Busulfan (Myleran®, Busulfex®)

Author: Marisa Healy, BSN, RN
Last Reviewed: August 25, 2023

Pronounce: bue-SUL-fan

Classification: Alkylating Agent

About: Busulfan (Myleran®, Busulfex®)

Busulfan kills cancer cells through a process called alkylation. Alkylation damages the DNA of cells, which prevents them from dividing and causes them to die. Since cancer cells divide faster and with less error-correcting than healthy cells, cancer cells are more sensitive to this damage.

How to Take Busulfan

Busulfan comes as a tablet to be taken by mouth. It also comes in an intravenous (IV, into a vein) form. The dose of the medication is based on your body size. It can be given alone or with other drugs.

Storage and Handling

Store your medication in the original, labeled container at room temperature and in a dry location (unless otherwise directed by your healthcare provider or pharmacist). This medication should not be stored in a pillbox. Keep containers out of reach of children and pets.

If a caregiver prepares your dose for you, they should consider wearing gloves or pour the pills directly from their container into the cap, a small cup, or directly into your hand. They should avoid touching the pills. They should always wash their hands before and after giving you the medication. Pregnant or nursing women should not prepare the dose for you. Ask your oncology team where to return any unused medication for disposal. Do not flush down the toilet or throw in the trash.

Where do I get this medication?

Busulfan is available through select specialty pharmacies. Your oncology team will work with your prescription drug plan to identify an in-network specialty pharmacy for distribution of this medication and shipment directly to your home.

Insurance Information

This medication may be covered under your prescription drug plan. Patient assistance may be available to qualifying individuals depending upon prescription drug coverage. Co-pay cards, which reduce the patient co-pay responsibility for eligible commercially (non-government sponsored) insured patients, may also be available. Your care team can help you find these resources, if they are available.

This medication is covered under Medicare part B for Medicare recipients. Make sure your pharmacist knows to process this prescription through your Medicare part B and NOT part D.

Possible Side Effects

There are a number of things you can do to manage the side effects of busulfan. Talk to your care team about these recommendations. They can help you decide what will work best for you. These are some of the most common or important side effects:

Low White Blood Cell Count (Leukopenia or Neutropenia)

White blood cells (WBC) are important for fighting infection. While receiving treatment, your WBC count can drop, putting you at a higher risk of getting an infection. You should let your doctor or nurse know right away if you have a fever (temperature greater than 100.4°F OR 38°C), sore throat or cold, shortness of breath, cough, burning with urination, or a sore that doesn't heal.

Tips to preventing infection:

  • Washing hands, both yours and your visitors, is the best way to prevent the spread of infection.
  • Avoid large crowds and people who are sick (i.e.: those who have a cold, fever or cough or live with someone with these symptoms).
  • When working in your yard, wear protective clothing including long pants and gloves.
  • Do not handle pet waste.
  • Keep all cuts or scratches clean.
  • Shower or bathe daily and perform frequent mouth care.
  • Do not cut cuticles or ingrown nails. You may wear nail polish, but not fake nails.
  • Ask your oncology care team before scheduling dental appointments or procedures.
  • Ask your oncology care team before you, or someone you live with, has any vaccinations.

Low Red Blood Cell Count (Anemia)

Your red blood cells are responsible for carrying oxygen to the tissues in your body. When the red cell count is low, you may feel tired or weak. You should let your oncology care team know if you experience any shortness of breath, difficulty breathing or pain in your chest. If the count gets too low, you may receive a blood transfusion.

Low Platelet Count (Thrombocytopenia)

Platelets help your blood clot, so when the count is low you are at a higher risk of bleeding. Let your oncology care team know if you have any excess bruising or bleeding, including nose bleeds, bleeding gums or blood in your urine or stool. If the platelet count becomes too low, you may receive a transfusion of platelets.

  • Do not use a razor (an electric razor is fine).
  • Avoid contact sports and activities that can result in injury or bleeding.
  • Do not take aspirin (salicylic acid), non-steroidal, anti-inflammatory medications (NSAIDs) such as Motrin/Advil (ibuprofen), Aleve (naproxen), Celebrex (celecoxib) etc. as these can all increase the risk of bleeding. Please consult with your healthcare team regarding use of these agents and all over the counter medications/supplements while on therapy.
  • Do not floss or use toothpicks and use a soft-bristle toothbrush to brush your teeth.

Nausea and/or Vomiting

Talk to your oncology care team so they can prescribe medications to help you manage nausea and vomiting. In addition, dietary changes may help. Avoid things that may worsen the symptoms, such as heavy or greasy/fatty, spicy or acidic foods (lemons, tomatoes, oranges). Try saltines, or ginger ale to lessen symptoms.

Call your oncology care team if you are unable to keep fluids down for more than 12 hours or if you feel lightheaded or dizzy at any time.

Mouth Ulcers (Mucositis)

Certain cancer treatments can cause sores or soreness in your mouth and/or throat. Notify your oncology care team if your mouth, tongue, inside of your cheek or throat becomes white, ulcerated, or painful. Performing regular mouth care can help prevent or manage mouth sores. If mouth sores become painful, your doctor or nurse can recommend a pain reliever.

  • Brush with a soft-bristle toothbrush or cotton swab twice a day.
  • Avoid mouthwashes that contain alcohol. A baking soda and/or salt with warm water mouth rinse (2 level teaspoons of baking soda or 1 level teaspoon of salt in an eight ounce glass of warm water) is recommended 4 times daily.
  • If your mouth becomes dry, eat moist foods, drink plenty of fluids (6-8 glasses), and suck on sugarless hard candy.
  • Avoid smoking and chewing tobacco, drinking alcoholic beverages, and citrus juices.

Decrease in Appetite or Taste Changes

Nutrition is an important part of your care. Cancer treatment can affect your appetite and, in some cases, the side effects of treatment can make eating difficult. Ask your oncology care team about nutritional counseling services at your treatment center to help with food choices.

  • Try to eat five or six small meals or snacks throughout the day, instead of 3 larger meals.
  • If you are not eating enough, nutritional supplements may help.
  • You may experience a metallic taste or find that food has no taste at all. You may dislike foods or beverages that you liked before receiving cancer treatment. These symptoms can last for several months or longer after treatment ends.
  • Avoid any food that you think smells or tastes bad. If red meat is a problem, eat chicken, turkey, eggs, dairy products, and fish without a strong smell. Sometimes cold food has less of an odor.
  • Add extra flavor to meat or fish by marinating it in sweet juices, sweet and sour sauce, or dressings. Use seasonings like basil, oregano, or rosemary to add flavor. Bacon, ham and onion can add flavor to vegetables.

Diarrhea

Your oncology care team can recommend medications to relieve diarrhea. Also, try eating low-fiber, bland foods, such as white rice and boiled or baked chicken. Avoid raw fruits, vegetables, whole-grain breads, cereals, and seeds. Soluble fiber is found in some foods and absorbs fluid, which can help relieve diarrhea. Foods high in soluble fiber include: applesauce, bananas (ripe), canned fruit, orange sections, boiled potatoes, white rice, products made with white flour, oatmeal, cream of rice, cream of wheat, and farina. Drink 8-10 glasses of non-alcoholic, un-caffeinated fluid a day to prevent dehydration.

Fever

Fever can be a side effect of this medication. Contact your care provider if you have a temperature of 100.4°F or 38°C or greater.

Difficulty Sleeping/Insomnia

Busulfan can cause difficulty falling or staying asleep. Talk with your provider about any problems you may have with sleep.

Pain/Aches and Headache

Your healthcare provider can recommend medications and other strategies to help relieve pain.

Electrolyte Abnormalities

This medication can affect the normal levels of electrolytes (potassium, magnesium, etc.) in your body. Your levels will be monitored using blood tests. If your levels become too low, your care team may prescribe specific electrolytes to be given by IV or taken by mouth. Do not take any supplements without first consulting with your care team.

High Blood Sugar

This medication can cause elevated blood sugar levels in patients with and without diabetes. Your oncology care team will monitor your blood sugar. If you develop increased thirst, urination or hunger, blurry vision, headaches or your breath smells like fruit, notify your healthcare team. Diabetics should monitor their blood sugar closely and report elevations to the healthcare team.

Rash

Some patients may develop a rash, scaly skin, or red itchy bumps. Use an alcohol-free moisturizer on your skin and lips; avoid moisturizers with perfumes or scents. Your oncology care team can recommend a topical medication if itching is bothersome. If your skin does crack or bleed, be sure to keep the area clean to avoid infection. Be sure to notify your oncology care team of any rash that develops, as this can be a reaction. They can give you more tips on caring for your skin.

Blood Clots, Heart Attack and Stroke

Busulfan can increase the risk of blood clots, stroke, and heart attack. Symptoms can include: swelling, redness or pain in an extremity, chest pain or pressure, pain in your arm, back, neck or jaw, shortness of breath, numbness or weakness on one side of the body, trouble talking, confusion, or mental status changes. If you experience any of these symptoms, you should contact your oncology care team immediately or go to an emergency room.

Fatigue

Fatigue is very common during cancer treatment and is an overwhelming feeling of exhaustion that is not usually relieved by rest. While on cancer treatment, and for a period after, you may need to adjust your schedule to manage fatigue. Plan times to rest during the day and conserve energy for more important activities. Exercise can help combat fatigue; a simple daily walk with a friend can help. Talk to your healthcare team for helpful tips on dealing with this side effect.

Constipation

There are several things you can do to prevent or relieve constipation. Include fiber in your diet (fruits and vegetables), drink 8-10 glasses of non-alcoholic fluids a day, and keep active. A stool softener once or twice a day may prevent constipation. If you do not have a bowel movement for 2-3 days, you should contact your healthcare team for suggestions to relieve the constipation.

Xerostomia (Dry Mouth)

This medication can cause xerostomia, also known as dry mouth. Xerostomia can be uncomfortable and can affect your speech, swallowing, and dental health. Ways to manage this side effect include:

  • Perform frequent oral hygiene with toothpaste containing fluoride.
  • Floss once a day, if your care team says you can.
  • Chew sugar-free gum or suck on sugar-free candy to stimulate saliva production.
  • Rinse your mouth frequently to keep the mouth moist.
  • Speak to your providers about over-the-counter and prescription gels and rinses that act as a saliva replacement.

Speak to your provider if this side effect continues to be a problem.

Allergic Reactions

In some cases, patients can have an allergic reaction to this medication. Signs of a reaction can include: shortness of breath or difficulty breathing, chest pain, rash, flushing or itching or a decrease in blood pressure. If you notice any changes in how you feel during the infusion, let your nurse know immediately.

Less common, but important side effects can include:

  • Seizures: Seizures can occur when busulfan is given in high doses. You will most likely be given medications to prevent seizures while you are receiving busulfan. Your doctor or nurse should be called immediately if you experience a loss of consciousness, twitching, tremors or uncontrollable shaking. If seizure activity begins others should help you to the ground, place pillows around you, and place you on your side if possible. Nothing should be forced into your mouth.
  • Lung Changes: This medication may cause pulmonary fibrosis (a scarring and stiffening of the lung tissue). This can occur several months to many years after starting this medication. Symptoms include cough, shortness of breath, or low-grade fever and may increase slowly over time or come on rapidly. Report any symptoms to your healthcare team.
  • Secondary Malignancies: There is a low risk of developing leukemia or other type of cancer due to treatment with this medication, which can occur many years after treatment. This is most often associated with repeated treatments or high doses. Your oncology care team will provide instructions on how to best follow up and be monitored for this.
  • Venous Occlusive Disease:In rare cases, busulfan can cause venous occlusive disease. Venous occlusive disease is a blockage of the small veins in the liver. Your healthcare provider will monitor for this using blood tests called liver function tests. Notify your healthcare provider if you notice yellowing of the skin or eyes, if your urine appears dark or brown, if there feels like there is fluid in your abdomen (belly), or if you have pain in your abdomen, as these can be signs of liver toxicity or venous occlusive disease.

Reproductive Concerns

This drug may affect your reproductive system, resulting in the menstrual cycle or sperm production becoming irregular or stopping permanently. Women may experience menopausal effects including hot flashes and vaginal dryness. In addition, the desire for sex may decrease during treatment.

Exposure of an unborn child to this medication could cause birth defects, so you should not become pregnant or father a child while on this medication. Effective birth control is necessary during treatment and 6 months after the last dose for women, and 3 months after the last dose for men. Even if your menstrual cycle stops or you believe you are not producing sperm. You may want to consider sperm banking or egg harvesting if you may wish to have a child in the future. Discuss these options with your oncology team. You should not breastfeed while taking this medication.

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