Treosulfan

Indications

Treosulfan is an anticancer medicine used together with Fludarabine as conditioning treatment before an allogeneic blood stem cell transplant (a transplant from a donor) in adults and children with certain blood cancers and some non-cancer blood disorders. In some countries it is also used to treat advanced ovarian cancer.

How It Works

Treosulfan is an alkylating agent. In the body it is converted into active substances that attach to DNA and cross-link its strands, which stops cells from copying their DNA and dividing. This kills cancer cells and clears the patient's bone marrow and immune cells, making room for the donor stem cells and helping prevent their rejection.

Dosage and Administration

Treosulfan is given by a doctor or nurse in a specialised hospital unit as an infusion into a vein, usually once a day for 3 days before the transplant. The dose is based on body surface area and the treatment plan, and is as prescribed by the transplant specialist.

Interactions

• Other chemotherapy and radiotherapy add to bone marrow and other toxic effects.
• Live vaccines must not be given during and for some time after treatment.
• Tell the team about all medicines, including Phenytoin and other anti-seizure medicines, as its effect on these is not fully known.

Contraindications

• Allergy to Treosulfan.
• Active uncontrolled infection.
• Severe heart, lung, liver or kidney disease that makes transplant conditioning unsafe.
• Pregnancy and breastfeeding.
• Use with live vaccines.

Side Effects

Very common effects include severe lowering of blood counts (risk of infection, bleeding and anaemia), nausea, vomiting, mouth sores (mucositis), diarrhoea, stomach pain, tiredness, fever, skin rash or darkening, and hair loss. Other possible effects include liver problems, lung inflammation, seizures in young children, heart rhythm problems, infertility and, later, new cancers.

Get urgent medical help if you have a fever or chills, bleeding or unusual bruising, severe mouth pain that stops eating or drinking, breathlessness, yellow skin or eyes, a seizure, or confusion.

Pregnancy and Lactation

Treosulfan can harm an unborn baby and must not be used in pregnancy. Women and men must use effective contraception during treatment and for at least 6 months after. Breastfeeding must be stopped. Treatment may cause permanent infertility; ask about sperm or egg storage beforehand.

Precautions and Warnings

• Given only by teams experienced in stem cell transplantation.
• Blood counts, liver and kidney function are monitored closely.
• Avoid contact with people who have infections and report fever immediately.
• Good mouth hygiene helps reduce mouth sores.

Overdose

Overdose mainly causes more severe bone marrow damage and gut toxicity. There is no specific antidote; supportive treatment is given in hospital.

Therapeutic Class

Antineoplastic alkylating agent; conditioning agent for stem cell transplant.

Storage

Store as directed on the pack, below 30°C and away from light. It is stored and prepared by hospital staff; do not refrigerate the prepared solution unless the pack says so.