Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)
- Determine whether very low-dose cytarabine can improve event-free survival (EFS) rates
in infants with high-risk transient myeloproliferative disorder (TMD), using high-risk
TMD patients from clinical trial COG-A2971 for historic comparison, and in infants with
intermediate-risk TMD, using intermediate-risk TMD patients from clinical trial
COG-A2971 for historic comparison.
- Maintain the current high overall EFS rate in low-risk TMD patients.
- Assess the toxicity of this regimen in these patients.
OUTLINE: This is a nonrandomized, multicenter, crossover study. Patients are stratified
according to disease risk (high or intermediate vs low).
- Group I (patients with high- or intermediate-risk transient myeloproliferative disorder
[TMD]): Patients receive very low-dose cytarabine subcutaneously twice daily on days
1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease
progression or unacceptable toxicity. Patients achieving stable disease or complete or
hepatic clinical remission undergo observation.
- Group II (patients with low-risk TMD): Patients are observed. If symptoms of
intermediate- or high-risk disease develop, patients may crossover to group I.
After completion of study treatment, patients are followed periodically for 10 years.
PROJECTED ACCRUAL: A total of 180 patients will be accrued for this study.
Allocation: Non-Randomized, Primary Purpose: Treatment
April D. Sorrell, MD
Cancer Institute of New Jersey