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Background <p>The previous Italian national trial (AIEOP-AML-2002/01) on children with acute myeloid leukemia (AML) achieved a 3-year overall (OS) and event-free survival (EFS) of 72.3% and 59.1%, respectively. In this study (AIEOP-AML-2013), we evaluated if refined patient’s stratification in 3 groups and a second induction with randomization could improve outcomes.</p> Methods <p>In the new AIEOP-AML-2013, patients were stratified into 3 groups [standard-(SR), intermediate-(IR) and high-risk (HR)] mainly according to genetic criteria and centralized assessment of multiparametric flow-cytometry measurable residual disease (MFC-MRD). The 1st induction course was common for all patients [idarubicin-cytarabine-etoposide (ICE)]. IR and HR patients were then randomized to receive either a 2nd ICE or the fludarabine-cytarabine- liposomal-doxorubicin (FLA-My) scheme as second induction. IR patients with HLA-compatible sibling, and all HR patients were consolidated with allogeneic hematopoietic stem cell transplantation (allo-HSCT), while the remaining IR and SR patients were consolidated with chemotherapy only. Data cut-off was February 1, 2024.</p> Results <p>From June/2015 to June/2022, 371 patients were enrolled in the trial. Twenty-six children (7%) experienced primary induction failure, while only 3 patients died during the 2 induction courses. The cumulative incidence of 3-year non-relapse mortality in continuous complete remission was 6.8%. The proportion of patients allocated to the SR, IR, and HR groups were 19.5%, 22%, and 58.5%, respectively. Three-year cumulative incidence of relapse was 18.9%. The 3-year probabilities of OS and EFS were 83.9% and 68.5%, respectively, both values being significantly better (<i>p</i> = 0.001) than those of the AIEOP-AML-2002/01 study. The 3-year OS of SR, IR, and HR patients were 97.0%, 84.2%, and 79.4%, respectively, (<i>p</i> = 0.01). The probability of EFS did not differ in IR and HR randomized to receive either a 2nd ICE or the FLA-My scheme course. Levels of MRD after the 1st and 2nd induction course strongly influenced the EFS probability.</p> Conclusions <p>A significant improvement in the outcomes of children with <i>de novo</i> AML was obtained with refined risk-stratification and risk-adapted therapy. FLA-My did not offer any advantage over repeating a 2nd ICE as 2nd induction course.</p> Trial registration <p>This study was registered in the European Clinical Trials Database (EudraCT 2014-000652-28).</p>

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Improved outcomes of the refined risk-stratification and risk-adapted therapy in children with acute myeloid leukemia: final results of the AIEOP AML 2013

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  • 发表日期：

    2026年4月20日

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  • Franco Locatelli,
  • Barbara Buldini,
  • Martina Pigazzi,
  • Carmelo Rizzari,
  • Giuseppe Menna,
  • Franca Fagioli,
  • Manuela Tumino,
  • Luca Lo Nigro,
  • Paolo D’Angelo,
  • Maria Giuseppina Cefalo,
  • Valeria Paganelli,
  • Luisa Strocchio,
  • Marco Becilli,
  • Marco Spinelli,
  • Giovanna Giagnuolo,
  • Francesco Saglio,
  • Nicola Santoro,
  • Concetta Micalizzi,
  • Marco Zecca,
  • Riccardo Masetti,
  • Pietro Merli

摘要

Background

The previous Italian national trial (AIEOP-AML-2002/01) on children with acute myeloid leukemia (AML) achieved a 3-year overall (OS) and event-free survival (EFS) of 72.3% and 59.1%, respectively. In this study (AIEOP-AML-2013), we evaluated if refined patient’s stratification in 3 groups and a second induction with randomization could improve outcomes.

Methods

In the new AIEOP-AML-2013, patients were stratified into 3 groups [standard-(SR), intermediate-(IR) and high-risk (HR)] mainly according to genetic criteria and centralized assessment of multiparametric flow-cytometry measurable residual disease (MFC-MRD). The 1st induction course was common for all patients [idarubicin-cytarabine-etoposide (ICE)]. IR and HR patients were then randomized to receive either a 2nd ICE or the fludarabine-cytarabine- liposomal-doxorubicin (FLA-My) scheme as second induction. IR patients with HLA-compatible sibling, and all HR patients were consolidated with allogeneic hematopoietic stem cell transplantation (allo-HSCT), while the remaining IR and SR patients were consolidated with chemotherapy only. Data cut-off was February 1, 2024.

Results

From June/2015 to June/2022, 371 patients were enrolled in the trial. Twenty-six children (7%) experienced primary induction failure, while only 3 patients died during the 2 induction courses. The cumulative incidence of 3-year non-relapse mortality in continuous complete remission was 6.8%. The proportion of patients allocated to the SR, IR, and HR groups were 19.5%, 22%, and 58.5%, respectively. Three-year cumulative incidence of relapse was 18.9%. The 3-year probabilities of OS and EFS were 83.9% and 68.5%, respectively, both values being significantly better (p = 0.001) than those of the AIEOP-AML-2002/01 study. The 3-year OS of SR, IR, and HR patients were 97.0%, 84.2%, and 79.4%, respectively, (p = 0.01). The probability of EFS did not differ in IR and HR randomized to receive either a 2nd ICE or the FLA-My scheme course. Levels of MRD after the 1st and 2nd induction course strongly influenced the EFS probability.

Conclusions

A significant improvement in the outcomes of children with de novo AML was obtained with refined risk-stratification and risk-adapted therapy. FLA-My did not offer any advantage over repeating a 2nd ICE as 2nd induction course.

Trial registration

This study was registered in the European Clinical Trials Database (EudraCT 2014-000652-28).