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慢性髓性白血病:第二部分——从商业角度看美国慢性期晚期或后续治疗线患者的护理成本

Chronic Myeloid Leukemia: Part II-Cost of Care Among Patients in Advanced Phases or Later Lines of Therapy in Chronic Phase in the United States from a Commercial Perspective.

作者信息

Atallah Ehab L, Maegawa Rodrigo, Latremouille-Viau Dominick, Rossi Carmine, Guérin Annie

机构信息

Medical College of Wisconsin, Milwaukee.

Novartis Pharmaceuticals Corporation, East Hanover, New Jersey.

出版信息

J Health Econ Outcomes Res. 2022 Aug 4;9(2):30-36. doi: 10.36469/001c.36976. eCollection 2022.

Abstract

Tyrosine kinase inhibitors (TKIs) are the standard-of-care treatment for chronic myeloid leukemia in chronic phase (CML-CP). Despite advances in therapy, there remains a proportion of patients with CML-CP that are refractory/intolerant to TKIs, and these patients cycle through multiple lines of therapy. Moreover, even with TKIs, some patients progress to accelerated phase/blast crisis (AP/BC), which is associated with particularly poor clinical outcomes. To describe real-world treatment patterns, healthcare resource utilization (HRU), and costs of patients with CML-CP reaching later lines of therapy or progressing to AP/BC in the United States. Adult CML patients from administrative claims data (January 1, 2000-June 30, 2019) were classified by health state: on third-line (CML-CP On Treatment), on fourth or later lines (CML-CP Post-Discontinuation), or progressed to AP/BC (CML-AP/BC). Outcomes were assessed by health state. There were 296 (4620 patient-months), 83 (1644 patient-months), and 949 (25 593 patient-months) patients classified in the CML-CP On Treatment, CML-CP Post-Discontinuation, and CML-AP/BC cohorts, respectively. Second-generation TKIs (nilotinib, dasatinib, and bosutinib) were most commonly used in the CML-CP On Treatment (69.1% of patient-months) and CML-CP Post-Discontinuation cohorts (59.1% of patient-months). Three-month outpatient incidence rates (IRs) were 7.6, 8.3, and 7.0 visits in the CML-CP On Treatment, CML-CP Post-Discontinuation, and CML-AP/BC cohort, respectively, with mean costs of $597 per service. Three-month inpatient IRs were 0.6, 0.7, and 1.4 days in the CML-CP On Treatment, CML-CP Post-Discontinuation, and CML-AP/BC cohort, respectively, with mean costs of $5892 per day. Mean hematopoietic stem cell transplantation cost was $352 333; mean 3-month terminal care cost was $107 013. Cost of CML care is substantial among patients with CML reaching third-line, fourth or later lines, or progressing to AP/BC, suggesting that the disease is associated with a significant economic and clinical burden. From third-line to fourth or later lines, HRU was observed to increase, and the incidence of inpatient days was particularly high for those who progressed to AP/BC. In this study, patients with CML cycling through TKIs in later lines of therapy or progressing to AP/BC experienced substantial HRU and costs, suggesting unmet treatment needs.

摘要

酪氨酸激酶抑制剂(TKIs)是慢性期慢性髓性白血病(CML-CP)的标准治疗方法。尽管治疗取得了进展,但仍有一部分CML-CP患者对TKIs难治/不耐受,这些患者需接受多线治疗。此外,即使使用TKIs,一些患者仍会进展为加速期/急变期(AP/BC),这与特别差的临床结局相关。为描述美国CML-CP患者接受后续治疗或进展为AP/BC后的真实世界治疗模式、医疗资源利用(HRU)及成本。根据行政索赔数据(2000年1月1日至2019年6月30日)将成年CML患者按健康状态分类:接受三线治疗(CML-CP治疗中)、接受四线或更后续治疗(CML-CP停药后)或进展为AP/BC(CML-AP/BC)。按健康状态评估结局。分别有296例(4620患者月)、83例(1644患者月)和949例(25593患者月)患者被分类到CML-CP治疗中、CML-CP停药后和CML-AP/BC队列。第二代TKIs(尼罗替尼、达沙替尼和博舒替尼)在CML-CP治疗中(占患者月的69.1%)和CML-CP停药后队列(占患者月的59.1%)中使用最为普遍。CML-CP治疗中、CML-CP停药后和CML-AP/BC队列的三个月门诊发病率(IRs)分别为7.6次、8.3次和7.0次就诊,每次服务平均成本为597美元。CML-CP治疗中、CML-CP停药后和CML-AP/BC队列的三个月住院IRs分别为0.6天、0.7天和1.4天,每天平均成本为5892美元。造血干细胞移植平均成本为352333美元;三个月终末期护理平均成本为107013美元。在达到三线、四线或更后续治疗或进展为AP/BC的CML患者中,CML治疗成本很高,这表明该疾病与巨大的经济和临床负担相关。从三线治疗到四线或更后续治疗,观察到HRU增加对于进展为AP/BC的患者,住院天数的发生率特别高。在本研究中,接受后续治疗或进展为AP/BC的CML患者经历了大量的HRU和成本,表明存在未满足的治疗需求。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/54a5/9353133/4e0d0b5d13c8/jheor_2022_9_2_36976_95598.jpg

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