Phase IIB Randomized Trial on the Use of 4-Aminopyridine in Guillain-Barré Syndrome.
Jay M Meythaler, Robert C Brunner, Jean Peduzzi
PMID 34179759WHAT IT FOUND
4-aminopyridine did not improve strength or walking compared with placebo in people with chronic Guillain-Barré syndrome.
Seven of nineteen participants dropped out, including three because of adverse effects such as tremor and dizziness.
Key findings
01Motor strength scores were numerically higher on placebo than on 4-aminopyridine, and the difference did not reach significance.
02There were no significant differences between 4-aminopyridine and placebo for grip strength, the 6-minute walk test, health status, depression, mood, or pain.
03Seven participants discontinued the study prematurely, including three because of adverse events likely related to 4-aminopyridine.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for OTs
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What it does not show
The study was very small, with only twelve participants completing both treatment arms, which severely limits statistical power. Most participants had high baseline function, creating a ceiling effect that made it difficult to detect improvements. Seven of nineteen participants dropped out, including three due to adverse effects, which may bias the results. The trial was designed primarily for safety, not efficacy, so it was not powered to detect clinical differences. Participants could often tell when they were on the active drug due to tingling sensations, which may have compromised blinding.
Declared interests
The study was conducted at the University of Alabama at Birmingham. The investigational medication was supplied by Scott Wepfer, RPh, at The Compounding Shoppe. No specific funding source or commercial conflict of interest declaration is provided in the text.
The easy way to misread this
Do not interpret the numerical trend toward better FIM scores on 4-aminopyridine as evidence of efficacy. The difference was not statistically significant, the study was underpowered, and the primary outcome of motor strength actually favored placebo.