Rating scales as outcome measures for clinical trials in neurology: problems, solutions, and recommendations

Hobart, Jeremy; Cano, Stefan; Zajicek, John; Thompson, Alan J.

doi:10.1016/s1474-4422(07)70290-9

Cited by 432 publications

(419 citation statements)

References 75 publications

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Section: Discussionmentioning

confidence: 99%

“…Scales with multiple, ordinal divisions are more sensitive to change than dichotomous scales35, 37 and their use has higher reliability because of reduced random error 32. Furthermore, a scale's validity is better assessed and improved when scores are assigned within multi‐item scales 32. We therefore recommend that a group of experts gather to discuss and refine the observations of horses with neurologic gait deficits30 based on a series of standardized videos of horses with confirmed spinal cord disease.…”

Section: Discussionmentioning

confidence: 99%

“…The PCA was performed on data derived from the questionnaire where the single rater ICC (Equation 1, Data S1) was >0.4, with the number of principal components determined from a scree plot 32, 33. ORs were calculated for the probability of positive answers in each of the questions correlating with either histopathologic changes consistent with pathology or of a horse being assigned a median ataxia grade ≥2.…”

Section: Methodsmentioning

confidence: 99%

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Rater Agreement on Gait Assessment during Neurologic Examination of Horses

Olsen

Dunkel

Barker

et al. 2014

Veterinary Internal Medicne

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BackgroundReproducible and accurate recognition of presence and severity of ataxia in horses with neurologic disease is important when establishing a diagnosis, assessing response to treatment, and making recommendations that might influence rider safety or a decision for euthanasia.ObjectivesTo determine the reproducibility and validity of the gait assessment component in the neurologic examination of horses.AnimalsTwenty‐five horses referred to the Royal Veterinary College Equine Referral Hospital for neurological assessment (n = 15), purchased (without a history of gait abnormalities) for an unrelated study (n = 5), or donated because of perceived ataxia (n = 5).MethodsUtilizing a prospective study design; a group of board‐certified medicine (n = 2) and surgery (n = 2) clinicians and residents (n = 2) assessed components of the equine neurologic examination (live and video recorded) and assigned individual and overall neurologic gait deficit grades (0–4). Inter‐rater agreement and assessment‐reassessment reliability were quantified using intraclass correlation coefficients (ICC).ResultsThe ICCs of the selected components of the neurologic examination ranged from 0 to 0.69. “Backing up” and “recognition of mistakes over obstacle” were the only components with an ICC > 0.6. Assessment‐reassessment agreement was poor to fair. The agreement on gait grading was good overall (ICC = 0.74), but poor for grades ≤ 1 (ICC = 0.08) and fair for ataxia grades ≥ 2 (ICC = 0.43). Clinicians with prior knowledge of a possible gait abnormality were more likely to assign a grade higher than the median grade.Conclusion and Clinical ImportanceClinicians should be aware of poor agreement even between skilled observers of equine gait abnormalities, especially when the clinical signs are subtle.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

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Rater Agreement on Gait Assessment during Neurologic Examination of Horses

Olsen

Dunkel

Barker

et al. 2014

Veterinary Internal Medicne

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“…A rating scale or instrument is composed of items, which are the fundamental units of measurement. Each item of the scale measures a manifest variable that is related to the latent variable, and takes on a specific value, depending on the magnitude of the latent variable in the respondent [2,3]. Therefore, rating scales use numerical values to represent the characteristics of the outcomes being measured.…”

Section: Introductionmentioning

confidence: 99%

“…The quality of the rating scale also has implications for sample size estimations in study design because sample size estimations take into account the expected effect size, or difference in outcome between the control and treatment groups [4]. The science of measuring and analyzing psychological variables is referred to as psychometrics, and the term clinimetrics is applied to the design, administration, and interpretation of tests to measure clinical and epidemiologic outcomes, such as symptoms or signs, disease progression, or ADLs [5,6]; these methods are increasingly being applied for accurate measurements of outcomes [3]. They are used to evaluate existing rating scales, as well as in the development of new scales.…”

Section: Introductionmentioning

confidence: 99%

The Spectrum of Functional Rating Scales in Neurology Clinical Trials

Narayanaswami

2017

Neurotherapeutics

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The selection of an appropriate outcome measure is crucial to the success of a clinical trial, in order to obtain accurate results, which, in turn, influence patient care and future research. Outcomes that can be directly measured are mortality/survival. More frequently, neurology clinical trials evaluate outcomes that cannot be directly measured, such as disability, cognitive function, or change in symptoms of the condition under study. These complex outcomes are abstract ideas or latent constructs and are measured using rating scales. Functional rating scales typically assess the ability of patients to perform tasks and roles for everyday life. Rating scales should be valid (measure what they are supposed to measure), reliable (provide similar results if administered under the same conditions), and responsive (able to detect clinically important changes over time). The clinical relevance of rating scales depends on their ability to detect a minimal clinically important difference, and should be distinguished from statistical significance. Most rating scales are ordinal scales and have limitations. Modern psychometric methods of Rasch analysis and item response theory, termed latent trait theory, are increasingly being utilized to convert ordinal data to interval measurements, both to validate existing scales and to develop new scales. Patient-reported outcomes are being increasingly used in clinical trials and have a role in clinical quality assessment. The PROMIS and NeuroQoL databases are excellent resources for rigorously developed and validated patientreported outcomes.

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Validation of the Charcot–Marie–Tooth disease pediatric scale as an outcome measure of disability

Burns

Ouvrier

Estilow

et al. 2012

Annals of Neurology

145

163

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Objective Charcot-Marie-Tooth disease (CMT) is a common heritable peripheral neuropathy. There is no treatment for any form of CMT although clinical trials are increasingly occurring. Patients usually develop symptoms during the first two decades of life but there are no established outcome measures of disease severity or response to treatment. We identified a set of items that represent a range of impairment levels and conducted a series of validation studies to build a patient-centered multi-item rating scale of disability for children with CMT. Methods As part of the Inherited Neuropathies Consortium, patients aged 3–20 years with a variety of CMT types were recruited from the USA, UK, Italy and Australia. Initial development stages involved: definition of the construct, item pool generation, peer review and pilot testing. Based on data from 172 patients, a series of validation studies were conducted, including: item and factor analysis, reliability testing, Rasch modeling and sensitivity analysis. Results Seven areas for measurement were identified (strength, dexterity, sensation, gait, balance, power, endurance), and a psychometrically robust 11-item scale constructed (Charcot-Marie-Tooth disease Pediatric Scale: CMTPedS). Rasch analysis supported the viability of the CMTPedS as a unidimensional measure of disability in children with CMT. It showed good overall model fit, no evidence of misfitting items, no person misfit and it was well targeted for children with CMT. Interpretation The CMTPedS is a well-tolerated outcome measure that can be completed in 25-minutes. It is a reliable, valid and sensitive global measure of disability for children with CMT from the age of 3 years.

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Rating scales as outcome measures for clinical trials in neurology: problems, solutions, and recommendations

Cited by 432 publications

References 75 publications

Rater Agreement on Gait Assessment during Neurologic Examination of Horses

Rater Agreement on Gait Assessment during Neurologic Examination of Horses

The Spectrum of Functional Rating Scales in Neurology Clinical Trials

Validation of the Charcot–Marie–Tooth disease pediatric scale as an outcome measure of disability

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