The US Food and Drug Administration (FDA) has approved treosolvan (Graphabex) in combination with fludarabine as a preparatory regimen for allogeneic hematopoietic stem cell transplantation in patients aged 1 year and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). ), Medixus announced Wednesday.
“This FDA approval provides a beneficial option for adult and pediatric patients, with the potential to enhance overall survival [OS] “With side effects being kept to a minimum,” Filippo Milano, MD, of Fred Hutch Cancer Center in Seattle, said in the company’s press release.
The system was evaluated in MC-FludT.14/L Trial IIwhich compared treosolvan and busulfan plus fludarabine in 570 patients. Eligible patients included adults with AML or MDS, Karnofsky performance status ≥60%, age ≥50 years or hematopoietic cell transplantation comorbidity score >2.
Compared with busulfan, the hazard ratio for OS was 0.67 (95% CI 0.51-0.90) in favor of treosulfan in the randomized population, 0.73 (95% CI 0.51-1.06) in patients with AML and 0.64 (95% CI 0.40-1.02). In patients with MDS.
The most common adverse events included musculoskeletal pain, stomatitis, fever, nausea, edema, infection, and vomiting. Selected grade 3 or 4 nonhematological laboratory abnormalities were increased gamma glutamyltransferase, bilirubin, alanine aminotransferase, aspartate aminotransferase, and creatinine.
Previously reported data The trial showed a 2-year event-free survival rate of 64% in the treosulfan group versus 50.4% in the busulfan group (p<0.0001).
Patients who received treosulfan also had an improvement in 2-year OS rate (87.5% vs. 69%, p=0.0082) and lower rates of transplant-related mortality (12.1% vs. 28.2%, p=0.020) and non-relapse mortality (11.4% vs. 22.6%, p=0.053) compared with patients who received busulfan. Two-year relapse rates were similar between the two groups (24.6% versus 23.3%, respectively). p=0.50).
