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Although hematopoietic stem cell transplantation (HSCT) has been widely used to treat pediatric patients with beta-thalassemia major, evidence showing whether this treatment improves health-related quality of life (HRQoL) is lacking. We used child-self and parent-proxy reports to prospectively evaluate HRQoL in 28 children with beta-thalassemia from Middle Eastern countries who underwent allogeneic HSCT in Italy. The PedsQL 4.0 Generic Core Scales were administered to patients and their parents 1 month before and 3, 6, and 18 months after transplantation. Two-year overall survival, thalassemia-free survival, mortality, and rejection were 89.3%, 78.6%, 10.9% and 14.3%, respectively. The cumulative incidence of acute and chronic graft-versus-host disease (GVHD) was 36% and 18%, respectively. Physical functioning declined significantly from baseline to 3 months after HSCT (median PedsQL score, 81.3 vs 62.5; P = .02), but then increased significantly up to 18 months after HSCT (median score, 93.7; P = .04). Agreement between child-self and parent-proxy ratings was high. Chronic GVHD was the most significant factor associated with lower HRQoL scores over time (P = .02). The child-self and parent-proxy reports showed improved HRQoL in the children with beta-thalassemia after HSCT. Overall, our study provides preliminary evidence-based data to further support clinical decision making in this area.

Original publication




Journal article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

Publication Date





861 - 866


Bone Marrow Transplant Center, R. Binaghi Hospital, Cagliari, Italy.


Humans, beta-Thalassemia, Graft vs Host Disease, Activities of Daily Living, Hematopoietic Stem Cell Transplantation, Sickness Impact Profile, Questionnaires, Incidence, Survival Analysis, Prospective Studies, Parents, Pediatrics, Health Status, Quality of Life, Adolescent, Child, Child, Preschool, Middle East, Italy, Female, Male