Background: Batten disease (juvenile neuronal ceroid lipofuscinosis [JNCL]) is an autosomal recessive neurodegenerative disorder characterized by blindness, seizures, and relentless decline in cognitive, motor, and be...Background: Batten disease (juvenile neuronal ceroid lipofuscinosis [JNCL]) is an autosomal recessive neurodegenerative disorder characterized by blindness, seizures, and relentless decline in cognitive, motor, and behavioral function. Onset is in the early school years, with progression to death typically by late adolescence. Development of a clinical instrument to quantify severity of illness is a prerequisite to eventual assessment of experimental therapeutic interventions Objective: To develop a clinical rating instrument to assess motor, behavioral, and functional capability in JNCL. Methods: A clinical rating instrument, the Unified Batten Disease Rating Scale (UBDRS), was developed by the authors to assess motor, behavioral, and functional capability in JNCL. Children with verified JNCL were evaluated independently by three neurologists. Intraclass correlation coefficients (ICCs) were used to estimate the interrater reliability for total scores in each domain. Interrater reliability for scale items was assessed with weighted κstatistics. Results: Thirty-one children with confirmed JNCL (10 boys, 21 girls) were evaluated. The mean age at symptom onset was 6.1 ±1.6 years, and the mean duration of illness was 9.0 ±4.4 years. The ICCs for the domains were as follows: motor=0.83, behavioral=0.68, and functional capability=0.85. Conclusions: The Unified Batten Disease Rating Scale (UBDRS) is a reliable instrument that effectively tests for neurologic function in blind and demented patients. In its current form, the UBDRS is useful for monitoring the diverse clinical findings seen in Batten disease.展开更多
目的分析改良呼吸困难指数(m MRC)评估和慢性阻塞性肺疾病评估测试量表(CAT)评分评估慢性阻塞性肺疾病(COPD)患者病情严重程度的临床效果。方法选择2014年4月至2015年6月接受诊治的75例COPD稳定期患者作为研究对象,分析m MRC评估、CAT...目的分析改良呼吸困难指数(m MRC)评估和慢性阻塞性肺疾病评估测试量表(CAT)评分评估慢性阻塞性肺疾病(COPD)患者病情严重程度的临床效果。方法选择2014年4月至2015年6月接受诊治的75例COPD稳定期患者作为研究对象,分析m MRC评估、CAT评分与COPD病情严重程度的相关性。结果 m MRC评估级别、CAT评分分值均与COPD患者病情严重程度存在显著相关性。结论 m MRC评估、CAT评分均可反映COPD患者病情的严重程度,两者均与COPD患者病情严重分级存在密切联系。展开更多
文摘Background: Batten disease (juvenile neuronal ceroid lipofuscinosis [JNCL]) is an autosomal recessive neurodegenerative disorder characterized by blindness, seizures, and relentless decline in cognitive, motor, and behavioral function. Onset is in the early school years, with progression to death typically by late adolescence. Development of a clinical instrument to quantify severity of illness is a prerequisite to eventual assessment of experimental therapeutic interventions Objective: To develop a clinical rating instrument to assess motor, behavioral, and functional capability in JNCL. Methods: A clinical rating instrument, the Unified Batten Disease Rating Scale (UBDRS), was developed by the authors to assess motor, behavioral, and functional capability in JNCL. Children with verified JNCL were evaluated independently by three neurologists. Intraclass correlation coefficients (ICCs) were used to estimate the interrater reliability for total scores in each domain. Interrater reliability for scale items was assessed with weighted κstatistics. Results: Thirty-one children with confirmed JNCL (10 boys, 21 girls) were evaluated. The mean age at symptom onset was 6.1 ±1.6 years, and the mean duration of illness was 9.0 ±4.4 years. The ICCs for the domains were as follows: motor=0.83, behavioral=0.68, and functional capability=0.85. Conclusions: The Unified Batten Disease Rating Scale (UBDRS) is a reliable instrument that effectively tests for neurologic function in blind and demented patients. In its current form, the UBDRS is useful for monitoring the diverse clinical findings seen in Batten disease.
文摘目的分析改良呼吸困难指数(m MRC)评估和慢性阻塞性肺疾病评估测试量表(CAT)评分评估慢性阻塞性肺疾病(COPD)患者病情严重程度的临床效果。方法选择2014年4月至2015年6月接受诊治的75例COPD稳定期患者作为研究对象,分析m MRC评估、CAT评分与COPD病情严重程度的相关性。结果 m MRC评估级别、CAT评分分值均与COPD患者病情严重程度存在显著相关性。结论 m MRC评估、CAT评分均可反映COPD患者病情的严重程度,两者均与COPD患者病情严重分级存在密切联系。
