OBJECTIVES : To evaluate the cost-effectiveness of gemtuzumab ozogamicin in combination with daunorubicin and cytarabine versus daunorubicin and cytarabine alone for the treatment of newly diagnosed acute myeloid leukaemia (AML) in Portugal.

METHODS : The cost-effectiveness analysis of gemtuzumab ozogamicin was based on a cohort state‑transition model, populated with ALFA-0701 trial data, designed to capture relapse free survival and overall survival according to induction therapy success. Age-specific mortality rates of Portuguese general population were considered for functionally cured patients. Utilities per health state and disutilities due to adverse events were based on the literature and on the general population for those functionally cured. Portuguese-specific disease management resource use was based on an expert panel and on Portuguese diagnosis-related group microdata. National legislation and official drug cost databases were the main sources for unit costs. The analysis was conducted from a payers’ perspective, assuming a lifetime horizon and a 5% annual discount rate for both costs and effects. Deterministic and probabilistic sensitivity analyses were conducted to assess the robustness of results.

RESULTS : Gemtuzumab ozogamicin enables a discounted gain of 0.97 life-years (LY) or 0.72 quality adjusted life years (QALY). Economic analysis shows that the higher costs of the gemtuzumab ozogamicin option are mainly due to higher treatment costs. The estimated incremental cost-effectiveness ratios are 23,916€/LY and 32,244€/QALY. Deterministic sensitivity analyses show that results are robust to most scenarios but slightly sensitive to utilities per health state.

CONCLUSIONS : Gemtuzumab ozogamicin in combination with daunorubicin and cytarabine for the treatment of AML versus daunorubicin and cytarabine alone increased LY and QALY at an incremental cost that allows to assess it as a cost-effective option when taking into consideration usually accepted thresholds in Portugal. Sensitivity analyses demonstrated the robustness of the results.