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Brief Communications
August 14, 2001
Letter to the Editor

A prospective natural history study of inclusion body myositis
Implications for clinical trials

August 14, 2001 issue
57 (3) 548-550

Abstract

Eleven patients with untreated inclusion body myositis (IBM) were prospectively studied during a 6-month period that included muscle strength, lean body mass, and muscle mass measurements. There was an overall quantifiable mean decline in percent of predicted normal muscle strength of 4% from baseline in a 6-month period, but one third of patients showed no change or slight improvements in strength. Short-term treatment trials in IBM will require large numbers of patients to detect slowing, arrest, or even slight improvement in muscle strength.

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References

1.
Griggs RC, Askanas V, DiMauro S, et al. Inclusion body myositis and myopathies. Ann Neurol . 1995; 38: 705–713.
2.
Personius KE, Pandya S, King WM, et al. Facioscapulohumeral dystrophy natural history study: standardization of testing procedures and reliability of measurements. The FSH DY Group Physical Therapy 1994;74:253–263.
3.
FSH-DY Study Group. A prospective, quantitative study of the natural history of facioscapulohumeral muscular dystrophy (FSHD): implications for therapeutic trials. Neurology . 1997; 48: 38–46.
4.
Griggs RC, Forbes G, Moxley RT, et al. The assessment of muscle mass in progressive neuromuscular disease. Neurology . 1983; 33: 158–165.
5.
Forbes GB, Gallup J, Hursh JB. Estimation of total body fat from potassium-40 content. Science . 1961; 133: 101.
6.
Lotz BP, Engel AG, Nishino H, et al. Inclusion body myositis. Observations in 40 patients. Brain . 1989; 112: 727–747.
7.
Lindberg C, Persson LI, Bjorkander J, et al. Inclusion body myositis: clinical, morphological, physiological and laboratory findings in 18 cases. Acta Neurol Scand . 1994; 89: 123–131.
8.
Peng A, Koffman BM, Malley JD, et al. Disease progression in sporadic inclusion body myositis: observations in 78 patients. Neurology . 2000; 55: 296–298.
9.
Leff RL, Miller FW, Hicks J, et al. The treatment of inclusion body myositis: a retrospective review and a randomised prospective trial of immunosuppressive therapy. Medicine . 1993; 72: 225–235.
10.
Griggs RC, Rose MR. Evaluation of treatment for sporadic inclusion body myositis. In: Askanas V, Serratrice G, Engel WK, eds. Inclusion body myositis and myopathies. Cambridge: Cambridge University Press, 1998:.
Letters to the Editor
29 October 2002
Reply to Mr. Gregg
Michael Rose
M. P. McDermott, C. A. Thornton, C. Palenski, W. B. Martens, and R. C. Griggs

We appreciate this thoughtful response particularly as it has the distinction of being a first published patient letter and from someone previously involved in one of our IBM trials. The question recognizes that there is a paucity of well-documented prospective studies of disease progression in IBM. This makes designing adequately powered clinical trials difficult and was the reason for conducting this natural history study [1]. We did base our study methods and subject numbers for both the published Avonex trial [2] and an ongoing higher-dose Avonex trial on these natural history data. Now that we have accumulated data from the placebo arm of both of these trials these data should be preferred to "natural history" data in the future planning of IBM clinical trials as they take into account potential "placebo effects". Published data from both Avonex trials will contain the necessary information to help any investigators design future trials of treatment for IBM.

It is relevant to this discussion to point out that combining such data from different sources (meta-analysis) is a recognized method of trying to make smaller studies and trials more informative. The ability to do this is considerably enhanced if common and agreed upon outcome measures are used.

1. Rose MR, McDermott MP, Thornton CA, Palenski C, Griggs RC. A prospective longitudinal natural history study of inclusion body myositis; implications for clinical trials. Neurology 2001; 57:548-550.

2. Muscle Study Group:, Randomized pilot trial of INF1a (Avonex) in patients with inclusion body myositis. Neurology 2001; 57:1566-1570.

29 October 2002
Question
Donald J Gregg, Engineer

I have Inclusion Body Myositis so I read your abstract with specific interest. Objective numbers on this diseases progress are lacking and your work represents an important step in developing this type of objective data.

My question concerns the use of data from other studies. Recognizing the ethical concerns and statistical validity, have you considered collecting the natural history portion of studies that focus on a specific treatment but used a placebo group as a control? In such cases. the placebo group might be a valid natural history group to supplement or augment your data with appropriate provisos.

I recently participated in a study using Avonex as a potential agent for the treatment of IBM. The Placebo group in this study underwent similar measurements as described in your abstract so might provide useful augmentation to your study if appropriate data release can be obtained. I note some of your colleagues are from Rochester NY where the study I refer to was carried out.

Just a thought. I'm an engineer/statistician not a physician or medical researcher.

Don Gregg

Information & Authors

Information

Published In

Neurology®
Volume 57Number 3August 14, 2001
Pages: 548-550
PubMed: 11502935

Publication History

Received: October 19, 2000
Accepted: March 25, 2001
Published online: August 14, 2001
Published in print: August 14, 2001

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Authors

Affiliations & Disclosures

M. R. Rose, MD, FRCP
From the Department of Neurology, King’s Neurosciences Centre, King’s College Hospital, London, UK (Dr. Rose); and Department of Neurology, University of Rochester Medical Center, NY (Drs. McDermott, Thornton, Martens, Griggs, and C. Palenski).
M. P. McDermott, PhD
From the Department of Neurology, King’s Neurosciences Centre, King’s College Hospital, London, UK (Dr. Rose); and Department of Neurology, University of Rochester Medical Center, NY (Drs. McDermott, Thornton, Martens, Griggs, and C. Palenski).
C. A. Thornton, MD
From the Department of Neurology, King’s Neurosciences Centre, King’s College Hospital, London, UK (Dr. Rose); and Department of Neurology, University of Rochester Medical Center, NY (Drs. McDermott, Thornton, Martens, Griggs, and C. Palenski).
C. Palenski, MS, RNC
From the Department of Neurology, King’s Neurosciences Centre, King’s College Hospital, London, UK (Dr. Rose); and Department of Neurology, University of Rochester Medical Center, NY (Drs. McDermott, Thornton, Martens, Griggs, and C. Palenski).
W. B. Martens
From the Department of Neurology, King’s Neurosciences Centre, King’s College Hospital, London, UK (Dr. Rose); and Department of Neurology, University of Rochester Medical Center, NY (Drs. McDermott, Thornton, Martens, Griggs, and C. Palenski).
R. C. Griggs, MD
From the Department of Neurology, King’s Neurosciences Centre, King’s College Hospital, London, UK (Dr. Rose); and Department of Neurology, University of Rochester Medical Center, NY (Drs. McDermott, Thornton, Martens, Griggs, and C. Palenski).

Notes

Address correspondence and reprint requests to Dr. M.R. Rose, Department of Neurology, King’s Neurosciences Centre, King’s College Hospital, Denmark Hill, London, UK SE5 9RS; e-mail: [email protected]

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  8. Inclusion body myositis: clinical features and pathogenesis, Nature Reviews Rheumatology, 15, 5, (257-272), (2019).https://doi.org/10.1038/s41584-019-0186-x
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