Background The Scale for the Assessment and Rating of Ataxia (SARA) was shown to be a reliable and valid measurement for patients with spinocerebellar ataxia (SCA). The Brazilian version and the Japanese version o...Background The Scale for the Assessment and Rating of Ataxia (SARA) was shown to be a reliable and valid measurement for patients with spinocerebellar ataxia (SCA). The Brazilian version and the Japanese version of SARA were favorable for good reliability and validity. This study aimed to translate SARA into Chinese and test its reliability and validity in measurement of cerebellar ataxia. Methods SARA was translated into Chinese. A total 39 patients with degeneration cerebellar ataxia were evaluated independently by two neurologists with the Chinese version of SARA. Then the patients were evaluated by one of above neurologists with International Cooperative Ataxia Rating Scale (ICARS). The statistical analyses were performed using SPSS 17.0 for Windows. Results The Cronbach's alpha coefficient of the Chinese version of SARA was 0.78, which represents a good internal consistence. The correlation coefficient of the Chinese version of SARA scores between the two evaluators was 0.86, illustrating that the inter-rater reliability of Chinese version of SARA was good. The correlation coefficient between the Chinese version of SARA and ICARS was 0.91, illustrating that the criterion validity of Chinese version of SARA was not bad. Conclusions The Chinese version of SARA is reliable and effective for the assessment of degeneration cerebellar ataxia. Compared with ICARS, the evaluation of Chinese version of SARA is more objective, the assessment time is shortened, and the maneuverability is better.展开更多
目的:探讨氢质子波谱(proton magnetic resonance spectroscopy,~1H-MRS)在脊髓小脑型共济失调3型/马查多–约瑟夫病(hereditary spinocerebellar ataxias 3 and Machado-Joseph,SCA3/MJD)诊断中的价值,同时探讨其与临床病程评分之间的...目的:探讨氢质子波谱(proton magnetic resonance spectroscopy,~1H-MRS)在脊髓小脑型共济失调3型/马查多–约瑟夫病(hereditary spinocerebellar ataxias 3 and Machado-Joseph,SCA3/MJD)诊断中的价值,同时探讨其与临床病程评分之间的联系。方法:SCA3/MJD起病患者、SCA3/MJD未起病患者及年龄匹配健康志愿者进行~1H-MRS检查,经数据后处理获得N–乙酰天门冬氨酸(N-acetylaspartate,NAA),肌酸(creatine,Cr),胆碱(choline-containing compounds,Cho),肌醇(myo-inositol,m I)值及NAA/Cr,Cho/Cr,m I/Cr比值。将以上数据进行对比研究;同时对于SCA3/MJD起病患者进行国际协作共济失调评估量表(International Cooperative Ataxia Rating Scale,ICARS)及共济失调等级量表(Scale for the Assessment and Rating of Ataxia,SARA)评估,计算NAA/Cr,Cho/Cr,m I/Cr比值与临床评分之间的相关性。结果:SCA3/MJD起病患者在桥脑及齿状核的NAA/Cr值较正常对照组明显减低。SCA3/MJD起病患者小脑齿状核NAA/Cr值与ICARS评分有明显相关性。结论:SCA3/MJD的病变主要位于小脑及脑干,ICARS评分与小脑病变的程度有相关性,能较好地反映临床表现的轻重程度。~1H-MRS对于SCA3/MJD的诊断有一定的应用前景。展开更多
Background: Spinocerebellar ataxia type 3 (SCA3) is the most common subtype of SCA worldwide, and runs a slowly progressive and unremitting disease course. There is currently no curable treatment available. Growing...Background: Spinocerebellar ataxia type 3 (SCA3) is the most common subtype of SCA worldwide, and runs a slowly progressive and unremitting disease course. There is currently no curable treatment available. Growing evidence has suggested that nerve growth factor (NGF) may have therapeutic effects in neurodegenerative diseases, and possibly also in SCA3. The objective of this study was to test the efficacy of NGF in SCA3 patients. Methods: We performed an open-label prospective study in genetically confirmed adult (〉18 years old) SCA3 patients. NGF was administered by intramuscular injection (18 μg once daily) fbr 28 days consecutively. All the patients were evaluated at baseline and 2 and 4 weeks after treatment using the Chinese version of the scale for assessment and rating of ataxia (SARA). Results: Twenty-one SCA3 patients ( 10 men and 11 women, mean age 39.14 ± 7.81 years, mean disease duration 4.14 ± 1.90 years, mean CAG repeats number 77,57 ± 2.27) were enrolled. After 28 days of NGF treatment, the mean total SARA score decreased significantly from a baseline of 8.48± 2.40 to 6.30 ± 1.87 (P 〈 0.001 ). Subsections SARA scores also showed significant improvements in stance (P = 0.003), speech (P = 0.023), finger chase (P = 0.015), fast alternating hand movements (P = 0.009), and heel-shin slide (P = 0.001). Conclusions: Our preliminary data suggest that NGF may be effective in treating patients with SCA3.展开更多
文摘Background The Scale for the Assessment and Rating of Ataxia (SARA) was shown to be a reliable and valid measurement for patients with spinocerebellar ataxia (SCA). The Brazilian version and the Japanese version of SARA were favorable for good reliability and validity. This study aimed to translate SARA into Chinese and test its reliability and validity in measurement of cerebellar ataxia. Methods SARA was translated into Chinese. A total 39 patients with degeneration cerebellar ataxia were evaluated independently by two neurologists with the Chinese version of SARA. Then the patients were evaluated by one of above neurologists with International Cooperative Ataxia Rating Scale (ICARS). The statistical analyses were performed using SPSS 17.0 for Windows. Results The Cronbach's alpha coefficient of the Chinese version of SARA was 0.78, which represents a good internal consistence. The correlation coefficient of the Chinese version of SARA scores between the two evaluators was 0.86, illustrating that the inter-rater reliability of Chinese version of SARA was good. The correlation coefficient between the Chinese version of SARA and ICARS was 0.91, illustrating that the criterion validity of Chinese version of SARA was not bad. Conclusions The Chinese version of SARA is reliable and effective for the assessment of degeneration cerebellar ataxia. Compared with ICARS, the evaluation of Chinese version of SARA is more objective, the assessment time is shortened, and the maneuverability is better.
文摘目的:探讨氢质子波谱(proton magnetic resonance spectroscopy,~1H-MRS)在脊髓小脑型共济失调3型/马查多–约瑟夫病(hereditary spinocerebellar ataxias 3 and Machado-Joseph,SCA3/MJD)诊断中的价值,同时探讨其与临床病程评分之间的联系。方法:SCA3/MJD起病患者、SCA3/MJD未起病患者及年龄匹配健康志愿者进行~1H-MRS检查,经数据后处理获得N–乙酰天门冬氨酸(N-acetylaspartate,NAA),肌酸(creatine,Cr),胆碱(choline-containing compounds,Cho),肌醇(myo-inositol,m I)值及NAA/Cr,Cho/Cr,m I/Cr比值。将以上数据进行对比研究;同时对于SCA3/MJD起病患者进行国际协作共济失调评估量表(International Cooperative Ataxia Rating Scale,ICARS)及共济失调等级量表(Scale for the Assessment and Rating of Ataxia,SARA)评估,计算NAA/Cr,Cho/Cr,m I/Cr比值与临床评分之间的相关性。结果:SCA3/MJD起病患者在桥脑及齿状核的NAA/Cr值较正常对照组明显减低。SCA3/MJD起病患者小脑齿状核NAA/Cr值与ICARS评分有明显相关性。结论:SCA3/MJD的病变主要位于小脑及脑干,ICARS评分与小脑病变的程度有相关性,能较好地反映临床表现的轻重程度。~1H-MRS对于SCA3/MJD的诊断有一定的应用前景。
基金This study was supported by grants from the National Natural Science Foundation of China grant,The Innovation Team Fund of the First Affiliated Hospital of Zhengzhou University and the National Natural Science Foundation of China
文摘Background: Spinocerebellar ataxia type 3 (SCA3) is the most common subtype of SCA worldwide, and runs a slowly progressive and unremitting disease course. There is currently no curable treatment available. Growing evidence has suggested that nerve growth factor (NGF) may have therapeutic effects in neurodegenerative diseases, and possibly also in SCA3. The objective of this study was to test the efficacy of NGF in SCA3 patients. Methods: We performed an open-label prospective study in genetically confirmed adult (〉18 years old) SCA3 patients. NGF was administered by intramuscular injection (18 μg once daily) fbr 28 days consecutively. All the patients were evaluated at baseline and 2 and 4 weeks after treatment using the Chinese version of the scale for assessment and rating of ataxia (SARA). Results: Twenty-one SCA3 patients ( 10 men and 11 women, mean age 39.14 ± 7.81 years, mean disease duration 4.14 ± 1.90 years, mean CAG repeats number 77,57 ± 2.27) were enrolled. After 28 days of NGF treatment, the mean total SARA score decreased significantly from a baseline of 8.48± 2.40 to 6.30 ± 1.87 (P 〈 0.001 ). Subsections SARA scores also showed significant improvements in stance (P = 0.003), speech (P = 0.023), finger chase (P = 0.015), fast alternating hand movements (P = 0.009), and heel-shin slide (P = 0.001). Conclusions: Our preliminary data suggest that NGF may be effective in treating patients with SCA3.