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BRIEF COMMUNICATIONS: M. R. Rose, M. P. McDermott, C. A. Thornton, C. Palenski, W. B. Martens, and R. C. Griggs A prospective natural history study of inclusion body myositis: Implications for clinical trials Neurology 2001; 57: 548-550 [Abstract] [Full text] [PDF]

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Reply to Mr. Gregg

Michael Rose, M. P. McDermott, C. A. Thornton, C. Palenski, W. B. Martens, and R. C. Griggs   (29 October 2002)

Question

Donald J Gregg   (29 October 2002)

Reply to Mr. Gregg 29 October 2002
Michael Rose King's College Hospital, London, M. P. McDermott, C. A. Thornton, C. Palenski, W. B. Martens, and R. C. Griggs Send Correspondence to journal: Re: Reply to Mr. Gregg m.r.rose{at}kcl.ac.uk Michael Rose, et al.	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.
Question 29 October 2002
Donald J Gregg, Engineer No Medical Affiliation Send Correspondence to journal: Re: Question dgregg000{at}sympatico.ca Donald J Gregg	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