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

Cancer - Tập 128 Số 13 - Trang 2441-2448 - 2022
Alberto Álvarez‐Larrán1, Marta Garrote1, Francisca Ferrer‐Marín2, Manuel Pérez‐Encinas3, María‐Isabel Mata‐Vázquez4, Beatríz Bellosillo5, Eduardo Arellano‐Rodrigo1, Montse Gómez6, Regina García7, Valentín García‐Gutiérrez8, Mercedes Gasior Kabat9, Beatriz Cuevas10, Anna Angona11, María Teresa Gómez‐Casares12, Clara Martínez13, Elena Magro14, Rosa Ayala15, Rafael Del Orbe16, Raúl Pérez‐López17, Laura Fox18, José‐María Raya19, Lucía Guerrero20, Carmen García‐Hernández21, Gonzalo Caballero22, Ilda Murillo23, Blanca Xicoy24, M.J. Ramírez25, Gonzalo Carreño‐Tarragona15, Juan Carlos Hernández‐Boluda6, Arturo Pereira1
1Hospital Clínic, Institut d'Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain
2Hospital Morales Messeguer, Universidad Católica San Antonio de Murcia, Murcia, Centro de Investigación Biomédica en Red de Enfermedades Raras Murcia Spain
3Hospital Clínico Universitario, Santiago de Compostela, Spain
4Hospital Costa del Sol, Marbella, Spain
5Hospital del Mar, Barcelona, Spain
6Hospital Clínico, Valencia, Spain
7Hospital Virgen de la Victoria, Málaga, Spain
8Hospital Ramón y Cajal (Instituto Ramón y Cajal de Investigación Sanitaria), Madrid, Spain
9Hospital La Paz, Madrid, Spain
10Hospital Universitario de Burgos, Burgos, Spain
11Hospital Josep Trueta Institut Català d'Oncologia Girona Spain
12Hospital Doctor Negrín, Las Palmas de Gran Canaria, Spain
13Hospital de la Santa Creu I Sant Pau, Barcelona, Spain
14Hospital Príncipe de Asturias, Alcalá de Henares, Spain
15Hospital Universitario 12 de Octubre, Madrid, Spain
16Hospital Universitario de Cruces, Barakaldo, Spain
17Hospital Virgen de la Arrixaca, Murcia, Spain
18Hospital Vall d'Hebron, Barcelona, Spain
19Hospital Universitario de Canarias, Santa Cruz de Tenerife, Spain
20Hospital Río Carrión, Palencia, Spain
21Hospital General, Alicante, Spain;
22Hospital Miguel Servet, Zaragoza, Spain
23Hospital San Jorge, Huesca, Spain
24Hospital Germans Trias i Pujol Institut Català d'Oncologia Josep Carreras Leukemia Research Institute Universitat Autònoma de Barcelona Badalona Spain
25Hospital General, Jerez de la Frontera, Spain

Tóm tắt

BackgroundRuxolitinib 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.MethodsA 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.ResultsPatients 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.ConclusionsThe 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.

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Cancer

Tập 128 Số 13

2441-2448

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