低剂量与传统剂量糖皮质激素治疗成人原发免疫性血小板减少症疗效和安全性比较

The comparison of efficacy and safety between low dose and conventional dose steroid therapy for adult patients with primary immune thrombocytopenia

目的 ：比较低剂量与传统剂量糖皮质激素治疗成人原发免疫性血小板减少症（ITP）的疗效和安全性。方法：对2011年1月1日至2013年8月31日在本院就诊的初治和既往激素敏感的复发ITP患者（n=49）给予口服醋酸泼尼松0.5 mg/（kg·d）,治疗3~4周,随后激素缓慢减量。同期接受醋酸泼尼松传统剂量[1 mg/（kg·d）]治疗的患者作为对照组（n=46）。随访资料收集截止期2014年8月31日。动态观察血小板计数,并监测生化指标、出血症状,记录不良事件。结果：低剂量组和传统剂量组治疗总体有效率（ORR）分别为81.6%和82.6%,其中初治的有效率分别为73.0%和80.0%,复发的有效率分别为94.7%和86.7%,2组间均没有统计学差异。2组患者血小板计数达到≥50×109/L和≥100×109/L中位时间均为2周,无统计学差异。血小板计数≥50×109/L维持时间大于6个月和12个月者,低剂量组分别为51.0%和30.6%,传统剂量组分别为45.7%和34.8%,没有统计学差异。2组患者治疗后出血表现均显著改善（均P〈0.000 1）。低剂量组不良事件发生率较低,失眠发生率较传统剂量组显著降低（P〈0.001）。结论：低剂量糖皮质激素治疗成人ITP疗效与传统剂量者相当,患者耐受性好。

Objective To compare the efficacy and safety between low dose （LD） and conventional dose （CD） steroid therapy for adult patients with primary immune thrombocytopenia （ITP）. Methods Adult ITP patients from Jan 1, 2011 to Aug 31, 2013 were enrolled. Prednisone at the dosage of 0.5 mg/（kg ·d） was given orally for 3 to 4 weeks and tapered gradually in LD group （n=49）. Patients received prednisone therapy at conventional dosage of 1 mg/（kg· d） were served as controls （CD group, n=46）. Follow-up was ended on Aug 31, 2014. Complete blood counts and biochemistry parameters were monitored. Bleeding and adverse events were documented. Results In LD and CD group, the overall response rate （ORR） was 81.6% and 82.6%, respectively. Response rates of initially treated patients and relapsed patients sensitive to steroids were 73.0% vs 80.0% and 94.7% vs 86.7%, respectively; these were of no statistical significance. The median time for platelet counts reaching ≥50×109/L and ≥100×109/L were 2 weeks in both LD and CD groups. Duration of platelet counts ≥50×109/L for ≥ 6 months and 〉i 12 months were 51.0% and 30.6% in LD group and 45.7% and 34.8% in CD group, respectively; these were of no statistical significance.. Bleeding symptoms ameliorated significantly in both groups after steroid therapy （P〈0.000 1）. Incidence of adverse events was lower in LD group. Insomnia occurred signifi- cantly less in LD group. Conclusions It is safe and effective to treat adult patients with primary immune thrombocytope- nia with LD steroid therapy.