How it is administered

Melphalan is available as a sterile powder for intravenous (IV) injection and as a tablet for oral administration, though the IV form is more commonly used in blood cancers. The IV form is typically reconstituted and diluted before being slowly infused over 15 to 30 minutes, often through a central venous line to avoid local tissue damage. Dosage and schedule depend on the specific indication and patient factors, such as kidney function.

How it works

Melphalan is an alkylating agent of the bischloroethylamine type. It works by binding to DNA in cancer cells, causing cross-linking between DNA strands. This prevents the cells from replicating and leads to cell death. Melphalan is active against both resting and rapidly dividing tumor cells, making it effective in treating blood cancers where cells are often dividing quickly.

Because melphalan targets DNA, it is not specific to cancer cells and can also affect normal cells, especially those that divide rapidly, such as bone marrow cells. This mechanism is why melphalan is effective in blood cancers like multiple myeloma, but also why it can cause significant side effects, particularly bone marrow suppression.

Who Should take it

Melphalan is indicated for patients with multiple myeloma, especially when oral therapy is not appropriate. It is also used as a high-dose conditioning treatment before stem cell transplantation in multiple myeloma patients.

Patients with other blood cancers may receive melphalan as part of a chemotherapy regimen, based on their doctor's recommendation. The decision to use melphalan depends on the type and stage of cancer, as well as the patient's overall health and kidney function.

Who should not take it

Melphalan should not be used in patients who have previously shown resistance to the drug or who have had severe hypersensitivity reactions (such as anaphylaxis) to melphalan. It is also contraindicated in patients with severe bone marrow suppression unless the benefits outweigh the risks and close monitoring is possible.

Pregnant women should not take melphalan due to the risk of harm to the fetus. Women of childbearing potential should use effective contraception during treatment and for several months after. Patients with severe kidney dysfunction may require dose adjustments or may not be suitable candidates for melphalan therapy.

Commonly used with

Melphalan is often used in combination with prednisone for the treatment of multiple myeloma. In the context of stem cell transplantation, it may be used with other supportive medications such as antiemetics (to prevent nausea) and antibiotics (to prevent infection during periods of low white blood cell counts).

It may also be part of multi-drug chemotherapy regimens, depending on the specific blood cancer being treated.

Commonly tested with

Melphalan has been tested in combination with prednisone in clinical trials for multiple myeloma. It is also tested as part of high-dose regimens before autologous stem cell transplantation. In some studies, it has been combined with other chemotherapy agents or with supportive therapies to manage side effects and improve outcomes.