Phase 3
Completed N=205
Combination Chemotherapy in Treating Young Patients With Down Syndrome and Acute Myeloid Leukemia or Myelodysplastic Syndromes
leukemia · Childhood Myelodysplastic Syndromes · de Novo Myelodysplastic Syndromes · Secondary Myelodysplastic Syndromes
Source: ClinicalTrials.gov NCT00369317 ↗
Enrolled (actual)
205
Serious AEs
1.5%
Results posted
Jan 2015
Primary outcomePrimary: Event-free Survival (EFS) at 3 Years — 90.1 percentage
Summary
This phase III trial is studying how well combination chemotherapy works in treating young patients with Down syndrome and acute myeloid leukemia or myelodysplastic syndromes. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Event-free Survival (EFS) at 3 Years |
90.1 | — |
| PRIMARY Overall Survival (OS) at 3 Years |
92.7 | — |
| SECONDARY Induction Remission Rate |
0.984615 | — |
| SECONDARY Percentage of Patients Experiencing Grade 3 or 4 Toxicity Assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 |
0.911765 | — |
| SECONDARY Prevalence of Leukemia Phenotype of DS Patients < 4 Years of Age at Diagnosis by Flow Cytometry |
0.45122 | — |
| SECONDARY Prevalence of of GATA1 Mutations of DS Patients < 4 Years of Age at Diagnosis |
0.891304 | — |
| SECONDARY Proportions of Patients in Morphologic Remission With Positive MRD by Flow Cytometry |
0.0935254 | — |
| SECONDARY Cytarabine Drug Sensitivity by R-Strip (MicroMath) Curve Fitting Program |
306.156034 | — |
| SECONDARY Gene Expression Profiles by Microarrays |
— | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis DS or DS mosaicism by karyotype or chromosomal analysis
- Diagnosis of myelodysplastic syndromes (MDS) with 90 days of age AND meets either of the following criteria:
- At least 30% blasts in the bone marrow regardless of time since resolution of TMD
- More than 8 weeks since resolution of TMD with ≥ 5% blasts in the bone marrow
- Immunophenotype required for study entry
- No promyelocytic leukemia
- Shortening fraction ≥ 27% by echocardiogram OR ejection fraction ≥ 50% by radionuclide angiogram
- Bilirubin ≤ 1.5 times upper limit of normal (ULN)
- AST or ALT 94%
- No prior chemotherapy, radiotherapy, or any antileukemic therapy
- Intrathecal cytarabine therapy given at diagnosis allowed
- Prior therapy for TMD allowed
Data sourced from ClinicalTrials.gov (NCT00369317). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.