A Multicenter, Randomized, Open-label, Parallel-group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)
Comparison/Control Interventions offered the physician three options:
- Best supportive care (BSC) alone,
- Low-dose cytarabine subcutaneously for 14 days every 28 to 42 days, or
- Standard chemotherapy administered for induction as a continuous intravenous infusion
of cytarabine over 7 days plus an anthracycline (daunorubicin, idarubicin, or
mitoxantrone) on Days 1, 2, and 3; and, for those eligible, 1 or 2 consolidation cycles
administered as continuous intravenous infusions of cytarabine for 3 to 7 days with the
same anthracycline that was used at induction on Days 1 and 2 (each cycle between 28 to
70 days from the start of the previous cycle).
All three options included best supportive care. Neither the experimental group
(azacitidine) nor any of the comparison/control options allowed use of erythropoietin.
Duration of Intervention: Patients will be treated until death, withdrawal, unacceptable
toxicity or conclusion of the study.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label
Kaplan-Meier Estimates for Median Time to Death From Any Cause
Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact.
Day 1 (randomization) to 42 months
United States: Food and Drug Administration
AZA PH GL 2003 CL 001
|Indiana University Cancer Center||Indianapolis, Indiana 46202-5265|
|Washington University School of Medicine||Saint Louis, Missouri 63110|
|Oregon Cancer Center||Portland, Oregon 97201-3098|
|Mount Sinai Medical Center||New York, New York 10029|
|Western Pennsylvania Cancer Institute||Pittsburgh, Pennsylvania 15224|
|Froedtert Memorial Lutheran Hospital||Milwaukee, Wisconsin 53226|
|Case Western Reserve University||Cleveland, Ohio 44106|
|University of Alabama School of Medicine||Birmingham, Alabama 35294|