Health-Related Quality of Life in Thai Children with Thalassemia as Evaluated by PedsQL and EQ-5D-Y: A Single-Center Experience.

Background: 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.

Methods: 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.

Results: 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.

Conclusion: 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.