Phase II study of AZD2171 for the treatment of patients with acute myelogenous leukemia.

6574 Background: Inhibition of vascular endothelial growth factor receptors (VEGFR) can block growth and trigger apoptosis in leukemia cells. AZD2171 (cediranib) is a highly potent, orally bioavailable, VEGFR-1/2 inhibitor. We conducted a phase II study of the efficacy of AZD2171 for the treatment of AML. Methods: Adults with AML (relapsed, refractory, or untreated > 60 years) were eligible if they had ≤ 2 prior induction courses, adequate organ function, and ECOG 0-2. The primary endpoint was proportion of responses according to the IWG criteria assessed at one and every 3 months. Pts with stable disease could remain on treatment. Pts were initially treated with cediranib 45 mg daily but due to toxicity, the starting dose was lowered to 30 mg daily. Results: 7 pts were treated at 45 mg; 4 of 7 pts experienced grade 3 toxicity (1 mucositis, 2 fatigue, and 1 hypertension) prompting lowering the starting dose to 30 mg for an additional 16 pts. A total of 23 pts (median age 70 years) were enrolled, and all a...