Impact of hematopoietic cell transplantation and quizartinib in newly diagnosed patients with acute myeloid leukemia and FMS-like tyrosine kinase 3–internal tandem duplications in the QuANTUM-First trial

QuANTUM-First (NCT02668653) was a randomized phase 3 trial in newly diagnosed FLT3-ITDQpositive acute myeloid leukemia (AML) patients treated with quizartinib or placebo plus standard induction and consolidation chemotherapy and/or allogeneic hematopoietic cell transplantation (allo-HCT), followed...

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Main Authors: Richard F. Schlenk, Pau Montesinos, Hee-Je Kim, Antonio Romero-Aguilar, Radovan Vrhovac, Elżbieta Patkowska, Pavel Žak, Po-Nan Wang, James Hanyok, Li Liu, Yasser Mostafa Kamel, Karima Imadalou, Arnaud Lesegretain, Jorge Cortes, Mikkael A. Sekeres, Herve Dombret, Sergio Amadori, Jianxiang Wang, Alexander E. Perl, Mark J. Levis, Harry P. Erba
Format: Article
Language:English
Published: Ferrata Storti Foundation 2025-03-01
Series:Haematologica
Online Access:https://haematologica.org/article/view/11974
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Summary:QuANTUM-First (NCT02668653) was a randomized phase 3 trial in newly diagnosed FLT3-ITDQpositive acute myeloid leukemia (AML) patients treated with quizartinib or placebo plus standard induction and consolidation chemotherapy and/or allogeneic hematopoietic cell transplantation (allo-HCT), followed by single-agent maintenance therapy. We evaluated the impact of allo-HCT performed in first complete remission (CR1) or composite CR1 (CRc1) on overall survival (OS), considering treatment randomization. Post-hoc extended Cox regression multivariable analyses were conducted in patients who achieved CR/CRc by the end of induction, including allo-HCT in CR1/CRc1 as a time-dependent variable to identify prognostic and predictive factors for OS. There were 297 patients with CR by the end of induction (quizartinib, n=147; placebo, n=150); of these, 157 (52.9%) underwent allo-HCT in CR1 (quizartinib, n=84; placebo, n=73). There were 368 patients with CRc by the end of induction (quizartinib, n=192; placebo, n=176); of these, 196 (53.3%) underwent allo-HCT in CRc1 (quizartinib, n=110; placebo, n=86). Multivariable analyses revealed quizartinib treatment and allo-HCT in either CR1 (hazard ratio [HR]=0.553, 95% confidence interval [CI]=0.383Q0.798, P=0.0015 and HR=0.527, 95% CI=0.349Q0.796, P=0.0023, respectively) or CRc1 (HR=0.645, 95% CI=0.470Q0.886, P=0.0068 and HR=0.557, 95% CI=0.391Q0.793, P=0.0012, respectively) as significant predictive factors for a longer OS. No new safety signals were identified. Patients who underwent protocol-specified allo-HCT in CR1/CRc1 experienced post-allo-HCTQrelated complications, mostly grade ≥2 graft-versus-host disease, as expected. This posthoc analysis further supports quizartinib and allo-HCT in CR1/CRc1 as an efficacious and well-tolerated treatment strategy for newly diagnosed FLT3-ITDQpositive AML patients fit for intensive chemotherapy.
ISSN:0390-6078
1592-8721