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
Álvarez Larrán, Alberto (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
Garrote, Marta (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
Ferrer-Marín, Francisca (Universidad Católica San Antonio de Murcia)
Pérez-Encinas, Manuel (Hospital Clínico Universitario)
Mata-Vazquez, M.Isabel (Hospital Costa del Sol (Marbella))
Bellosillo Paricio, Beatriz (Hospital del Mar (Barcelona, Catalunya))
Arellano-Rodrigo, Eduardo (Institut d'Investigacions Biomèdiques August Pi i Sunyer)
Gómez, Montse (Hospital Clínico)
García, Regina (Hospital Universitario Virgen de la Victoria (Màlaga, Andalusia))
Garcia-Gutierrez, Valentin (Instituto Ramón y Cajal de Investigación Sanitaria (Madrid))
Gasior, Mercedes (Hospital Universitario La Paz (Madrid))
Cuevas, Beatriz (Hospital Universitario de Burgos)
Angona, Anna (Hospital Universitari de Girona Doctor Josep Trueta)
Gómez-Casares, María Teresa (Hospital Universitario de Gran Canaria Dr. Negrín)
Martínez, Clara (Institut d'Investigació Biomèdica Sant Pau)
Magro, Elena (Hospital Príncipe de Asturias)
Ayala, Rosa (Hospital Universitario 12 de Octubre (Madrid))
Del Orbe Barreto, Rafael Andrés (Hospital Universitario de Cruces (Barakaldo, País Basc))
Pérez-López, Raúl (Hospital Universitario Virgen de la Arrixaca (Múrcia))
Fox, Maria Laura (Hospital Universitari Vall d'Hebron)
Raya, José-Maria (Hospital Universitario de Canarias (La Laguna))
Guerrero, Lucía (Hospital Río Carrión)
García-Hernández, Carmen (Hospital General)
Caballero, Gonzalo (Hospital Universitario Miguel Servet (Saragossa))
Murillo, Ilda (Hospital San Jorge)
Xicoy, Blanca (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Ramírez, M.José (Hospital General)
Carreño-Tarragona, Gonzalo (Hospital Universitario 12 de Octubre (Madrid))
Hernandez-Boluda, Juan Carlos (Hospital Clínico)
Pereira, Aarturo (Institut d'Investigacions Biomèdiques August Pi i Sunyer)

Fecha:	2022
Resumen:	Background: 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. Methods: 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. Results: 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. Conclusions: The results suggest that ruxolitinib treatment for PV patients with resistance/intolerance to hydroxyurea may reduce the incidence of arterial thrombosis. Lay Summary: 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.
Ayudas:	Instituto de Salud Carlos III PI18/01472 Instituto de Salud Carlos III PI18/00205 Instituto de Salud Carlos III PI21/00231
Nota:	Altres ajuts: Novartis.
Derechos:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Lengua:	Anglès
Documento:	Article ; recerca ; Versió publicada
Materia:	Hemorrhage ; Myelofibrosis ; Myeloproliferative neoplasms ; Polycythemia vera ; Ruxolitinib ; Therapy ; Thrombosis
Publicado en:	Cancer, Vol. 128 Núm. 13 (january 2022) , p. 2441-2448, ISSN 1097-0142

DOI: 10.1002/cncr.34195
PMID: 35417564

8 p, 187.8 KB

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