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.
Haiyan Liang, Yang Yang, Lin Pan, Yanni Xie, Yongrong Lai
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引用次数: 0
Abstract
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.
背景:本横断面研究评估了接受输血和铁螯合治疗的中国β-地中海贫血(β-TM)患者的健康相关生活质量(HRQOL)结局和影响HRQOL的因素。研究设计和方法:采用儿童生活质量量表(PedsQL)4.0通用核心量表对198例4-18岁的患者和431名健康对照者进行评估。采用关联分析和线性回归分析,分析社会人口学因素和临床因素与hrqolscore的关系。结果:与健康同龄人相比,患者在所有领域的PedsQL评分均显着降低(p p = 0.034)。铁螯合治疗和输血前血红蛋白水平超过7.5 g/dL是总总评分的显著阳性预测因子,而两种合并症的存在被确定为显著的阴性预测因子(p = 0.037, p p = 0.001)。结论:β-TM患者需要进一步加强输血和铁螯合治疗等规范化治疗。此外,改善营养状况和预防和治疗合并症可能有利于患者的HRQOL。
期刊介绍:
Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.