acute promyelocytic leukemia
Conditions
Interventions
None listed
Sponsors
AIEOP- Associazione Italiana Ematologia Oncologia Pediatrica
Eligibility
Age
No minimum to 17 Years
Inclusion criteria
Inclusion criteria: - Newly diagnosed APL confirmed by the presence of PML/RARa fusion gene - Age
Exclusion criteria
Exclusion criteria: - Patients with a clinical diagnosis of APL but subsequently found to lack PML/RARa rearrangement should be withdrawn from the study and treated with an alternative protocol - Significant liver dysfunction (bilirubin serum levels >3 mg/dL, ALT/AST serum levels >5x the normal values) - Creatinine serum levels >2 times the normal value for age - Significant arrhythmias, ECG abnormalities, other cardiac contraindications (L-FEV
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint of the study is event-free survival (EFS). This cumulative endpoint includes the following events: • no achievement of hematological complete remission after induction therapy; • no achievement of molecular remission after three consolidation courses (molecular resistance); • relapse (hematological/molecular); • death, including early death, at 2 years from diagnosis. | — |
Secondary
| Measure | Time frame |
|---|---|
| • Rate of hematological CR after induction • Rate of early and aplastic death during induction • Overall survival (OS) • Cumulative incidence of either hematological and molecular relapse (CIR) • Incidence of hematological and non-hematological toxicity • Kinetics of MRD clearance • Rate of molecular remission after 3 consolidation cycles • Assessment of PML/RARa transcript level reduction during treatment • Toxicity - hematological and non-hematological • Supportive care requirements • Total hospitalization days during therapy and health economic impact | — |
Countries
Netherlands
Outcome results
None listed