Center for Health Research, Kaiser Permanente Northwest, Portland, Oregon.
Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, Maryland.
J Natl Compr Canc Netw. 2019 Oct 1;17(10):1166-1172. doi: 10.6004/jnccn.2019.7303.
Oral tyrosine kinase inhibitors (TKIs) have been the standard of care for chronic myeloid leukemia (CML) since 2001. However, few studies have evaluated changes in the treatment landscape of CML over time. This study assessed the long-term treatment patterns of oral anticancer therapies among patients with CML.
This retrospective cohort study included patients newly diagnosed with CML between January 1, 2000, and December 31, 2016, from 10 integrated healthcare systems. The proportion of patients treated with 5 FDA-approved oral TKI agents-bosutinib, dasatinib, imatinib, nilotinib, and ponatinib-in the 12 months after diagnosis were measured, overall and by year, between 2000 and 2017. We assessed the use of each oral agent through the fourth-line setting. Multivariable logistic regression estimated the odds of receiving any oral agent, adjusting for sociodemographic and clinical characteristics.
Among 853 patients with CML, 81% received an oral agent between 2000 and 2017. Use of non-oral therapies decreased from 100% in 2000 to 5% in 2005, coinciding with imatinib uptake from 65% in 2001 to 98% in 2005. Approximately 28% of patients switched to a second-line agent, 9% switched to a third-line agent, and 2% switched to a fourth-line agent. Adjusted analysis showed that age at diagnosis, year of diagnosis, and comorbidity burden were statistically significantly associated with odds of receiving an oral agent.
A dramatic shift was seen in CML treatments away from traditional, nonoral chemotherapy toward use of novel oral TKIs between 2000 and 2017. As the costs of oral anticancer agents reach new highs, studies assessing the long-term health and financial outcomes among patients with CML are warranted.
自 2001 年以来,口服酪氨酸激酶抑制剂(TKI)一直是慢性髓性白血病(CML)的标准治疗方法。然而,很少有研究评估随着时间的推移 CML 治疗领域的变化。本研究评估了 CML 患者长期口服抗癌治疗的模式。
这项回顾性队列研究纳入了 2000 年 1 月 1 日至 2016 年 12 月 31 日期间来自 10 个综合医疗系统的新诊断为 CML 的患者。测量了诊断后 12 个月内接受 5 种美国食品药品监督管理局批准的口服 TKI 药物(博舒替尼、达沙替尼、伊马替尼、尼洛替尼和泊那替尼)的患者比例,分别按年份和总体进行测量,时间范围为 2000 年至 2017 年。我们通过第四线治疗评估了每种口服药物的使用情况。多变量逻辑回归调整了社会人口统计学和临床特征,估计了接受任何口服药物的可能性。
在 853 名 CML 患者中,81%在 2000 年至 2017 年期间接受了口服药物治疗。非口服治疗的使用率从 2000 年的 100%下降到 2005 年的 5%,这与 2001 年伊马替尼使用率从 65%上升到 2005 年的 98%相一致。大约 28%的患者转为二线药物治疗,9%转为三线药物治疗,2%转为四线药物治疗。调整分析表明,诊断时的年龄、诊断年份和合并症负担与接受口服药物治疗的可能性具有统计学显著相关性。
2000 年至 2017 年间,CML 治疗发生了从传统非口服化疗向新型口服 TKI 的显著转变。随着口服抗癌药物的成本达到新的高点,有必要对 CML 患者的长期健康和经济结果进行研究。