Anfas Muneeb, Sana Inam, Ijaz Ali, Abdullah Ali Alshehri, Maham Siddiq, Salamat Ali
Pediatric β-TM patients reported moderate overall adherence to ICT. Clinical outcomes showed improvement in anemia and increased iron overload. HRQoL was moderately affected, especially in school functioning. Patients with low adherence had lower HRQoL and increased serum ferritin levels, reflecting suboptimal clinical outcomes.
BACKGROUND: Thalassemia is a chronic blood disorder requiring lifelong blood transfusions and iron chelation therapy (ICT) to reduce iron overload.
PURPOSE: In Pakistan, limited evidence exists on how medication adherence, clinical outcomes, and health-related quality of life (HRQoL) interact simultaneously in β-thalassemia major (β-TM) children. This study therefore aimed to evaluate these variables and examine their interrelationships in pediatric β-TM patients receiving ICT.
METHODS: A cross-sectional observational study was conducted among 622 patients aged 5-12 years across six thalassemia centers in Punjab, Pakistan (September 2024-February 2025). For data collection, Treatment Adherence Scale (TAS) and Paediatric Quality of Life Inventory™ (PedsQL™) 4.0 Generic Core Scales were used. Clinical outcomes were assessed via hematological and iron overload parameters. Statistical analyses, including non-parametric tests, Friedman test, multiple regression analysis, and Spearman's rank correlation, with p < 0.05 considered significant.
RESULTS: The total adherence score was 6 IQR (3-8). Clinical outcomes showed significant improvement in hemoglobin levels, while serum ferritin remained elevated. Younger age, urban residency, and deferasirox monotherapy were significantly associated with higher adherence and HRQoL. The total QoL score was 67.3 IQR (50-84.4). Family history, deferoxamine use, and serum ferritin were identified as negative predictors of adherence and HRQoL, whereas urban residency, splenectomy, and higher adherence to ICT were positive predictors of HRQoL. A significant positive correlation was found between adherence and HRQoL (ρ = 0.420, p < 0.001), while serum ferritin was negatively correlated with adherence (ρ = -0.476, p < 0.001) and HRQoL (ρ = -0.329, p < 0.001).
CONCLUSION: Pediatric β-TM patients reported moderate overall adherence to ICT. Clinical outcomes showed improvement in anemia and increased iron overload. HRQoL was moderately affected, especially in school functioning. Patients with low adherence had lower HRQoL and increased serum ferritin levels, reflecting suboptimal clinical outcomes.