Vagelos College of Physicians and Surgeons, Columbia University Medical School, New York, New York, USA.
Princess Maxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Br J Haematol. 2023 Oct;203(1):17-22. doi: 10.1111/bjh.19072. Epub 2023 Aug 29.
Immune thrombocytopenia (ITP) in children is a relatively mild and self-limited disorder with the majority of children demonstrating normalization of platelet count by 12 months from diagnosis. Because of this, many children with ITP can be observed without the need for treatment. When needed, treatment with either intravenous immunoglobulin (IVIG) or corticosteroids is highly effective (>80% IVIG and >95% corticosteroids). For those children who require second-line therapies, response rates of >60% are seen with both the thrombopoietin-receptor agonists and rituximab. Despite this, some children will have 'refractory' ITP (rITP) with poor or transient responses to platelet-raising therapies. Here, we review the clinical features of rITP in children, outline proposed classifications and explore potential predictors for children with rITP.
儿童免疫性血小板减少症 (ITP) 是一种相对较轻且自限性疾病,大多数儿童在诊断后 12 个月内血小板计数恢复正常。正因为如此,许多 ITP 患儿可以不进行治疗而得到观察。当需要治疗时,静脉注射免疫球蛋白 (IVIG) 或皮质类固醇的治疗效果非常显著 (>80% IVIG 和 >95%皮质类固醇)。对于那些需要二线治疗的患儿,血小板生成素受体激动剂和利妥昔单抗的反应率>60%。尽管如此,一些患儿仍会出现“难治性” ITP (rITP),对血小板升高治疗的反应较差或短暂。在此,我们回顾了儿童 rITP 的临床特征,概述了提出的分类,并探讨了 rITP 患儿的潜在预测因素。