This prospective non-randomized multicenter trial created based on protocol ML DS 2006 and aimed at standardization of current therapy approaches and creating a national network for diagnostic, treatment and monitoring of children (0-18 years) with AML and Down syndrome in Russia. Based on the results the investigators expect to increase long-term overall and event-free survival in children with AML and DS and reduce the immediate and remote toxicity of chemotherapy by reducing the dose load of chemotherapeutic drugs.
The study protocol therapy for all patients includes four chemotherapy blocks:
Course 1 AIE (cytarabine/idarubicin/etoposide) Course 2 AI (cytarabine/idarubicin) Course 3 HAD (high -dose cytarabine (1g)/daunorubicin) Course 4 HA (high-dose cytarabine) Safety to be monitored based on CTCAE v5.0
Age 0-18 years
Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)
Signed informed consent
Children with Down syndrome and acute lymphoblastic leukemia (ALL)
Severe comorbidities with contraindications to the treatment according to the protocol
Pre-treatment >14 days with intensive induction therapy
Refusal of all therapy or important elements of therapy
Single group
4 chemotherapy blocks: Course 1 AIE (cytarabine/idarubicin/etoposide), Course 2 AI (cytarabine/idarubicin), Course 3 HAD (high-dose cytarabine (1g)/daunorubicin), Course 4 HA (high-dose cytarabine).
Drug4 chemotherapy blocks: Course 1 AIE (cytarabine/idarubicin/etoposide), Course 2 AI (cytarabine/idarubicin), Course 3 HAD (high-dose cytarabine (1g)/daunorubicin), Course 4 HA (high-dose cytarabine).
event-free survival
Response rate; event-free survival (EFS)
relapse-free survival (RFS)
overall survival (OS)
treatment-related mortality
CTCAE
Number of adverse events by Common Terminology Criteria for Adverse Events (CTCAE) 5.0
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