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Measuring upper limb function in MS: Which existing patient reported outcomes are fit for purpose?

ABSTRACT: Background:Multiple Sclerosis (MS) clinical trials increasingly focus on progressive and advanced MS, with upper limb function (ULF) as a key outcome. Within clinical trials, Patient Reported Outcomes (PROs) quantify clinical variables and establish meaningfulness of changes. Scientific standards and regulatory criteria (from Food and Drug Administration [FDA]) require PROs be "fit-for-purpose": well-defined and reliable measures of specific concepts in defined contexts. Objective:To identify, from literature, existing PROs measuring ULF and determine which satisfy scientific and regulatory clinical trials requirements. Method:We screened PubMed/Web of Science using multiple relevant terms. Abstracts and full texts were screened using suitability criteria. PRO development papers were evaluated using recently expanded Consensus Standards for Measurement Instruments (COSMIN) criteria for content development. Results:We identified 3619 articles; 485 used 24 different ULF PROs. No PRO satisfied scientific and regulatory requirements as a well-defined measure of a clearly defined construct in a specific clinical context. Conclusions:Existing ULF PROs don't meet fit-for-purpose criteria. MS clinical trials require new measures with greater emphasis on patient engagement to derive theoretical frameworks, concepts of interest, and contexts of use followed by systematic literature searches, expert input, and qualitative research to support item generation. Until then, trials will miss aspects of meaningful within-patient change and thereby misrepresent (likely underestimating) treatment effects.

SUBMITTER: Close J

PROVIDER: S-EPMC7125352 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature

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Measuring upper limb function in MS: Which existing patient reported outcomes are fit for purpose?

Close James J Baines Kathryn K Burke Laurie L Hobart Jeremy J

eNeurologicalSci 20200316

<h4>Background</h4>Multiple Sclerosis (MS) clinical trials increasingly focus on progressive and advanced MS, with upper limb function (ULF) as a key outcome. Within clinical trials, Patient Reported Outcomes (PROs) quantify clinical variables and establish meaningfulness of changes. Scientific standards and regulatory criteria (from Food and Drug Administration [FDA]) require PROs be "fit-for-purpose": well-defined and reliable measures of specific concepts in defined contexts.<h4>Objective</h4 ...[more]

PMID: 32258444

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Project description:BACKGROUND:Breast cancer is the most common cancer in women worldwide. Major breast cancer surgery especially with axillary lymph node dissection (ALND), is associated with upper limb functional decline. Majority of studies are conducted in Western population and may not be applicable to Asians. This pilot study aims to evaluate whether major breast surgery results in upper limb functional impairment in a cohort of Asian women with breast cancer. METHODS:This is a prospective cohort study of 41 patients who underwent 44 major breast surgeries from April 2018 to August 2019. Main inclusion criteria were patients over 21?years of age undergoing major breast surgery for breast cancer. Major breast surgery was defined as wide local excision (WLE) or mastectomy. Main exclusion criteria were patients with pre-existing neurological or rheumatological co-morbidities affecting upper limb function or previous trauma with resulting deformities to the upper limbs. Patients underwent early rehabilitation from post-operative day 1. Shoulder flexion and abduction active range of motion (AROM) and QuickDASH disability score were assessed 1?week before surgery, post-operative week 2 and week 6. Baseline demographics and peri-operative data were also collected. RESULTS:Median age was 62.5?years. There were 16 (36.4%) wide local excisions and 28 (63.6%) simple mastectomies. Two (4.5%) cases had neoadjuvant chemotherapy. Fifteen (34.1%) cases had ALND. At post-operative week 6, shoulder flexion was comparable to baseline (p?=?0.775), while abduction improved from baseline (p?=?0.016). However, QuickDASH disability score was significantly worse at post-operative week 6 compared to baseline (median score 2.5 vs 0, p?=?0.027). Subgroup analysis of patients with ALND demonstrated significantly worse QuickDASH disability score at post-operative week 6 (p?=?0.010) but not for patients with only sentinel lymph node biopsy (p?=?0.396). CONCLUSION:This pilot study in an Asian cohort found that patients were able to regain AROM of shoulder after major breast surgery at post-operative week 6 but had a worse QuickDASH disability score, especially in the subgroup with ALND. Aggressive and early rehabilitation should be encouraged. However, a longer follow-up is required to evaluate long term functional outcomes.

Project description:AimsThe aims of this review were (i) to evaluate whether patient-reported outcome measures used in clinical studies for assessing sensation after mastectomy and breast reconstruction are suitable for this purpose, and (ii) to explore whether any measures used for assessing sensation after non-oncologic breast surgery are worth modifying for use in post-mastectomy patients.MethodsPRISMA guidelines were followed (PROSPERO number CRD42020178066). We searched six databases for studies of oncologic (i.e., therapeutic, prophylactic, and reconstructive) and non-oncologic breast surgery (e.g., breast reduction) in which sensation was assessed with a patient-reported outcome measure. From the selected studies, we extracted eligible measures, evaluated their fitness for purpose, and summarized the content of sensation-specific items.ResultsOf 6728 articles identified, we selected 135 studies that used 124 eligible patient-reported outcome measures. For 97% of these measures, details regarding development and measurement properties were unavailable. Four (3%) validated measures-the Sensory Disturbances subscale of the Breast Cancer Sequelae Cause Scales, the Discomfort subscale of the Breast Sensation Assessment Scale (BSAS), Didier et al.'s questionnaire for "Assessment of the patients' satisfaction with cosmetic results, physical and emotional impact of mastectomy", and the Breast Specific Pain subscale of the Breast Cancer Treatment Outcomes Scale (BCTOS)-each contain at least one item pertaining to breast sensation, but target different concepts of interest. In total, the measures feature 215 sensation-specific items, most of which concern symptom severity (97%) as opposed to impact on daily functioning (3%).ConclusionPatient-reported outcome measures used in clinical studies for assessing sensation after mastectomy and breast reconstruction are unsuitable for this purpose: they are either non-validated or non-specific. We failed to identify any measures for use in non-oncologic breast surgery populations worth modifying. To collect meaningful, patient-relevant data regarding sensation after mastectomy, it is pertinent that future clinical trials adopt psychometrically robust, specific patient-reported outcome measures.

Project description:Importance:Aesthetic eye treatments can dramatically change a person's appearance, but outcomes are rarely measured from the patient perspective. The patient perspective could be measured using an eye-specific patient-reported outcome measure. Objective:To describe the development and psychometric evaluation of FACE-Q scales and an adverse effect checklist designed to measure outcomes following cosmetic eye treatments. Design, Setting, and Participants:Pretreatment and posttreatment patients 18 years and older who had undergone facial aesthetic procedures were recruited from plastic surgery clinics in United States and Canada and completed FACE-Q scales between June 6, 2010, and July 14, 2014. We used Rasch Measurement Theory, a modern psychometric approach, to refine the scales and to examine psychometric properties. Main Outcomes and Measures:The FACE-Q Eye Module, which has 4 scales that measure appearance of the eyes, upper and lower eyelids, and eyelashes. Scale scores range from 0 (worst) to 100 (best). The module also includes a checklist measuring postblepharoplasty adverse effects. Results:Overall, 233 patients (81% response rate) 18 years and older participated. Adverse effects included being bothered by eyelid scars, dry eyes, and eye irritation. In Rasch Measurement Theory analysis, each scale's items had ordered thresholds and good item fit. Person Separation Index and Cronbach ? were greater than or equal to 0.83. Higher scores on the eye scales correlated with fewer adverse effects (range, -0.26 to -0.36). In the pretreatment group, older age correlated with lower scores (range, -0.42 to -0.51) on the scales measure appearance of the eyes and upper and lower eyelids. Compared with the pretreatment group, posttreatment participants reported significantly better scores on the scales measuring appearance of eyes overall, as well as upper and lower eyelids. Conclusions and Relevance:The FACE-Q Eye Module can be used in clinical practice, research and quality improvement to collect evidence-based outcomes data. Level of Evidence:NA.

Project description:Importance: Given the importance of apathy for stroke, we felt it was time to scrutinize the psychometric properties of the commonly used Starkstein Apathy Scale (SAS) for this purpose. Objectives: The objectives were to: (i) estimate the extent to which the SAS items fit a hierarchical continuum of the Rasch Model; and (ii) estimate the strength of the relationships between the Rasch analyzed SAS and converging constructs related to stroke outcomes. Methods: Data was from a clinical trial of a community-based intervention targeting participation. A total of 857 SAS questionnaires were completed by 238 people with stroke from up to 5 time points. SAS has 14 items, rated on a 4-point scale with higher values indicating more apathy. Psychometric properties were tested using Rasch partial-credit model, correlation, and regression. Items were rescored so higher scores are interpreted as lower apathy levels. Results: Rasch analysis indicated that the response options were disordered for 8/14 items, pointing to unreliability in the interpretation of the response options; they were consequently reduced from 4 to 3. Only 9/14 items fit the Rasch model and therefore suitable for creating a total score. The new rSAS was deemed unidimensional (residual correlations: < 0.3), reasonably reliable (person separation index: 0.74), with item-locations uniform across time, age, sex, and education. However, 30% of scores were > 2 SD above the standardized mean but only 2/9 items covered this range (construct mistargeting). Apathy (rSAS/SAS) was correlated weakly with anxiety/depression and uncorrelated with physical capacity. Regression showed that the effect of apathy on participation and health perception was similar for rSAS/SAS versions: R2 participation measures ranged from 0.11 to 0.29; R2 for health perception was ∼0.25. When placed on the same scale (0-42), rSAS value was 6.5 units lower than SAS value with minimal floor/ceiling effects. Estimated change over time was identical (0.12 units/month) which was not substantial (1.44 units/year) but greater than expected assuming no change (t: 3.6 and 2.4). Conclusion: The retained items of the rSAS targeted domains of behaviors more than beliefs and results support the rSAS as a robust measure of apathy in people with chronic stroke.

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Measuring upper limb function in MS: Which existing patient reported outcomes are fit for purpose?

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Measuring upper limb function in MS: Which existing patient reported outcomes are fit for purpose?

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