Class: Chemotherapy

Generic Name: Melphalan (MEL-fah-lan), L-PAM, L-Sarcolysin, Phenylalanine Mustard
Trade Name: Alkeran®

How is this drug used? Melphalan is FDA approved for the treatment of multiple myeloma, breast cancer, ovarian cancer, and in treatment regimens prior to bone marrow or stem cell transplantation.  It is important for patients to remember that physicians have the ability to prescribe medication for conditions other than those for which the drug has been approved by the FDA. Patients who have received a prescription of this drug for a condition other than which it is approved may wish to discuss this issue with their physician.

What is the mechanism of action? Melphalan belongs to a class of agents referred to as alkylating agents and is a derivative of nitrogen mustard. It produces its anti-cancer effects by causing a chemical reaction with DNA in cells. The chemical reaction, called interstrand cross-linking, inhibits the cancer cell to grow or replicate and/or ultimately causes cellular death.

How is melphalan given (administered)? Melphalan may be administered orally or in a vein (intravenous). The route of administration, as well as the doses, depends upon the condition being treated, the overall health of the patient, the particular regimen being used, and the height and weight of the patient.

How are patients monitored? Patients will usually have scheduled meetings with their healthcare provider while they are being treated with melphalan. Typically, blood will be drawn to check levels of blood cells and to monitor functions of some organ systems, such as the kidneys or liver. Physical examinations, scans, and/or other measures may be performed to assess side effects and response to therapy.

Uncommonly, patients may experience an allergic-type reaction during administration of melphalan.  Patients are monitored closely for these symptoms, and if they experience difficulty breathing, wheezing, swelling of the throat or facial features or hives, they should tell their healthcare provider immediately, as these symptoms are indicative of an allergic reaction, which needs immediate attention.

What are the common (occur in 30% or more of patients) side effects of treatment with melphalan?

• Low white blood cell levels – increases the risk of infection
• Low red blood cell levels – increases the risk of anemia and blood transfusions
• Low platelet levels – increases the risk of bleeding
• Nausea and vomiting

What are the less common (occur in 10% to 29% of patients) side effects of treatment with melphalan?

• Allergic reaction—difficulty breathing, swelling of throat or facial features, hives, itching, wheezing, low blood pressure, fast heart rate
• Mouth sores or ulcers
• Hair loss
• Diarrhea
• Kidney abnormalities
• Changes in heart rhythm
• Loss of fertility

This is not a complete list of side effects. Some patients may experience other side effects that are not listed here. Patients may wish to discuss with their physician the other less common side effects of this drug, some of which may be serious.

Some side effects may require medical attention. Other side effects do not require medical attention and may go away during treatment. Patients should check with their physician about any side effects that continue or are bothersome.

What are possible late side effects of treatment with melphalan? With the use of this drug, there is a risk of developing side effects months or years after treatment has been completed. In uncommon instances, a secondary malignancy may occur as a result of receiving melphalan. A secondary malignancy is a new and unrelated cancer that occurs in an individual as a result of previous treatment with radiation or chemotherapy.  Patients treated with melphalan should discuss the risk of late occurring side effects with their physician.

What can patients do to help alleviate or prevent discomfort and side effects?

• Pay careful attention to the physician’s instructions and inform the physician of any side effects.
• Maintain adequate rest and nutrition.
• Wear sunscreen and protective clothing and try to minimize sun exposure.
• Drink plenty of fluids. (Patients should ask their physician about the amount of liquid to consume during a day.)
• If possible, avoid large crowds or people who are sick or not feeling well, as this drug may leave some patients susceptible to infection.
• Wash hands often to reduce the risk of infection.
• Eat small meals frequently to help alleviate nausea.
• If patients have been prescribed an anti-nausea medication, they should be sure to take the prescribed doses.
• Avoid activities that may cause injury or bruising.
• Use a soft toothbrush and an electric razor to prevent cuts on the mouth or skin.
• For mouth sores, patients should rinse their mouth three times a day with a salt and soda solution (8 ounces of water mixed with ½ to 1 tsp baking soda and/or ½ to 1 tsp salt) and brush their teeth with a soft toothbrush to help prevent the development of mouth sores.

Are there any special precautions patients should be aware of before starting chemotherapy?

• If patients are being treated with melphalan as an oral agent, they should take the drug on an empty stomach – 1 hour before eating or 2 hours after eating. However, patients should speak with their healthcare provider regarding scheduling of taking melphalan.
• Patients should inform their physician if they are pregnant, breastfeeding or planning a family in the near future. This drug may cause birth defects. It is important to use some kind of birth control while undergoing treatment. Also, patients may want to talk to their physician if they are considering having children in the future, since some drugs may cause fertility problems.
• It is important that patients inform their physician of any pre-existing conditions (chicken pox, heart disease, kidney disease, liver disease, lung disease, etc.) as they may worsen with this drug.
• Patients should inform their physician of any other medication they are taking (whether prescription or over-the-counter, including vitamins, herbs, etc.) as they may interfere with treatment.
• Patients should check with their physician before starting any new drug or nutritional supplement.
• Patients should inform their physician of any known drug or food allergies or any reactions to medications they have experienced in the past.

When should patients notify their physician?

• Difficulty breathing, wheezing
• Swelling of the throat
• Swelling of facial features
• Hives, skin rash, itching
• Flu or cold-like symptoms: fever, chills, sore throat, cough
• Signs of infection – redness, swelling, pus, tenderness
• Prolonged nausea or vomiting
• Extreme or prolonged diarrhea
• Painful urination
• Yellowing of the skin or eyes
• Prolonged or severe mouth sores
• Unexplained bleeding or bruising
• Black, tarry stools
• Blood in the urine or stool
• Extreme, prolonged fatigue
• Redness, pain or swelling at injection site
• Unusual lumps or masses
• Weight loss

What is a package insert?
A package insert is required by the FDA and contains a summary of the essential scientific information needed for the safe and effective use of the drug for healthcare providers and consumers.  A package insert typically includes information regarding specific indications, administration schedules, dosing, side effects, contraindications, results from some clinical trials, chemical structure, pharmacokinetics and metabolism of the specific drug. By carefully reviewing the package insert, you will get the most complete and current information about how to safely use this drug. If you do not have the package insert for the drug you are using, your pharmacist or physician may be able to provide you with a copy.

Copyright © 2010 CancerConnect Last updated 07/10.

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