Thalidomide use in pediatric patients

OBJECTIVE: To provide a detailed overview of thalidomide use in pediatric patients. DATA SOURCES: English-language articles were identified through a MEDLINE search (1966-February 2001); key terms included thalidomide, child, graft-versus-host disease, cancer, HIV, Crohn's disease, Behcet's disease, and lupus erythematosus. References cited in those articles were also evaluated. DATA SYNTHESIS: Thalidomide appears to be effective in patients with chronic, not acute, graft-versus-host disease (GVHD) and in healing aphthous ulcers in patients with HIV infection. Limited case reports suggest efficacy of thalidomide in the treatment of cutaneous manifestations of Behcet's disease, Crohn's disease, and lupus in children; however, the recurrence of disease is almost universal on drug discontinuation. CONCLUSIONS: Thalidomide should be used as a last resort when all other therapies fail, preferably in male or prepubescent female patients.