Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation

Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative treatment in pediatric patients with beta thalassemia major (beta-TM). However, little data are available regarding patients and their parents ' health-related quality of life (HRQoL) after the procedure. As such, we investigated the HRQoL of pediatric patients with beta-TMafter HSCT compared to that of patients treated with blood transfusions and iron chelation. The health-related quality of life of 43 beta-TM pediatric patients and 43 parents were evaluated using the Pediatric Quality of Life Inventory (PedsQL). A total of 25 patients underwent HSCT: 15 from a sibling and 10 from an HLA-matched donor. The median follow-up time from HSCT was 5 years (range 1-13 years). The mean ages at the survey were 10.1 years (range 5-15) and 9.6 years (range 5-15) for transfused and transplanted patients, respectively. A significant reduction inHRQoLwas reported in the group of transfused patients comparedwith that of patients transplanted in the following PedsQL domains: children ' s and parents ' physical functions, Delta = 15.4, p = 0.009 and Delta = 11.3, p = 0.002, respectively; children ' s and parents ' emotional functioning, Delta = 15.2, p = 0.026 and Delta = 15.2, p = 0.045, respectively; child ' s and parents ' school functioning, Delta = 25, p = 0.005 and Delta = 22.5, p = 0.011, respectively; total child and parents scores, Delta = 14.5, p = 0.004 and Delta = 13.2, p = 0.005, respectively. The results of a multivariable analysis showed that the HSCT procedure was significantly associated with a higher total child PedsQL score (adjusted mean difference = 15.3, p = 0.001) and a higher total parent PedsQL score (adjusted mean difference = 14.1, p = 0.006). We found no significant difference in the HRQoL measured after sibling or unrelated human leukocyte antigen (HLA)-matched HSCT. Finally, a significant positive correlation across all the PedsQL domains was found between the scores reported by the children and those reported by their parents. In conclusion, our study shows that HSCT in pediatric patients with fi-TMis associated with a good overall HRQoL profile. This information further supports physicians when counseling patients and their parents before the HSCT procedure.

Automated summary

This study investigated the health-related quality of life (HRQoL) of pediatric patients with beta thalassemia major (beta-TM) after hematopoietic stem cell transplantation (HSCT), compared to those treated with blood transfusions. The results showed that HSCT was associated with a higher HRQoL in various domains, and there was no significant difference in HRQoL between sibling or unrelated HLA-matched HSCT. The study highlights the importance of considering HSCT as a treatment option for pediatric patients with beta-TM and provides valuable information for physicians to counsel patients and parents.