Melphalan Flufenamide (Pepaxto®)

Author: OncoLink Team
Last Reviewed:

Pronounced: MEL-fa-lan floo-FEN-a-mide

Classification: Alkylating Agent

About: Melphalan Flufenamide (Pepaxto®)

Melphalan Flufenamide damages 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, in general, divide faster and with less error-correcting than healthy cells, cancer cells are more sensitive to this damage.

How to Take Melphalan Flufenamide 

This medication is given as an intravenous (IV, into a vein) infusion. How often you receive this medication will be determined by your provider. 

Possible Side Effects

There are a number of things you can do to manage the side effects of melphalan flufenamide. 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 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.

Infection and Low White Blood Cell Count (Leukopenia or Neutropenia)

This medication can cause life threatening infections, with or without a decrease in white blood cell counts. 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 bath 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.

Kidney Problems

This medication can cause kidney problems, including an increased creatinine level, which your oncology care team may monitor for using blood tests. Notify your healthcare provider if you notice decreased urine output, blood in the urine, swelling in the ankles, or loss of appetite. 

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.

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 lesson 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.

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 on non-alcoholic, un-caffeinated fluid a day to prevent dehydration. For medications with serious diarrhea complications: Diarrhea can be a serious side effect that can lead to dehydration. Notify your healthcare provider if you develop diarrhea.

Less common, but important side effects can include:

  • Secondary Cancers: A secondary cancer is one that develops as a result of cancer treatment for another cancer. This is quite rare, but you should be aware of the risk. In most cases, a secondary cancer related to chemotherapy is a blood cancer (leukemia, lymphoma). This can occur years after treatment. This is most often associated with repeated treatments or high doses. Your provider will monitor your labs closely. Consider having a complete blood count with differential checked annually by your healthcare provider if you received high risk therapies. 

Reproductive Concerns

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 for at least 6 months after treatment for women and 3 months after treatment for men. Even if your menstrual cycle stops or you believe you are not producing sperm, you could still be fertile and conceive. You should not breastfeed while taking this medication or for 1 week after your last dose.

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