- In a phase 2 study of patients with relapsed/refractory (R/R) and newly diagnosed acute myeloid leukemia (AML) who were unsuitable for intensive therapy, pazopanib was well tolerated but demonstrated limited efficacy.
Why this matters
- After complete response, most patients with AML experience relapse within 1-2 years of initiating therapy; new treatment options are needed.
- Pazopanib is an oral adenosine triphosphate (ATP)-competitive tyrosine kinase inhibitor (TKI) currently approved for the treatment of renal cancer.
- Phase 2 study to investigate pazopanib in 25 patients with AML who were ineligible for intensive therapy.
- Median patient age: 76 (range, 52-86) years.
- 15 (75%) patients had R/R disease; 25% had newly diagnosed disease.
- Funding: GSK; Novartis, Germany.
- No significant decrease in bone marrow (BM) microvessel density (P=.677).
- Pazopanib-associated grade 3 adverse events included nausea (n=2), fatigue (n=3), hypertension (n=1), and increased blood bilirubin (n=1).
- 10% of patients achieved partial response (defined as >50% reduction in elevated blast counts in peripheral blood and bone marrow vs baseline).
- Median PFS: 65 (95% CI, 29-105) days.
- Median OS: 191 (95% CI, 87-435) days.
- 35% 1-year survival.
- Single-group study design.