Prospective Assessment of Health-Related Quality of Life in Pediatric Patients with Beta-Thalassemia following Hematopoietic Stem Cell Transplantation

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 5 .02), but
then increased significantly up to 18 months after HSCT (median score, 93.7; P 5.04). Agreement between
child-self and parent-proxy ratings was high. Chronic GVHD was the most significant factor associated with
lower HRQoL scores over time (P5.02). 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.