Introduction to Treosulfan-Grafapex: What kind of medicine is it used to treat?

Treosulfan（Treosulfan)-Grafapex is an alkylating agent mainly used to treat acute myeloid leukemia（AML) andmyelodysplastic syndrome (MDS) preparation plan for allogeneic hematopoietic stem cell transplantation (alloHSCT), applicable to adults and children 1 year and older. The drug, used in combination with fludarabine, is designed to use conditioning therapy to remove residual cancer cells from the patient's body and weaken their immune system, thereby reducing the risk of rejection of the donor cells.

As a DNA alkylating agent, Grafapex interferes with DNA replication and RNA transcription, destroying nucleic acid functions and leading to cell death. FDA approval was supported by data from the MC-Flud T.14/L trial II (NCT00822393), which compared triosulfan plus fludarabine (n=280) with busulfan plus fludarabine (n=290). The results showed that patients taking the trioxofan regimen had a 33% lower risk of death overall (HR, 0.67; 95% CI, 0.51-0.90), patients with acute myeloid leukemia had a 27% lower risk of death (hazard ratio, 0.73; 95% CI, 0.51-1.06), and patients with MDS had a 36% lower risk (HR, 0.64; 95% CI is 0.40-1.02).

Grafapex was administered via intravenous infusion in combination with fludarabine for three days (days -4, -3 and -2) before administration of the hematopoietic stem cells. Common adverse reactions include musculoskeletal pain, stomatitis, pyrexia, nausea, edema, infection, and vomiting. In addition, non-hematological laboratory abnormalities also require attention, including increases in GGT, bilirubin, ALT, AST, and creatinine. There are also warnings and precautions that may cause seizures, dermatoses, injection site reactions, and local tissue necrosis, as well as an increased risk of secondary malignancies and embryonic-fetal toxicity.

Reference materials:https://www.drugs.com/grafapex.html