scholarly journals Validation of MRC Centre MRI calf muscle fat fraction protocol as an outcome measure in CMT1A

Neurology ◽  
2018 ◽  
Vol 91 (12) ◽  
pp. e1125-e1129 ◽  
Author(s):  
Jasper M. Morrow ◽  
Matthew R.B. Evans ◽  
Tiffany Grider ◽  
Christopher D.J. Sinclair ◽  
Daniel Thedens ◽  
...  
Keyword(s):  
Outcome Measure ◽  
Intraclass Correlation ◽  
Calf Muscle ◽  
Charcot Marie Tooth Disease ◽  
Leg Muscles ◽  
Mri Protocol ◽  
Fat Fraction ◽  
Test Retest Reliability ◽  
Healthy Calf ◽  
Independent Cohort

ObjectiveTo translate the quantitative MRC Centre MRI protocol in Charcot-Marie-Tooth disease type 1A (CMT1A) to a second site; validate its responsiveness in an independent cohort; and test the benefit of participant stratification to increase outcome measure responsiveness.MethodsThree healthy volunteers were scanned for intersite standardization. For the longitudinal patient study, 11 patients with CMT1A were recruited with 10 patients rescanned at a 12-month interval. Three-point Dixon MRI of leg muscles was performed to generate fat fraction (FF) maps, transferred to a central site for quality control and analysis. Clinical data collected included CMT Neuropathy Score.ResultsTest-retest reliability of FF within individual healthy calf muscles at the remote site was excellent: intraclass correlation coefficient 0.79, limits of agreement −0.67 to +0.85 %FF. In patients, mean calf muscle FF was 21.0% and correlated strongly with disease severity and age. Calf muscle FF significantly increased over 12 months (+1.8 ± 1.7 %FF, p = 0.009). Patients with baseline FF >10% showed a 12-month FF increase of 2.9% ± 1.3% (standardized response mean = 2.19).ConclusionsWe have validated calf muscle FF as an outcome measure in an independent cohort of patients with CMT1A. Responsiveness is significantly improved by enrolling a stratified patient cohort with baseline calf FF >10%.

scholarly journals Development of MRC Centre MRI calf muscle fat fraction protocol as a sensitive outcome measure in Hereditary Sensory Neuropathy Type 1

2019 ◽  
Vol 90 (8) ◽  
pp. 895-906 ◽  
Author(s):  
Umaiyal Kugathasan ◽  
Matthew R B Evans ◽  
Jasper M Morrow ◽  
Christopher D J Sinclair ◽  
John S Thornton ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Outcome Measure ◽  
Sensory Neuropathy ◽  
Intramuscular Fat ◽  
Calf Muscle ◽  
Fat Accumulation ◽  
Hereditary Sensory Neuropathy ◽  
Fat Fraction

ObjectivesHereditary sensory neuropathy type 1 (HSN1) is a rare, slowly progressive neuropathy causing profound sensory deficits and often severe motor loss. L-serine supplementation is a possible candidate therapy but the lack of responsive outcome measures is a barrier for undertaking clinical trials in HSN1. We performed a 12-month natural history study to characterise the phenotype of HSN1 and to identify responsive outcome measures.MethodsAssessments included Charcot-Marie-Tooth Neuropathy Score version 2 (CMTNSv2), CMTNSv2-Rasch modified, nerve conduction studies, quantitative sensory testing, intraepidermal nerve fibre density (thigh), computerised myometry (lower limbs), plasma 1-deoxysphingolipid levels, calf-level intramuscular fat accumulation by MRI and patient-based questionnaires (Neuropathic Pain Symptom Inventory and 36-Short Form Health Survey version 2 [SF-36v2]).Results35 patients with HSN1 were recruited. There was marked heterogeneity in the phenotype mainly due to differences between the sexes: males generally more severely affected. The outcome measures that significantly changed over 1 year and correlated with CMTNSv2, SF-36v2-physical component and disease duration were MRI determined calf intramuscular fat accumulation (mean change in overall calf fat fraction 2.36%, 95% CI 1.16 to 3.55, p=0.0004), pressure pain threshold on the hand (mean change 40 kPa, 95% CI 0.7 to 80, p=0.046) and myometric measurements of ankle plantar flexion (median change −0.5 Nm, IQR −9.5 to 0, p=0.0007), ankle inversion (mean change −0.89 Nm, 95% CI −1.66 to −0.12, p=0.03) and eversion (mean change −1.61 Nm, 95% CI −2.72 to −0.51, p=0.006). Intramuscular calf fat fraction was the most responsive outcome measure.ConclusionMRI determined calf muscle fat fraction shows validity and high responsiveness over 12 months and will be useful in HSN1 clinical trials.

scholarly journals Enfeeblement in Essential Tremor: Development and Validation of a New Psychometric Measure

2019 ◽  
Vol 32 (3) ◽  
pp. 145-151 ◽  
Author(s):  
Daniel Trujillo Diaz ◽  
Tess E. K. Cersonsky ◽  
Sarah Kellner ◽  
Maria Anna Zdrodowska ◽  
Ruby Hickman ◽  
...  
Keyword(s):  
Essential Tremor ◽  
Neurological Disorders ◽  
Convergent Validity ◽  
Outcome Measure ◽  
Intraclass Correlation ◽  
Floor Effect ◽  
Future Studies ◽  
Body Of Knowledge ◽  
Test Retest Reliability ◽  
Development And Validation

Introduction: Despite a growing body of knowledge regarding essential tremor (ET), past studies have fallen short in capturing the full impact of ET on patients and caregivers. We propose enfeeblement (i.e., having the qualities of being prematurely old, helpless, or debilitated) as a novel clinical outcome measure in ET. Due to the lack of enfeeblement scales for ET in the literature, we developed and validated an enfeeblement scale for ET. Methods: The Essential Tremor Enfeeblement Survey (ETES) consists of eight 5-point Likert-type scale questions and is designed to be a caregiver-reported outcome. Results: Enfeeblement scores showed a floor effect of 15.3%, no ceiling effects, and demonstrated good overall test–retest reliability (intraclass correlation coefficient = 0.73), favorable internal consistency (Cronbach α coefficient = 0.92) and good convergent validity. Conclusion: The ETES has robust properties. Aside from future studies of enfeeblement in ET, enfeeblement should be explored more broadly as a psychometric measure across other neurological disorders.

scholarly journals Development and validation of a new patient-reported outcome measure for peripheral nerve disorders of the hand, the I-HaND© Scale

2018 ◽  
Vol 43 (8) ◽  
pp. 864-874 ◽  
Author(s):  
Mark Ashwood ◽  
Christina Jerosch-Herold ◽  
Lee Shepstone
Keyword(s):  
Outcome Measure ◽  
Intraclass Correlation ◽  
Principal Component ◽  
Patient Reported Outcome ◽  
Face To Face ◽  
Patient Reported Outcome Measure ◽  
Patient Reported ◽  
Test Retest Reliability ◽  
Development And Validation ◽  
The Impact

Following guidelines from the Patient-Centred Outcomes Research Institute and using a mixed methods study, a new patient-reported outcome measure (PROM) for both nerve trauma and compression affecting the hand, the Impact of a Hand Nerve Disorders (I-HaND) Scale, was developed. Face-to-face interviews with 14 patients and subsequent pilot-testing with 61 patients resulted in the development of the 32-item PROM. A longitudinal validation study with 82 patients assessed the psychometric properties of the I-HaND. Content and construct validity was confirmed by cognitive interviews with patients and through principal component analysis. The I-HaND has high internal consistency (α = 0.98) and excellent test–retest reliability (intraclass correlation coefficient = 0.97). Responsiveness statistics showed that the I-HaND can detect change over 3 months and discriminate between improvers and non-improvers. We conclude that the I-HaND can be used as a PROM for people with a range of hand nerve disorders.

scholarly journals The Quantification of Hop Landing Balance Using Trunk-Mounted Accelerometry

2019 ◽  
Vol 28 (8) ◽  
Author(s):  
Jonathan M. Williams ◽  
Michael Gara ◽  
Carol Clark
Keyword(s):  
Outcome Measure ◽  
Intraclass Correlation ◽  
Minimal Detectable Change ◽  
Detectable Change ◽  
Balance Performance ◽  
Clinical Measurement ◽  
Injury Prediction ◽  
Test Retest Reliability ◽  
University Undergraduates ◽  
Acceleration Profile

Context:Balance is important for injury prediction, prevention, and rehabilitation. Clinical measurement of higher level balance function such as hop landing is necessary. Currently, no method exists to quantify balance performance following hopping in the clinic.Objective:To quantify the sacral acceleration profile and test–retest reliability during hop landing.Participants:A total of 17 university undergraduates (age 27.6 [5.7] y, height 1.73 [0.11] m, weight 74.1 [13.9] kg).Main Outcome Measure:A trunk-mounted accelerometer captured the acceleration profile following landing from hopping forward, medially, and laterally. The path length of the acceleration traces were computed to quantify balance following landing.Results:Moderate to excellent reliability (intraclass correlation coefficient .67–.93) for hop landing was established with low to moderate SEM (4%–16%) and minimal detectable change values (13%–44%) for each of the hop directions. Significant differences were determined in balance following hop landing from the different directions.Conclusion:The results suggest that hop landing balance can be quantified by trunk-mounted accelerometry.

scholarly journals Development of a patient-oriented outcome measure for patients with hand and elbow disorder: HandQ

2020 ◽  
Vol 28 (3) ◽  
pp. 230949902095915
Author(s):  
Kosuke Uehara ◽  
Toshiki Miura ◽  
Ohe Takashi ◽  
Tokuhide Doi ◽  
Sakae Tanaka ◽  
...  
Keyword(s):  
Internal Consistency ◽  
Graphical Model ◽  
Outcome Measure ◽  
High Reliability ◽  
Intraclass Correlation ◽  
Score Test ◽  
Reliability And Validity ◽  
Specific Outcome ◽  
Disease Specific Outcome Measure ◽  
Test Retest Reliability

Purpose: The Disabilities of the Arm, Shoulder, and Hand (DASH) is the most widely used patient-oriented outcome measure for the upper extremities in the world, and high reliability and validity of it has already been confirmed. However, there are several problems with using the DASH, some of which are culturally related. We aimed to (1) develop a patient-oriented disease-specific outcome measure for patients with disorders of the hand and elbow, which we call the HandQ and (2) examine the practical applicability, reliability, and validity of the HandQ for any patient with disorders of the hand and elbow. Methods: A total of 216 patients were surveyed with the HandQ, as well as the Hand20 and the DASH to assess psychometric characteristics. Results: There were no considerable floor and ceiling effects regarding the total HandQ score. Test–retest reliability and internal consistency determined using the intraclass correlation coefficient (0.942) and Cronbach’s α test (0.961) were excellent. The HandQ was well correlated with the Hand20 and the DASH scores. Scree plot showed unidimensionality of the HandQ, and the graphical model showed the questionnaire items of the HandQ had reasonable correlation among each item. Conclusions: The HandQ has a sufficient reliability and internal consistency, and an excellent validity, and was shown to be able to be practically applicable in all patients with hand and elbow disorders.

scholarly journals Development of a patient-oriented outcome measure for patients with hand and elbow disorder: HandQ

2019 ◽  
Author(s):  
Kosuke Uehara ◽  
Toshiki Miura ◽  
Takashi Ohe ◽  
Tokuhide Doi ◽  
Sakae Tanaka ◽  
...  
Keyword(s):  
Internal Consistency ◽  
Graphical Model ◽  
Outcome Measure ◽  
Intraclass Correlation ◽  
Reliability And Validity ◽  
Psychometric Characteristics ◽  
Specific Outcome ◽  
Disease Specific Outcome Measure ◽  
Test Retest Reliability ◽  
Disease Specific

Abstract Background: We aimed to develop a patient-oriented disease-specific outcome measure for patients with disorders of the hand and elbow, which we call the HandQ, and examine the practical applicability, reliability and validity of the HandQ for any patient with disorders of the hand and elbow. Methods: A total of 216 patients were surveyed with the HandQ, as well as the Hand20 and the DASH to assess psychometric characteristics. Results: Test-retest reliability and internal consistency determined using the intraclass correlation coefficient (0.942) and Cronbach’s alpha test (0.961) were excellent. The HandQ was well correlated with the other questionnaires. Scree plot showed uni-dimensionality of the HandQ, and the graphical model showed the questionnaire items of the HandQ had reasonable correlation among each item. Conclusions: The HandQ has a sufficient reliability and internal consistency, and an excellent validity, and was shown to be able to be practically applicable in all patients with hand and elbow disorders.

Test-Retest-Reliability of Computer-Based Metamorphopsia Measurement in Macular Diseases

2020 ◽  
Author(s):  
Daniela Claessens ◽  
Alexander K. Schuster ◽  
Ronald V. Krüger ◽  
Marian Liegl ◽  
Laila Singh ◽  
...  
Keyword(s):  
Macular Edema ◽  
Correlation Coefficient ◽  
Intraclass Correlation Coefficient ◽  
Intraclass Correlation ◽  
Measuring Method ◽  
Repeated Measurements ◽  
Retest Reliability ◽  
Macular Diseases ◽  
Computer Based ◽  
Test Retest Reliability

AbstractIn this study, the test-retest-reliability as one aspect of reliability of metamorphopsia measurements using a computer-based measuring method was determined in patients with macular diseases. Metamorphopsia amplitude, position, and area were quantified using AMD – A Metamorphopsia Detector software (app4eyes GmbH & Co. KG, Germany) in patients with diabetic, myopic, or uveitic macular edema, intermediate or neovascular age-associated macular degeneration, epiretinal membrane, vitelliform maculopathy, Irvine-Gass syndrome, or macular edema due to venous retinal occlusion. The intraclass correlation coefficient (ICC) was calculated in order to determine the repeatability of two repeated measurements and was used as an indicator of the reliability of the measurements. In this study, metamorphopsia measurements were conducted on 36 eyes with macular diseases. Metamorphopsia measurements made using AMD – A Metamorphopsia Detector software were highly reliable and repeatable in patients with maculopathies. The intraclass correlation coefficient of all indices was excellent (0.95 – 0.97). For diseases of the vitreoretinal interface or macular diseases with intra- or subretinal edema, this metamorphopsia measurement represents a supplement for visual function testing in the clinic, as well as in clinical studies.

scholarly journals Validation of a menstrual pictogram and a daily bleeding diary for assessment of uterine fibroid treatment efficacy in clinical studies

2020 ◽  
Vol 4 (1) ◽  
Author(s):  
Claudia Haberland ◽  
Anna Filonenko ◽  
Christian Seitz ◽  
Matthias Börner ◽  
Christoph Gerlinger ◽  
...  
Keyword(s):  
Uterine Fibroid ◽  
Full Range ◽  
Intraclass Correlation ◽  
Phase Iii ◽  
Measurement Properties ◽  
Retest Reliability ◽  
Response Options ◽  
Patient Global Impression ◽  
Patient Reported ◽  
Test Retest Reliability

Abstract Background To evaluate the psychometric and measurement properties of two patient-reported outcome instruments, the menstrual pictogram superabsorbent polymer-containing version 3 (MP SAP-c v3) and Uterine Fibroid Daily Bleeding Diary (UF-DBD). Test-retest reliability, criterion, construct validity, responsiveness, missingness and comparability of the MP SAP-c v3 and UF-DBD versus the alkaline hematin (AH) method and a patient global impression of severity (PGI-S) were analyzed in post hoc trial analyses. Results Analyses were based on data from up to 756 patients. The full range of MP SAP-c v3 and UF-DBD response options were used, with score distributions reflecting the cyclic character of the disease. Test-retest reliability of MP SAP-c v3 and UF-DBD scores was supported by acceptable intraclass correlation coefficients when stability was defined by the AH method and Patient Global Impression of Severity (PGI-S) scores (0.80–0.96 and 0.42–0.94, respectively). MP SAP-c v3 and UF-DBD scores demonstrated strong and moderate-to-strong correlations with menstrual blood loss assessed by the AH method. Scores increased in monotonic fashion, with greater disease severities, defined by the AH method and PGI-S scores; differences between groups were mostly statistically significant (P < 0.05). MP SAP-c v3 and UF-DBD were sensitive to changes in disease severity, defined by the AH method and PGI-S. MP SAP-c v3 and UF-DBD showed a lower frequency of missing patient data versus the AH method, and good agreement with the AH method. Conclusions This evidence supports the use of the MP SAP-c v3 and UF-DBD to assess clinical efficacy endpoints in UF phase III studies replacing the AH method.

scholarly journals Cross-cultural translation and validation of the Spanish version of the patellofemoral pain and osteoarthritis subscale of the KOOS (KOOS-PF)

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Juan Pablo Martinez-Cano ◽  
Daniel Vernaza-Obando ◽  
Julián Chica ◽  
Andrés Mauricio Castro
Keyword(s):  
Intraclass Correlation ◽  
Patellofemoral Pain ◽  
Spanish Version ◽  
Minimal Detectable Change ◽  
Global Rating ◽  
Minimal Important Change ◽  
Specific Patient ◽  
Retest Reliability ◽  
Patient Reported ◽  
Test Retest Reliability

Abstract Objective The aim of this study was to translate to Spanish the patellofemoral pain and osteoarthritis subscale of the knee injury and osteoarthritis outcome score (KOOS-PF) and validate this Spanish version of a disease-specific patient-reported outcome measure (PROM) for patellofemoral pain. Results The KOOS-PF was translated to Spanish and sixty patients with patellofemoral pain and/or osteoarthritis accepted to complete the questionnaire. 1-week later 58 patients answered the questions again for the test–retest reliability validation and finally 55 patients completed 1-month later for the responsiveness assessment. The Spanish version showed very good internal consistency (Cronbach’s alpha: 0.93) and test–retest reliability (intraclass correlation coefficient: 0.82). Responsiveness was confirmed, showing a strong correlation with the global rating of change (GROC) score (r 0.64). The minimal detectable change was 11.1 points, the minimal important change was 17.2 points, and there were no floor or ceiling effects for the score.

scholarly journals Maximal Isometric or Eccentric Hamstring Strength—Which Test Modality Might Be More Suitable for Assessments in Youth Alpine Ski Racers?

2021 ◽  
Vol 18 (4) ◽  
pp. 2138
Author(s):  
Roland Luchner ◽  
Lisa Steidl-Müller ◽  
Martin Niedermeier ◽  
Christian Raschner
Keyword(s):  
Mixed Model ◽  
Intraclass Correlation ◽  
Test Procedure ◽  
Knee Injuries ◽  
Racing Performance ◽  
Age Appropriate ◽  
Mean Differences ◽  
Fitness Programs ◽  
Test Retest Reliability ◽  
Ski Racers

Background: Physical fitness is an important component in the development of youth alpine ski racers. To write systematically planned and age-appropriate fitness programs athletes need to be physically tested at regular intervals at an early age. Although well-developed hamstring muscle strength is important for alpine ski racing performance and the prevention of serious knee injuries, it has not been well investigated, especially in youth athletes. Accordingly, the first aim of the present study was to assess the test-retest reliability of the maximum bilateral eccentric (MBEHS) and unilateral isometric (MUIHS) hamstring tests. The second aim of the present study was to assess whether the results of these two methods correlate and if it is possible to commit to one of the two methods to provide an economic test procedure. Methods: The first study included 26 (14 females/12 males) youth alpine ski racers aged between 12 and 13 years. All athletes performed two MBEHS and two MUIHS tests, 7 days apart. The intraclass correlation coefficient (ICC 3,1) and their 95% confidence intervals based on a consistency two-way mixed model were used to estimate the reliability of the two different test modalities. The second study included 61 (27 females/34 males) youth alpine ski racers aged between 10 and 13 years. All athletes performed one MBEHS and one MUIHS test. Bland-Altman plots and the 95% limits of agreement as well as correlations by Pearson (r) between the different test modalities were assessed. Results: In study 1 “poor” to “moderate” (MBEHS right leg 0.79 (0.58–0.90); left leg 0.83 (0.66–0.92); MUIHS right leg 0.78 (0.56–0.89); left leg 0.66 (0.37–0.83)) ICC values and 95% confident intervals were obtained. Standard error of measurement (SEM) between trails was between 18.3 and 25.1 N. Smallest detectable difference (SDD) was between 50.8 and 69.5 N. In study 2 mean differences between MBEHS and MUIHS was around 20 N with higher values for MBEHS. Significant moderate-to-strong correlations were found between the test modalities (r = 0.74–0.84, p <0.001). Conclusions: The MBEHS test has higher ICC values, lower CV values, higher SEM values and lower SDD values than the MUIHS test. All this suggests that the MBEHS test is more suitable than the MUIHS test to determine the maximum hamstring force in young alpine ski racers.

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