Key clinical point: Addition of tosedostat to low-dose cytosine arabinoside (LDAC) did not confer any additional clinical benefit compared with LDAC alone in older patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy.
Major finding: There was no significant benefit of adding tosedostat to LDAC vs. LDAC alone on overall response (25% vs. 18%; P = .22) and 2-year overall survival (16% vs. 12%; P = .8). Overall rates of grade 3 or higher toxicity were low, but diarrhea and cardiac toxicity were significantly higher with tosedostat.
Study details: Findings are from LI-1 trial including 243 older (age, 60 years or older) patients with de novo or secondary AML or high-risk myelodysplastic syndrome who were unfit for intensive chemotherapy. Patients were randomly allocated to receive either LDAC (n=121) or LDAC+tosedostat (n=122).
Disclosures: This study was supported by CTI Biopharma, Blood Cancer UK, the Haematology Clinical Trials Unit, and the Centre for Trials Research, Cardiff University. No author disclosures were reported.
Source: Dennis M et al. Br J Haematol. 2021 May 7. doi: 10.1111/bjh.17501 .