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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
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 resist...
Autores principales: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9324831/ https://www.ncbi.nlm.nih.gov/pubmed/35417564 http://dx.doi.org/10.1002/cncr.34195 |
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author | Alvarez‐Larrán, Alberto Garrote, Marta Ferrer‐Marín, Francisca Pérez‐Encinas, Manuel Mata‐Vazquez, M. Isabel Bellosillo, Beatriz Arellano‐Rodrigo, Eduardo Gómez, Montse García, Regina García‐Gutiérrez, Valentín Gasior, Mercedes Cuevas, Beatriz Angona, Anna Gómez‐Casares, María Teresa Martínez, Clara M. Magro, Elena Ayala, Rosa del Orbe‐Barreto, Rafael Pérez‐López, Raúl Fox, Maria Laura Raya, José‐María Guerrero, Lucía García‐Hernández, Carmen Caballero, Gonzalo Murillo, Ilda Xicoy, Blanca Ramírez, M. José Carreño‐Tarragona, Gonzalo Hernández‐Boluda, Juan Carlos Pereira, Arturo |
author_facet | Alvarez‐Larrán, Alberto Garrote, Marta Ferrer‐Marín, Francisca Pérez‐Encinas, Manuel Mata‐Vazquez, M. Isabel Bellosillo, Beatriz Arellano‐Rodrigo, Eduardo Gómez, Montse García, Regina García‐Gutiérrez, Valentín Gasior, Mercedes Cuevas, Beatriz Angona, Anna Gómez‐Casares, María Teresa Martínez, Clara M. Magro, Elena Ayala, Rosa del Orbe‐Barreto, Rafael Pérez‐López, Raúl Fox, Maria Laura Raya, José‐María Guerrero, Lucía García‐Hernández, Carmen Caballero, Gonzalo Murillo, Ilda Xicoy, Blanca Ramírez, M. José Carreño‐Tarragona, Gonzalo Hernández‐Boluda, Juan Carlos Pereira, Arturo |
author_sort | Alvarez‐Larrán, Alberto |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-9324831 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-93248312022-07-30 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 Alvarez‐Larrán, Alberto Garrote, Marta Ferrer‐Marín, Francisca Pérez‐Encinas, Manuel Mata‐Vazquez, M. Isabel Bellosillo, Beatriz Arellano‐Rodrigo, Eduardo Gómez, Montse García, Regina García‐Gutiérrez, Valentín Gasior, Mercedes Cuevas, Beatriz Angona, Anna Gómez‐Casares, María Teresa Martínez, Clara M. Magro, Elena Ayala, Rosa del Orbe‐Barreto, Rafael Pérez‐López, Raúl Fox, Maria Laura Raya, José‐María Guerrero, Lucía García‐Hernández, Carmen Caballero, Gonzalo Murillo, Ilda Xicoy, Blanca Ramírez, M. José Carreño‐Tarragona, Gonzalo Hernández‐Boluda, Juan Carlos Pereira, Arturo Cancer Original Articles 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. John Wiley and Sons Inc. 2022-04-13 2022-07-01 /pmc/articles/PMC9324831/ /pubmed/35417564 http://dx.doi.org/10.1002/cncr.34195 Text en © 2022 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Original Articles Alvarez‐Larrán, Alberto Garrote, Marta Ferrer‐Marín, Francisca Pérez‐Encinas, Manuel Mata‐Vazquez, M. Isabel Bellosillo, Beatriz Arellano‐Rodrigo, Eduardo Gómez, Montse García, Regina García‐Gutiérrez, Valentín Gasior, Mercedes Cuevas, Beatriz Angona, Anna Gómez‐Casares, María Teresa Martínez, Clara M. Magro, Elena Ayala, Rosa del Orbe‐Barreto, Rafael Pérez‐López, Raúl Fox, Maria Laura Raya, José‐María Guerrero, Lucía García‐Hernández, Carmen Caballero, Gonzalo Murillo, Ilda Xicoy, Blanca Ramírez, M. José Carreño‐Tarragona, Gonzalo Hernández‐Boluda, Juan Carlos Pereira, Arturo 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 |
title | 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 |
title_full | 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 |
title_fullStr | 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 |
title_full_unstemmed | 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 |
title_short | 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 |
title_sort | 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 |
topic | Original Articles |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9324831/ https://www.ncbi.nlm.nih.gov/pubmed/35417564 http://dx.doi.org/10.1002/cncr.34195 |
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