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Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

ABSTRACT: Dysferlinopathy is a muscular dystrophy with a highly variable functional disease progression in which the relationship of function to some patient reported outcome measures (PROMs) has not been previously reported. This analysis aims to identify the suitability of PROMs and their association with motor performance.Two-hundred and four patients with dysferlinopathy were identified in the Jain Foundation's Clinical Outcome Study in Dysferlinopathy from 14 sites in 8 countries. All patients completed the following PROMs: Individualized Neuromuscular Quality of Life Questionnaire (INQoL), International Physical Activity Questionnaire (IPAQ), and activity limitations for patients with upper and/or lower limb impairments (ACTIVLIMs). In addition, nonambulant patients completed the Egen Klassifikation Scale (EK). Assessments were conducted annually at baseline, years 1, 2, 3, and 4. Data were also collected on the North Star Assessment for Limb Girdle Type Muscular Dystrophies (NSAD) and Performance of Upper Limb (PUL) at these time points from year 2. Data were analyzed using descriptive statistics and Rasch analysis was conducted on ACTIVLIM, EK, INQoL. For associations, graphs (NSAD with ACTIVLIM, IPAQ and INQoL and EK with PUL) were generated from generalized estimating equations (GEE). The ACTIVLIM appeared robust psychometrically and was strongly associated with the NSAD total score (Pseudo R ² 0.68). The INQoL performed less well and was poorly associated with the NSAD total score (Pseudo R ² 0.18). EK scores were strongly associated with PUL (Pseudo R ² 0.69). IPAQ was poorly associated with NSAD scores (Pseudo R ² 0.09). This study showed that several of the chosen PROMs demonstrated change over time and a good association with functional outcomes. An alternative quality of life measure and method of collecting data on physical activity may need to be selected for assessing dysferlinopathy.

SUBMITTER: Mayhew AG

PROVIDER: S-EPMC8961025 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature

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Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

Mayhew Anna G AG James Meredith K MK Moore Ursula U Sutherland Helen H Jacobs Marni M Feng Jia J Lowes Linda Pax LP Alfano Lindsay N LN Muni Lofra Robert R Rufibach Laura E LE Rose Kristy K Duong Tina T Bello Luca L Pedrosa-Hernández Irene I Holsten Scott S Sakamoto Chikako C Canal Aurélie A Sánchez-Aguilera Práxedes Nieves N Thiele Simone S Siener Catherine C Vandevelde Bruno B DeWolf Brittney B Maron Elke E Gordish-Dressman Heather H Hilsden Heather H Guglieri Michela M Hogrel Jean-Yves JY Blamire Andrew M AM Carlier Pierre G PG Spuler Simone S Day John W JW Jones Kristi J KJ Bharucha-Goebel Diana X DX Salort-Campana Emmanuelle E Pestronk Alan A Walter Maggie C MC Paradas Carmen C Stojkovic Tanya T Mori-Yoshimura Madoka M Bravver Elena E Díaz-Manera Jordi J Pegoraro Elena E Mendell Jerry R JR Straub Volker V

Frontiers in neurology 20220310

Dysferlinopathy is a muscular dystrophy with a highly variable functional disease progression in which the relationship of function to some patient reported outcome measures (PROMs) has not been previously reported. This analysis aims to identify the suitability of PROMs and their association with motor performance.Two-hundred and four patients with dysferlinopathy were identified in the Jain Foundation's Clinical Outcome Study in Dysferlinopathy from 14 sites in 8 countries. All patients comple ...[more]

PMID: 35359643

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Project description:BackgroundObtaining patients' views of their health and outcomes of interventions utilising patient-reported outcome measures (PROMs) is a well-established method, but there is still uncertainty about the impact of PROMs on services and patient care. Studies are now needed of alternative ways of implementing PROMs. This paper describes a case study of the introduction of a new PROM to assess musculoskeletal (MSK) problems, known as the Musculoskeletal Health Questionnaire (MSK-HQ).MethodsFollowing an invitation from the Arthritis Research UK (ARUK), 11 groups and organisations agreed to become 'partners' in piloting the MSK-HQ. Twenty-nine interviews and a focus group were carried out with key informants from the partners. Interviews were supplemented with some documentary evidence of partners' meetings. Data were coded and analysed with NVivo software V.10. Analysis was carried out via a framework method.ResultsParticipants reported positive evidence that the MSK-HQ is feasible and practical for use in patient care with content that helped health professionals identify and address patients' main presenting problems. Although mediated and reported through health professionals' judgments, the questionnaire was also seen as very relevant and acceptable to a wide spectrum of patients.There was also broad support for the view that whilst the MSK-HQ is relevant to individual patient care, it could also, when aggregated, reflect the experiences of patients as a group and be used as evidence for third parties concerned with the provision and commissioning of services.The main difficulties revealed by the case study were in the form of logistics and sustainability. It was recognised that electronic systems would be more effective for administration and data processing but they were not feasible to develop and implement within reasonable timelines and available budgets. A sustainable approach to using the PROM required significant long-term commitment of budget, a coherent system, and active support from diverse organisations.ConclusionsThe current study supports the view that a bottom-up approach is a promising method to generate PROM-related insights that are relevant to patients and health professionals. The partnership approach to developing and using PROMs may have wider relevance and potential as a model of implementation.

Project description:IntroductionSmoking cessation following lung cancer diagnosis is recommended to improve patient outcomes. Electronic Patient Reported Outcome Measures (ePROMs) may be useful for identifying and facilitating cessation support in patients that continue to smoke after a cancer diagnosis. The primary aim was to investigate the level of agreement between clinician-reported and self-reported patient smoking status during the first visit to a cancer centre (I). Secondary aims included investigating differences between cancer-specific characteristics between never smokers and current/ex-smokers (IIA), and the self-reported frequency of smoking cessation after diagnosis of lung cancer (IIB).MethodsRetrospective single-centre study carried out at a tertiary cancer centre in the UK. Lung cancer patients that completed at least one ePROM questionnaire within 6 weeks of their first visit to the cancer centre (baseline), between February 2019 and February 2020, were included in the study. All ePROM questionnaires included a question regarding smoking status to allow comparison with the clinician records. Patients were offered these electronic questionnaires at each subsequent visit to the hospital.ResultsOf 195 patients included, 24 (12.3%) demonstrated discordance between clinician-reported and self-reported smoking status at the baseline assessment. Clinician-reported 'current smokers' were more likely to be discordant with self-reported smoking status, compared with clinician-reported 'ex-smokers' and 'never smokers' (P = 0.017). Never smokers were more likely to have adenocarcinoma (P < 0.005), present with stage IV cancer (P = 0.023) and receive treatment with palliative intention (P = 0.042), compared with current and ex-smokers. Of those that were reported by clinicians as being current smokers, 9/38 (23.7%) were self-reported ex-smokers. A sub-group of 137 patients completed at least one additional ePROM questionnaire after the baseline and were included in the smoking cessation analysis. Thirty-eight patients were clinician-reported as 'current smokers' at baseline. Of these, 9 subsequently stopped smoking, 17 continued and 3 had short periods of cessation, identified through self-reporting.ConclusionIn most cases, there is concordance between clinician- and self-reported smoking status. A small area of discordance was identified where clinicians reported some patients as 'current smokers', whilst patients self-reported having stopped. The causes for this were not explored and require further investigation. This study supports the use of ePROMs as a helpful tool to assess smoking status, and it can be used to identify patients for smoking cessation referral.

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.

Dataset Information

Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

Publications

Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

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