Characteristics and outcomes of adult patients in the PETHEMA registry with relapsed or refractory FLT3-ITD mutation-positive acute myeloid leukemia

This retrospective study investigated outcomes of 404 patients with relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) enrolled in the PETHEMA registry, pre-approval of tyrosine kinase inhibitors. Most patients (63%) had receive...

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Autores: Martínez-Cuadrón, David, Serrano, Josefina, Mariz, José, Gil, Cristina, Tormo, Mar, Martínez-Sánchez, Pilar, Rodríguez-Arbolí, Eduardo, García-Boyero, Raimundo, Rodríguez-Medina, Carlos, Martínez-Chamorro, Carmen, Polo, Marta, Bergua, Juan, Aguiar, Eliana, Amigo, María Luz, Herrera, Pilar, Alonso-Domínguez, Juan Manuel, Bernal, Teresa, Espadana, Ana, Sayas, María-José, Algarra, Lorenzo, Vidriales, Maria Belén, Vasconcelos, Graça, Vives, Susana, Pérez-Encinas, Manuel, López, Aurelio, Noriega, Víctor, García-Fortes, María, Chillón, M. del Carmen, López, Juan A., Boluda, Blanca, Rodríguez-Veiga, Rebeca, Martínez-López, Joaquín, Barragán, Eva, Sanz, Miguel Ángel, Montesinos, Pau
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2022
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:dnet:digitalcsic_::9027719b642fd2efc6fa07bce70605a4
Acceso en línea:http://hdl.handle.net/10261/288789
Access Level:acceso abierto
Palabra clave:Acute myeloid leukemia
FLT3-ITD mutation
Real-world outcomes
Relapsed/refractory disease
Salvage therapy
Descripción
Sumario:This retrospective study investigated outcomes of 404 patients with relapsed/refractory (R/R) FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) enrolled in the PETHEMA registry, pre-approval of tyrosine kinase inhibitors. Most patients (63%) had received first-line intensive therapy with 3 + 7. Subsequently, patients received salvage with intensive therapy (n = 261), non-intensive therapy (n = 63) or supportive care only (n = 80). Active salvage therapy (i.e., intensive or non-intensive therapy) resulted in a complete remission (CR) or CR without hematological recovery (CRi) rate of 42%. More patients achieved a CR/CRi with intensive (48%) compared with non-intensive (19%) salvage therapy (p < 0.001). In the overall population, median overall survival (OS) was 5.5 months; 1- and 5-year OS rates were 25% and 7%. OS was significantly (p < 0.001) prolonged with intensive or non-intensive salvage therapy compared with supportive therapy, and in those achieving CR/CRi versus no responders. Of 280 evaluable patients, 61 (22%) had an allogeneic stem-cell transplant after they had achieved CR/CRi. In conclusion, in this large cohort study, salvage treatment approaches for patients with FLT3-ITD mutated R/R AML were heterogeneous. Median OS was poor with both non-intensive and intensive salvage therapy, with best long-term outcomes obtained in patients who achieved CR/CRi and subsequently underwent allogeneic stem-cell transplant.

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