OBJECTIVES: To evaluate the clinical effectiveness of ruxolitinib as compared to best available treatment (BAT) based on control of leucocytosis and thus prolongation of life-years, in polycythaemia (PV) patients, with hydroxy-urea (HU) resistance/intolerance.

METHODS: A Markov model demonstrating the progression of HU resistant/intolerant PV patients through health states defined by the presence (>15x10/L) or absence of leucocytosis, was adapted to the Turkish treatment and epidemiology setting. The model time horizon was 15 years. The cycle length was defined as three months. Clinical data, derived from Phase III RESPONSE trial, were reviewed by a local expert panel. The model relied on white blood cell control as a prognostic marker for overall survival (OS). The statistical relation between leucocytosis and OS was derived from the literature. Utility values were based on international literature. The patient population modelled is assumed to be 61 years old in average, 65.8% male, and mean duration of disease 9.24 years, HU resistant/intolerant ratio 35%/65% respectively, proportion of patients without leucocytosis at the beginning 43%. The patients are treated with ruxolitinib or BAT (HU 59%, interferon 11%, other medications 13% and phlebotomy only 17%).

RESULTS: Patients treated with ruxolitinib experienced additional life-years without leucocytosis compared with patients on BAT (6.47 vs. 4.39 years). OS for patients treated with ruxolitinib was 1.15 years longer than patients treated with BAT (8.21 vs. 7.06 years). When the duration of life-years was adjusted for quality of life, patients treated with ruxolitinib experienced an incremental QALY gain of 1.38 years compared to patients treated with BAT (6.67 vs. 5.29 QALYs).

CONCLUSIONS: Modelled survival outcomes demonstrate the improvements in quality-of-life and overall survival for HU intolerant/resistant patients who are treated with ruxolitinib compared to BAT. Therefore, ruxolitinib may be considered in PV patients who experience resistance or intolerance to HU, in Turkey.