Central Manchester University Hospitals NHS Foundation Trust, NIHR/Wellcome Trust Manchester CRF, Royal Manchester Children's Hospital, University of Manchester, Manchester, England.
Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.
Br J Haematol. 2019 Apr;185(1):102-106. doi: 10.1111/bjh.15732. Epub 2018 Dec 27.
The PETIT (Eltrombopag in Pediatric Patients with Thrombocytopenia from Chronic ITP) trial showed that in children aged 1-17 years with chronic or persistent immune thrombocytopenia (ITP), eltrombopag improved platelet counts, decreased clinically significant bleeding and reduced rescue medication need. We report the health-related quality of life (HRQoL) results from the PETIT study using the Kids' ITP Tools (KIT). A limitation was that PETIT was not powered for the HRQoL analysis. Eltrombopag did not impact children's HRQoL assessed by the KIT. Although median KIT scores in children treated with eltrombopag with platelet responses were numerically higher compared with non-responders in some age groups, the interquartile ranges overlapped.
PETIT(Eltrombopag 在患有慢性 ITP 的血小板减少症的儿科患者中的试验)表明,在年龄为 1-17 岁的患有慢性或持续性免疫性血小板减少症 (ITP) 的儿童中,艾曲波帕可提高血小板计数,减少临床显著出血,并减少抢救药物的需求。我们使用 Kids' ITP Tools (KIT) 报告了来自 PETIT 研究的与健康相关的生活质量 (HRQoL) 结果。一项限制是 PETIT 没有针对 HRQoL 分析进行调整。艾曲波帕并未影响 KIT 评估的儿童 HRQoL。虽然在一些年龄组中,血小板反应性治疗的儿童的 KIT 中位数评分数值高于无反应者,但四分位间距重叠。