Pokorska-Śpiewak Maria, Dobrzeniecka Anna, Talarek Ewa, Aniszewska Małgorzata, Pluta Magdalena, Marczyńska Magdalena, Indolfi Giuseppe
From the Department of Children's Infectious Diseases, Medical University of Warsaw, Warsaw, Poland.
Department of Pediatric Infectious Diseases, Regional Hospital of Infectious Diseases in Warsaw, Warsaw, Poland.
Pediatr Infect Dis J. 2025 May 1;44(5):405-410. doi: 10.1097/INF.0000000000004675. Epub 2024 Dec 20.
The aim of this study was to assess the health-related quality of life (HRQL) of children with chronic hepatitis C (CHC) at 1 year after the effective treatment with sofosbuvir/velpatasvir (SOF/VEL).
All 50 patients treated for CHC with a fixed dose SOF/VEL in the noncommercial, nonrandomized, open-label PANDAA-PED study achieved sustained virologic response at 12 weeks after the end of treatment. Evaluation of HRQL at 1-year posttreatment was compared with the baseline (before the treatment) assessment. KIDSCREEN-27 questionnaires, which included 5 dimensions of HRQL, for child self-reporting and parent proxy reporting were used. The normal range for the population was set to T values of 40-60 points. Child-parent agreement was analyzed using the intraclass correlation coefficient (ICC).
Mean T values were within the normal range for all HRQL dimensions. A significant improvement in "autonomy & parent relation" in children's self-assessment (from 48.3 to 51.5, P = 0.03) was observed. In parent proxy assessment, a significant decrease occurred in "school" dimension (from 49.5 to 45.8, P = 0.03), which was not revealed at 3-month posttreatment. Older age was associated with worse HRQL scores in all dimensions. Evaluation of the ICC for child self-reports versus parent proxy reports revealed poor-to-moderate agreement for most single measures, lower than at 3-month posttreatment analysis.
This is the first study to present the long-term influence of treatment with direct-acting antivirals on patient-reported outcomes in children. At 1 year after effective treatment with SOF/VEL, an improvement in some areas of children's well-being was revealed, which may indicate also some patient-reported outcomes benefits of direct-acting antiviral therapy. Despite the improvement in the child self-report of "autonomy & parent relation," there was a more pronounced discrepancy between children self-reports and parents proxy reports in all dimensions of HRQL. Older patients' age correlated with worse HRQL assessment. If this finding is mediated by the duration of hepatitis C virus infection, it would support recommendation for the treatment of younger children.
本研究旨在评估索磷布韦/维帕他韦（SOF/VEL）有效治疗1年后慢性丙型肝炎（CHC）患儿的健康相关生活质量（HRQL）。
在非商业性、非随机、开放标签的PANDAA-PED研究中，所有50例接受固定剂量SOF/VEL治疗CHC的患者在治疗结束后12周均实现了持续病毒学应答。将治疗后1年的HRQL评估结果与基线（治疗前）评估结果进行比较。使用KIDSCREEN-27问卷，该问卷包括HRQL的5个维度，用于儿童自我报告和家长代理报告。设定人群正常范围为T值40 - 60分。使用组内相关系数（ICC）分析儿童与家长的一致性。
所有HRQL维度的平均T值均在正常范围内。在儿童自我评估中，“自主性与亲子关系”有显著改善（从48.3升至51.5，P = 0.03）。在家长代理评估中，“学校”维度有显著下降（从49.5降至45.8，P = 0.03），这在治疗后3个月时未显现。年龄较大与所有维度的HRQL评分较差相关。对儿童自我报告与家长代理报告的ICC评估显示，大多数单项测量的一致性较差至中等，低于治疗后3个月分析时的情况。
这是第一项展示直接作用抗病毒药物治疗对儿童患者报告结局的长期影响的研究。在使用SOF/VEL有效治疗1年后，发现儿童幸福感的某些方面有所改善，这也可能表明直接作用抗病毒治疗在患者报告结局方面有一些益处。尽管儿童自我报告的“自主性与亲子关系”有所改善，但在HRQL的所有维度上，儿童自我报告与家长代理报告之间仍存在更明显的差异。年龄较大的患者HRQL评估较差。如果这一发现是由丙型肝炎病毒感染持续时间介导的，那么将支持对年幼儿童进行治疗的建议。
