Department of Pediatric Hematology, University Medical Center Utrecht, Utrecht, The Netherlands.
Department of Pediatrics, Flevo Hospital, Almere, The Netherlands.
Blood. 2018 Aug 30;132(9):883-891. doi: 10.1182/blood-2018-02-830844. Epub 2018 Jun 26.
Management of children with newly diagnosed immune thrombocytopenia (ITP) consists of careful observation or immunomodulatory treatment. Observational studies suggest a lower risk for chronic ITP in children after intravenous immunoglobulin (IVIg) treatment. In this multicenter randomized trial, children aged 3 months to 16 years with newly diagnosed ITP, platelet counts 20 × 10/L or less, and mild to moderate bleeding were randomly assigned to receive either a single infusion of 0.8 g/kg IVIg or careful observation. Primary outcome was development of chronic ITP, which at the time of study initiation was defined as a platelet count lower than 150 × 10/L after 6 months. Two hundred six children were allocated to receive IVIg (n = 102) or careful observation (n = 104). Chronic ITP occurred in 18.6% of the patients in the IVIg group and 28.9% in the observation group (relative risk [RR], 0.64; 95% confidence interval [CI], 0.38-1.08). Platelet counts lower than 100 × 10/L at 12 months (current definition of chronic ITP) were observed in 10% of children in the IVIg group and 12% in the observation group (RR, 0.83; 95% CI, 0.38-1.84). Complete response rates in the first 3 months were significantly higher in the IVIg group. Immunoglobulin G Fc receptor IIb genetic variations were associated with early complete response in both groups. Grade 4 to 5 bleeding occurred in 9% of the patients in the observation group vs 1% in the IVIg group. This trial was registered at www.trialregister.nl as NTR 1563.
新诊断的免疫性血小板减少症（ITP）患儿的治疗包括密切观察或免疫调节治疗。观察性研究表明，静脉注射免疫球蛋白（IVIg）治疗后，儿童慢性 ITP 的风险较低。在这项多中心随机试验中，3 个月至 16 岁新诊断的 ITP 患儿，血小板计数为 20×10/L 或更低，且有轻至中度出血，被随机分配接受单次 0.8 g/kg IVIg 输注或密切观察。主要结局是发生慢性 ITP，在研究开始时定义为 6 个月后血小板计数低于 150×10/L。206 名患儿被分配接受 IVIg（n=102）或密切观察（n=104）。IVIg 组中 18.6%的患儿发生慢性 ITP，观察组中 28.9%的患儿发生慢性 ITP（相对风险 [RR]，0.64；95%置信区间 [CI]，0.38-1.08）。12 个月时血小板计数低于 100×10/L（目前慢性 ITP 的定义），IVIg 组有 10%的患儿，观察组有 12%的患儿（RR，0.83；95%CI，0.38-1.84）。IVIg 组在前 3 个月内完全缓解率显著更高。两组患儿的免疫球蛋白 G Fc 受体 IIb 遗传变异与早期完全缓解相关。观察组中 9%的患儿发生 4 级至 5 级出血，IVIg 组中 1%的患儿发生 4 级至 5 级出血。该试验在 www.trialregister.nl 上注册为 NTR 1563。
