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

Clinical study of pulsed high-dose dexamethasone treatment in 38 children with primary immune thrombocytopenic purpura

目的探讨大剂量地塞米松（HDD）静脉滴注，对原发免疫性血小板减少症（ITP）患儿的疗效及安全性。方法2013年9月至2014年9月38例一线治疗无效的ITP患儿接受HDD治疗。用药方案：地塞米松0.6 mg·kg^-1·d^-1×4 d（最大剂量40 mg/d），冲击治疗每疗程间隔28 d，共6个疗程。结果①38例患儿中男26例，女12例，中位月龄为54（6-151）个月，中位病程为6（1-72）个月；新诊断ITP 9例，持续性ITP 13例，慢性ITP 16例；治疗前中位PLT为16.3（1.0-30.0）×10^9/L。②中位随访时间180（90-554）d, 17例（44.7%）获得治疗反应[完全反应（CR）7例（18.4%），有效（R）10例（26.3%）]，中位起效时间为80.5（23-245）d; 17例获得CR/R患儿中3例失效复发，中位反应持续时间为63（37-67）d; 21例（55.3%）无效（NR），但其中18例（85.7%）患儿出血症状好转。③用药过程中仅1例患儿出现轻度可逆性不良反应。④CR/R组患儿外周血CD4^＋CD25^＋Foxp3^＋T细胞比例高于NR组[（7.54±1.50）％对（5.69±1.95）%，P=0.049]。单因素分析显示骨髓巨核细胞〈300个患儿HDD疗效较好（P=0.049）。结论HDD冲击治疗是ITP患儿一种比较理想的二线治疗选择；骨髓巨核细胞计数〈300个、CD4^＋CD25^＋Foxp3^＋T细胞比例高的患儿可能更适用于该疗法。

ObjectiveTo evaluate the efficacy and safety of pulsed high-dose dexamethasone（HDD）treatment in children with primary immune thrombocytopenic purpura（ITP）.MethodITP 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.Results①A 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. ③ Only 1 patient had mild reversible side effects during treatment. ④ The 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）.ConclusionPulsed 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.