Melphalan (U.S. brand name Alkeran) is a chemotherapy drug commonly used in treating multiple myeloma. It belongs to the class of drugs known as alkylating agents, which inhibit DNA and RNA synthesis, causing the death of both dividing and non-dividing tumor cells. It has been used to treat MM since the 1960s.

How Is It Used?

In the U.S., melphalan is most commonly used in the treatment of MM as the conditioning agent in autologous stem cell transplant. It is given as an intravenous infusion (IV) at a high dose to ablate (wipe out the cells within) the bone marrow, where myeloma grows, the day before the patient's own hematopoietic (blood-making) stem cells are given back to restore bone marrow function.

It is also used, most commonly outside the U.S., as an oral agent to treat myeloma in combination with Velcade® and prednisone (VMP), and in combination with thalidomide and prednisone (MPT).

Possible Side Effects

Like most traditional chemotherapy agents, which kill all rapidly-dividing cells in the body, IV high-dose melphalan commonly causes the following side effects:

  • nausea
  • vomiting,
  • mouth sores
  • hair loss,
  • rash
  • itching
  • infertility, and
  • suppression of the bone marrow's ability to make new blood cells, particularly white blood cells (which can cause increased risk of infection) and platelets (which can cause an increased risk of bleeding).

As an oral agent given in combination therapy at lower doses, melphalan is well tolerated, and is often used to treat older, frail patients.

With prolonged use, however, melphalan may result in the following conditions:

  • scarring of lung tissue (pulmonary fibrosis),
  • a severe, potentially fatal form of respiratory disease (interstitial pneumonitis),
  • cardiac arrest,
  • myelodysplasia, or
  • leukemia.

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