A recent multicentre study has highlighted the potential of venetoclax combined with decitabine as an effective first-line treatment for older patients with acute myeloid leukaemia (AML) who are eligible for allogeneic haematopoietic stem-cell transplantation (HSCT). Conducted across 20 centres in Italy, the phase 2 trial involved 93 patients aged 60 to 74 with newly diagnosed, intermediate to high-risk AML.
The therapy demonstrated impressive results, with 69% of participants achieving complete remission (CR) after induction. Furthermore, 57% of all patients went on to undergo allogeneic HSCT in complete remission. Notably, 83% of patients who reached CR proceeded with HSCT. However, five patients relapsed before transplantation, and four died due to relapse complications.
The trial used venetoclax, an oral medication, alongside intravenous decitabine. The regimen involved a gradual dose increase of venetoclax and standard decitabine cycles, with two to four cycles total. The study found that adverse events, including infections, neutropenia, and cardiac issues, were prevalent, with 53% of participants experiencing grade ≥3 side effects. Seven deaths occurred due to infections, including pneumonia and COVID-19.
Despite the challenges, the findings suggest that this treatment combination improves the feasibility of allogeneic HSCT in older AML patients, providing an effective bridge to transplantation and enhancing long-term outcomes for a difficult-to-treat population.
Helena Bradbury, EMJ
Reference
Frangoul H et al. Exagamglogene autotemcel for severe sickle cell disease. N Engl J Med. 2024;390:1649-62.
