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Cost-effectiveness analysis for midostaurin versus standard of care in acute myeloid leukemia in the United Kingdom

机译:联合王国急性髓性白血病中豚鼠与护理标准的成本效益分析

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Midostaurin (MIDO) has been proposed for the treatment of newly-diagnosed adult patients with FMS-like tyrosine kinase 3 mutation-positive (FLT3+) acute myeloid leukemia (AML) in combination with standard chemotherapy. The cost-effectiveness of MIDO and standard of care (SOC) followed by MIDO monotherapy was compared to SOC alone for newly-diagnosed FLT3+?AML in the UK. A partitioned survival model was developed from a UK public healthcare system perspective to compare the cost-effectiveness of MIDO plus SOC and SOC over a lifetime horizon. The model included the following health states/partitions: induction, consolidation, monotherapy, complete remission (CR), relapse, stem cell transplantation (SCT), SCT recovery, and post-SCT recovery. Data on CR, overall survival, and adverse events were obtained from a Phase III clinical trial. Overall?survival was extrapolated beyond the trial horizon using a 'cure model' approach and data from the Office for National Statistics. Utilities were identified via a systematic review. Routine care utilization was obtained from the National Institute for Health and Care Excellence single technology appraisal for azacitidine in AML (TA399). The costs of drugs and administration, adverse events, hospitalizations, physician visits, and end-of-life care were incorporated. Incremental life years (LYs) and quality-adjusted life years (QALYs) gained by patients on MIDO and SOC versus SOC were 1.67 and 1.47, respectively. At an incremental cost of £54,072 over a lifetime horizon, the ICER was £32,465 per LY and £36,826 per QALY. Sensitivity analyses were generally consistent with the base case findings. With limited treatments in FLT3+?AML, MIDO represents a clinically significant advance in the management of newly-diagnosed AML. Using a threshold of £50,000 per QALY for end-of-life treatment, MIDO was shown to be a cost-effective option for newly-diagnosed FLT3+?AML.
机译:已提出中豚(MIDO)用于治疗新诊断的成年患者的FMS样酪氨酸激酶3突变阳性(FLT3 +)急性髓性白血病(AML)与标准化疗组合。将MIDO单疗法的MIDO和护理标准(SOC)的成本效益与SOC单独进行,用于在英国新诊断的FLT3 +?AML。从英国公共医疗保健系统的角度开发了一个分区生存模型,以比较MIDO Plus SoC和SoC的成本效益在一生的范围内。该模型包括以下健康状态/分区:归纳,固结,单药治疗,完整缓解(Cr),复发,干细胞移植(SCT),SCT恢复和后SCT恢复。来自CR,总体存活和不良事件的数据是从III期临床试验中获得的。总体而言?使用来自国家统计办公室的“治疗模型”方法和数据,外汇超越试验视图。通过系统审查确定公用事业。常规护理利用是从AML(TA399)中氮杂氨基的国家健康和护理卓越单一技术评估研究所获得。纳入了药物和行政,不良事件,住院,医生访问以及终生护理的成本。患者在MIDO和SOC与SOC上获得的增量寿命年(LYS)和质量调整的生命年(QALYS)分别为1.67和1.47。在终身视线上以54,072英镑的增量成本,ICER为每LY为32,465英镑,每QALY 36,826英镑。敏感性分析通常与基础案例结果一致。在FLT3 +的有限治疗中,MIDO代表了新诊断的AML管理中的临床上显着进展。使用每QALY 50,000英镑的阈值进行寿命终身治疗,MIDO被证明是新诊断的FLT3 + aml的成本效益。

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