Journal of Clinical Oncology, Vol 22, No 12 (June 15), 2004: pp. 2477-2488
© 2004 American Society of Clinical Oncology.
DOI: 10.1200/JCO.2004.10.127
Thalidomide: Current Role in the Treatment of Non-Plasma Cell Malignancies
Shaji Kumar,
Thomas E. Witzig,
S. Vincent Rajkumar
From the Division of Hematology, Department of Internal Medicine, Mayo Clinic and Foundation, Rochester, MN
Address reprint requests to S. Vincent Rajkumar, MD, Division of Hematology, Mayo Clinic, 200 First St SW, Rochester, MN 55905; e-mail: rajks{at}mayo.edu
Thalidomide, initially introduced as a sedative, was withdrawn from the market in the early 1960s after it was found to be a teratogen. However, it later found use as an investigational agent in the treatment of erythema nodosum leprosum, oral ulcers, graft versus host disease, and wasting associated with the human immunodeficiency syndrome. Its antiangiogenic properties were recognized in the early 1990s during a period where the importance of angiogenesis became increasingly apparent as a critical step in the in the proliferation and spread of malignant neoplasms. This led to the evaluation of thalidomide as an antiangiogenic agent in the treatment of several cancers. Thalidomide has already become part of standard therapy for the treatment of patients with relapsed and refractory multiple myeloma. It has also been found to have varying degree of benefit in various other malignancies. Although more clinical trials are needed, Kaposi's sarcoma and myelofibrosis represent other malignancies in which thalidomide has already demonstrated promising activity. The mechanism of action of thalidomide in cancer is still unclear, but do appear to be mediated by several other mechanisms in addition to its anti-angiogenic properties. This article reviews the current status of thalidomide for the treatment of non-plasma-cell malignancies.
Supported in part by grants CA85818, CA93842, CA100080, and CA62242 from the National Cancer Institute, Bethesda, MD. Also supported in part by the Multiple Myeloma Research Foundation, the Goldman Philanthropic Partnerships, and the Leukemia and Lymphoma Society.
The authors have received research support for clinical trials using thalidomide from Celgene.
Authors' disclosures of potential conflicts of interest are found at the end of this article.

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