Analysis of the cost-effectiveness of treatment strategies for CML with incorporation of treatment discontinuation

Yamamoto, Chiyo; Nakashima, Hirotomo; Ikeda, Takashi; Kawaguchi, Shinichiro; Toda, Yumiko; Ito, Shoko; Mashima, Kiyomi; Nagayama, Takashi; Umino, Kento; Minakata, Daisuke; Nakano, Hirofumi; Morita, Kaoru; Yamasaki, Ryo; Sugimoto, Miyuki; Ishihara, Yuko; Ashizawa, Masahiro; Hatano, Kaoru; Sato, Kazuya; Oh, Iekuni; Fujiwara, Shin-ichiro; Ueda, Masuzu; Ohmine, Ken; Muroi, Kazuo; Kanda, Yoshinobu

doi:10.1182/bloodadvances.2019000745

Cited by 31 publications

(30 citation statements)

References 32 publications

(56 reference statements)

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“…The potential for a prolonged TFR and discontinuation of all treatment are also involved in these issues. Several studies have analyzed cost effectiveness of frontline treatments in various scenarios including strategies to increase the rate of sustained DMR to potentially increase the rate of TFR [66][67][68][69]. The conclusion is that generic imatinib is the costeffective initial treatment strategy for CML-CP.…”

Section: Tki Costs and Cost Effectivenessmentioning

confidence: 99%

European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia

et al. 2020

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The therapeutic landscape of chronic myeloid leukemia (CML) has profoundly changed over the past 7 years. Most patients with chronic phase (CP) now have a normal life expectancy. Another goal is achieving a stable deep molecular response (DMR) and discontinuing medication for treatment-free remission (TFR). The European LeukemiaNet convened an expert panel to critically evaluate and update the evidence to achieve these goals since its previous recommendations. First-line treatment is a tyrosine kinase inhibitor (TKI; imatinib brand or generic, dasatinib, nilotinib, and bosutinib are available firstline). Generic imatinib is the cost-effective initial treatment in CP. Various contraindications and side-effects of all TKIs should be considered. Patient risk status at diagnosis should be assessed with the new EUTOS long-term survival (ELTS)score. Monitoring of response should be done by quantitative polymerase chain reaction whenever possible. A change of treatment is recommended when intolerance cannot be ameliorated or when molecular milestones are not reached. Greater than 10% BCR-ABL1 at 3 months indicates treatment failure when confirmed. Allogeneic transplantation continues to be a therapeutic option particularly for advanced phase CML. TKI treatment should be withheld during pregnancy. Treatment discontinuation may be considered in patients with durable DMR with the goal of achieving TFR.

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Section: Tki Costs and Cost Effectivenessmentioning

confidence: 99%

European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia

et al. 2020

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“…Cost-effectiveness analyses in the US and Japan simulating the clinical course of 10 years of CML treatment, mapped cost estimate probabilities starting with imatinib, dasatinib, nilotinib, or any TKI according to the physician’s choice. This model demonstrated a value advantage for imatinib-first sequential treatment strategies over the initial use of second-generation agents even after factoring in drug discontinuation at the 2-year DMR target ( 29 ). While the cost of imatinib has varied over the years, data suggests that generic imatinib could be purchased at a daily cost as low as $20 in 2018.…”

Section: Counterpoint: the Case Against Using Second Generation Tkis As Initial Therapy For Cp CMLmentioning

confidence: 97%

Debating Frontline Therapy in Chronic Myeloid Leukemia

Ramanathan

Keiffer

2021

Front. Oncol.

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“…The high cost of TKIs has become a major limitation in terms of patient and national medical economics. Hence, recent reports on various CML-CP treatment models have analyzed the treatment value of 2GTKIs and generic drugs such as imatinib from the perspective of medical expense [49][50][51]. Shih et al performed a decision analysis to compare the cost effectiveness of generic imatinib and 2GTKI as the first-line treatment for CML-CP [49].…”

Section: Medical Expensesmentioning

confidence: 99%

Which Tyrosine Kinase Inhibitors Should Be Selected as the First-Line Treatment for Chronic Myelogenous Leukemia in Chronic Phase?

Ono

2021

Cancers

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With the use of tyrosine kinase inhibitors (TKIs), chronic myelogenous leukemia in chronic phase (CML-CP) has been transformed into a non-fatal chronic disease. Hence, "treatment-free remission (TFR)" has become a possible treatment goal of patients with CML-CP. Currently, four types of TKIs (imatinib, nilotinib, dasatinib, and bosutinib) are used as the first-line treatment for newly diagnosed CML-CP. However, the second-generation TKI (2GTKI), the treatment response of which is faster and deeper than that of imatinib, is not always recommended as the first-line treatment for CML-CP. Factors involved in TKI selection in the first-line treatment of CML-CP include not only patients’ medical background, but also patients’ choice regarding the desired treatment goal (survival or TFR?). Therefore, it is important that clinicians select an appropriate TKI to successfully achieve the desired treatment goal for each patient, while minimizing the development of adverse events. This review compares the pros and cons of using imatinib and 2GTKI for TKI selection as the first-line treatment for CML-CP, mainly considering treatment outcomes, medical history (i.e., desire for pregnancy, aging factor, and comorbidity), and cost. The optimal use of 2GTKIs is also discussed.

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Analysis of the cost-effectiveness of treatment strategies for CML with incorporation of treatment discontinuation

Abstract: Key Points The cost of TKI for treatment of CML can be substantially saved by treatment discontinuation in patients who achieved DMR. Starting treatment with imatinib is the most cost-effective strategy even after incorporation of treatment discontinuation.

Cited by 31 publications

References 32 publications

European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia

European LeukemiaNet 2020 recommendations for treating chronic myeloid leukemia

Debating Frontline Therapy in Chronic Myeloid Leukemia

Which Tyrosine Kinase Inhibitors Should Be Selected as the First-Line Treatment for Chronic Myelogenous Leukemia in Chronic Phase?

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