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.
Susan Hau is a distinguished researcher in the field of cancer cell therapy, with a particular focus on T cell-based approaches and cancer vaccines. Her work spans several innovative treatment modalities, including CAR T-cell therapy, TIL (Tumor-Infiltrating Lymphocyte) therapy, and NK (Natural Killer) cell therapy.
Hau's expertise lies in cancer cell biology, where she has made significant contributions to understanding the complex interactions between immune cells and tumors.
Her research aims to enhance the efficacy of immunotherapies by manipulating the tumor microenvironment and exploring novel ways to activate and direct immune responses against cancer cells.
Throughout her career, Hau has collaborated with leading professors and researchers in the field of cancer treatment, both in the United States and China.
These international experiences have broadened her perspective and contributed to her innovative approach to cancer therapy development.
Hau's work is particularly focused on addressing the challenges of treating advanced and metastatic cancers. She has been involved in clinical trials evaluating the safety and efficacy of various immunotherapy approaches, including the promising Gamma Delta T cell therapy.
