The Epidemiology of Myasthenia Gravis

Lawrence H. Phillips II, M.D.

Disclosures

Semin Neurol. 2004;24(1) 

In This Article

Future Areas for Epidemiologic Research in MG

A population-based evaluation of treatment has not been done. The present emphasis on evidence-based medicine usually requires that any treatment be subjected to a standard of proof of efficacy from data obtained in a controlled clinical trial. There have been very few clinical trials conducted in patients with MG, and the only large-scale randomized controlled clinical trial has been for the use of cyclosporine.[16,17] The current state of treatment for MG is thus based more on the individual preference of the clinician than it is on hard evidence. A population-based overview of current treatments in use would be a good first step in providing some much-needed evidence for rational treatment decisions. In the absence of head-to-head comparisons of treatments, a survey that established the frequency of use of various treatments as well as the rate of clinical response and the occurrence of side effects would be of great use for helping guide decisions between different drugs where there is no clear evidence of superiority of any one drug. The term "equipose" is used to describe the situation where the available evidence does not clearly favor one treatment approach over another.

One area of treatment that would benefit from information that might come from a population-based survey is that of thymectomy. There have been several attempts over the years to design a clinical trial of thymectomy in MG, but so far none have been organized or performed successfully. The bulk of the information available about the efficacy has come from accumulated clinical experience in the form of surgeons' case series. Data from multicenter databases has established that some patients with MG do benefit from thymectomy, but only the most general information about which patients are most likely to do so is available.[18]

Finally, the true impact of MG on the individual patient cannot be measured in terms of muscle strength alone. There are social, financial, and emotional factors that affect each patient in different ways. Research is just beginning into quality-of-life issues in patients with MG.[19] Physicians are unaccustomed to performing research in this area, but it is an important part of the overall picture of the disease in our patients. Hopefully, there will be ongoing research into such nontraditional areas, as economic and social factors will certainly be factors in future treatment decisions.

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