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Leukemia, and Myelodysplastic Syndrome (MDS)
The Treatment of Down Syndrome Children With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) Under the Age of 4 YearsUnique Dataset IDnci-data-384ClinicalTrial.gov IDNCT00369317
Clinical Trial Title
The Treatment of Down Syndrome Children With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) Under the Age of 4 Years
Trial Summary and Conditions
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.
See data dictionary for more details.
PRIMARY OBJECTIVES: I. Determine the event-free survival (EFS) and overall survival rates in pediatric patients with Down syndrome (DS) and acute myeloid leukemia AML or myelodysplastic syndromes MDS treated with induction therapy comprising cytarabine, daunorubicin hydrochloride, thioguanine, and asparaginase followed by intensification therapy comprising cytarabine and etoposide. II. Determine if the EFS rate in these patients can be increased with an intensified course of cytarabine therapy during induction therapy, compared to the EFS rate of patients in protocol COG-A2971. III. Determine if the number of intrathecal chemotherapy treatments can be reduced in these patients. IV. Determine if the total cumulative anthracycline dose can be reduced in these patients. SECONDARY OBJECTIVES: I. Determine the type and degree of treatment-related toxicity in these patients. II. Determine the prevalence of leukemia phenotype and globin transcription factor 1 (GATA1) mutations of DS patients < 4 years of age at diagnosis. III. Determine the relationship of GATA1 mutations with leukemia phenotype and EFS rates of DS patients < 4 years of age at diagnosis. IV. Determine the relationship of minimal residual disease monitored by flow cytometry and remission status during and after completion of therapy based on bone marrow morphology. V. Examine parameters of in vitro drug sensitivity and in vivo Ara-C pharmacokinetics. VI. Examine gene expression profiles by microarrays and the relationship to leukemia phenotype and outcome. VII. Examine the relationship of functional polymorphisms in phase I and phase II detoxification genes and DNA repair pathways that may modify susceptibility to leukemia and outcome of therapy in DS children. VIII. Assess the effect of karyotypic abnormalities on survival. IX. Establish a DS leukemia cell bank for future biological studies.
Primary: Event-free Survival (EFS) at 3 Years [ Time from study entry to induction failure, relapse, or death assessed at 3 years. ] Overall Survival (OS) at 3 Years [ Time from study entry to death, assessed at 3 years. ] Secondary: Induction Remission Rate [ End of induction therapy (day 112) ] 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 [ From the beginning of induction therapy to the end of intensification therapy ] Prevalence of Leukemia Phenotype of DS Patients < 4 Years of Age at Diagnosis by Flow Cytometry [ At the start of therapy ] Prevalence of of GATA1 Mutations of DS Patients < 4 Years of Age at Diagnosis [ At baseline and at the end of therapy (intensification) or disease relapse ] Proportions of Patients in Morphologic Remission With Positive MRD by Flow Cytometry [ After Induction I therapy (day 28 from start of therapy) ] Cytarabine Drug Sensitivity by R-Strip (MicroMath) Curve Fitting Program [ Days 1, 2, 8, and 9 of induction II ] Cytarabine Drug Sensitivity by R-Strip (MicroMath) Curve Fitting Program [ Days 1, 2, 8, and 9 of induction II ] Cytarabine Drug Sensitivity by R-Strip (MicroMath) Curve Fitting Program [ Days 1, 2, 8, and 9 of induction II ] Gene Expression Profiles by Microarrays [ At baseline and at the time of relapse (if available) ]
Below are the clinical trial(s) associated with this dataset (all links are to ClinicalTrials.Gov):
NCT00369317-D1 is the only dataset associated with this publication. The dataset contains the CONSORT flow of patient enrollment, baseline characteristics, adverse events, MRD, cytogenetics, and primary outcome analyses for patients on AAML0431 as reported in the manuscript. Also, data for patients on A2971 are provided in order to compare brief baseline characteristics and outcome to patients on AAML0431.
Available Downloads: NCT00369317-D1
To gain access to the data and analytic tools click here.
Data Dictionary: NCT00369317-D1-Data-Dictionary.pdf