Treosulfan (By injection)

Treosulfan (tree-oh-SUL-fan)

Used before an allogeneic hematopoietic stem cell transplant (HSCT) to treat acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

Brand Name(s):

Grafapex

There may be other brand names for this medicine.

When This Medicine Should Not Be Used:

This medicine is not right for everyone. You should not receive it if you had an allergic reaction to treosulfan.

How to Use This Medicine:

Injectable

• Your doctor will prescribe your dose and schedule. This medicine is given through an IV placed in a vein. It must be given slowly, so the IV will have to remain in place for at least 2 hours.
• You will receive this medicine while you are in a hospital or cancer treatment center. A nurse or other trained health professional will give you this medicine.
• Your doctor will give you other medicines (medicine to prevent vomiting) before you receive and during treatment with this medicine.

Drugs and Foods to Avoid:

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

• Some medicines can affect how treosulfan works. Tell your doctor if you are using midazolam.

Warnings While Using This Medicine:

• This medicine may cause birth defects if either partner is using it during conception or pregnancy. Tell your doctor right away if you or your partner becomes pregnant. If you can get pregnant, your doctor may do tests to make sure you are not pregnant within 7 days before receiving this medicine. Use an effective birth control during treatment and for at least 6 months after the last dose. Males who are receiving this medicine, with female partners who can become pregnant, must use effective birth control during treatment and for at least 3 months after the last dose.
• Do not breastfeed during treatment and for 1 week after the last dose.
• Tell your doctor if you have kidney disease, liver disease, blood problems, or any infection.
• This medicine may cause the following problems:
  • Seizures
  • Injection site reactions and tissue necrosis
  • Increased risk of secondary cancer
• This medicine may cause skin reactions (including rash, dermatitis). Always clean sweaty areas (including armpit, groin, genital area, inframammary line) with a washcloth and clean water. Do not apply any cream during treatment. Do not wear tight clothing.
• This medicine may make you bleed, bruise, or get infections more easily. Take precautions to prevent illness and injury. Wash your hands often.
• This medicine could cause infertility. Talk with your doctor before using this medicine if you plan to have children.
• Medicines used to treat cancer are very strong and can have many side effects. Before receiving this medicine, make sure you understand all the risks and benefits. It is important for you to work closely with your doctor during your treatment.
• Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments.

Possible Side Effects While Using This Medicine:

Call your doctor right away if you notice any of these side effects:

• Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
• Dark urine or pale stools, nausea, vomiting, loss of appetite, stomach pain, yellow skin or eyes
• Fever, chills, cough, sore throat, body aches
• Muscle or bone pain
• Pain, itching, burning, swelling, or a lump under your skin where the needle is placed
• Rapid weight gain, swelling in your hands, ankles, or feet
• Seizures
• Unusual bleeding, bruising, or weakness

If you notice these less serious side effects, talk with your doctor:

• Darkening of the skin
• Sores or white patches on your lips, mouth, or throat

If you notice other side effects that you think are caused by this medicine, tell your doctor

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088

Last Updated: 5/4/2025