Phase II Trial of Arsenic Trioxide and Dose-Escalated Cholecalciferol in Myelodysplastic Syndrome
- Determine the complete response rate and the rate of hematological improvement in
patients with myelodysplastic syndromes treated with arsenic trioxide and
cholecalciferol (vitamin D).
- Determine the safety of this regimen in these patients.
- Determine the time to progression to acute myeloid leukemia, defined as blast ≥ 20%, in
patients treated with this regimen.
- Determine overall survival and progression-free survival of patients treated with this
- Determine the effect of this regimen on bone marrow and peripheral blood mononuclear
cell apoptosis and p21 protein expression in these patients.
OUTLINE: This is an open-label, nonrandomized study.
Patients receive oral cholecalciferol (vitamin D)* once daily on days 1-28. Patients also
receive arsenic trioxide IV over 1-4 hours on days 1-5 (week 1) and then twice weekly for 3
weeks (weeks 2-4) for course 1 and twice weekly for 4 weeks for all subsequent courses.
Courses repeat every 28 days for up to 12 months in the absence of disease progression or
NOTE: * Patients who do not achieve a complete hematologic response receive escalating doses
of cholecalciferol (vitamin D) at 3, 6, and 9 months during therapy in the absence of
disease progression and unacceptable toxicity.
At the completion of study treatment, patients are followed for survival.
PROJECTED ACCRUAL: A total of 25-60 patients will be accrued for this study.
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Complete response rate
Istvan Molnar, MD
Comprehensive Cancer Center of Wake Forest University
United States: Institutional Review Board
|Wake Forest University Comprehensive Cancer Center||Winston-Salem, North Carolina 27157-1096|