Treosulfan
Treosulfan is an alkylating chemotherapeutic agent used primarily in the treatment of ovarian cancer and as part of conditioning regimens before stem cell transplantation. It works by cross-linking DNA strands, preventing cancer cells from dividing and ultimately causing their death.
Treosulfan is a chemotherapy drug used for ovarian cancer and stem cell transplant conditioning that kills cancer cells by damaging their DNA.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- L01AB02
- RxNorm
- 38508
- Generic
- Treosulfan
- Class
- atc-l01
- Brands
- Grafapex
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- injection, capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Treosulfan is primarily used to treat advanced ovarian cancer, particularly epithelial ovarian cancer. It is also employed as part of conditioning regimens prior to allogeneic hematopoietic stem cell transplantation in patients with hematological malignancies such as leukemia, myelodysplastic syndromes, and lymphomas.
Treosulfan is a prodrug that is converted to diepoxybutane in the body. As an alkylating agent, it cross-links DNA strands by forming covalent bonds with the nitrogenous bases in DNA. This DNA damage prevents replication and transcription, leading to cell cycle arrest and apoptosis in rapidly dividing cells, including cancer cells.
Treosulfan is administered intravenously and is contraindicated in pregnancy due to its teratogenic potential. Common side effects include bone marrow suppression, nausea, vomiting, and fatigue. Due to its cytotoxic nature, handling requires appropriate safety precautions. Treatment should be supervised by oncologists experienced in chemotherapy with regular monitoring of blood counts and organ function.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 25, 2026. · Read our editorial policy