Elron Eyal, Yacobovich Joanne, Efros Orly, Tanous Osama, Levy-Mendelovich Sarina, Shamba Esti, Steinberg-Shemer Orna, Goldberg Tracie, Izraeli Shai, Gilad Oded
Department of Neonatology, Schneider Children's Medical Center of Israel, Petah Tikva, Israel.
School of Medicine, Tel Aviv University, Tel Aviv, Israel.
Ther Adv Hematol. 2024 Sep 17;15:20406207241279202. doi: 10.1177/20406207241279202. eCollection 2024.
Treatment of pediatric immune thrombocytopenia (ITP) is guided by the risk of bleeding. Intravenous immunoglobulin (IVIg) is one of the first-line therapy options for new-onset pediatric ITP. However, the exact optimal dose of IVIg has not been determined.
This retrospective cohort study included all hospitalized children with newly diagnosed ITP receiving IVIg as first-line therapy during 2010-2020. We compared the safety and efficacy of two common IVIg dose regimens, 1 and 2 g/kg. Outcomes were short and long-term treatment responses and adverse events to the different doses.
A total of 168 children were included in our cohort. Eighty-two children were treated with 1 g/kg of IVIg and 86 with 2 g/kg. There was no difference in sustained response (platelet count > 20 × 10, > 14 days) between the groups (74.3% vs 76.7%, respectively, = 0.72) and maximal platelet counts following treatment ( = 0.44). No difference was found regarding the percentage of chronic ITP between the two groups (24.4% in the 1 g/kg group as compared to 17.4% in the 2 g/kg group; = 0.34). Logistic regression analysis demonstrated there was no effect of the IVIg dose on treatment failure and development of chronic ITP. As anticipated, 47.7% of adverse events were in the 2 g/kg group and 32.9% in the 1 g/kg group, with borderline statistical significance ( = 0.06).
The initial treatment of newly diagnosed pediatric ITP using a 1 g/kg IVIg regimen may give comparable results to the double dose of 2 g/kg in attaining a prolonged safe hemostatic threshold, without impacting the incidence of chronic disease.
儿童免疫性血小板减少症（ITP）的治疗以出血风险为指导。静脉注射免疫球蛋白（IVIg）是新发性儿童ITP的一线治疗选择之一。然而，IVIg的确切最佳剂量尚未确定。
这项回顾性队列研究纳入了2010年至2020年期间所有住院的新诊断ITP儿童，他们接受IVIg作为一线治疗。我们比较了两种常见IVIg剂量方案（1和2 g/kg）的安全性和有效性。结果是不同剂量的短期和长期治疗反应以及不良事件。
我们的队列共纳入168名儿童。82名儿童接受1 g/kg的IVIg治疗，86名儿童接受2 g/kg的治疗。两组之间的持续反应（血小板计数>20×10，>14天）（分别为74.3%对76.7%，P=0.72）和治疗后的最大血小板计数（P=0.44）没有差异。两组之间慢性ITP的百分比没有差异（1 g/kg组为24.4%，2 g/kg组为17.4%；P=0.34）。逻辑回归分析表明，IVIg剂量对治疗失败和慢性ITP的发生没有影响。正如预期的那样，47.7%的不良事件发生在2 g/kg组，32.9%发生在1 g/kg组，具有边缘统计学意义（P=0.06）。
对于新诊断的儿童ITP，使用1 g/kg IVIg方案进行初始治疗在达到延长的安全止血阈值方面可能与2 g/kg的双倍剂量产生可比的结果，而不会影响慢性病的发生率。
