Sinlapamongkolkul Phakatip, Surapolchai Pacharapan
Department of Pediatrics, Faculty of Medicine, Thammasat University, Thailand.
Mediterr J Hematol Infect Dis. 2020 Jul 1;12(1):e2020036. doi: 10.4084/MJHID.2020.036. eCollection 2020.
Thalassemia remains a challenging chronic disease in Thailand, but national prenatal screening, along with better treatment and management, may have improved health-related quality of life HRQoL for pediatric patients. We aimed to measure the HRQoL of transfusion-dependent TDT and non-transfusion dependent NTDT of these pediatric patients at our institute.
We included all patients 2 - 18 years old, with TDT and NTDT, using the Pediatric Quality of Life Inventory 4.0 Generic Core Scales PedsQL and the EuroQol Group's Five Dimensions for Youth EQ-5D-Y instruments. Patients and caregivers responded as appropriate for age.
Mean PedsQL total summary scores TSSSD of child self-reports and parent proxy-reports were 81.00 10.94 and 78.84 16.72 from 150 participants. Mean EQ-5D-Y VAS SD for children was 89.27 11.56 and 86.72 10.62 for parent proxies. The most problematic EQ-5D-Y dimension was "having pain or discomfort". These scores had significant correlations between the child and parental proxy perspectives, as well as between the PedsQL and EQ-5D-Y. An age of 8 - 12 years and oral chelation therapy predicted lower self-reported PedsQL TSS. Parental proxy-report predictors for reduced PedsQL TSS and EQ-5D-Y VAS were primary school education for children, parental proxy secondary school education, Universal Coverage insurance, and TDT.
HRQoL scores of our pediatric thalassemia patients had improved from the previous decade, and these findings may represent our better standard of care. Some sociodemographic and clinical characteristics may present negative impacts on HRQoL. More exploration is needed to understand predictors and further improve HRQoL, especially for TDT patients.
地中海贫血在泰国仍然是一种具有挑战性的慢性疾病，但全国范围内的产前筛查，以及更好的治疗和管理，可能已经改善了儿科患者与健康相关的生活质量（HRQoL）。我们旨在测量我院这些儿科患者中依赖输血的重型地中海贫血（TDT）和非依赖输血的轻型地中海贫血（NTDT）患者的HRQoL。
我们纳入了所有2至18岁的TDT和NTDT患者，使用儿童生活质量量表4.0通用核心量表（PedsQL）和欧洲生活质量研究组的青少年五维度量表（EQ-5D-Y）。患者和照顾者根据年龄进行相应回答。
150名参与者的儿童自我报告和家长代理报告的PedsQL总总结得分（TSSSD）平均值分别为81.00±10.94和78.84±16.72。儿童的EQ-5D-Y视觉模拟量表（VAS）平均值为89.27±11.56，家长代理报告的平均值为86.72±10.62。EQ-5D-Y中最成问题的维度是“有疼痛或不适”。这些得分在儿童和家长代理视角之间，以及PedsQL和EQ-5D-Y之间存在显著相关性。8至12岁的年龄和口服螯合疗法预示着自我报告的PedsQL TSS较低。家长代理报告中，儿童小学教育、家长代理中学教育、全民覆盖保险和TDT是PedsQL TSS和EQ-5D-Y VAS降低的预测因素。
我们儿科地中海贫血患者的HRQoL得分较前十年有所提高，这些结果可能代表了我们更好的护理标准。一些社会人口学和临床特征可能对HRQoL产生负面影响。需要更多的探索来了解预测因素并进一步改善HRQoL，特别是对于TDT患者。
