Acute myeloid leukemia in Down syndrome
Conditions
Interventions
LR: Induction 1 (CET) -> Intensification L-1 (CET) -> Intensification L-2 (CT) -> Intensification L-3 (CT). SR: Induction 1 (CET) -> Intensification S-1 (CET) -> Intensification S-2 (CT) -> Intensific
Sponsors
Taga Takashi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients who participate in the AML-D16 study must meet all of the following criteria: (1) Patients with Down syndrome and newly diagnosed myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). (2) Children aged 4 months or older at diagnosis of MDS or AML. (3) Prior to participation in the AML-D16 study, patients must be enrolled in the CHM-14. (4)Each patient and patient's guardians, or patient's guardians must sign a written informed consent.
Exclusion criteria
Exclusion criteria: Patients were excluded if they met at least one of the following criteria: (1) Age at diagnosis of MDS or AML older than 18 years old. (2) Severe intracranial hemorrhage (grade 3 or more of CTCAE version 4.0). (3) Poorly controlled diabetes. (4) Intractable heart failure. (5) Uncontrollable infection. (6) pregnant or possibility pregnant. (7) History of congenital or acquired immunodeficiency syndrome. (8) Central nervous system (CNS) leukemia (diagnostic lumbar puncture is performed only in case strongly suspected of CNS infiltration). (9) Acute promyelocytic leukemia. (10) BCR-ABL positive AML. (11) Acute leukemias of ambiguous lineage including mixed phenotype acute leukemia. (12) Myeloid sarcoma. (13) History of previous chemotherapy (except for administration of cytarabine for the treatment of transient myeloproliferative disorder) or radiation therapy. (14) Patients who are evaluated with ineligible by attending physician, principal investigator, and co-investigators.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 3-year event-free survival (EFS) for children with low risk (LR) AML-DS | — |
Secondary
| Measure | Time frame |
|---|---|
| (1) 3-year EFS and overall survival (OS) for overall subjects. (2) 3-year EFS for children with each risk group; standard risk (SR), high risk (HR) or minimal residual disease (MRD)-high risk (MHR). (3) 3-year cumulative incidence of relapse for children with each risk group. (4) 3-year OS for children with each risk group. (5) Remission rate for overall subjects. (6) Positive rate of MRD after the initial induction therapy and at the end of chemotherapy (MRDs are also measured after induction 2 and 3 for HR group; measured after intensification 2 (MH-2) for MHR group). (7) Incidence of clinically relevant adverse events for overall subjects or children with each risk group. (8) Quality of life for patients evaluated by patients' families. (9) 3-year EFS and OS for children with HR group combined with HR patients who enrolled in the previous study AML-D05 and D11. | — |
Contacts
Public ContactDaisuke Hasegawa
St. Luke's International Hospital
Outcome results
None listed