FDA Approval of Treosulfan for AML and MDS Transplant Preparation
On January 21, 2025, the Food and Drug Administration sanctioned treosulfan (Grafapex, medac GmbH), an alkylating agent, in conjunction with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients aged 1 year and older diagnosed with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Effectiveness and Safety The efficacy was assessed in the MC-FludT.14/L Trial II (NCT00822393), a randomized active-controlled study comparing treosulfan to busulfan in conjunction with fludarabine as a preparative regimen for allogeneic transplantation. Eligible patients comprised adults aged 18 to 70 years with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS), a Karnofsky performance level of 60% or higher, and either an age of 50 years or older or a hematopoietic cell transplantation comorbidity index (HCTCI) score exceeding 2. A total of 570 patients were randomized to receive either treosulfan (n=280) or busulfan (n=290). The primary effectiveness outcome measure was overall survival (OS), defined as the duration from randomization to death from any cause. The hazard ratio for overall survival (stratified by donor type and risk category) relative to busulfan was 0.67 (95% CI: 0.51, 0.90) in the randomized cohort, 0.73 (95% CI: 0.51, 1.06) in patients with acute myeloid leukemia (AML), and 0.64 (95% CI: 0.40, 1.02) in patients with myelodysplastic syndromes (MDS). The predominant adverse effects (≥20%) included musculoskeletal discomfort, stomatitis, pyrexia, nausea, edema, infection, and vomiting. Identified Grade 3 or 4 nonhematological laboratory abnormalities included elevated GGT, elevated bilirubin, elevated ALT, elevated AST, and elevated creatinine.
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