Oral azacitidine

The randomized, double-blind, placebo-controlled phase III QUAZAR AML-001 trial (NCT01757535) provided the basis for regulatory approval of oral azacitidine as maintenance therapy for AML.[20] The study included 472 patients with AML who were aged 55 years or older, were within 4 months of first CR or CR with incomplete hematologic recovery after induction chemotherapy with or without consolidation treatment, and were not candidates for allogeneic HCT. Oral azacitidine or placebo was administered at 300 mg daily for 14 days every 28 days. Treatment was continued until disease progression or unacceptable toxicity.

The randomized, double-blind, placebo-controlled phase III QUAZAR AML-001 trial (NCT01757535) provided the basis for regulatory approval of oral azacitidine as maintenance therapy for AML.[20] The study included 472 patients with AML who were aged 55 years or older, were within 4 months of first CR or CR with incomplete hematologic recovery after induction chemotherapy with or without consolidation treatment, and were not candidates for allogeneic HCT. Oral azacitidine or placebo was administered at 300 mg daily for 14 days every 28 days. Treatment was continued until disease progression or unacceptable toxicity.

For patients who received azacitidine, the median OS from randomization was 24.7 months (95% CI, 18.7−30.5) compared with 14.8 months (95% CI, 11.7−17.6) for patients receiving placebo (HR, 0.69; 95% CI, 0.55−0.86; P = .0009).[20][Level of evidence A1]