"Assessing Long-Term Health-Related Quality of Life in Children with β-Thalassemia Major Following Hematopoietic Stem Cell Transplantation" - A Cross-Sectional Comparative Study.

This study aimed to compare the health-related quality of life (HRQoL) of children under 18 years with β-thalassemia major (β-TM) who are at least 2 years post-hematopoietic stem cell transplantation (HSCT) with that of children receiving conventional therapy. It was a cross-sectional comparative study involving 41 patients in the transplant (Tx) group and 59 patients in the non-transplant (NTx) group, both matched by age and sex. HRQoL was assessed using the PedsQL 4.0 Generic Core Scales. The median age at HRQoL assessment was 13 years for the Tx group and 10 years for the NTx group. In the Tx group, the median time from transplant to HRQoL assessment was 25.1 months, with a median follow-up of 38.2 months post-transplant (range 24.5-96.2 months). The Tx group had significantly higher HRQoL scores across all domains-physical, emotional, social, school functioning, psychosocial health, and overall health-compared to the NTx group (p < 0.05). Additionally, patients in the Tx group with serum ferritin levels below 2000 mg/dl reported better scores (p < 0.05). However, the presence of graft-versus-host disease negatively impacted HRQoL, especially in the social domain (p = 0.04). HSCT significantly improves long-term HRQoL in children with β-TM.