Chemotherapy for Acute Myeloid Leukemia in Young Patients with Down Syndrome
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, it mentions that children who have previously received chemotherapy, radiation therapy, or any anti-leukemic therapy are not eligible, except for cytarabine for treating transient myeloproliferative disorder.
What data supports the effectiveness of the drug for treating acute myeloid leukemia in young patients with Down syndrome?
Research shows that children with Down syndrome and myeloid leukemia have better survival rates when treated with less intensive chemotherapy regimens, including drugs like cytarabine and daunorubicin, due to their heightened sensitivity to these drugs. A study reported a 5-year event-free survival rate of 89.9% and overall survival of 93.0% for patients treated with a modified regimen, highlighting the effectiveness of these drugs in this specific group.12345
What safety data exists for chemotherapy treatments in young patients with Down syndrome and acute myeloid leukemia?
Children with Down syndrome treated for myeloid leukemia using reduced-dose chemotherapy have shown favorable survival rates while minimizing toxicity. Common side effects of treatments like Vyxeos, which includes daunorubicin and cytarabine, are hypersensitivity, febrile neutropenia (low white blood cell count with fever), and prolonged neutropenia (low white blood cell count) and thrombocytopenia (low platelet count).13678
What makes this chemotherapy treatment for acute myeloid leukemia in young patients with Down syndrome unique?
This treatment is unique because it combines multiple drugs, including asparaginase and cytarabine, which have shown increased sensitivity in Down syndrome patients, allowing for potentially better outcomes with reduced toxicity compared to standard treatments. The approach also involves optimizing drug dosing and scheduling to improve survival rates while minimizing side effects.135910
What is the purpose of this trial?
This trial studies a chemotherapy treatment that adjusts based on how well patients respond initially. It targets younger patients with Down syndrome who have certain types of blood cancer. The treatment aims to effectively kill cancer cells while reducing side effects.
Research Team
Jason N Berman
Principal Investigator
Children's Oncology Group
Eligibility Criteria
This trial is for young patients with Down syndrome who have newly diagnosed acute myeloid leukemia or myelodysplastic syndrome. They must not have received any anti-leukemic therapy except cytarabine for transient myeloproliferative disease, and they should not have promyelocytic leukemia. Patients need to show a certain percentage of blasts in their bone marrow or blood and meet specific diagnostic criteria.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Induction I
Patients receive cytarabine intrathecally and intravenously, daunorubicin hydrochloride, and thioguanine orally. Induction I continues for a minimum of 28 days.
Induction II
Patients receive cytarabine, daunorubicin hydrochloride, and thioguanine. Induction II continues for a minimum of 28 days.
Induction III
Patients receive cytarabine, daunorubicin hydrochloride, and thioguanine as in Induction II. Induction III continues for a minimum of 28 days.
Intensification I
Patients receive cytarabine and etoposide. Intensification I continues for a minimum of 28 days.
Intensification II
Patients receive cytarabine and etoposide. Intensification II continues for a minimum of 28 days.
Follow-up
Participants are monitored for safety and effectiveness after treatment. Follow-up includes visits at 1 month, monthly for 12 months, every 3 months for 12 months, every 6 months for 3 years, and annually for 10 years.
Treatment Details
Interventions
- Asparaginase
- Asparaginase Erwinia chrysanthemi
- Cytarabine
- Daunorubicin Hydrochloride
- Etoposide
- Mitoxantrone Hydrochloride
- Thioguanine
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Oncology Group
Lead Sponsor
National Cancer Institute (NCI)
Collaborator