What happens to intolerant, relapsed or refractory chronic myeloid leukemia patients without access to clinical trials?

Objective: To assess clinical outcomes of intolerant, relapsed or refractory patients who could not be treated with new tyrosine kinase inhibitors or experimental therapies. Methods: A retrospective cohort of 90 chronic myeloid leukemia patients in all phases of the disease treated with imatinib mes...

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Detalles Bibliográficos
Autores: Bosi, Guilherme Rasia, Fogliatto, Laura Maria, Costa, Tito Emílio Vanelli, Grokoski, Kamila Castro, Pereira, Mariana Pinto, Bugs, Nathan, Kalil, Marco Antônio Baptista, Fraga, Christina Garcia da Silva, Daudt, Liane Esteves, Silla, Lucia Mariano da Rocha
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2019
País:Brasil
Institución:Universidade Federal do Rio Grande do Sul (UFRGS)
Repositorio:Repositório Institucional da UFRGS
Idioma:inglés
OAI Identifier:oai:www.lume.ufrgs.br:10183/203983
Acceso en línea:http://hdl.handle.net/10183/203983
Access Level:acceso abierto
Palabra clave:Leucemia mielogênica crônica BCR-ABL positiva
Análise de sobrevida
Mesilato de imatinib
Dasatinibe
Chronic myeloid leukemia
Imatinib mesylate
Dasatinib
Nilotinib
Survival analysis
Descripción
Sumario:Objective: To assess clinical outcomes of intolerant, relapsed or refractory patients who could not be treated with new tyrosine kinase inhibitors or experimental therapies. Methods: A retrospective cohort of 90 chronic myeloid leukemia patients in all phases of the disease treated with imatinib mesylate as their first TKI therapy, and with dasatinib or nilotinib as the next line of therapy. We evaluated clinical outcomes of these patients, with special focus on the group that needed more than two therapy lines. Results: Thirty-nine percent of patients were refractory or intolerant to imatinib. An 8-year overall survival rate of the patients who went through three or more lines of treatment was significantly lower, compared to those who were able to maintain imatinib as their first-line therapy (83% and 22%, respectively p < 0.01). Decreased overall survival was associated with advanced-phase disease (p < 0.01), failure to achieve major molecular response in first-line treatment (p < 0.01) and interruption of first-line treatment due to any reason (p = 0.023). Failure in achieving complete cytogenetic response and major molecular response and treatment interruption were associated with the progression to the third-line treatment. Conclusion: The critical outcome observed in relapsed, intolerant or refractory chronic phase CML patients reflects the unmet need for this group of patients without an alternative therapy, such as new drugs or experimental therapies in clinical trials. Broader access to newer treatment possibilities is a crucial asset to improve survival among CML patients, especially those refractory or intolerant to first-line therapies.