According to researchers at The University of Texas MD Anderson Cancer Center, 80% of patients with previously untreated or relapsed/refractory advanced-phase chronic myeloid leukemia (CML)—including both accelerated or myeloid blast phases of the disease—or Philadelphia chromosome-positive acute myeloid leukemia (AML) achieved a bone marrow remission when treated with a novel combination of decitabine, venetoclax and ponatinib. Findings from the Phase II clinical trial, published in The Lancet Haematology , represent an important step forward for patients with advanced-phase CML, who tend to have poor outcomes. Limited data on a standard-of-care approach to treating the disease, highlights the need for investigations into additional therapeutics.
"Over the last decade, there have been very few studies that evaluated a regimen to treat this rare disease and identify a potential standard-of-care treatment," said principal investigator Nicholas Short, M.D., associate professor of Leukemia.
"It is important that we get these patients into a state of marrow remission as this will allow them to be considered for a stem cell transplant . We were able to achieve this response in 80% of patients on this trial." A total of 20 patients were enrolled in the trial, with 14 having myeloid blast-phase CML, four with accelerated-phase CML, and two with Philadelphia chromosome-positive AML.
Overall, 50% of patients achieved a complete remission or complete remission with an incomplete he.