Response-guided chemotherapy for pediatric acute myeloid leukemia without hematopoietic stem cell transplantation in first complete remission: Results from protocol DB AML-01

Barbara de Moerloose, Ardine Reedijk, Geertruida H. de Bock, Tim Lammens, Valerie de Haas, Barbara Denys, Laurence Dedeken, Marry M. van den Heuvel-Eibrink, Maroeska te Loo, Anne Uyttebroeck, An van Damme, Jutte van der Werff-ten Bosch, Jozsef Zsiros, Gertjan Kaspers, Eveline de Bont

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Background: Children with acute myeloid leukemia (AML) have a 70% survival rate with treatment regimens containing high doses of cytarabine and anthracyclines and, in some, hematopoietic stem cell transplantation (allo-HSCT). Procedure: In this multicenter Dutch–Belgian protocol (DB AML-01), 112 children with de novo AML were included. Treatment was stratified according to day 15 bone marrow response after the first induction course. Poor responders received a second course without delay while good responders awaited hematological recovery. Patients achieving CR after two induction courses continued with three consolidation courses without HSCT in CR1. Results: The overall remission rate was 93.5%. After a median follow-up of 4.1 years, three-year event-free survival (EFS) was 52.6% (95% CI, 42.9%–61.3%), three-year cumulative incidence of relapse 39.7% (95% CI, 30.1%–49.0%), and three-year overall survival (OS) 74.0% (95% CI, 64.8%–81.2%). Significantly more events occurred in patients with high WBC at diagnosis or FLT3-ITD/NPM1-WT, whereas core binding factor (CBF) leukemia had a significantly better EFS. KMT2A rearrangements and age > 10 years negatively impacted OS. Conclusions: DB AML-01 response-guided therapy results in a favorable OS, particularly for children with CBF leukemia, children younger than 10 years or with initial WBC counts below 100 × 109/L. Outcome of patients with FLT3-ITD/NPM1-WT remains poor and warrants alternative treatment strategies.
Original languageEnglish
Article numbere27605
Pages (from-to)e27605
JournalPediatric Blood and Cancer
Issue number5
Early online date2019
Publication statusPublished - 1 May 2019


  • DB AML-01
  • HSCT
  • anthracycline
  • outcome
  • pediatric AML

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