treosulfan at a glance
Medicine class
Alkylating antineoplastic prodrug
Main role
Conditioning before allogeneic haematopoietic stem cell transplantation
How it is given
Intravenous infusion under an experienced transplant specialist
Key expected effect
Temporary profound suppression of blood-cell production before transplanted stem cells restore it
What is treosulfan used for?
The selected UK product is licensed with fludarabine as conditioning treatment before an allogeneic stem cell transplant.
Adults with malignant or non-malignant diseases.
Children older than one month with malignant or non-malignant diseases.
How does treosulfan work?
Treosulfan is converted into active epoxide compounds that cross-link DNA. This damages and depletes bone-marrow precursor and immune cells, helping prepare the body for donor stem cells.
treosulfan preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 1 g, 5 g
How to take treosulfan
Treosulfan is prepared by trained staff and given into a vein as part of a specialist transplant programme. Follow the transplant team’s eating, drinking and mouth-care instructions.
Tell the team about all medicines, supplements, vaccines, allergies and current infection symptoms.
Report pain, burning, redness or swelling at the infusion site immediately.
Follow prescribed mouth-care measures because soreness and ulcers are very common.
treosulfan side effects
Common or expected effects
- Very low white cells, red cells and platelets
- Sore mouth or mouth ulcers
- Nausea, vomiting, diarrhoea or abdominal discomfort
- Fatigue and reduced appetite
- Fever or infections
- Rash, itching or hair loss
- Changes in liver blood tests
Get urgent medical advice
- Fever, shivering or other infection symptoms
- Unexpected or severe bleeding
- New or worsening breathlessness, chest pain or coughing up blood
- Markedly reduced urine, blood in urine or painful urination
- Yellow skin or eyes, abdominal swelling or significant liver-test abnormalities
- Pain, burning, swelling or redness around the infusion line
treosulfan in pregnancy
Treosulfan is contraindicated during pregnancy because it damages DNA and fetal harm cannot be excluded. Sexually active people who could conceive or father a child must use effective contraception during treatment and for six months afterwards. Discuss pregnancy or fertility preservation with the specialist team before treatment whenever possible.
treosulfan while breastfeeding
Breastfeeding must be stopped during treatment because it is unknown whether treosulfan enters breast milk and risk to the child cannot be excluded. The transplant and maternity teams can advise when breastfeeding could be reconsidered.
treosulfan interactions
The transplant team should review all prescribed, over-the-counter and complementary products before treosulfan. Important examples include:
Tacrolimus and other narrow-therapeutic-index CYP3A4 or CYP2C19 substrates
Treosulfan may alter exposure; these medicines should not be given during treosulfan treatment.
Live vaccines
Administration is contraindicated because profound immune suppression may allow vaccine-related infection.
Fludarabine
This is an intended component of the licensed conditioning combination, although treosulfan’s effect on fludarabine pharmacokinetics is unknown.
Common questions about treosulfan
Answers are fully visible for fast scanning and source review.
Is treosulfan chemotherapy?
Yes. It is an alkylating chemotherapy used here for conditioning before a donor stem cell transplant.
Is it used only for cancer?
No. The selected product is licensed before transplantation for malignant and non-malignant diseases.
Why are blood tests needed?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.