Purpose: The effectiveness of pulsed high-dose oral dexamethasone therapy in children with refractory chronic idiopathic thrombocytopenic purpura (ITP) is evaluated. Patients and 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 (≤50 x 109/L and <150 x 109/L) were observed in three patients (43%). At the end of the sixth cycle, two patients (29%) had complete responses (>150 x 109/L) and one had a partial response. However, only one patient (14%) remained partially responsive I 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.
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