VIALE-A trial: Long-term efficacy and safety follow-up

The combination of venetoclax (Ven) + azacitidine (Aza) was approved in 2018 for the treatment of patients with newly diagnosed acute myeloid leukemia, aged ≥75 years old, or ineligible for intensive chemotherapy. The phase III VIALE-A trial (NCT02993523) evaluating Ven + Aza vs placebo + Aza met its primary endpoint of improved overall survival (OS).

Recently, Pratz et al.¹ published a long-term efficacy and safety follow-up of the VIALE-A trial in American Journal of Hematology. We summarize the key results below.

Study design¹

• The VIALE-A trial was a multicenter, randomized, placebo-controlled, double-blind study.
• Patients were randomized 2:1 to receive Ven + Aza or placebo + Aza.
• The primary endpoint of the long-term follow-up was OS.

Key findings¹

• N = 431 (intent-to-treat population)
  • N = 286 (Ven + Aza)
  • n = 145 (placebo + Aza)
• The median follow-up was 43.2 months.
• The median OS across patient subgroups was superior for Ven + Aza vs placebo + Aza (Figure 1).

Figure 1. Median OS in the overall patient population and patient subgroups in the VIALE-A trial* 

Aza, azacitidine; OS, overall survival; Ven, venetoclax.
^*Adapted from Pratz et al.^1
†Ven + Aza (n = 61), placebo + Aza (n = 28).
^‡Ven + Aza (n = 23), placebo + Aza (n = 11).
^§Ven + Aza (n = 40), placebo + Aza (n = 18).

• The estimated 24-month OS rate for Ven + Aza was 37.5% vs 16.9% for placebo + Aza.
• The complete response (CR) rate for patients treated with Ven + Aza was 38.8% vs 17.9% for placebo + Aza (p < 0.001).
• The rate of CR/CR with incomplete count recovery in patients treated with Ven + Aza was 66.8% vs 29% for placebo + Aza (p < 0.001).
• Any-grade hematologic and gastrointestinal treatment-emergent adverse events were more frequent in patients treated with Ven + Aza (n = 283) vs placebo + Aza (n =144) (Table 1).

Table 1. Any-grade hematologic and GI TEAEs in ≥20% of patients*

• Treatment discontinuations due to treatment-emergent adverse events occurred in 30% of patients treated with Ven + Aza vs 22% for placebo + Aza.