Buchbinder David, Nugent Diane, Hsieh Loan
Division of Hematology, CHOC Children's Hospital and UC Irvine Medical Center, CA, USA.
Drug Des Devel Ther. 2017 Mar 30;11:1055-1063. doi: 10.2147/DDDT.S113191. eCollection 2017.
Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia. In approximately one-third of cases, the duration of thrombocytopenia will extend beyond 12 months consistent with a diagnosis of chronic ITP. Minor bleeding manifestations are common in chronic ITP while severe or life-threatening bleeding complications are uncommon. Moreover, spontaneous resolution occurs in the majority of children with chronic ITP necessitating treatment in only those children with ongoing bleeding manifestations or impairment in health-related quality of life (HRQOL). The characterization of thrombopoietin (TPO) and remarkable advancements in our understanding of the pathophysiology of ITP has led to the development of a new class of agents, the TPO-receptor agonists that have documented efficacy in the amelioration of thrombocytopenia and bleeding manifestations in chronic ITP. Romiplostim is a second-generation TPO-receptor agonist that has undergone limited evaluation in the treatment of chronic ITP in children. Evolving data suggest that romiplostim may be a safe and effective agent in the treatment of chronic ITP in children. Additional data are needed to confirm its ability to increase platelet counts, decrease bleeding manifestation, and improve the HRQOL of children and caregivers impacted by chronic ITP.
原发性免疫性血小板减少症（ITP）是一种以孤立性血小板减少为特征的自身免疫性疾病。在大约三分之一的病例中，血小板减少的持续时间将超过12个月，符合慢性ITP的诊断。轻微出血表现在慢性ITP中很常见，而严重或危及生命的出血并发症则不常见。此外，大多数慢性ITP儿童会自发缓解，仅那些有持续出血表现或健康相关生活质量（HRQOL）受损的儿童需要治疗。血小板生成素（TPO）的特性以及我们对ITP病理生理学认识的显著进展导致了一类新型药物的开发，即TPO受体激动剂，已证明其在改善慢性ITP患者的血小板减少和出血表现方面有效。罗米司亭是一种第二代TPO受体激动剂，在儿童慢性ITP治疗中的评估有限。不断发展的数据表明，罗米司亭可能是治疗儿童慢性ITP的一种安全有效的药物。需要更多数据来证实其增加血小板计数、减少出血表现以及改善受慢性ITP影响的儿童及其照料者HRQOL的能力。
