Thakur Yash, Meshram Revat J, Taksande Amar
Pediatrics, Jawaharlal Nehru Medical College, Datta Meghe Institute of Higher Education and Research, Wardha, IND.
Cureus. 2024 Sep 18;16(9):e69635. doi: 10.7759/cureus.69635. eCollection 2024 Sep.
Immune thrombocytopenia (ITP) in paediatric patients is a complex and heterogeneous disorder characterized by isolated thrombocytopenia and an increased risk of bleeding. The diagnosis of ITP involves a careful exclusion of other causes of thrombocytopenia, supported by clinical evaluation and laboratory findings. Management strategies have evolved significantly, emphasizing individualized treatment approaches based on disease severity, bleeding risk, and patient-specific factors. This comprehensive review provides an in-depth analysis of the current diagnostic criteria, including the role of novel biomarkers and genetic testing in distinguishing ITP from other haematological disorders. We also explore the latest therapeutic options, ranging from observation and first-line treatments such as corticosteroids and intravenous immunoglobulin (IVIG) to second-line therapies, including thrombopoietin receptor agonists and immunosuppressive agents. The review addresses the challenges of managing chronic ITP in pediatric patients, focusing on balancing treatment efficacy with the potential side effects and long-term outcomes. Additionally, we discuss the emerging role of personalized medicine in optimizing care for children with ITP, highlighting recent advances in targeted therapies and the potential for future research to refine diagnostic and treatment paradigms to refine diagnostic and treatment paradigms further.
儿童免疫性血小板减少症(ITP)是一种复杂的异质性疾病,其特征为孤立性血小板减少和出血风险增加。ITP的诊断需要仔细排除其他血小板减少的原因,并辅以临床评估和实验室检查结果。管理策略已发生显著演变,强调基于疾病严重程度、出血风险和患者特定因素的个体化治疗方法。本综述全面深入地分析了当前的诊断标准,包括新型生物标志物和基因检测在鉴别ITP与其他血液系统疾病中的作用。我们还探讨了最新的治疗选择,从观察以及皮质类固醇和静脉注射免疫球蛋白(IVIG)等一线治疗,到二线治疗,包括血小板生成素受体激动剂和免疫抑制剂。该综述探讨了儿童慢性ITP管理面临的挑战,重点在于平衡治疗效果与潜在副作用及长期预后。此外,我们讨论了个性化医疗在优化ITP患儿护理方面的新兴作用,强调靶向治疗的最新进展以及未来研究进一步完善诊断和治疗模式的潜力。