How it is administered

Melphalan is available as an intravenous (IV) injection and as an oral tablet, though IV administration is more common for multiple myeloma, especially when oral therapy is not appropriate. The IV formulation is supplied as a powder that is reconstituted and diluted before infusion. It is typically administered over 15 to 30 minutes via a central venous line or a fast-running IV infusion to minimize the risk of tissue damage if the drug leaks outside the vein.

How it works

Melphalan is an alkylating agent, meaning it works by attaching alkyl groups to DNA within cancer cells. This process leads to cross-linking of DNA strands, which prevents the cells from replicating and ultimately causes cell death. Melphalan is active against both resting and rapidly dividing tumor cells, making it effective for treating cancers like multiple myeloma.

The drug's cytotoxicity is mainly due to its ability to form interstrand cross-links in DNA, especially at the N7 position of guanine bases. This disrupts the normal function and division of cancer cells, leading to their destruction. Melphalan is metabolized primarily by hydrolysis and is eliminated from the body mainly through chemical breakdown, with only a small portion excreted in urine.

Who Should take it

Melphalan is indicated for the palliative treatment of patients with multiple myeloma, particularly when oral therapy is not suitable. It is also used as a high-dose conditioning treatment before autologous stem cell transplantation in multiple myeloma patients.

Patients who have not responded to other therapies or who are undergoing stem cell transplantation may benefit from melphalan. The decision to use melphalan should be made by a healthcare provider experienced in treating blood cancers, as the drug requires careful dosing and monitoring due to its potential side effects.

Who should not take it

Melphalan should not be used in patients whose disease has previously shown resistance to this medication. It is also contraindicated in individuals with a known hypersensitivity or severe allergic reaction to melphalan.

Women who are pregnant should not take melphalan, as it can cause harm to the unborn baby. Patients with severe bone marrow suppression or those who cannot be closely monitored for blood counts may not be suitable candidates for melphalan therapy. Always discuss your full medical history with your healthcare provider before starting treatment.

Commonly used with

Melphalan is often used in combination with other medications for multiple myeloma, such as prednisone (a corticosteroid). In high-dose regimens, it is used as part of the conditioning treatment before autologous stem cell transplantation, sometimes alongside other supportive medications like antiemetics to manage nausea and vomiting.

It may also be used with other chemotherapeutic agents or as part of combination regimens in clinical trials.

Commonly tested with

Melphalan has been tested in combination with prednisone in clinical trials for multiple myeloma. It has also been evaluated alongside other chemotherapy agents and in combination with stem cell transplantation protocols. In some studies, it has been tested with cyclosporine, though this combination may increase the risk of kidney problems.

Other agents that have been studied with melphalan include BCNU (carmustine), which may increase the risk of lung toxicity when used together.