ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 284

Validation of the Sporadic Inclusion Body Myositis Physical Functioning Assessment

Valerie Williams1, Theresa Coles2, Carla DeMuro2, Sandra Lewis2, Nicole Williams2, Stuart Yarr2, Victoria Barghout3, Linda Lowes4, Lindsay Alfano4, Bob Goldberg5, Ari Gnanasakthy2, Gorana Capkun-Niggli6 and Brian Tseng7, 1Psychometrics, RTI Health Solutions, North Carolina, NC, 2RTI Health Solutions, North Carolina, NC, 3VEB HealthCare LLC, Morristown, NJ, 4Nationwide Children’s Hospital, Columbus, OH, 5The Myositis Association, Alexandria, VA, 6Novartis Pharmaceuticals Corporation, Basel, Switzerland, 7Novartis Pharmaceuticals Corporation, East Hanover, NJ

Meeting: 2015 ACR/ARHP Annual Meeting

Date of first publication: September 29, 2015

Keywords:

Session Information

Date: Sunday, November 8, 2015

Title: Muscle Biology, Myositis and Myopathies Poster

Session Type: ACR Poster Session A

Session Time: 9:00AM-11:00AM

Background/Purpose: Patient-reported outcomes (PROs) assess disease symptoms and impact from the patient’s perspective. This research aimed to establish the validity, reliability, and responsiveness of the sporadic Inclusion Body Myositis (sIBM) Physical Functioning Assessment (sIFA) for measuring physical functioning in sIBM patients.

Methods: The development and psychometric evaluation of the sIFA followed the Food and Drug Administration’s PRO guidance. Data were collected from three small, non-interventional, observational studies of sIBM patients in the United States, with a subset of 31 patients followed longitudinally for 1 year. Patients completed the sIFA either electronically or using paper forms. The Improved Health Assessment Questionnaire (IHAQ), as well as objective measures of physical functioning, such as the 6‑Minute Walk Distance (6MWD) and Short Physical Performance Battery, were included to evaluate construct validity. Measures of reliability (Cronbach’s alpha, test-retest intraclass correlations), construct validity (correlations, analyses of variance), and responsiveness (effect size estimates) were computed.

Results: Cronbach’s alpha (range: 0.86–0.91) and test-retest reliability (0.91) were highly satisfactory. Correlations with the IHAQ and 6MWD supported convergent validity; for example, the correlations between the sIFA and IHAQ activities scores were strong (range: 0.73–0.85), but the correlations between the sIFA and IHAQ pain scores were weak (range: 0.19–0.29). The sIFA also correlated strongly with the 6MWD (range: -0.59 to -0.60). sIBM patients who were able to walk without assistive devices scored significantly better (p<0.01) on the sIFA (range: 36.0–47.1) than those requiring power mobility or wheelchairs (range: 54.9–71.5) and patients who walked >400 meters on the 6MWD scored significantly better (p<0.01) on the sIFA (range: 30.0–31.9) than those who walked <300 meters on the 6MWD (range: 56.3–59.5), demonstrating the discriminating ability of the sIFA. Although estimates of responsiveness were small, indicating slow progression of functional impairment, preliminary evidence suggests that the sIFA can be used to detect functional change in sIBM patients.

Conclusion: The psychometric analysis of the sIFA strongly supports its reliability, validity, and responsiveness for assessing the impact of sIBM on patient-reported physical function. The sIFA has the potential to facilitate a more comprehensive evaluation of treatment benefit in sIBM patients.

Disclosure: V. Williams, Novartis Pharmaceutical Corporation, RTI Health Solutions, 9; T. Coles, Novartis Pharmaceutical Corporation, RTI Health Solutions, 9; C. DeMuro, Novartis Pharmaceutical Corporation, RTI Health Soultions, 9; S. Lewis, Novartis Pharmaceutical Corporation, RTI Healthcare Solutions, 9; N. Williams, Novartis Pharmaceutical Corporation, RTI Healthcare Solutions, 9; S. Yarr, Novartis Pharmaceutical Corporation, RTI Health Solutions, 9; V. Barghout, Novartis Pharmaceutical Corporation, 5; L. Lowes, Novartis Pharmaceutical Corporation, 5; L. Alfano, Nationwide Children's Hospital, Columbus, OH, USA, 5; B. Goldberg, None; A. Gnanasakthy, Novartis Pharmaceutical Corporation, RTI Healthcare Solutions, 9; G. Capkun-Niggli, Novartis Pharmaceutical Corporation, 3; B. Tseng, Novartis Pharmaceutical Corporation, 3.

To cite this abstract in AMA style:

Williams V, Coles T, DeMuro C, Lewis S, Williams N, Yarr S, Barghout V, Lowes L, Alfano L, Goldberg B, Gnanasakthy A, Capkun-Niggli G, Tseng B. Validation of the Sporadic Inclusion Body Myositis Physical Functioning Assessment [abstract]. Arthritis Rheumatol. 2015; 67 (suppl 10). https://acrabstracts.org/abstract/validation-of-the-sporadic-inclusion-body-myositis-physical-functioning-assessment/. Accessed .

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