姚婉茹, 张纪水, 吴润晖. 综合评价体系在中国儿童血友病治疗中的发展与应用[J]. 罕见病研究, 2022, 1(4): 380-385. DOI: 10.12376/j.issn.2097-0501.2022.04.004
引用本文: 姚婉茹, 张纪水, 吴润晖. 综合评价体系在中国儿童血友病治疗中的发展与应用[J]. 罕见病研究, 2022, 1(4): 380-385. DOI: 10.12376/j.issn.2097-0501.2022.04.004
YAO Wanru, ZHANG Jishui, WU Runhui. Development and Application of Comprehensive Evaluation System in the Treatment of Children with Hemophilia in China[J]. Journal of Rare Diseases, 2022, 1(4): 380-385. DOI: 10.12376/j.issn.2097-0501.2022.04.004
Citation: YAO Wanru, ZHANG Jishui, WU Runhui. Development and Application of Comprehensive Evaluation System in the Treatment of Children with Hemophilia in China[J]. Journal of Rare Diseases, 2022, 1(4): 380-385. DOI: 10.12376/j.issn.2097-0501.2022.04.004

综合评价体系在中国儿童血友病治疗中的发展与应用

Development and Application of Comprehensive Evaluation System in the Treatment of Children with Hemophilia in China

  • 摘要: 作为遗传性出血性疾病,血友病以自发性出血为特征,特别是关节出血。其治疗结局的综合评价体系由3个方面组成,分别是出血、肌肉骨骼的结构(影像学)和功能、社会活动参与情况,通过多学科检测及相应量表完成评估。其中血友病患者的生活质量评估尤为重要,评估方式涵盖通用问卷及血友病专用问卷,其中血友病儿童专用生活质量评估工具加拿大血友病结局-儿童生活评估工具(CHO-KLAT)应用最为广泛。本文对综合评价体系的发展和应用进行简述。

     

    Abstract: Being a hereditary bleeding disorder, hemophilia is characterized by spontaneous bleeding, especially joint bleeding. The treatment outcome is a comprehensive evaluation system of the following three aspects: bleeding, musculoskeletal structure (imaging), and function-activity participation. Multidisciplinary testing and corresponding scales are needed in the assessment. Among them, the quality of life assessment of hemophilia patients is particularly important, through general questionnaires and hemophilia-specific questionnaires. Canadian hemophilia outcomes-Kids' life assessment tool(CHO-KLAT), a special quality of life assessment tool for children with hemophilia, is the most widely used. This paper briefly describes the development and application of comprehensive evaluation system of the children with the disease.

     

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