Thalidomide in gastrointestinal disorders

Drugs. 2001;61(6):777-87. doi: 10.2165/00003495-200161060-00006.

Abstract

Thalidomide was originally marketed as a sedative, but was removed from the market in 1961 after it was associated with an epidemic of severe birth defects. Subsequently, it has been shown to have therapeutic efficacy in a number of the gastrointestinal tract conditions characterised by immune dysregulation. The exact mechanism of the immunosuppressive effects of thalidomide is unknown; proposed mechanisms include inhibition of tumour necrosis factor alpha release and inhibition of angiogenesis. In chronic graft versus host disease, use of high dose thalidomide (1200 mg/day) may bring about a response in 20% of patients with refractory disease. Thalidomide 200 mg/day helps eradicate ulcers in 50% of patients with HIV-associated oral aphthous ulceration. In Behçet's disease, thalidomide 100 to 300 mg/day can decrease the number of mucocutaneous ulcers, although full remission occurs in less than 20% of patients. In Crohn's disease, thalidomide 50 to 300 mg/day may decrease the severity of mucosal disease and prompt closure of fistulae. Patients to be placed on thalidomide therapy must practice either abstinence or strict birth control; women must undergo regular pregnancy testing and utilise 2 forms of contraception. Other adverse effects include sedation (present in nearly all patients), symptomatic neuropathy (present in approximately 20%), and skin rashes. Given the potential toxicity, thalidomide use should generally be limited to clinical protocols with institutional review board oversight.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Behcet Syndrome / drug therapy
  • Cell Adhesion / drug effects
  • Colitis, Ulcerative / drug therapy
  • Crohn Disease / drug therapy
  • Gastrointestinal Diseases / drug therapy*
  • Graft vs Host Disease / drug therapy
  • HIV Infections / drug therapy
  • Humans
  • Neovascularization, Physiologic / drug effects
  • Thalidomide / adverse effects
  • Thalidomide / pharmacology
  • Thalidomide / therapeutic use*

Substances

  • Thalidomide