Treatment Status and Quality-of-Life Assessment of Transfusion-Dependent Thalassemia Managed with Standard-of-Care or Hematopoietic Stem Cell Transplantation in Southern Jiangxi, China: A Cross-Sectional Study

This study aimed to evaluate health-related quality of life (HRQoL) and its determinants in patients with transfusion-dependent thalassemia (TDT) to inform clinical decision-making and patient-centered outcomes. A cohort of 111 patients aged 2-17.9 years from Ganzhou, Jiangxi Province, was assessed using the Chinese versions of the Pediatric Quality of Life Inventory 4.0, with concurrent analysis of sociodemographic, clinical, and genetic parameters. Comparative analysis revealed that TDT patients who had undergone hematopoietic stem cell transplantation (HSCT) had statistically significant improvements in total HRQoL scores and across four specific subdomains-physical, emotional, social, and school functioning-compared with transfusion-dependent controls. Effect size analysis indicated the most pronounced intergroup differences in physical and school functioning, exceeding established minimal clinically important difference thresholds. After adjusting for age and ferritin levels via multivariable linear regression, ongoing transfusion dependence and the presence of complications were identified as independent predictors of reduced HRQoL scores. In conclusion, HSCT is associated with a significant improvement in quality of life for patients with TDT. These findings underscore the importance of standardizing transfusion protocols and proactively managing complications to optimize long-term outcomes in TDT care.