Abstract
Background: Thalassemia is a hereditary hemolytic anemia disease. Most Chinese thalassemia patients can be diagnosed in initial episode, due to the fact that the prevention and treatment of thalassemia has achieved remarkable effects in recent two decades. The research of new therapies have also made considerable progress. Patients with transfusion-dependent thalassemia, especially those with β-Thalassemia Major(β-TM), require regular blood transfusions and iron chelation therapy. This places a large burden on patients and their families. The burden is not only physical pain, but also includes psychological and social dimensions. The unmet needs of the patient population are also worth studying. However, the overall QoL of Chinese thalassemia patients and its affecting factors remain unclear. Aims: The aim of this study was to investigate the level of QoL and ascertain its influencing factors in children with β-TM in China. Methods: The study data came from a nationwide and cross-sectional survey of patients with thalassemia in China (NCSTC, Ethical ID: IIT2021045-EC-1). Participants were recruited from 9 provinces and 1 municipality. Recruitment was based on the Beijing AngelMom Charity Foundation. Multistage stratified cluster sampling method was used for the sampling. The study used a web-based questionnaire to collect the QoL of children and the burden level of caregivers. The QoL was assessed using the Pediatric Quality of Life Inventory (PedsQL4.0), which contained 23 items in 4 dimensions (physical, emotional, social, and school functioning). The caregiver’s burden level was assessed by Zarit Burden Interview-22 (ZBI-22). The multiple linear regressions were used to analyze. Results: A total of 519 children (aged 2-17 years) with β-TM were obtained from NCSTC. The mean of PedsQL total score was 65.7 (standard deviation, 18.1). In the multi-adjusted linear regression model, participants with younger age (2-4 years= 72.1; 5-7 years= 67.8; 8-12 years = 64.3; 13-17 years= 60.5), without complications (with complications vs. no complications: 61.6 vs. 75.4), shorter course of disease (< 8 years vs. s 8 years: 67.6 vs. 63.8), without iron chemotherapy (with iron chemotherapy vs. without chemotherapy: 64.9 vs. 68.8), with lower caregiver burden level (0-20: 79.3, 21-40: 68.5, 41-60: 57.7, 61-80: 46.1), and receive transplantation treatment (with HLA transplantation vs. without HLA transplantation: 71.9 vs. 64.0) have higher PedsQL total score. The younger age, without complications, with shorter course of disease, with lower caregiver burden level, and receive transplantation treatment were all related to higher performance of physical, emotional, social, and school functioning (Figure 1). Image:Summary/Conclusion: The health-related QoL assessed by PedsQL scores in Chinese children with β-TM were lower than that in previous studies. Multiple factors were associated with the QoL of Chinese children with β-TM, and should be comprehensively paying attention to physical, emotional, social, and school functioning in β-TM patients.
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