Posted: Tuesday, March 17, 2020
In an open-label, non-inferiority, phase III trial, Dietrich Wilhelm Beelen, MD, of the West German Cancer Centre, University Hospital of Essen, Germany, and colleagues studied the effects of preparative regimens before allogenic haemopoietic stem cell transplantation in patients with acute myeloid leukemia (AML). Treosulfan was found to be as effective as busulfan when used in combination with fludarabine preceding stem cell transplantation. The results of their study were reported in The Lancet Haematology.
A total of 476 patients were enrolled and randomly assigned 1:1 to receive either busulfan and transplantation (n = 240) or treosulfan and transplantation (n = 221; 1 patient received treosulfan alone). Both the busulfan and treosulfan groups also received fludarabine.
The primary outcome was event-free survival two years after receiving a conditioning treatment prior to transplantation. Safety was assessed in all patients who received treatment; efficacy was assessed in all patients who received both treatment and transplantation. Serious adverse events were reported in up to 8% of patients in both groups.
The median follow-up was 15.4 months for patients treated with treosulfan and 17.4 months for those treated with busulfan. The 2-year event-free survival was 64% in the treosulfan group and 50% in the busulfan group.
“Improved outcomes in patients treated with the treosulfan-fludarabine regimen suggest its potential to become a standard preparative regimen,” concluded Dr. Beelen and colleagues.
Disclosure: For full disclosures of the study authors, visit thelancet.com.