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Tawil R, Griggs R, Thornton C, Kissel J, Mendell J, Genge A, Karpati G, Jackson C, Rose M, Amato A, Barohn R, Saperstein D, McDermott MP, Pandya S, Wahlen D, King W, Venturini A, Myers D, Prisley S, Tagerman M, O'Brien M, Herbelin L, Cos L, Downing K, Plesiak R, Barefield F, Morrison C, Briemberg H, Holloway R, Peterson D, Janciuras J, Martens W, Blood C.
University of Texas Health Science Center, San Antonio, TX, USA. jacksonce@uthscsa.edu
We developed a disease-specific, 10-point functional rating scale for patients with inclusion body myositis (IBMFRS). The IBMFRS was utilized as a secondary outcome measure in a multicenter pilot trial of the clinical safety and tolerability of high-dose beta interferon-1a. In this trial, 28 IBM patients completed the IBMFRS at baseline and monthly for 6 months. The IBMFRS showed statistically significant correlations (P < 0.001) with maximal voluntary isometric contraction, manual muscle testing, handgrip dynamometry, and the amyotrophic lateral sclerosis (ALS) functional rating scale (ALSPRS). Compared to these other outcome measures, the IBMFRS was also the most sensitive measure of change over the course of the study.
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