Abstract

Chronic myeloid leukaemia (CML) is a clonal myeloproliferative disorder of pluripotent stem cells, first described by John Hughes Bennett in 1845. Previously, the treatment options, such as hydroxicarbamide, interferon, busulfan and radiotherapy, only controlled the disease while the novel therapeutic options increased significantly the rate of remissions. Untreated patients in chronic phase (CML-CP) inevitably progressed to CML-BC (blast crisis), an aggressive form of acute leukaemia. Treatment with imatinib mesylate was associated with high response rate and an improved overall survival rate, especially when used in chronic phase. Options for patients resistant to imatinib include either dose increase or use of dasatinib or nilotinib. The objective of the current study was to analyse the impact of the severity and frequency of the adverse drug reactions on treatment results and to determine the treatment costs with imatinib, dasatinib, and nilotinib. An epidemiology and safety model based on information about the registered patients with CML in Bulgaria was established. Treatment costs for each therapy option and the treatment costs of adverse drug reactions (ADR) were determined for 6 months, and 1 year time horizons, from the payer’s perspective. The results confirmed that the frequency and severity of ADRs influenced the total pharmacotherapy costs. Nilotinib is the preferred therapeutic and economic alternative.

Key words: Chronic myeloid leukaemia, adverse drug reactions, nilotinib, dasatinib, imatinib.