Hematopoietic stem cell transplantation (HSCT) is a curative treatment in primary immune deficiencies (PID), but at the same time increases the risk of treatment-related morbidity and problems affecting the child’s daily functioning. This study aimed to assess the quality of life (QoL) of children with PID after HSCT and to analyze the factors influencing their QoL.
This was a cross-sectional study, enrolling 25 patients followed after HSCT. The QoL was assessed using two assisted questionnaires for parents and children/adolescents: generic Pediatric Quality of Life (PedsQL 4.0) and the additional PedsQL “stem cell transplant” (SCT) module.
The mean age was 79.73 months ± 60.15 months (10 months-213 months). The overall score for the QoL reported by parents was well correlated with children’s responses. The overall QoL after HSCT was lower than the standards defined for healthy subjects. The most affected area after transplant was the academic function having the lowest score. The patient’s physical capacity was lower than that of the healthy subjects. The post-transplant emotional state and social relations were not altered with a score close to the norms of healthy subjects. The QoL assessed by the specific SCT module was also satisfactory, with overall parent and patient scores well correlated. Worry problems were most reported by parents and especially those of children under 4 years old. Communication had the lowest score in older children and adolescents. The PedsQL SCT module quality of life score in patients who received a non-myeloablative conditioning regimen was significantly greater than that in patients who had myeloablative treatment (p = 0.008). QoL was better far from HSCT (r = 0.8; p = 0.012).
After HSCT, patients generally do not achieve an especially physical QoL identical to healthy subjects. However, HSCT recipients maintain normal emotional and social functioning. In addition to monitoring physical function and HSCT-related symptoms, monitoring should also consider overall psychosocial functioning. Knowing the factors influencing the quality of life of children receiving HSCT is essential to improve long-term outcomes.
