Melphalan chemical structure
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Melphalan

Melphalan hydrochloride ( AlkeranĀ®) is a chemotherapy drug belonging to the class of nitrogen mustard alkylating agents. It is used primarily to treat multiple myeloma and ovarian cancer, and occasionally malignant melanoma. Otherwise known as L-Phenylalanine Mustard, or L-PAM, melphalan is a phenylalanine derivative of mechlorethamine. The agent was first investigated as a possible drug for use in melanoma however it did not work but was found to be of use in myeloma. more...

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Administration

Oral or intravenous; dosing varies by purpose and route of administration as well as patient weight.

Side effects

Common side effects include:

  • Nausea and vomiting
  • Bone marrow suppression, including
    • Decreased white blood cell count causing increased risk of infection
    • Decreased platelet count causing increased risk of bleeding

Less common side effects include:

  • Severe allergic reactions
  • Scarring of lung tissue (usually only with prolonged use)
  • Hair loss
  • Rash
  • Itching

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Thalidomode as a potential treatment for scleromyxedema
From Journal of Drugs in Dermatology, 7/1/04

Caradonna S, Jacobe H. Arch Dermatol 2004 Mar; 140(3):277-80.

The authors present a case of a 39-year-old female with a history of scleromyxedema for about one year. The patient's only complaint was of limited joint mobility. A skin biopsy was performed on the patient's skin to evaluate the sclerotic plaques on her body, and her results showed dermal thickening with displacement of collagen bundles by mucin, with spindled fibroblasts consistent with scleromyxedema.

The patient was started on prednisone and melphalan with no improvement. She was then changed from melphalan to cyclophosphamide and continued on prednisone with minimal change. Later on a trial of cladribine was instituted with no success and afterwards the patient was maintained on prednisone with no improvement. After seven years of various treatments, she was treated with thalidomide 100 mg/d and after just six months of therapy she started to notice increase mobility and less skin thickness. A repeat biopsy at one year of thalidomide therapy demonstrated a decrease in mucin.

JDD ARTICLE EVALUATION

Scleromyxedema is a disease of adults that mainly affects patients of 30-50 years of age. The pathogenesis is unknown and treatment modalities are sparse and unsuccessful. In the past melphalan has been known to clear the disease but long term use is associated with hematologic malignancies. Thalidomide offers hope to patients with scleromyxedema, its mechanism of action is unknown but it is believed that it acts through TNF alpha inhibition. In the past it has been shown to be effective in neutrophilic dermatosis and in animal models it may represent a useful anti-neoplastic agent.

Although an interesting case report, we would hope for a follow up studies to compare thalidomide with other treatment modalities in a double-blinded randomized study. All in all, this represents a new regimen, albeit off-label, for the treatment of scleromyxedema.

WASHINGTON WHISPERS APPEARS IN EACH ISSUE AND PROVIDES A SUMMARY AND CRITICAL EVALUATION OF THE LATEST DRUG TRIALS, STUDIES, AND REACTIONS AVAILABLE TO THE MEDICAL COMMUNITY, AS COLLATED FROM A WEALTH OF INDUSTRY SOURCES.

COPYRIGHT 2004 Journal of Drugs in Dermatology, Inc.
COPYRIGHT 2005 Gale Group

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