Lightening the load for paediatric ITP: The importance of health-related quality of life.

IF 5.1 2区 医学 Q1 HEMATOLOGY British Journal of Haematology Pub Date : 2024-08-27 DOI:10.1111/bjh.19719
Cindy E Neunert, Michele P Lambert
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Abstract

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 (Online ahead of print). doi: 10.1111/bjh.19662.

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减轻小儿 ITP 的负担:与健康相关的生活质量的重要性。
Klaassen 等人在论文中介绍了他们对修订后的儿童 ITP 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)的有效测量方法。免疫性血小板减少症是一种获得性自身免疫性疾病,会导致血小板减少,并使儿童面临大量出血的风险。值得庆幸的是,大多数ITP患儿病程短暂,没有或仅有轻微出血症状。严重出血事件或慢性疾病发展的罕见性为临床试验设计带来了挑战,因此,替代性药效测量方法极为重要。相关评论Dhir 等人儿童免疫性血小板减少症的生活质量:修订儿童 ITP 工具 (KIT)。Doi: 10.1111/bjh.19662.
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来源期刊
CiteScore
8.60
自引率
4.60%
发文量
565
审稿时长
1 months
期刊介绍: The British Journal of Haematology publishes original research papers in clinical, laboratory and experimental haematology. The Journal also features annotations, reviews, short reports, images in haematology and Letters to the Editor.
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