Health-related quality of life in pediatric patients with β-thalassemia major after hematopoietic stem cell transplantation.

Our main purpose was to evaluate health-related quality of life (HRQOL) in β-thalassemia major patients who underwent transplantation from September 2012 to November 2019. The PedsQL 4.0 scale proxy version was administered to 221 transplant patients aged 5-18 years. Their HRQOL results in the proxy-report were compared with those in the proxy-report of 429 matched healthy peers and 198 matched nontransplant patients. Compared with their healthy peers, the transplant patients exhibited worse HRQOL in the physical health domain (P < 0.001), school domain (P < 0.001) and overall scores (P = 0.006). Patients within 4 years after transplantation exhibited physical functioning (P < 0.001), school functioning (P < 0.001) and overall HRQOL damage (P = 0.001); the scores across all domains for patients more than 4 years after transplantation were not significantly different from those for the healthy controls. The transplant patients rated their HRQOL for all domains better than the nontransplant patients (P < 0.001). The HRQOL of patients after human leukocyte antigen (HLA)-matched related and HLA-matched unrelated donor transplantation were not significantly different. Chronic graft-versus-host disease and two or more comorbidities were independently negatively associated with overall HRQOL outcomes (P = 0.032 and P < 0.001, respectively). In conclusion, patients more than 4 years after transplantation achieve an HRQOL equal to that of their healthy peers.