Ven/Aza bridging to allo-HSCT in R/R AML: A real-world VENAURA registry study

Results from a multicenter, retrospective VENAURA registry study, evaluating post-transplant outcomes in 75 patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) after venetoclax + azacitidine (Ven/Aza) salvage, were published in Bone Marrow Transplantation by Tauveron-Jalenques et al. The primary endpoint was overall survival (OS). Secondary endpoints included cumulative incidence of relapse (CIR), non-relapse mortality (NRM), refined graft-versus-host disease (GvHD) and relapse-free survival (GRFS), and rates of acute (a) and chronic (c) GvHD.

Key data: At a median follow-up of 16.9 months, the estimated 2‑year OS was 61.4% (95% confidence interval [CI], 49.5–68.1%), and the median OS was not reached (NR). Post-transplant OS among patients in molecular relapse salvaged with Ven/Aza and bridged to allo-HSCT was 100%. The 2‑year CIR was 35.1% (95% CI, 20–50.2%) and 2‑year NRM was 10.6% (95% CI, 9.8–23.3%). The cumulative incidence of Grade 2–4 aGvHD at Day 100 was 12%, with a median time to aGvHD of 53 days. The 2‑year cumulative incidence of any grade cGvHD was 22.6%, with a median time to cGvHD of 6 months. Positive donor cytomegalovirus (CMV) status was associated with higher risk of cGvHD (p = 0.02) vs CMV negativity. Overall median GRFS was 39.7 months with a 2‑year estimated GRFS rate of 53.1%.

Key learning: Ven/Aza represents a feasible and well-tolerated bridge-to-transplant strategy in patients with R/R AML. Prospective, randomized studies are warranted to validate this approach.