Venetoclax with low-dose cytarabine safe, effective for untreated acute myeloid leukaemia

In patients with untreated acute myeloid leukaemia (AML) ineligible for intensive chemotherapy, the combination of venetoclax with low-dose cytarabine (+LDAC) may be a safe and effective treatment option, inducing high rates of response and reducing mortality risk, a recent study has found.

The researchers conducted a follow-up of the VIALE-C study, a randomized, double-blind, placebo-controlled phase III trial that compared venetoclax+LDAC vs placebo+LDAC. The present analysis included 211 patients (143 on venetoclax, 68 on placebo), in whom survival outcomes were assessed over a median follow-up of 17.5 months.

After follow-up, median overall survival (OS) was longer in venetoclax patients (8.4 vs 4.1 months), corresponding to a significantly lower risk of mortality (hazard ratio [HR], 0.70, 95 percent confidence interval [CI], 0.50–0.98; p=0.04). Adjusting for covariates did not attenuate this effect.  

The venetoclax combination therapy was also superior to placebo in terms of secondary efficacy endpoints, including the rate of investigator-assessed complete response (CR; 28.0 percent vs 7.4 percent), and CR with incomplete (48.3 percent vs 13.2 percent) or partial (48.3 percent vs 14.7 percent; p<0.001 for all) haematologic recovery.

In terms of safety, 99 percent of both study arms developed at least one adverse event (AE). Common side effects included neutropaenia, thrombocytopaenia, and nausea, all of which occurred at marginally higher rates in the venetoclax arm. Nevertheless, safety profiles were comparable between treatments, with no differences in grade ≥3 AEs and death, suggesting that venetoclax was a safe treatment option.