Expert review of hematology · 2026
Health-related quality of life and associated factors in Chinese pediatric patients with β-thalassemia major receiving transfusion and iron chelation therapy: a cross-sectional study.
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Liang Haiyan, Yang Yang, Pan Lin, Xie Yanni, Lai Yongrong
Аннотация
BACKGROUND: This cross-sectional study assessed health-related quality of life (HRQOL) outcomes and the factors affecting HRQOL in Chinese patients with β-thalassemia major (β-TM) receiving transfusion and iron chelation therapy.
RESEARCH DESIGN AND METHODS: The Pediatric Quality of Life Inventory (PedsQL)4.0 Generic Core Scales were administered to 198 patients and 431 heathy controls aged 4-18 years old. The relationship of sociodemographic and clinical factors with HRQOLscores were analyzed using association and linear regression analyses.
RESULTS: Compared with healthy peers, patients exhibited significantly lower PedsQL scores across all domains (p < 0.001). Patients with Body Mass Index (BMI) below the 50th percentile (the median BMI for Chinese children and adolescents) exhibited poorer HRQOL scores than those with BMI ator above this threshold in physical functioning (p = 0.034). Iron chelation therapy and pre-transfusion hemoglobin levels exceeding 7.5 g/dL emerged as significant positive predictors of total summary score, while the presence of two comorbidities was identified as significant negative predictors (p = 0.037, p < 0.001 and p = 0.001, respectively).
CONCLUSIONS: Standardized treatment involving blood transfusion and iron chelation therapy in β-TM patients requires further enhancement. Additionally, improving nutritional status and preventing and treating comorbidities may benefit patients' HRQOL.
RESEARCH DESIGN AND METHODS: The Pediatric Quality of Life Inventory (PedsQL)4.0 Generic Core Scales were administered to 198 patients and 431 heathy controls aged 4-18 years old. The relationship of sociodemographic and clinical factors with HRQOLscores were analyzed using association and linear regression analyses.
RESULTS: Compared with healthy peers, patients exhibited significantly lower PedsQL scores across all domains (p < 0.001). Patients with Body Mass Index (BMI) below the 50th percentile (the median BMI for Chinese children and adolescents) exhibited poorer HRQOL scores than those with BMI ator above this threshold in physical functioning (p = 0.034). Iron chelation therapy and pre-transfusion hemoglobin levels exceeding 7.5 g/dL emerged as significant positive predictors of total summary score, while the presence of two comorbidities was identified as significant negative predictors (p = 0.037, p < 0.001 and p = 0.001, respectively).
CONCLUSIONS: Standardized treatment involving blood transfusion and iron chelation therapy in β-TM patients requires further enhancement. Additionally, improving nutritional status and preventing and treating comorbidities may benefit patients' HRQOL.