Myeloid leukemia with Down syndrome Myeloid leukemia with Down syndrome
Conditions
Interventions
Based on the age at diagnosis, the initial induction therapy is administered as follows
Patients under 2 years of age: cytarabine, etoposide and pirarubicin (CET)
Patients aged 2 years and older: azacitidine and CET (AZA-CET)
After the initial induction therapy, patients are stratified
Sponsors
Hasegawa Daisuke
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) Newly diagnosed myeloid leukemia with Down syndrome (ML-DS) including mosaicism DS (2) Age 4 months to <18 years at diagnosis (3) Patients who registered JPLSG-CHM-14 and have JPLSG number (4) Written informed consent from patient and guardian, or guardian
Exclusion criteria
Exclusion criteria: (1) Severe intracranial hemorrhage (grade 3 or more of CTCAE version 5.0) (2) Uncontrolled diabetes (3) Intractable heart failure (presence of congenital heart disease itself is not a exclusion criteria) (4) Uncontrolled infection (5) Pregnant or possibility of pregnancy (6) History of congenital or acquired immunodeficiencies (7) Central nervous system (CNS) leukemia (diagnostic lumbar puncture is performed only in case strongly suspected of CNS infiltration) (8) Acute promyelocytic leukemia (9) BCR::ABL1 positive AML (10) Acute leukemias of ambiguous lineage including mixed phenotype acute leukemia (11) Myeloid sarcoma (12) History of previous chemotherapy (except for chemotherapy for transient myeloproliferative disorder) or radiation therapy (13) Any inappropriate status judged by the primary investigators and co-investigators
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 3-year event-free survival (EFS) in the non-LR group | — |
Secondary
| Measure | Time frame |
|---|---|
| 3-year overall survival (OS), cumulative incidence of relapse (CIR), and non-relapse mortality (NRM) in the non-LR group 3-year OS, EFS, CIR, and NRM in the entire cohort 3-year OS, EFS, CIR, and NRM in each risk group Remission rate and positive rate of FCM-MRD in the entire cohort after the initial induction therapy Remission rate and positive rate of FCM-MRD in patients aged 2 years and older after the initial induction therapy Remission rate and positive rate of FCM-MRD in patients under 2 years of age after the initial induction therapy Positive rate of FCM-MRD after intensification therapy 1 and 2, and at the end of therapy in the MRD-HR group Remission rate after induction therapy 2 and 3, and positive rate of FCM-MRD after the initial induction therapy, induction therapy 2, induction therapy 3, and at the end of therapy in the HR group Incidence of myelosuppression, adverse events, treatment reductions, and deaths during the initial induction therapy in patients under 2 years of age, and in patients aged 2 years and older Incidence of myelosuppression, adverse events, delay in starting the subsequent treatment course, and treatment reductions during each treatment course in each risk group | — |
Contacts
Public ContactDaisuke Hasegawa
St. Luke's International Hospital
Outcome results
None listed