How it is administered

Treosulfan is administered as an intravenous (IV) infusion. It is supplied as a white, sterile, lyophilized powder for injection in glass vials containing either 1 g or 5 g of treosulfan. The powder is reconstituted and diluted before being given as a two-hour infusion, typically over three consecutive days as part of a preparative regimen for stem cell transplantation.

How it works

Treosulfan is an alkylating agent, which means it works by adding alkyl groups to DNA. This process damages the DNA of cells, preventing them from dividing and growing. In blood cancers such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), this action helps destroy abnormal blood cells and suppresses the immune system to prepare the body for a stem cell transplant.

Treosulfan is a prodrug, meaning it is converted in the body to active compounds that exert the cytotoxic (cell-killing) effects. It depletes hematopoietic stem cells (the cells that make new blood cells) and has both immunosuppressive and antitumor activities. This makes it particularly useful in conditioning regimens before allogeneic hematopoietic stem cell transplantation (alloHSCT), where the goal is to eliminate diseased cells and suppress the immune system to reduce the risk of transplant rejection.

Who Should take it

Treosulfan is indicated for use in combination with fludarabine as part of a preparative (conditioning) regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT). It is approved for adult and pediatric patients (1 year of age and older) with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

This medication is specifically used in patients who are undergoing stem cell transplantation, either because their disease is not responding to other treatments or as part of planned therapy for these blood cancers. The goal is to prepare the bone marrow to accept new, healthy stem cells from a donor.

Who should not take it

Treosulfan should not be taken by patients who have a known hypersensitivity (allergic reaction) to any component of the drug product.

It is also not recommended for use in pregnant women due to the risk of harm to the fetus, and breastfeeding should be avoided during treatment and for one week after the last dose. Patients with Fanconi anemia or other DNA breakage disorders should not use treosulfan, as the safety and efficacy have not been established and there is an increased risk of secondary malignancies. Patients with severe renal or hepatic impairment were not studied and should avoid use unless specifically directed by their healthcare provider.

Commonly used with

Treosulfan is most commonly used in combination with fludarabine as part of a conditioning regimen before allogeneic hematopoietic stem cell transplantation. In some cases, other supportive medications such as antiemetics (for nausea), anticonvulsants (for seizure prevention), and antibiotics, antivirals, or antifungals (to prevent or treat infections) may be used alongside treosulfan.

Commonly tested with

Treosulfan has been tested in clinical trials in combination with fludarabine, and compared to other conditioning regimens such as busulfan plus fludarabine. In some studies, antithymocyte globulin (ATG) is also used for patients with unrelated donors, and graft-versus-host disease prophylaxis with cyclosporine and methotrexate is common.