학술논문
Characteristics and Outcomes of Adult Patients in the PETHEMA Registry with Relapsed or Refractory FLT3-ITD Mutation-Positive Acute Myeloid Leukemia
Document Type
article
Author
David Martínez-Cuadrón; Josefina Serrano; José Mariz; Cristina Gil; Mar Tormo; Pilar Martínez-Sánchez; Eduardo Rodríguez-Arbolí; Raimundo García-Boyero; Carlos Rodríguez-Medina; Carmen Martínez-Chamorro; Marta Polo; Juan Bergua; Eliana Aguiar; María L. Amigo; Pilar Herrera; Juan M. Alonso-Domínguez; Teresa Bernal; Ana Espadana; María J. Sayas; Lorenzo Algarra; María B. Vidriales; Graça Vasconcelos; Susana Vives; Manuel M. Pérez-Encinas; Aurelio López; Víctor Noriega; María García-Fortes; María C. Chillón; Juan I. Rodríguez-Gutiérrez; María J. Calasanz; Jorge Labrador; Juan A. López; Blanca Boluda; Rebeca Rodríguez-Veiga; Joaquín Martínez-López; Eva Barragán; Miguel A. Sanz; Pau Montesinos; on behalf of the PETHEMA Group
Source
Cancers, Vol 14, Iss 11, p 2817 (2022)
Subject
Language
English
ISSN
14112817
2072-6694
2072-6694
Abstract
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.