標靶治療於急性骨髓性白血病成本效益分析

急性骨髓性白血病(Acute myeloid leukemia, AML)在各種成人血癌裡是發生率最高的白血病，以癌症整體存活狀況來看，急性骨髓性白血病患者存活率低，其標準治療策略是7+3方案，七天阿糖胞苷(Cytarabine)加上三天蒽環類藥物(Anthracycline)。然而「7+3」方案的密集誘導化療有時會造成患者身體負荷過重，特別在年長或嚴重共病的患者。到目前已有多種臨床試驗採用新型的藥物來進行治療，自2017年至今已有十種藥物獲得美國Food and Drug Administration (FDA)，包括Venetoclax及針對FLT3抑制劑, IDH1/2抑制劑等標靶藥物或其他新穎治療藥物運用於臨床試驗中。大部分的標靶治療價格昂貴且未獲台灣FDA核准或不在台灣全民健康保險範圍內，因此本研究提供治療急性骨髓性白血病患者在透過各項分子檢測及標靶治療上的成本效益分析。基於決策分析建立模型，所建構之馬可夫模型 (Markov Model) 提供量性決策分析，並考慮決策過程中的多項不確定因素，評估相較於「7+3」方案治療，使用標靶治療或者利用危險分層對高風險族群之標靶治療結合中低風險族群之「7+3」方案之精準方案的成本效益分析。效益部分利用新治療和「7+3」方案治療患者所得的生活品質調整後生命年(Quality-adjusted life year, QALY)進行估計；成本部分包括基因檢測、治療及住院等等。本研究利用臺灣本土參數所建構的成本效益分析得到標靶治療與精準治療策略相較於「7+3」方案治療的增量成本效益比(Incremental cost-effectiveness ratio, ICER) ，亦即當每多增加一單位的生活品質調整生命年時將會分別需要9,696美元及11,334美元，低於2021年3倍的國民人均生產毛額98,361美元，機率性成本效益分析顯示當願付成本超過7500美元時精準策略為最具成本效益的抉擇，當願付成本超過7500美元時，標靶治療為最可能具成本效益之策略。以美國情境進行分析時，65歲男性標靶治療比上「7+3」方案治療的ICER為 65,628美元，金額小於付費意願(Willingness to pay, WTP)閥值98,361 美元，因此標靶治療對急性骨髓性白血病患者來說是一種具有成本效益的選擇。

關鍵字

效益分析；急性骨髓性白血病

並列摘要

Acute myeloid leukemia (AML) is the most common acute adult leukemia and poor survival in the patients new diagnosed with AML. The standard treatment for treating AML is ‘7+3’ regimen, over 4 decades already, which is 7 days of Cytarabine and 3 days of an Anthracycline. However, the intensive induction chemotherapy like ‘7+3’ is sometimes unbearable, especially in elderly patients. Many clinical trials have designed to use novel agents for an alternative treatment. Ten agents received approved by U.S. Food and Drug Administration (FDA) for various indications, including several targeted therapies such as Venetoclax, FLT3 inhibitors and IDH inhibitors, they are available in clinical trials for treating AML patients since 2017. However, in most of targeted therapies are expensive and still not in the coverage of National Health Insurance in Taiwan. This study aimed to provide a cost effectiveness analysis for genetic screening and targeted therapies for treating AML patients. Based on the decision-analytical modelling, our goal is to evaluate the cost effectiveness of using new treatment like targeted therapies with ‘7+3’ standard treatment through the modelling of Markov models for providing quantitative analysis and considering the uncertain factors during the decision making process. We used the empirical results from Taiwanese data for the parameters of Markov decision analysis. Compared with ‘7+3’ standard treatment, the incremental cost-effectiveness ratio (ICER) for personalized strategy and target therapy was $9,696 and $11,334, respectively, for one additional quality-adjusted life year (QALY) gained. Both ICERs were lower than three times of GDP per capita, 98,361. Analysis with the probabilistic approach shows the same results.

並列關鍵字

Cost-effectiveness analysis ； CEA ； AML

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