%0 Journal Article
%A Martínez Cuadrón, David
%A Serrano, Josefina
%A Mariz, José
%A Gil, Cristina
%A Tormo, Mar
%A Martínez Sánchez, María Del Pilar
%A Rodríguez Arbolí, Eduardo
%A García Boyero, Raimundo
%A Rodríguez Medina, Carlos
%A Martínez Chamorro, Carmen
%A Polo, Marta
%A Bergua, Juan
%A Aguiar, Eliana
%A Amigo, María 
%A Herrera, Pilar
%A Alonso Domínguez, Juan Manuel
%A Bernal, Teresa
%A Espadana, Ana
%A Sayas, María 
%A Algarra, Lorenzo
%A Vidriales, María B.
%A Vasconcelos, Graça
%A Vives, Susana
%A Pérez Encinas, Manuel 
%A López, Aurelio
%A Noriega Concepción, Víctor
%A García Fortes, María
%A Chillón, María 
%A Rodríguez Gutiérrez, Juan 
%A Calasanz, María
%A Labrador, Jorge
%A López, Juan
%A Boluda, Blanca
%A Rodríguez Veiga, Rebeca
%A Martínez López, Joaquín
%A Barragán, Eva
%A Sanz, Miguel
%A Montesinos, Pau
%T Characteristics and Outcomes of Adult Patients in the PETHEMA Registry with Relapsed or Refractory FLT3-ITD Mutation-Positive Acute Myeloid Leukemia
%D 2022
%@ 2072-6694
%U https://hdl.handle.net/20.500.14352/72154
%X 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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