How it Works

Melphalan is most frequently used during the autologous stem cell transplant process for multiple myeloma patients. Melphalan is an alkylating agent, which are most active in the resting phase of the cell. 

Melphalan chemically alters the DNA nucleotide guanine through alkylation, and causes linkages between strands of DNA. This chemical alteration inhibits DNA synthesis and RNA synthesis, functions necessary for cells to survive. These changes cause cytotoxicity in both dividing and non-dividing tumor cells.

As a palliative treatment, it helps control myeloma cell propagation. Melphalan also is used at higher doses as myeloablative chemotherapy prior to ASCT. At these doses, melphalan is more potent against myeloma cells, but also drastically reduces the number of healthy blood-forming cells in the bone marrow.

How it’s Administered

Melphalan can be given as a conditioning treatment for stem cell transplant. The melphalan (IV) injection is usually given for two days in a row before stem cell transplant.

Melphalan for stem cell transplant is given as an infusion through a central intravenous (IV) line placed into a large vein. A healthcare provider will give you this injection. This medicine must be given slowly, and the infusion can take up to 30 minutes to complete. Tell your caregivers if you feel any burning, pain, or swelling around the IV needle when melphalan is injected.

Melphalan, in some instances, can be given orally. 

As a palliative treatment for multiple myeloma, a melphalan injection is usually given every 2 to 4 weeks. Melphalan oral is usually taken daily, and your doctor may occasionally ask you to stop taking it for a short time.

Who Should Take Melphalan

Melphalan is currently approved for multiple myeloma, ovarian cancer, and neuroblastoma rhabdomyosarcoma breast cancer. It is also used in some conditioning regimens prior to bone marrow transplant. 

Who Shouldn’t Take Melphalan

• Women who are pregnant
• Women who are breastfeeding
• Men and women who are trying to conceive a child / get pregnant. Barrier methods of contraception, such as condoms, are recommended. Discuss with your doctor when you may safely become pregnant or conceive a child after therapy.
• Before starting melphalan treatment, make sure you tell your doctor about any other medications you are taking (including prescription, over-the-counter, vitamins, herbal remedies, etc.). Do not take aspirin or products containing aspirin unless your doctor expressly permits this.
• Do not receive any kind of immunization or vaccination without your doctor's approval while taking melphalan.

The Most Common Side Effects of Taking Melphalan Include: 

The following side effects are common (occurring in greater than 30%) for patients taking melphalan:

• Low blood counts.  Your white and red blood cells and platelets may temporarily decrease.  This can put you at increased risk for infection, anemia, and/or bleeding.  This effect may be prolonged and cumulative
• Nausea and vomiting. (mild with regular dosing, can be severe with high-dose therapy)

These side effects are less common side effects (occurring in about 10-29%) of patients receiving melphalan:

• Allergic reaction (rare with pill form, happens in about 10% of patients treated with IV form).
• Mouth sores (mild with regular dosing, can be severe with high-dose therapy)
• Diarrhea (mild with regular dosing, can be severe with high-dose therapy).
• Loss of fertility.  Meaning, your ability to conceive or father a child may be affected by melphalan.  Discuss this issue with your healthcare provider
• Hair loss (rare with regular dosing, common with high-dose used for bone marrow transplant)
• Nephrotoxicity (see kidney problems): Uncommon with regular doses, seen when melphalan is given in high-doses used for bone marrow transplant
• Arrhythmias (see heart problems): Uncommon with regular doses, seen when melphalan is given in high-doses used for bone marrow transplant

Delayed effects:

• After taking melphalan, there is a slight risk of developing a blood cancer such as leukemia or myelodysplasia.  Talk to your doctor about this risk

Commonly Used With 

• Cytoxan
• Prednisone
• Thalomid

Commonly Being Tested With

• Proteasome Inhibitors: Bortezomib, Carfilzomib
• Targeted Inhibitors: Selinexor
• Immunomodulators (IMiDs): Lenalidomide
• Alkylating Agents: Cyclophosphamide
• Monoclonal Antibodies:​​ Daratumumab

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