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, María Del Pilar; Rodríguez Arbolí, Eduardo; García Boyero, Raimundo; Rodríguez Medina, Carlos; Martínez Chamorro, Carmen; Polo, Marta; Bergua, Juan; Aguiar, Eliana; Amigo, María; Herrera, Pilar; Alonso Domínguez, Juan Manuel; Bernal, Teresa; Espadana, Ana; Sayas, María; Algarra, Lorenzo; Vidriales, María B.; Vasconcelos, Graça; Vives, Susana; Pérez Encinas, Manuel; López, Aurelio; Noriega Concepción, Víctor; García Fortes, María; Chillón, María; Rodríguez Gutiérrez, Juan; Calasanz, María; Labrador, Jorge; López, Juan; Boluda, Blanca; Rodríguez Veiga, Rebeca; Martínez López, Joaquín; Barragán, Eva; Sanz, Miguel; Montesinos, Pau
Tipo de documento: Artículo
Fecha de publicación:2022
País:España
Institución:Universidad Complutense de Madrid (UCM)
Repositorio:Docta Complutense
Lenguaje:Inglés
OAI Identifier:oai:docta.ucm.es:20.500.14352/72154
Acceso en línea:https://hdl.handle.net/20.500.14352/72154
Nivel de acceso:Acceso abierto
Palabra clave:616-006.04
616.15
Acute myeloid leukemia
FLT3-ITD mutation
Real-world outcomes
Relapsed/refractory disease
Salvage therapy
Medicina
Oncología
32 Ciencias Médicas
3201.01 Oncología
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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