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How it is administered
Treosulfan is given 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 medication is reconstituted and then infused over 2 hours each day for three consecutive days (Day -4, -3, and -2) prior to allogeneic stem cell transplantation. The typical dose is 10 g/m² of body surface area per day.
Premedication with antiemetics is recommended before the first dose and continued throughout the course of treosulfan administration.
How it works
Treosulfan is an alkylating agent, which means it works by attaching alkyl groups to DNA. This process, called DNA alkylation, damages the DNA in cells, preventing them from dividing and growing. This is particularly effective against rapidly dividing cells, such as cancer cells found in acute myeloid leukemia (AML).
Treosulfan is a prodrug, meaning it is converted in the body into its active forms, which then exert cytotoxic (cell-killing) effects. In addition to its anti-tumor activity, treosulfan also depletes hematopoietic stem cells and has immunosuppressive effects. These properties make it useful as part of a preparative regimen before allogeneic hematopoietic stem cell transplantation, helping to create space in the bone marrow and suppress the immune system to reduce the risk of transplant rejection.
Common side effects
- Musculoskeletal pain
- Stomatitis (mouth sores)
- Fever (pyrexia)
- Nausea
- Edema (swelling)
- Infection
- Vomiting
- Rash
- Diarrhea
- Headache
- Febrile neutropenia (fever with low white blood cell count)
- Abdominal pain
- Hypertension (high blood pressure)
- Hemorrhage (bleeding)
- Fatigue
- Constipation
- Tachycardia (fast heart rate)
- Hepatotoxicity (liver toxicity)
All patients will experience myelosuppression (low blood counts), which is an intended effect to prepare for transplantation. Serious but less common side effects include seizures, skin disorders, injection site reactions, and risk of secondary cancers.
Who Should take it
Treosulfan is indicated for use in combination with fludarabine as part of a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and children (1 year of age and older) with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
This medication is used to prepare the body for receiving new stem cells by reducing the number of existing bone marrow cells and suppressing the immune system. It is especially used for patients who are about to undergo a stem cell transplant as part of their treatment for AML or MDS.
Who should not take it
Treosulfan should not be used in patients who have a known hypersensitivity (allergic reaction) to any component of the drug product.
Additionally, it is 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 at least one week after the last dose. Patients with severe kidney or liver impairment, or those with certain genetic disorders like Fanconi anemia, may be at increased risk for adverse effects and should discuss alternative options with their healthcare provider.
Commonly used with
Treosulfan is most commonly used in combination with fludarabine as part of the preparative regimen for allogeneic hematopoietic stem cell transplantation.
Other medications that may be used alongside treosulfan during the transplant process include antiemetics (to prevent nausea), antibiotics, antivirals, antifungals (to prevent infections), and immunosuppressive drugs such as cyclosporine and methotrexate for graft-versus-host disease prophylaxis.
Commonly tested with
Treosulfan has been tested in clinical trials in combination with fludarabine, compared against regimens using busulfan plus fludarabine for preparative regimens before stem cell transplantation in AML and MDS patients.
In some studies, antithymocyte globulin was also used for patients with unrelated donors, and supportive care medications (such as cyclosporine and methotrexate) were included for graft-versus-host disease prevention.