Objective: The effectiveness of pulsed high-dose oral dexamethasone therapy in children with refractory chronic idiopathic thrombocytopenic purpura (ITP) is evaluated.

Methods: Seven children (5 to 16 years old) who were refractory to 2 to 5 conventional standard therapies were included in the study. Dexamethasone was administered orally at a dosage of 40 mg/m2 per day (maximum 40 mg/day) for 4 consecutive days as a cycle. The cycle was repeated once a month for 6 months.

Results: One month after the first cycle, partial responses of platelet counts (> or = 50 x 10(9)/L and < 150 x 10(9)/L) were observed in three patients (43%). At the end of the sixth cycle, two patients (29%) had complete responses (> 150 x 10(9)/L) and one had a partial response. However, only one patient (14%) remained partially responsive 1 year after completion of therapy.

Conclusions: In contrast to what was observed in adults, this preliminary study suggests that pulsed high-dose oral dexamethasone therapy was not uniformly effective in children with chronic ITP.