Flores Adolfo, Klaassen Robert J, Buchanan George R, Neunert Cindy E
Department of Pediatrics, Children's Medical Center Dallas, University of Texas Southwestern Medical Center, Dallas, Texas.
Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada.
Pediatr Blood Cancer. 2017 Aug;64(8). doi: 10.1002/pbc.26405. Epub 2017 Jan 23.
Relationships between clinical/demographic factors and health-related quality of life (HRQoL) in childhood immune thrombocytopenia (ITP) remain poorly understood. Recent studies reveal conflicting information about factors that contribute to HRQoL.
This was a prospective, single-institution, cohort study of newly diagnosed children with ITP. Serial evaluations of HRQoL were performed using the Kid's ITP Tools (KIT), scored from 0 (worst) to 100 (best), at enrollment and 1 week, 6 months, and 12 months following diagnosis. All visits included bleeding severity grading. Relationships between HRQoL and platelet count, treatment, bleeding severity, and course of disease were examined.
A total of 99 children with newly diagnosed ITP were evaluable for analysis. KIT scores were low at diagnosis for parents (median 26, range 15-43) and children (median 65, range 55-81) and were not influenced by age or platelet count. At diagnosis, children who received treatment had lower platelet counts (P = 0.005), more severe hemorrhage (P < 0.0125), and lower HRQoL by parent, child, and proxy reporting (P < 0.05). Oral bleeding negatively impacted proxy-reported disease burden at diagnosis (P = 0.01). Persistence of disease and lower platelet counts at 6 and 12 month visits were the only factors noted to consistently impact quality of life beyond diagnosis for both parents and children.
HRQoL is low at diagnosis but significantly improves over time. Patients with ongoing disease and lower platelet counts continue to have significant disease burden.
儿童免疫性血小板减少症（ITP）的临床/人口统计学因素与健康相关生活质量（HRQoL）之间的关系仍未得到充分理解。最近的研究揭示了有关影响HRQoL的因素的相互矛盾的信息。
这是一项对新诊断的ITP患儿进行的前瞻性、单机构队列研究。使用儿童ITP工具（KIT）对HRQoL进行系列评估，评分范围为0（最差）至100（最佳），在入组时以及诊断后1周、6个月和12个月进行。所有访视均包括出血严重程度分级。研究了HRQoL与血小板计数、治疗、出血严重程度和病程之间的关系。
共有99例新诊断的ITP患儿可纳入分析。诊断时，父母的KIT评分较低（中位数26，范围15 - 43），儿童的KIT评分也较低（中位数65，范围55 - 81），且不受年龄或血小板计数影响。诊断时，接受治疗的儿童血小板计数较低（P = 0.005），出血更严重（P < 0.0125），根据父母、儿童和代理人报告，其HRQoL较低（P < 0.05）。口腔出血对诊断时代理人报告的疾病负担有负面影响（P = 0.01）。疾病持续存在以及在6个月和12个月访视时血小板计数较低是唯一被指出在诊断后持续影响父母和儿童生活质量的因素。
诊断时HRQoL较低，但随时间显著改善。疾病持续且血小板计数较低的患者继续承受重大疾病负担。
