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南非慢性粒细胞白血病酪氨酸激酶抑制剂治疗策略的成本效益

Cost-effectiveness of tyrosine kinase inhibitor treatment strategies for chronic myeloid leukemia in South Africa.

作者信息

Woudberg Rochelle, Sinanovic Edina

机构信息

Health Economics Unit, School of Public Health, University of Cape Town, Cape Town, South Africa.

出版信息

Front Pharmacol. 2025 Jan 7;15:1511603. doi: 10.3389/fphar.2024.1511603. eCollection 2024.

Abstract

BACKGROUND

The treatment of chronic myeloid leukemia through tyrosine kinase inhibitors (TKIs) has achieved promising efficacy and safety outcomes, however the costs are associated with a substantial economic burden. The objective of this study was to develop a Markov model with a 20-year time horizon to assess the cost effectiveness of TKIs from a public healthcare system perspective in South Africa.

METHODS

We constructed a Markov model to compare three strategies in which treatment was initiated with either imatinib, nilotinib, or dasatinib. Treatment was switched to another TKI in the case of intolerance or resistance to the initial TKI. Effectiveness and utility data were obtained from published literature. Cost data was obtained from local sources for generic imatinib and branded second-generation TKIs and based on national tariffs. Outcomes were reported in total costs and quality-adjusted life years (QALYs). Outcomes were based on calculated incremental cost effectiveness ratios (ICERs) and compared to a willingness-to-pay (WTP) threshold. Sensitivity analyses were conducted to determine the robustness of the model outcomes.

RESULTS

The base-case results showed that imatinib was favored over nilotinib and dasatinib by having the lowest cost at $120 719.55 and providing 5.93 QALYs. Compared to imatinib strategy, nilotinib had an ICER of $26 620.27 per QALY and dasatinib had an ICER of $35 934.94 per QALY, both exceeding the WTP threshold of $18 760 per QALY gained. The sensitivity analysis indicated the robustness of the results.

CONCLUSION

Imatinib remains the most cost-effective first-line treatment for adults diagnosed with CML in South Africa, with a high probability of being cost-effective across a range of WTP thresholds. Nilotinib and Dasatinib, though offering clinical benefits, their affordability remains a challenge within the current healthcare system and should remain reserved for second-line treatment.

摘要

背景

通过酪氨酸激酶抑制剂(TKIs)治疗慢性髓性白血病已取得了有前景的疗效和安全性结果,然而其成本带来了巨大的经济负担。本研究的目的是建立一个为期20年的马尔可夫模型,从南非公共医疗系统的角度评估TKIs的成本效益。

方法

我们构建了一个马尔可夫模型,以比较三种治疗策略,即分别起始使用伊马替尼、尼洛替尼或达沙替尼进行治疗。若对初始TKI不耐受或耐药,则更换为另一种TKI。有效性和效用数据来自已发表的文献。成本数据来自当地通用伊马替尼和第二代品牌TKI的来源,并基于国家关税。结果以总成本和质量调整生命年(QALYs)报告。结果基于计算的增量成本效益比(ICERs),并与支付意愿(WTP)阈值进行比较。进行敏感性分析以确定模型结果的稳健性。

结果

基础病例结果显示,伊马替尼以120719.55美元的最低成本和5.93个QALYs优于尼洛替尼和达沙替尼。与伊马替尼策略相比,尼洛替尼的ICER为每QALY 26620.27美元,达沙替尼的ICER为每QALY 35934.94美元,两者均超过每获得一个QALY 18760美元的WTP阈值。敏感性分析表明结果具有稳健性。

结论

在南非,伊马替尼仍然是诊断为慢性髓性白血病的成人最具成本效益的一线治疗药物,在一系列WTP阈值范围内具有很高的成本效益可能性。尼洛替尼和达沙替尼虽然提供临床益处,但在当前医疗系统中其可负担性仍然是一个挑战,应保留用于二线治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f0b4/11746066/cb56146edd56/fphar-15-1511603-g001.jpg

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