Melphalan is an anticancer drug that belongs to the class of medications called alkylating agents. FDA approved it initially in 1964 for treating multiple myeloma, advanced breast cancer, ovarian cancer, polycythemia vera, childhood neuroblastoma, malignant melanoma, and soft tissue sarcoma. 

Melphalan is available as a tablet and injection.  It is used to treat multiple myeloma (a type of cancer of the bone marrow). It is also used to treat advanced ovarian cancer. It is also indicated to treat childhood neuroblastoma (cancer of the nervous system), malignant melanoma (skin cancer), and soft tissue sarcoma (cancer of the muscle, fat, fibrous tissue, blood vessels, or other supporting tissues of the body). It is also indicated to treat polycythemia vera ( a disease that increases the number of red cells in your blood. This makes the blood thicken and causes blood clots). It works by reducing the number of abnormal cells your body makes. 

Melphalan is available as a tablet and injection. Melphalan injection will be administered to you by a doctor or other healthcare professional in a hospital setting trained in cancer chemotherapy. This injection can be given as an infusion into your vein, or it will be given as a perfusion to a particular part of your body through an artery. If you are prescribed the tablet. Take the tablet at the same time, with or without food. Do not break, chew, crush, or break the tablet. Swallow the whole tablet with water. Your doctor will decide the dose, route of administration, and duration of the therapy based on your disease status, severity, and other factors such as body weight, other disease conditions, and age.  

The common side effects include decreased blood cell count, nausea, vomiting, diarrhea, mouth ulcers, and hair loss. Serious side effects include cardiac arrest, liver inflammation, hypersensitivity skin reactions, acute renal failure, and lung inflammation. 

This drug is indicated to cause serious allergic reactions. Report to your physician immediately if you have a rash, lumps or hives on the skin, swollen face, eyelids, or lips, sudden wheeziness, and chest tightness after consuming the medicine. Melphalan can cause fetal harm. Inform your physician if you are pregnant or planning to get pregnant during the treatment. This medicine may cause infertility that is due to ovarian or testicular suppression. 

Inform your physician if you have had radiotherapy or cancer chemotherapy recently or have a kidney problem. Do not take Melphalan if you are allergic to melphalan or any of the other ingredients. Inform your healthcare provider about all the medicines you take, including prescription medications, over-the-counter medications, nutritional and vitamin supplements, and herbal products. Certain medications interact with melphalan and reduce its effectiveness by causing undesirable side effects. Use effective contraception during the treatment. Do not breastfeed during the treatment with this medicine because it may cause harm to the baby. Your healthcare professional may advise you to take certain blood tests to monitor the effectiveness and side effects of the therapy. 

Store Melphalan tablets and injection in a refrigerator, 2° to 8°C. Protect the medicine from light and excessive moisture. Keep the medicine out of reach of children and pets. If you forgot to take the tablet, simply take your next dose at the scheduled time. Do not take a double dose to make up for a forgotten dose. If you missed the injection dosing schedule, inform your healthcare professional immediately and reschedule. This medicine may cause changes in your blood levels. Your doctor may advise you to take certain blood tests during the treatment to monitor any unwanted side effects. This medicine may you to bleed, bruise, or get infections more easily. Take precautions to prevent illness and injury. 

1. Melphalan 50 mg Powder and Solvent for Solution for Injection/Infusion - Summary of Product Characteristics (SmPC) - (emc) [Internet]. www.medicines.org.uk. [revised on 2020] [cited 2023 Apr 7]. Available from: https://www.medicines.org.uk/emc/product/927/smpc
2. HIGHLIGHTS OF PRESCRIBING INFORMATION - Melphalan injection [revised on 2017] [cited 2023 Apr 7]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/207155s001lbl.pdf
3. Melphalan 2 mg Tablets - Summary of Product Characteristics (SmPC) - (emc) www.medicines.org.uk. [revised on 2022] [cited 2023 Apr 7]. Available from: https://www.medicines.org.uk/emc/product/3806/smpc
4. ALKERAN ® 2 - melphalan [revised on 2021] [cited 2023 Apr 7]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2003/14691slr024_alkeran_lbl.pdf
5. KD Tripathi, Essentials of Medical Pharmacology, Anticancer drugs, 7th edition, 2013, 860.

The drug information on this page is not a substitute for medical advice. It is meant for educational purposes only. For further details, consult your doctor about your medical condition to know if you can receive this treatment.