FV 1180. The Unified Batten Disease Rating Scale: An International Collaboration Testing Validation and Reliability in an Independent Cohort of CLN3-Patients

2018 ◽  
Author(s):  
Miriam Nickel ◽  
Jonathan Mink ◽  
Eva Wibbeler ◽  
Christoph Schwering ◽  
Angela Schulz
Keyword(s):  
International Collaboration ◽  
Rating Scale ◽  
Batten Disease ◽  
Disease Rating ◽  
Independent Cohort

The unified Batten disease rating scale (UBDRS): Validation and reliability in an independent sample

2018 ◽  
Vol 123 (2) ◽  
pp. S95
Author(s):  
Jonathan W. Mink ◽  
Miriam Nickel ◽  
Eva Wibbeler ◽  
Christoff Schwering ◽  
Angela Schulz
Keyword(s):  
Rating Scale ◽  
Batten Disease ◽  
Disease Rating

scholarly journals The CLN3 Disease Staging System

Neurology ◽  
2020 ◽  
Vol 94 (23) ◽  
pp. e2436-e2440 ◽  
Author(s):  
Margaux C. Masten ◽  
Justin D. Williams ◽  
Jennifer Vermilion ◽  
Heather R. Adams ◽  
Amy Vierhile ◽  
...  
Keyword(s):  
Rating Scale ◽  
Batten Disease ◽  
Staging System ◽  
Severe Stage ◽  
Severity Scores ◽  
Potential Applications ◽  
Disease Rating ◽  
Cln3 Disease ◽  
Disease Stages ◽  
Disease Specific

ObjectiveTo develop a disease-specific staging system for CLN3 disease and to test the hypothesis that salient and discrete clinical features of CLN3 disease may be used to define disease stages by analyzed data from an 18-year-long natural history study.MethodsA proposed staging system, the CLN3 Staging System (CLN3SS), was based on salient and clinically meaningful endpoints. The relationships between stage and age, stage and Unified Batten Disease Rating Scale (UBDRS) physical severity score, and stage and UBDRS capability impairment subscale scores were determined. We used t tests to determine whether the stages were significantly different from each other on the basis of age and scores.ResultsData were analyzed from 322 evaluations in 108 individuals. There were significant differences among the stages based on age and severity scores. For individuals with longitudinal data, no individual reverted to a less severe stage over time.ConclusionThe CLN3SS is a disease-specific staging system that can be used to classify individuals into specific strata based on age and disease severity. The CLN3SS has potential applications in clinical trials for cohort stratification.

Hamburg Late Infantile neuronal ceroid lipofucinosis scale (H-LINCLS) vs. the Unified Batten Disease Rating Scale (UBDRS): Comparison and cross-validation

2020 ◽  
Vol 129 (2) ◽  
pp. S105
Author(s):  
Margaux C. Masten ◽  
Heather R. Adams ◽  
Amy Vierhile ◽  
Erika F. Augustine ◽  
Jonathan W. Mink
Keyword(s):  
Cross Validation ◽  
Rating Scale ◽  
Batten Disease ◽  
Disease Rating

Feasibility and reliability of telemedicine administration of the Unified Batten Disease Rating Scale

2011 ◽  
Vol 102 (2) ◽  
pp. S11
Author(s):  
Jennifer Cialone ◽  
Erika F. Augustin ◽  
Nicole J. Newhouse ◽  
Amy Vierhile ◽  
Frederick J. Marshall ◽  
...  
Keyword(s):  
Rating Scale ◽  
Batten Disease ◽  
Disease Rating

scholarly journals Salivary caffeine in Parkinson’s disease

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Giorgio Leodori ◽  
Maria Ilenia De Bartolo ◽  
Daniele Belvisi ◽  
Alessia Ciogli ◽  
Andrea Fabbrini ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Rating Scale ◽  
De Novo ◽  
Oral Intake ◽  
Caffeine Intake ◽  
Motor Complications ◽  
Disease Rating ◽  
Caffeine Metabolism ◽  
Movement Disorder Society

AbstractWe aimed to investigate salivary caffeine content, caffeine absorption and metabolism in Parkinson’s disease (PD) and verify whether salivary caffeine can be used as a biomarker of PD. We enrolled 98 PD patients and 92 healthy subjects. Caffeine and its major metabolite, paraxanthine, were measured in saliva samples collected before and 4 h after the oral intake of caffeine (100 mg). We measured caffeine absorption as the normalized increase in caffeine levels, and caffeine metabolism as the paraxanthine/caffeine ratio. The Movement Disorder Society Unified Parkinson's Disease Rating Scale part III, the Hoehn & Yahr, the presence of motor complications, and levodopa equivalent dose (LED) were assessed and correlated with caffeine levels, absorption, and metabolism. The effects of demographic and environmental features possibly influencing caffeine levels were also investigated. Caffeine levels were decreased in patients with moderate/advanced PD, while caffeine levels were normal in patients with early and de-novo PD, unrelated to caffeine intake. Caffeine absorption and metabolism were normal in PD. Decreased salivary caffeine levels in PD were associated with higher disease severity, longer duration, and the presence of motor complications, no significant association was found with LED. Salivary caffeine decrease correlates with PD progression.

scholarly journals A Comparison of Pain between Parkinson’s Disease and Multiple System Atrophy: A Clinical Cross-Sectional Survey

2019 ◽  
Vol 2019 ◽  
pp. 1-6
Author(s):  
He-Yang You ◽  
Lei Wu ◽  
Hai-Ting Yang ◽  
Chen Yang ◽  
Xiao-Ling Ding
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Multiple System Atrophy ◽  
Rating Scale ◽  
Depression Scale ◽  
Healthy Controls ◽  
Cross Sectional Survey ◽  
Cross Sectional ◽  
Disease Rating ◽  
Vas Scores

Background. Pain is frequent in Parkinson’s disease (PD) and Parkinson-plus syndrome. This study aimed to assess the prevalence, characteristics, therapy (especially the effect of dopaminergic therapy), and associated symptoms of pain in Parkinson's disease and multiple system atrophy (MSA) patients. Methods. Seventy-one PD patients, sixty-five MSA patients, and forty age-matched healthy controls were enrolled and evaluated by using the German pain questionnaire and visual analogue scale (VAS). In addition, the influence of pain in PD patients on anxiety, depression, and the quality of life was assessed with the Hospital Anxiety and Depression Scale (HADS) and Parkinson’s Disease Questionnaire (PDQ-39). Results. Compared to that of the healthy controls, the PD and MSA patients had a significantly higher presence of pain (P<0.01, P<0.01). PD patients had a higher presence of pain than MSA patients (P=0.007). No difference in VAS scores was observed between the PD and MSA patients (P=0.148). A total of 21 PD patients (42.85%) with pain and 13 MSA patients (43.33%) with pain received treatment. A total of 13 PD patients with pain and 6 MSA patients with pain had an improved pain intensity after using dopaminergic medication. The differences in the disease duration, Hoehn and Yahr stages, and scores on the Unified Parkinson’s Disease Rating Scale motor score, HAD-D, HAD-A, and PDQ-39 were significant between the PD patients with and without pain. Conclusion. PD and MSA patients are prone to pain with insufficient treatment. Pain interventions should be provided as soon as possible to improve the patient’s life.

scholarly journals The Parkinson’s Disease Comprehensive Response (PDCORE): a composite approach integrating three standard outcome measures

2020 ◽  
Vol 2 (2) ◽  
Author(s):  
Matthias Luz ◽  
Alan Whone ◽  
Niccolò Bassani ◽  
Richard K Wyse ◽  
Glenn T Stebbins ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Cell Line ◽  
Glial Cell ◽  
Neurotrophic Factor ◽  
Rating Scale ◽  
Initial Development ◽  
Disease Rating ◽  
Recent Phase ◽  
Data Source

Abstract There is an increasing need for improved endpoints to assess clinical trial effects in Parkinson’s disease. We propose the Parkinson’s Disease Comprehensive Response as a novel weighted composite endpoint integrating changes measured in three established Parkinson’s outcomes, including: OFF state Movement Disorder Society Unified Parkinson’s Disease Rating Scale Motor Examination scores; Motor Experiences of Daily Living scores; and total good-quality ON time per day. The data source for the initial development of the composite described herein was a recent Phase II trial of glial cell line-derived neurotrophic factor. A wide range of clinically derived relative weights was assessed to normalize for differentially scoring base rates with each endpoint component. The Parkinson’s disease comprehensive response, in contrast to examining practically defined OFF state Unified Parkinson’s Disease Rating Scale Motor Examination scores alone, showed stability over 40 weeks in placebo patients, and all 432 analyses in this permutation exercise yielded significant differences in favour of glial cell line-derived neurotrophic factor. The findings were consistent with results obtained employing three different global statistical test methodologies and with patterns of intra-patient change. Based on our detailed analyses, we conclude it worth prospectively evaluating the clinical utility, validity and regulatory feasibility of using clinically supported final Parkinson’s disease comprehensive response formulas (for both the Unified Parkinson’s Disease Rating Scale-based and Movement Disorders Society-Unified Parkinson’s Disease Rating Scale-based versions) in future disease-modifying Parkinson’s trials. Whilst the data source employed in the initial development of this weighted composite score is from a recent Phase II trial of glial cell line-derived neurotrophic factor, we wish to stress that the results are not described to provide post hoc evidence of the efficacy of glial cell line-derived neurotrophic factor but rather are presented to further the debate of how current regulatory approved rating scales may be combined to address some of the recognized limitations of using individual scales in isolation.

scholarly journals The perception of apathy by caregivers of patients with dementia in Parkinson's disease

2016 ◽  
Vol 10 (4) ◽  
pp. 339-343 ◽  
Author(s):  
Carlos Henrique Ferreira Camargo ◽  
Rafael Arthur Serpa ◽  
Thiago Matnei ◽  
Jivago Szpoganicz Sabatini ◽  
Hélio Afonso Ghizoni Teive
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Rating Scale ◽  
Neuropsychiatric Symptoms ◽  
Parkinson’S Disease Dementia ◽  
Disease Rating

ABSTRACT Background: Apathy is one of the main neuropsychiatric symptoms in patients with Parkinson's disease (PD) and is associated with Parkinson's disease dementia (PDD). Objective: To identify the characteristics of apathy in individuals with PDD according to caregiver perception. Methods: Thirty-nine patients with PD according to MDS criteria for PDD were included. The following scales were used: the Hoehn and Yahr, the Unified Parkinson's Disease Rating Scale III, Scales for Outcomes in Parkinson's Disease-Cognition (SCOPA Cog), the Montgomery-Åsberg Depression Rating Scale (MADRS) and the Apathy Evaluation Scale (AES). Results: A total of 97.4% of the patients showed results consistent with apathy. Analysis of question 14 of the AES revealed no correlation with the total result of all the questions [r=-1293, r²=0.0167, 95%CI (-0.4274 to 0.1940), P=0.2162], however, there was a correlation of responses to the same question with depression data on the MADRS scale [r=-0.5213, r²=0.2718, 95%CI (-0.7186 to -0.2464), P=0.00033]. Conclusion: Apathy is a disorder associated with PDD. However, the scoring scheme of the AES questions can lead to different interpretations of caregiver responses, highlighting limitations of the tool for use in studies of PDD.

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