Clinica Pediatrica, 1Servizio di Farmacia, IRCCS “Burlo Garofolo”, Università di Trieste
Key words: Thalidomide, Pharmacology, Therapeutic use, Adverse event
The Authors provide an overview of thalidomide: mechanism of action, pharmacokinetics, efficacy and safety profile, with particular reference to experiences reported with pediatric patients. The review was carried out using the MEDLINE database for the studies, particularly RCTs, published from 1966 to February 2003. The exact mechanism of action of thalidomide has not yet been understood. Thalidomide is an immunomodulating drug with antiinflammatory properties. It also inhibits angiogenesis and has a sedative and hypnotic effect. A total of 47 randomized controlled trials (RCT) have been published regarding adult and pediatric patients, 4 of which studied patients aged from 0-18 years. Indications for use of thalidomide in pediatric patients is not based on RCTs, but on non-controlled observational studies of a limited number of cases. In pediatric patients thalidomide has been used in the following conditions: chronic graft-versus host disease (GVHD), aphthous ulcers in patients with HIV, Behçet’s syndrome, systemic lupus erythematosus, and Crohn’s disease. In the adult patient, thalidomide has been also used in the treatment of certain cancers and particularly of multiple myeloma. The most frequent side effects sonnolence, peripheral neuropathy, constipation, headaches, a maculopapular, pruritic rash. In conclusion, little is known about the efficacy of the drug when compared to conventional treatments, and about dosage, duration of treatment and safety profile. Large multi-centre studies are needed to define the use of the drug in pediatric patients.
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