the follow-up period was $8,974 (SD = $11,562); cost increased by the number of AEs, from $5,144 (SD = $5,409) among those with 1-2 AEs to $10,077 (SD = $12,542) among those with ≥6 AEs.
Over two-thirds of patients survived at least 2 years after starting their first observed therapy for CLL. Our findings highlight considerable susceptibility to AEs and unmet medical need in Medicare patients with CLL treated in routine practice. Medicare incurred substantial economic burden following initiation of systemic therapy, and patients with greater numbers of AEs accounted disproportionately for the high overall cost of CLL management.
关于慢性淋巴细胞白血病（CLL）患者的总生存期（OS）和不良事件（AE）的信息主要来自临床试验。因此，我们进行了一项基于人群的回顾性队列研究，以评估医疗保险患者在真实世界实践中接受 CLL 治疗的 OS、AE 发生率和经济负担。
从医疗保险索赔数据库中选择了 2013 年至 2015 年期间接受了≥1 种全身治疗的 CLL 患者，并从首次观察到的全身治疗（索引日期）开始随访，直至 2016 年 12 月或死亡。Kaplan-Meier 法估计接受每种最常见治疗的患者的 OS。在所有观察到的治疗线中评估接受这些治疗的患者的 AE。从医疗保险系统的角度评估全因直接医疗费用。
在所有观察到的治疗线中，有 7965 名符合条件的患者，伊布替尼单药治疗（Ibr；n=2708）、苯丁酸氮芥单药治疗（Clb；n=1620）和苯达莫司汀/利妥昔单抗（BR；n=1485）是最常见的治疗方法。对于首次观察到的治疗，Ibr、Clb 和 BR 接受者的 24 个月 OS 估计值分别为 69%（95%CI=68%-71%）、68%（95%CI=65%-71%）和 79%（95%CI=77%-81%）。在任何观察到的治疗线中接受这些治疗的患者中最常记录的 AE 是中性粒细胞减少症、高血压、贫血和感染。对于所有患者，在随访期间的平均每月全因费用为 8974 美元（SD=5144 美元）；费用随着 AE 数量的增加而增加，从有 1-2 个 AE 的患者的 5409 美元（SD=5409 美元）增加到有≥6 个 AE 的患者的 12542 美元（SD=12542 美元）。
超过三分之二的患者在开始接受 CLL 的首次观察治疗后至少存活了 2 年。我们的研究结果表明，在常规实践中接受治疗的医疗保险 CLL 患者存在相当大的 AE 易感性和未满足的医疗需求。医疗保险在开始全身治疗后承担了巨大的经济负担，AE 数量较多的患者对 CLL 管理的总体高成本不成比例地做出了贡献。
