Neunert Cindy E, Lambert Michele P
Columbia University Irving Medical Center, New York City, New York, USA.
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Br J Haematol. 2024 Oct;205(4):1265-1266. doi: 10.1111/bjh.19719. Epub 2024 Aug 27.
In their paper, Klaassen et al. present their findings on the revised Kids ITP Tools (KIT). This important work provides an update to the validated measure for evaluating health-related quality of life (HRQoL) in children with immune thrombocytopenia (ITP). ITP, an acquired autoimmune disorder, results in thrombocytopenia and places children at risk for significant bleeding. Thankfully, the majority of children with ITP will have a brief disease course and no or mild bleeding symptoms. The rarity of severe bleeding events or the development of chronic disease provides a challenge with regard to clinical trial design, making alternative measures of pharmacological efficacy extremely important. Commentary on: Dhir et al. Quality of life in childhood immune thrombocytopenia: Revision of the Kids' ITP Tools (KIT). Br J Haematol 2024; 205:1489-1496.
在他们的论文中，克拉斯森等人展示了他们关于修订版儿童免疫性血小板减少症工具（KIT）的研究结果。这项重要工作更新了用于评估免疫性血小板减少症（ITP）患儿健康相关生活质量（HRQoL）的经过验证的测量方法。ITP是一种获得性自身免疫性疾病，会导致血小板减少，并使儿童面临严重出血的风险。幸运的是，大多数ITP患儿的病程较短，没有出血症状或仅有轻微出血症状。严重出血事件或慢性病的发生较为罕见，这给临床试验设计带来了挑战，因此药理学疗效的替代测量方法极为重要。评论：迪尔等人。儿童免疫性血小板减少症的生活质量：儿童ITP工具（KIT）的修订。《英国血液学杂志》2024年；205:1489 - 1496。
