Investigation of the psychometric properties of the inclusion body myositis functional rating scale with rasch analysis

Ramdharry, Gita; Morrow, Jasper M.; Hudgens, Stacie; Skorupinska, Iwona; Gwathmey, Kelly; Currence, Melissa; Herbelin, Laura; Jawdat, Omar; Pasnoor, Mamatha; McVey, April; Barohn, Richard J.; Burns, Ted M.; Dimachkie, Mazen M.; Amato, Anthony A.; Hanna, Michael G.; Machado, Pedro

doi:10.1002/mus.26521

Cited by 12 publications

(4 citation statements)

References 17 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The IBM-FRS and sIFA cover many disabling aspects of the disease, but they have limitations. Rasch analysis of IBM-FRS showed multidimensionality, possible unequal importance of the items on the scale, and redundancy of some items (19,61). Three Rasch modified IBM-FRS scorings have been proposed, but none of them has been validated (61).…”

Section: Discussionmentioning

confidence: 99%

“…Rasch analysis of IBM-FRS showed multidimensionality, possible unequal importance of the items on the scale, and redundancy of some items (19,61). Three Rasch modified IBM-FRS scorings have been proposed, but none of them has been validated (61). Moreover, even the modified upper extremity IBM-FRS does not correlate well with hand function when compared to PRO measures (62).…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Current status of clinical outcome measures in inclusion body myositis: a systematised review

Roy

Lucchini

Lilleker

et al. 2022

Clinical and Experimental Rheumatology

View full text Add to dashboard Cite

Objective. Sporadic inclusion body myositis (IBM) is a debilitating idiopathic inflammatory myopathy (IIM) which affects hand function, ambulation, andswallowing. There is no approved pharmacological therapy for IBM, and there is a lack of suitable outcome measure to assess the effect of an intervention. The IBM scientific interest group under IMACS reviewed the previously used outcome measures in IBM clinical studies to lay the path for developing a core set of outcome measures in IBM. Methods. In this systematised review, we have extracted all outcome measures reported in IBM clinical studies to determine what measures were being used and to assess the need for optimising outcome measures in IBM. Results. We found 13 observational studies, 17 open-label clinical trials, and 15 randomised control trials (RCTs) in IBM. Six-minute walk distance, IBMfunctional rating scale (IBM-FRS), quantitative muscle testing, manual muscle testing, maximal voluntary isometric contraction testing, and thigh muscle volume measured by MRI were used as primary outcome measures. Twelve different outcome measures of motor function were used in IBM clinical trials. IBM-FRS was the most used measure of functionality. Swallowing function was reported as a secondary outcome measure in only 3 RCTs. Conclusion. There are inconsistencies in using outcome measures in clinical studies in IBM. The core set measures developed by the IMACS group for other IIMs are not directly applicable to IBM. As a result, there is an unmet need for an IBM-specific core set of measures to facilitate the evaluation of new potential therapeutics for IBM.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Current status of clinical outcome measures in inclusion body myositis: a systematised review

Roy

Lucchini

Lilleker

et al. 2022

Clinical and Experimental Rheumatology

View full text Add to dashboard Cite

show abstract

“…Yet, we are unaware of any study that has directly investigated the predictive validity between IBM-FRS, or more specifically the swallowing item, and validated measures of swallowing physiology. In fact, one study that employed Rasch analysis recommended removal of the swallowing item to improve the ability to discriminate severity ratings in patients with IBM and because swallowing was unrelated to the construct of the overall scale [13]. However, dysphagia is an important and clinically relevant symptom in IBM that could be analyzed as a separate single-item tool.…”

Section: Updates On Assessment Of Dysphagia In Inclusion Body Myositismentioning

confidence: 99%

Update on the evaluation and management of dysphagia in sporadic inclusion body myositis

Garand,

Malandraki,

Dimachkie

2023

Current Opinion in Otolaryngology &Amp; Head &Amp; Neck Surgery

Self Cite

View full text Add to dashboard Cite

Purpose of review Dysphagia is a common symptom of sporadic inclusion body myositis (IBM), affecting disease trajectory and patient quality-of-life. Despite this, it is considerably understudied. The purpose of this review is to summarize current evidence related to the evaluation and management of dysphagia in IBM. We highlight a patient case involving a multidisciplinary management approach, and we encourage continued exploration of exercises for delaying progression and improving impairments in patients with IBM and dysphagia. Recent findings Recent investigations confirm that dysphagia in IBM is a debilitating and complex symptom that warrants timely evaluation and management. Further, they highlight the lack of validation of standardized swallowing-related metrics specifically for IBM and the limited evidence supporting a consensus of management approaches. Small scale research and clinical anecdotal data support a multidisciplinary and multipronged patient-centered approach, including rehabilitative exercise protocols, for dysphagia management in IBM. Summary A paucity exists in the literature to effectively guide clinical decision-making for patients with IBM and dysphagia. Given this, it is our belief that a careful multidisciplinary and multipronged patient-centered approach is critical for dysphagia management in IBM. Prospective, longitudinal research on the underlying mechanisms of swallowing dysfunction using advanced and validated swallowing-related outcome measures is urgently needed.

show abstract

“…However, deficiencies in these tools are apparent, particularly with regard to assessment of hand function. There have been several attempts to create optimized tools which will require additional validation [63,64].…”

Section: Using Sensitive and Clinically Relevant Outcome Measuresmentioning

confidence: 99%

In Pursuit of an Effective Treatment: the Past, Present and Future of Clinical Trials in Inclusion Body Myositis

Snedden

Lilleker

Chinoy

2021

Curr Treat Options in Rheum

View full text Add to dashboard Cite

Purpose of review No clinical trial in sporadic inclusion body myositis (IBM) thus far has shown a clear and sustained therapeutic effect. We review previous trial methodology, explore why results have not translated into clinical practice, and suggest improvements for future IBM trials. Recent findings Early trials primarily assessed immunosuppressive medications, with no significant clinical responses observed. Many of these studies had methodological issues, including small participant numbers, nonspecific diagnostic criteria, short treatment and/or assessment periods and insensitive outcome measures. Most recent IBM trials have instead focused on nonimmunosuppressive therapies, but there is mounting evidence supporting a primary autoimmune aetiology, including the discovery of immunosuppression-resistant clones of cytotoxic T cells and anti-CN-1A autoantibodies which could potentially be used to stratify patients into different cohorts. The latest trials have had mixed results. For example, bimagrumab, a myostatin blocker, did not affect the 6-min timed walk distance, whereas sirolimus, a promotor of autophagy, did. Larger studies are planned to evaluate the efficacy of sirolimus and arimoclomol. Summary Thus far, no treatment for IBM has demonstrated a definite therapeutic effect, and effective treatment options in clinical practice are lacking. Trial design and ineffective therapies are likely to have contributed to these failures. Identification of potential therapeutic targets should be followed by future studies using a stratified approach and sensitive and relevant outcome measures.

show abstract

Investigation of the psychometric properties of the inclusion body myositis functional rating scale with rasch analysis

Cited by 12 publications

References 17 publications

Current status of clinical outcome measures in inclusion body myositis: a systematised review

Current status of clinical outcome measures in inclusion body myositis: a systematised review

Update on the evaluation and management of dysphagia in sporadic inclusion body myositis

In Pursuit of an Effective Treatment: the Past, Present and Future of Clinical Trials in Inclusion Body Myositis

Contact Info

Product

Resources

About