Martinez-Cuadron, DavidSerrano, JosefinaMariz, JoseGil, CristinaTormo, MarMartinez-Sanchez, PilarRodriguez-Arboli, EduardoGarcia-Boyero, RaimundoRodriguez-Medina, CarlosMartinez-Chamorro, CarmenPolo, MartaBergua, JuanAguiar, ElianaAmigo, Maria LHerrera, PilarAlonso-Dominguez, Juan MBernal, TeresaEspadana, AnaSayas, Maria JAlgarra, LorenzoVidriales, María BVasconcelos, GraçaVives, SusanaPerez-Encinas, Manuel MLopez, AurelioNoriega, VictorGarcia-Fortes, MaríaChillon, Maria CRodriguez-Gutierrez, Juan ICalasanz, Maria JLabrador, JorgeLopez, Juan ABoluda, BlancaRodriguez-Veiga, RebecaMartinez-Lopez, JoaquinBarragan, EvaSanz, Miguel AMontesinos, Pau2023-05-032023-05-032022-05-31Martínez-Cuadrón D, Serrano J, Mariz J, Gil C, Tormo M, Martínez-Sánchez P, et al. Characteristics and Outcomes of Adult Patients in the PETHEMA Registry with Relapsed or Refractory FLT3-ITD Mutation-Positive Acute Myeloid Leukemia. Cancers (Basel). 2022 Jun 6;14(11):28172072-6694http://hdl.handle.net/10668/20901This 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.enAttribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/FLT3-ITD mutationAcute myeloid leukemiaReal-world outcomesRelapsed/refractory diseaseSalvage therapyTyrosine Kinase InhibitorsPrior AuthorizationLeukemia, Myeloid, AcuteStem Cell TransplantationHematopoietic Stem Cell Transplantationresearch article35681796open accessAutorización previaLeucemia mieloide agudaProteínas Tirosina QuinasasTrasplante de células madreTrasplante de células madre hematopoyéticas10.3390/cancers14112817PMC9179309https://www.mdpi.com/2072-6694/14/11/2817/pdf?version=1654571500https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9179309/pdf