More than three decades after its withdrawal from the world marketplace,
thalidomide is attracting growing interest because of its reported immunomodulatory and anti-inflammatory properties. Current evidence indicates that
thalidomide reduces the activity of the inflammatory
cytokine tumor necrosis factor (
TNF)-alpha by accelerating the degradation of its
messenger RNA.
Thalidomide also inhibits angiogenesis. Recently, the
drug was approved for sale in the United States for the treatment of
erythema nodosum leprosum, an inflammatory complication of
Hansen's disease. However, it has long been used successfully in several other dermatologic disorders, including
aphthous stomatitis, Behçet's syndrome, chronic cutaneous
systemic lupus erythematosus, and
graft-versus-host disease, the apparent shared characteristic of which is immune dysregulation. Many recent studies have evaluated
thalidomide in patients with human immunodeficiency virus (
HIV) infection; the
drug is efficacious against oral
aphthous ulcers, HIV-associated
wasting syndrome, HIV-related
diarrhea, and
Kaposi's sarcoma. To prevent teratogenicity, a comprehensive program has been established to control access to the
drug, including registration of prescribing physicians, dispensing pharmacies, and patients; mandatory informed consent and education procedures; and limitation of the quantity of
drug dispensed. Clinical and, in some patients, electrophysiologic monitoring for
peripheral neuropathy is indicated with
thalidomide therapy. Other adverse effects include sedation and
constipation. With appropriate safeguards,
thalidomide may benefit patients with a broad variety of disorders for which existing treatments are inadequate.