NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia
The overall objective is to improve the cure rate of pediatric patients with newly diagnosed
acute myeloid leukemia (AML). The specific aims are as follows:
1.1 Therapeutic aims
To improve the event-free survival (EFS) of AML patients who undergo risk-adapted therapy.
To improve the overall survival (OS) by reserving stem cell transplantation (SCT) to
high-risk patients based on cytogenetics and response to induction therapy.
To compare the outcome of SCT using HLA-matched sibling donor (MSD) or HLA-matched unrelated
To assess the efficacy and toxicity of Gemtuzumab ozogamicin (GO, Mylotarg) as post
1.2 Biologic aims
To study minimal residual disease (MRD) levels in blood and bone marrow (BM) at defined time
points and to study the prognostic impact of MRD.
To test in vitro cellular drug resistance at diagnosis and relapse, and correlate these data
to background factors and clinical outcome.
To secure storage of biological material from diagnosis for future biologic studies
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Event free survival
Henrik Hasle, MD
Department of Pediatrics, Aarhus University Hospital Skejby, Aarhus, Denmark
Denmark: National Board of Health