Acute Myeloid Leukemia
Conditions
Keywords
AML, Children, Gemtuzumab ozogamicin, Stem cell transplantation
Brief summary
The overall objective is to improve the cure rate of children with newly diagnosed acute myeloid leukemia (AML) who undergo risk-adapted therapy. Stem cell transplantation (SCT) is reserved to high-risk patients defined by cytogenetics and response to chemotherapy. The efficacy and toxicity of Gemtuzumab ozogamicin (GO, Mylotarg) will be evaluated.
Detailed description
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 donor (MUD). To assess the efficacy and toxicity of Gemtuzumab ozogamicin (GO, Mylotarg) as post consolidation therapy. 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
Interventions
DRUGGemtuzumab ozogamicin
Two courses of Gemtuzumab vs. no further therapy
Sponsors
University of Aarhus
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Days to 18 Years
Healthy volunteers
No
Inclusion criteria
* AML as defined by the diagnostic criteria, * Age \< 19 years at time of study entry, * Written informed consent
Exclusion criteria
* Previous chemo- or radiotherapy, * AML secondary to previous bone marrow failure syndrome, * Down syndrome (DS), * Acute promyelocytic leukemia (APL), * Juvenile myelomonocytic leukemia (JMML), * Myelodysplastic syndrome (MDS), * Fanconi anemia, * Positive pregnancy test
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Event free survival | 5 years |
Secondary
| Measure | Time frame |
|---|---|
| Overall survival | 5 years |
Countries
Denmark
Outcome results
None listed