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Comparison of Consolidation Strategies for Pediatric Patients With Acute Myeloid Leukemia

Phase 3
Completed
Conditions
AML
Pediatric AML
Registration Number
NCT06211452
Lead Sponsor
Grupo Argentino de Tratamiento de la Leucemia Aguda
Brief Summary

GATLA 8-AML´07 trial is an multicenter phase III dose-optimization trial for the treatment of acute myeloid leukemias in children and adolescents. Patients are treated with a combination of intensive chemotherapy in combination with intrathecal-injection by CNS and haematopoietic stem cell transplantation.

The patients are stratified in a standard-group (SR) and a high risk-group (HR). SR was defined as FAB (French-American-British) M1/M2 with Auer rods; FAB M4eo or favorable cytogenetics \[t(8;21)/AML1-ETO or inv(16) or t(16;16) and/or CBFB/MYH11)\]; bone marrow blasts ≤5% on day 15. HR was defined as all others. SR patients were reclassified to the HR group if FLT3-ITD positive.

Based on the experience of the BFM group, it was decided to randomly evaluate whether the six-drug conventional consolidation stage can be replaced with the use of a consolidation based by block therapy on drugs of proven efficacy in AML with the aim of reducing residual disease, and the toxicity of this stage. Patients are randomized once the double induction is completed into those who will receive the conventional consolidation phase and those who will receive consolidation with the combination of high doses cytarabine and two different anthracyclines sequentially.

Detailed Description

All patients in the trial, whether standard risk (SR) or high risk (HR), receive 2 Induction courses: AIE and HAM and then are randomized to continue with the consolidation phase: a conventional consolidation (Arm A) with 6 drugs vs 2 blocks of chemotherapy that are incorporated in this protocol (AI and haM) (Arm B) and then an Intensification phase with high doses of cytarabine and etoposide. The difference between the SR and AR groups is that the former don't continue with the maintenance phase, while those in HR receive 1 year of Maintenance if they don't have a histoidentical family donor. SR patients according to cytogenetics and with \< 5% blasts in the bone marrow on day 15, haven´t indication for TCHP in 1st CR. HR patients, with a histoidentical family donor, will be transplanted in the 1st. CR after the Consolidation phases or the AI and haM blocks, according to the treatment arm that corresponded to them by randomization

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
258
Inclusion Criteria
  • De novo AML
  • FAB M0-M7
  • Primary myelosarcomas or acute mixed lineage leukemia / biphenotypic leukemia (predominantly myeloid).
  • Written informed consent from patients, parents, or guardians was obtained at study entry in accordance with the Declaration of Helsinki
Exclusion Criteria
  • AML as secondary malignancy
  • Children with Down's syndrome
  • Accompanying diseases which do not allow therapy according to the protocol
  • Pre-treatment for more than 14 days with another intensive induction therapy
  • Patients with APL
  • Patients with myelodysplasia
  • Patients with blast crisis of Chronic Myeloid Leukemia
  • Patients who die before starting treatment

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Primary Outcome Measures
NameTimeMethod
Determine if two-cycle consolidation reduced the cumulative incidence of relapse compared to conventional consolidation (CONS) among newly diagnosed pediatric patients with AML.72 months

The GATLA 8-LMA-P'07 was a randomized trial including pediatric AML children in Argentina; the design was based on AML-BFM 98 with minor modifications.(10) Similar to AML-BFM 98, the trial compared two approaches to consolidation. The standard regimen was a 6-week consolidation phase (CONS), which was also used in AML-BFM 93 vs. the comparator regimen of two short chemotherapy cycles (two-cycle) with cytarabine plus idarubicin (AI) and high dose cytarabine plus mitoxantrone (haM). AML-BFM 98 previously demonstrated that haM was tolerable.10 We hypothesized that the two-cycle consolidation (two-cycle) would be associated with lower relapse rates related to dose intensification compared to the standard CONS approach.

Secondary Outcome Measures
NameTimeMethod
Evaluate Event-free survival (EFS) of pediatric patients with AML.72 months

EFS was calculated from date of randomization to first event (death, resistant leukemia, relapse or secondary malignancy) or date of last follow-up.

Evaluate Overall Survival (OS) of pediatric patients with AML.72 months

OS was also calculated from date of randomization to date of death or date of last follow-up.

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