RT Journal Article T1 Frequency and prognostic value of resistance/intolerance to hydroxycarbamide in 890 patients with polycythaemia vera. A1 Alvarez-Larrán, Alberto A1 Kerguelen, Ana A1 Hernández-Boluda, Juan C A1 Pérez-Encinas, Manuel A1 Ferrer-Marín, Francisca A1 Bárez, Abelardo A1 Martínez-López, Joaquín A1 Cuevas, Beatriz A1 Mata, M Isabel A1 García-Gutiérrez, Valentín A1 Aragües, Pilar A1 Montesdeoca, Sara A1 Burgaleta, Carmen A1 Caballero, Gonzalo A1 Hernández-Rivas, J Angel A1 Durán, M Antonia A1 Gómez-Casares, M Teresa A1 Besses, Carles A1 Grupo Español de Enfermedades Mieloproliferativas Filadelfia Negativas (GEMFIN), K1 hydroxycarbamide K1 myeloproliferative neoplasms K1 polycythaemia vera K1 resistance/intolerance K1 response criteria AB The clinical significance of resistance/intolerance to hydroxycarbamide (HC) was assessed in a series of 890 patients with polycythaemia vera (PV). Resistance/intolerance to HC was recorded in 137 patients (15·4%), consisting of: need for phlebotomies (3·3%), uncontrolled myeloproliferation (1·6%), failure to reduce massive splenomegaly (0·8%), development of cytopenia at the lowest dose of HC to achieve a response (1·7%) and extra-haematological toxicity (9%). With a median follow-up of 4·6 years, 99 patients died, resulting in a median survival of 19 years. Fulfilling any of the resistance/intolerance criteria had no impact on survival but when the different criteria were individually assessed, an increased risk of death was observed in patients developing cytopenia [Hazard ratio (HR): 3·5, 95% confidence interval (CI): 1·5-8·3, P = 0·003]. Resistance/intolerance had no impact in the rate of thrombosis or bleeding. Risk of myelofibrotic transformation was significantly higher in those patients developing cytopenia (HR: 5·1, 95% CI: 1·9-13·7, P = 0·001) and massive splenomegaly (HR: 9·1, 95% CI: 2·3-35·9, P = 0·002). Cytopenia at the lowest dose required to achieve a response was also an independent risk factor for transformation to acute leukaemia (HR: 20·3, 95% CI: 5·4-76·5, P YR 2015 FD 2015-12-21 LK https://hdl.handle.net/10668/26696 UL https://hdl.handle.net/10668/26696 LA en DS RISalud RD Apr 6, 2025