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An important goal of the treatment of relapsing and remitting multiple sclerosis (RRMS) is to prevent the accumulation of irreversible disability
Recurrences and new lesions on MRI scans of the head often occur in the RRMS trial cohort, so it is simple to compare the frequency of recurrence and the MRI results between treatment groups in RCTs
This suggests that the most commonly used clinical outcome indicator, 3M CDW, may be a poor indicator of irreversible deterioration of disability
Current RCTs use the statistical technique of survival analysis to investigate the time of disability deterioration, and display the Kaplan-Meier survival curve in trial publications to record the accumulation of disability
In this way, they studied EDSS as a disability outcome, including different presentation methods and statistical processing of the outcome data, and compared it with the alternative outcome measures T25FW and NHPT
They used the CombiRx data set, a large three-stage RCT that compared EDSS with the alternative results T25FW and NHPT
CombiRx includes 1,008 participants
During the three-year follow-up, the deterioration of EDSS was as frequent as the improvement of a similar definition, and the improvement rate and the deterioration rate increased simultaneously
During the three-year follow-up, the deterioration of EDSS was as frequent as the improvement of a similar definition, and the improvement rate and the deterioration rate increased simultaneously
Using the Kaplan-Meier method, the proportion of patients with worsening events rose steadily until it reached several times the number of events seen with a more conservative analysis method
For the 36-month 3-month CDW, the EDSS estimated by the "Kaplan-Meier" method is 2.
The main significance of this study is that it has aroused people's attention to the use of EDSS as a standard disability outcome in the RRMS trial, and indicated that T25FW may be a more useful measure
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These findings are related to the design and evaluation of RCTs
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These findings are related to the design and evaluation of RCTs
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Original source:
Koch MW, Mostert JP, Wolinsky JS, Lublin FD, Uitdehaag B, Cutter GR.
Comparison of the EDSS, Timed 25-Foot Walk, and the 9-Hole Peg Test as Clinical Trial Outcomes in Relapsing-Remitting Multiple Sclerosis.
Neurology.
Published online August 25, 2021:10.
1212/WNL.
0000000000012690.
doi:10.
1212/WNL.
0000000000012690.
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