Objectives To investigate the impact of gene therapy on the health-related quality of life (HRQoL) of pediatric patients with transfusion-dependent β-thalassemia (TDT), and to assess the changes in anxiety and depression levels among their family caregivers during the therapy.
Methods A total of 9 pediatric patients with TDT and their family caregivers were enrolled in this prospective cohort study from July 2020 to July 2023. The Chinese version of the Pediatric Quality of Life Inventory 4.0 (PedsQL 4.0) was used to compare changes in children's HRQoL before and 6 months after receiving gene therapy. The Hamilton Anxiety Rating Scale (HAMA) and Hamilton Depression Rating Scale (HAMD) were used to assess the anxiety and depression levels in the family caregivers before and after children's gene therapy.
Results The TDT patient cohort included 3 males and 6 females with a median age of 12 years (range: 5-16 years). The total PedsQL 4.0 score post-gene therapy was significantly higher than the pre-gene therapy score (post: 90.46±11.42, pre: 76.45±10.48, t=-7.79, P<0.001). Scores for both physical function and school performance at 6 months post-gene therapy were also higher than those pre-gene therapy (physical function: t=-7.17, school performance: t=-5.36, both P<0.001). Prior to gene therapy, 3 family caregivers had mild anxiety and 7 had mild to moderate depression. After gene therapy, 1 caregiver had mild anxiety and 1 had mild depression. There was a statistically significant difference in the HAMD score before and after gene therapy (t=3.98, P=0.004), with medians of 9 (range: 4-15) and 4 (range: 2-8), respectively.
Conclusions The HRQoL of pediatric TDT patients improved 6 months after gene therapy, and their family caregivers experienced decreased anxiety and depression. Gene therapy can effectively enhance the health outcomes of both TDT patients and their caregivers.
No relevant conflicts of interest to declare.
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