大剂量地塞米松治疗38例原发免疫性血小板减少症患儿的临床研究

OBJECTIVE: To evaluate the efficacy and safety of pulsed high-dose dexamethasone (HDD) treatment in children with primary immune thrombocytopenic purpura (ITP). METHODS: ITP children who failed to first-line therapy from September 2013 to September 2014 were given pulsed HDD treatment, dexamethasone was administered at a dosage of 0.6 mg ·kg(−1)·d(−1) (maximum 40 mg/d) for 4 consecutive days. The cycle was repeated every 28 days for 6 months. RESULTS: ①A total of 38 cases were enrolled, 26 boys and 12 girls, median age was 54(6–151) months, median duration of disease was 6(1–72) months, 9 cases was newly diagnosed ITP, 13 cases with persistent ITP, 16 cases with chronic ITP. Median platelet count before treatment was 16.3(1.0–30.0)×10(9)/L. ②A median follow-up time was 180(90–554) days. Treatment response was obtained in 17 cases (44.7%), including 7 cases (18.4%) with complete response (CR), 10 cases (26.3%) response (R); the median time to response was 80.5(23–245) days. Of 17 CR/R cases, 3 turned to no response, with a median duration of response 63(37–67) days. Of 38 cases, 21(55.3%) was no response, but the bleeding symptoms in 85.7% of this group improved. ③Only 1 patient had mild reversible side effects during treatment. ④The percentage of CD4 (+)CD25 (+)Foxp3(+)T cells is higher in effective group than that in ineffective group[(7.54±1.50)% vs(5.69±1.95)%, P=0.049]. Univariate analyses suggested that the efficacy of HDD treatment in children with megakaryocyte count <300/slide is better than that >300/slide (P=0.049). CONCLUSION: Pulsed HDD treatment is a comparatively safe and effective choice for children with ITP who failed to first-line therapy. Children with less than 300 megakaryocytes or higher CD4(+)CD25(+)Foxp3(+)T cells may be more suitable for the therapy.