Recombinant Human Interferon Alpha-2A Therapy in Children with Chronic Immune Thrombocytopenic Purpura.

Background: In a prospective study, 11 children with chronic immune thrombocytopenic purpura between ages 3 and 18 years were treated with recombinant human interferon alpha2a (rhIFNalpha-2a).Patients and Methods: A dose of 3 x 10(6) U/m(2) three times weekly for 4 to 5 weeks (one cycle) was administered. Patients were treated with one to four cycles of rhIFNa-2a, and the outcomes were measured initially and 18 to 30 months after the last cycle.Results: Good therapeutic responses (defined as platelet count >100 x 10(9)/L) lasting for 18 to 30 months from the last interferon cycle were achieved in 6 of the 11 (55%) patients, including one with a probable spontaneous remission. Fair responses (platelet count 31-60 x 10(9)/L) for 18 months were achieved in 3 of the 11 (27%) patients. Only two patients, each treated only with one interferon cycle, exhibited no response. Side effects of treatment included fever and a flulike syndrome, which were usually present during the first 14 days of therapy only.Conclusions: Interferon-a appears to be an effective therapeutic approach to children with chronic immune thrombocytopenic purpura, with the potential of sustained long-term remission. A randomized, placebo-controlled study is needed to confirm its role in this population.