RT Journal Article
T1 Real-world analysis of main clinical outcomes in patients with polycythemia vera treated with ruxolitinib or best available therapy after developing resistance/intolerance to hydroxyurea.
A1 Alvarez-Larrán, Alberto
A1 Garrote, Marta
A1 Ferrer-Marín, Francisca
A1 Pérez-Encinas, Manuel
A1 Mata-Vazquez, M Isabel
A1 Bellosillo, Beatriz
A1 Arellano-Rodrigo, Eduardo
A1 Gómez, Montse
A1 García, Regina
A1 García-Gutiérrez, Valentín
A1 Gasior, Mercedes
A1 Cuevas, Beatriz
A1 Angona, Anna
A1 Gómez-Casares, María Teresa
A1 Martínez, Clara M
A1 Magro, Elena
A1 Ayala, Rosa
A1 Del Orbe-Barreto, Rafael
A1 Pérez-López, Raúl
A1 Fox, Maria Laura
A1 Raya, José-María
A1 Guerrero, Lucía
A1 García-Hernández, Carmen
A1 Caballero, Gonzalo
A1 Murillo, Ilda
A1 Xicoy, Blanca
A1 Ramírez, M José
A1 Carreño-Tarragona, Gonzalo
A1 Hernández-Boluda, Juan Carlos
A1 Pereira, Arturo
A1 MPN Spanish Group (Grupo Español de Enfermedades Mieloproliferativas Filadelfia Negativas), 
K1 hemorrhage
K1 myelofibrosis
K1 myeloproliferative neoplasms
K1 polycythemia vera
K1 ruxolitinib
K1 therapy
K1 thrombosis
AB Ruxolitinib is approved for patients with polycythemia vera (PV) who are resistant/intolerant to hydroxyurea, but its impact on preventing thrombosis or disease-progression is unknown. A retrospective, real-world analysis was performed on the outcomes of 377 patients with resistance/intolerance to hydroxyurea from the Spanish Registry of Polycythemia Vera according to subsequent treatment with ruxolitinib (n = 105) or the best available therapy (BAT; n = 272). Survival probabilities and rates of thrombosis, hemorrhage, acute myeloid leukemia, myelofibrosis, and second primary cancers were calculated according to treatment. To minimize biases in treatment allocation, all results were adjusted by a propensity score for receiving ruxolitinib or BAT. Patients receiving ruxolitinib had a significantly lower rate of arterial thrombosis than those on BAT (0.4% vs 2.3% per year; P = .03), and this persisted as a trend after adjustment for the propensity to have received the drug (incidence rate ratio, 0.18; 95% confidence interval, 0.02-1.3; P = .09). There were no significant differences in the rates of venous thrombosis (0.8% and 1.1% for ruxolitinib and BAT, respectively; P = .7) and major bleeding (0.8% and 0.9%, respectively; P = .9). Ruxolitinib exposure was not associated with a higher rate of second primary cancers, including all types of neoplasia, noncutaneous cancers, and nonmelanoma skin cancers. After a median follow-up of 3.5 years, there were no differences in survival or progression to acute leukemia or myelofibrosis between the 2 groups. The results suggest that ruxolitinib treatment for PV patients with resistance/intolerance to hydroxyurea may reduce the incidence of arterial thrombosis. Ruxolitinib is better than other available therapies in achieving hematocrit control and symptom relief in patients with polycythemia vera who are resistant/intolerant to hydroxyurea, but we still do not know whether ruxolitinib provides an additional benefit in preventing thrombosis or disease progression. We retrospectively studied the outcomes of 377 patients with resistance/intolerance to hydroxyurea from the Spanish Registry of Polycythemia Vera according to whether they subsequently received ruxolitinib (n = 105) or the best available therapy (n = 272). Our findings suggest that ruxolitinib could reduce the incidence of arterial thrombosis, but a disease-modifying effect could not be demonstrated for ruxolitinib in this patient population.
YR 2022
FD 2022-04-13
LK http://hdl.handle.net/10668/21618
UL http://hdl.handle.net/10668/21618
LA en
DS RISalud
RD Jul 4, 2025