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Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study

Two recent phase 3 trials showed that outcomes for relapsed/refractory (R/R) FLT3-mutated acute myeloid leukemia (AML) patients may be improved by a single-agent tyrosine kinase inhibitor (TKI) (i.e., quizartinib or gilteritinib). In the current study, we retrospectively investigated the characteris...

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Autores principales: Dumas, Pierre-Yves, Bertoli, Sarah, Bérard, Emilie, Largeaud, Laetitia, Bidet, Audrey, Delabesse, Eric, Leguay, Thibaut, Leroy, Harmony, Gadaud, Noémie, Rieu, Jean Baptiste, Vial, Jean-Philippe, Vergez, François, Lechevalier, Nicolas, Luquet, Isabelle, Klein, Emilie, Sarry, Audrey, de Grande, Anne-Charlotte, Pigneux, Arnaud, Récher, Christian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7465142/
https://www.ncbi.nlm.nih.gov/pubmed/32722211
http://dx.doi.org/10.3390/cancers12082044
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author Dumas, Pierre-Yves
Bertoli, Sarah
Bérard, Emilie
Largeaud, Laetitia
Bidet, Audrey
Delabesse, Eric
Leguay, Thibaut
Leroy, Harmony
Gadaud, Noémie
Rieu, Jean Baptiste
Vial, Jean-Philippe
Vergez, François
Lechevalier, Nicolas
Luquet, Isabelle
Klein, Emilie
Sarry, Audrey
de Grande, Anne-Charlotte
Pigneux, Arnaud
Récher, Christian
author_facet Dumas, Pierre-Yves
Bertoli, Sarah
Bérard, Emilie
Largeaud, Laetitia
Bidet, Audrey
Delabesse, Eric
Leguay, Thibaut
Leroy, Harmony
Gadaud, Noémie
Rieu, Jean Baptiste
Vial, Jean-Philippe
Vergez, François
Lechevalier, Nicolas
Luquet, Isabelle
Klein, Emilie
Sarry, Audrey
de Grande, Anne-Charlotte
Pigneux, Arnaud
Récher, Christian
author_sort Dumas, Pierre-Yves
collection PubMed
description Two recent phase 3 trials showed that outcomes for relapsed/refractory (R/R) FLT3-mutated acute myeloid leukemia (AML) patients may be improved by a single-agent tyrosine kinase inhibitor (TKI) (i.e., quizartinib or gilteritinib). In the current study, we retrospectively investigated the characteristics and real-world outcomes of R/R FLT3-internal tandem duplication (ITD) acute myeloid leukemia (AML) patients in the Toulouse-Bordeaux DATAML registry. In the study, we included 316 patients with FLT3-ITD AML that received intensive chemotherapy as a first-line treatment. The rate of complete remission (CR) or CR without hematological recovery (CRi) was 75.2%, and 160 patients were R/R after a first-line TKI-free treatment (n = 294). Within the subgroup of R/R patients that fulfilled the main criteria of the QUANTUM-R study, 48.9% received an intensive salvage regimen; none received hypomethylating agents or low-dose cytarabine. Among the R/R FLT3-ITD AML patients with CR1 durations < 6 months who received intensive TKI-free treatment, the rate of CR or CRi after salvage chemotherapy was 52.8%, and these results allowed a bridge to be transplanted in 39.6% of cases. Finally, in this QUANTUM-R standard arm-matched cohort, the median overall survival (OS) was 7.0 months and 1-, 3- and 5-year OS were 30.2%, 23.7% and 21.4%, respectively. To conclude, these real-world data show that the intensity of the second-line treatment likely affects response and transplantation rates. Furthermore, the results indicate that including patients with low-intensity regimens, such as low-dose cytarabine or hypomethylating agents, in the control arm of a phase 3 trial may be counterproductive and could compromise the results of the study.
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spelling pubmed-74651422020-09-04 Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study Dumas, Pierre-Yves Bertoli, Sarah Bérard, Emilie Largeaud, Laetitia Bidet, Audrey Delabesse, Eric Leguay, Thibaut Leroy, Harmony Gadaud, Noémie Rieu, Jean Baptiste Vial, Jean-Philippe Vergez, François Lechevalier, Nicolas Luquet, Isabelle Klein, Emilie Sarry, Audrey de Grande, Anne-Charlotte Pigneux, Arnaud Récher, Christian Cancers (Basel) Article Two recent phase 3 trials showed that outcomes for relapsed/refractory (R/R) FLT3-mutated acute myeloid leukemia (AML) patients may be improved by a single-agent tyrosine kinase inhibitor (TKI) (i.e., quizartinib or gilteritinib). In the current study, we retrospectively investigated the characteristics and real-world outcomes of R/R FLT3-internal tandem duplication (ITD) acute myeloid leukemia (AML) patients in the Toulouse-Bordeaux DATAML registry. In the study, we included 316 patients with FLT3-ITD AML that received intensive chemotherapy as a first-line treatment. The rate of complete remission (CR) or CR without hematological recovery (CRi) was 75.2%, and 160 patients were R/R after a first-line TKI-free treatment (n = 294). Within the subgroup of R/R patients that fulfilled the main criteria of the QUANTUM-R study, 48.9% received an intensive salvage regimen; none received hypomethylating agents or low-dose cytarabine. Among the R/R FLT3-ITD AML patients with CR1 durations < 6 months who received intensive TKI-free treatment, the rate of CR or CRi after salvage chemotherapy was 52.8%, and these results allowed a bridge to be transplanted in 39.6% of cases. Finally, in this QUANTUM-R standard arm-matched cohort, the median overall survival (OS) was 7.0 months and 1-, 3- and 5-year OS were 30.2%, 23.7% and 21.4%, respectively. To conclude, these real-world data show that the intensity of the second-line treatment likely affects response and transplantation rates. Furthermore, the results indicate that including patients with low-intensity regimens, such as low-dose cytarabine or hypomethylating agents, in the control arm of a phase 3 trial may be counterproductive and could compromise the results of the study. MDPI 2020-07-24 /pmc/articles/PMC7465142/ /pubmed/32722211 http://dx.doi.org/10.3390/cancers12082044 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Dumas, Pierre-Yves
Bertoli, Sarah
Bérard, Emilie
Largeaud, Laetitia
Bidet, Audrey
Delabesse, Eric
Leguay, Thibaut
Leroy, Harmony
Gadaud, Noémie
Rieu, Jean Baptiste
Vial, Jean-Philippe
Vergez, François
Lechevalier, Nicolas
Luquet, Isabelle
Klein, Emilie
Sarry, Audrey
de Grande, Anne-Charlotte
Pigneux, Arnaud
Récher, Christian
Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study
title Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study
title_full Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study
title_fullStr Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study
title_full_unstemmed Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study
title_short Real-World Outcomes of Patients with Refractory or Relapsed FLT3-ITD Acute Myeloid Leukemia: A Toulouse-Bordeaux DATAML Registry Study
title_sort real-world outcomes of patients with refractory or relapsed flt3-itd acute myeloid leukemia: a toulouse-bordeaux dataml registry study
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7465142/
https://www.ncbi.nlm.nih.gov/pubmed/32722211
http://dx.doi.org/10.3390/cancers12082044
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