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Better CMT outcome measurement is Wayne State University physician's goal
Date:12/7/2011

ves with vitamin C in large quantities, and researchers wanted to see if the therapy actually was safe and effective in a randomized trial.

Trials in Australia, France, Italy, England and the Netherlands concluded that ascorbic acid, given as a medication, was safe but ineffective.

"One concern with those trials was that outcome measures varied," Ramchandren said. Some researchers measured a combined outcome score called the Charcot-Marie-Tooth Neuropathy Score, which was developed by Michael Shy, M.D., WSU professor of neurology and Ramchandren's mentor for her current study. Others looked at whether the speed of nerve conduction improved.

Because financial constraints typically limit trials to one or two years, Ramchandren said, an outcome measurement is needed that could change within that time period.

"A good outcome measure will show changes in scores in a short time period so that, when used in a trial, if a drug is effective, we should see those scores improve," she said. "However, if you use an outcome measure that barely budges in a short time period, there's not much chance you will get a dramatic improvement in scores, even if you have a good medication. So, with the recent CMT trials, it may very well be that vitamin C is ineffective, but we can't say for sure when there are doubts about what we are measuring."

With this grant, she will use data from an ongoing consortium study by Shy that collects natural history data on children and adult CMT patients to see how the disease progresses over time. Shy is mentoring Ramchandren as she develops a quality-of-life instrument to measure how CMT impacts children's lives.

Using a questionnaire format, Ramchandren will try to determine what kinds of physical activities CMT restrains. Questions also will address the disease's effects on social roles, such as playing with friends and interaction with families and teachers, as well as emotional behavior, depre
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Contact: Julie O'Connor
julie.oconnor@wayne.edu
313-577-8845
Wayne State University - Office of the Vice President for Research
Source:Eurekalert

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