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芦可替尼停药后骨髓纤维化患者的特征和结局。

Patient characteristics and outcomes after ruxolitinib discontinuation in patients with myelofibrosis.

机构信息

Medicine, Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

Strategic Market Access, Janssen Global Services, LLC, Raritan, NJ, USA.

出版信息

J Med Econ. 2020 Jul;23(7):721-727. doi: 10.1080/13696998.2020.1741381. Epub 2020 Mar 31.

Abstract

This retrospective analysis evaluates morbidities, outcomes and associated risk factors in patients with myelofibrosis (MF) after ruxolitinib discontinuation, using Truven Health Analytics MarketScan (TR), Optum integrated virtual electronic health records and claims databases (OP), and Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database (SM). A total of 290 patients with MF between 2006 and 2015 (using ICD-9 and ICD-O-3 codes), who were treated with and discontinued ruxolitinib were identified. Only patients with ≥90 days of medical history prior to index diagnosis (TR + OP) and Part A, B, and D enrollment at the time of index diagnosis (SM) were included. Morbidities were assessed during the 30-day period each following ruxolitinib initiation, prior to and post ruxolitinib discontinuation. Cumulative incidence of cytopenias and efficacy outcomes were evaluated from baseline. Median age of patients was 68 years, with equal proportion of either gender. Median time to ruxolitinib discontinuation was 284 days and median follow-up after discontinuation was 70.9 days. The majority of patients were diagnosed with anemia and >30% of the patients received RBC transfusions during 30-day period prior to and the 30-day period post ruxolitinib discontinuation. After ruxolitinib discontinuation, half of the patients developed cytopenias. The median treatment progression-free survival, and overall survival after ruxolitinib discontinuation were 6.0 (4.4, 8.3) months and 11.1 (8.4, 14.5) months, respectively. Age at ruxolitinib discontinuation (HR [95% CI] = 2.071 [1.320, 3.248]), Charlson Comorbidity Index score (HR [95% CI] = 1.172 [1.093, 1.257]) and gender (HR [95% CI] = 1.620 [1.108, 2.369]) increased the risk of treatment progression (start of the subsequent treatment regimen) or death. Results from this large, retrospective, US population-based outcome analysis of MF patients show an increase in morbidity burden and identifies the risk factors of survival outcomes among real-world patients who have discontinued ruxolitinib.

摘要

这项回顾性分析使用 Truven Health Analytics MarketScan(TR)、Optum 综合虚拟电子健康记录和索赔数据库(OP)以及 Surveillance, Epidemiology, and End Results(SEER)-Medicare 链接数据库(SM),评估了接受鲁索替尼治疗后停药的骨髓纤维化(MF)患者的发病率、结局和相关风险因素。共纳入 2006 年至 2015 年间 290 名使用 ICD-9 和 ICD-O-3 代码诊断为 MF 的患者,这些患者接受了鲁索替尼治疗并停药。仅纳入在索引诊断前有≥90 天病史(TR+OP)且在索引诊断时同时参加了 A 部分、B 部分和 D 部分的患者(SM)。在鲁索替尼起始后的 30 天内、鲁索替尼停药前和停药后评估发病率。从基线评估细胞减少症的累积发生率和疗效结局。患者的中位年龄为 68 岁,性别比例相等。鲁索替尼停药的中位时间为 284 天,停药后中位随访时间为 70.9 天。大多数患者被诊断为贫血,在鲁索替尼停药前 30 天和停药后 30 天期间,超过 30%的患者接受了红细胞输注。鲁索替尼停药后,一半的患者出现细胞减少症。鲁索替尼停药后,中位无进展生存和总生存分别为 6.0(4.4,8.3)个月和 11.1(8.4,14.5)个月。鲁索替尼停药时的年龄(HR [95%CI] = 2.071 [1.320,3.248])、Charlson 合并症指数评分(HR [95%CI] = 1.172 [1.093,1.257])和性别(HR [95%CI] = 1.620 [1.108,2.369])增加了治疗进展(开始后续治疗方案)或死亡的风险。这项来自美国大型回顾性人群基础结局分析的结果表明,MF 患者的发病率负担增加,并确定了停止鲁索替尼治疗的真实世界患者的生存结局的风险因素。

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