The latest results of a Phase II study evaluating the addition of venetoclax to the intensive chemotherapy treatment of cladribine, idarubicin and cytarabine (CLIA) as a frontline therapy demonstrated high rates of disease control and remissions in younger patients with newly diagnosed AML and high-risk myelodysplastic syndrome (MDS).
In the study, 96% of patients responded to treatment and 90% had no measurable disease detected in a bone marrow sample.
Patrick Reville, M.D., instructor of Leukemia, presented updated results and longer-term follow-up data at ASH 2022.
“Venetoclax has been a breakthrough for AML patients that are ineligible for intensive therapy. This data continues to demonstrate the benefit of including venetoclax with the CLIA induction regimen,” Reville said. “This regimen is leading to unprecedented response and measurable residual disease-negativity rates. As we continue to follow participants, we are encouraged by their long-term outcomes and survival.”
The single-centre, single-arm trial enroled 67 patients with a median age of 48.
Sixty patients had AML and four patients had high-risk MDS. In addition, three patients had a mixed-phenotype acute leukaemia (MPAL).
The composite complete response rate was 96% across all patients and 100% for patients with both MDS and MPAL with a myeloid predominant clone.
Most patients went on to receive a subsequent allogeneic stem cell transplant (alloSCT), including 70% of those who responded to treatment.
Encouragingly, with a median follow-up of just over two years, the median duration of response, event-free survival and overall survival have not yet been reached.
At 12 months, the estimated event-free survival rate is 70% and the estimated overall survival rate is 86%.
Seventy-four percent of responding patients are estimated to have an ongoing response at 12 months.
The most common non-haematologic adverse event that participants experienced was febrile neutropenia, which was managed.
Researchers continue to follow patients and study this treatment regimen as a safe and effective induction treatment strategy for this patient population.