Patients with progressive multiple sclerosis (MS) and their clinicians see clear improvements during treatment with a high dose of biotin, show further results from the phase III trial of the therapy.
After 12 months of treatment, the average clinical global impression score was 4.05 in the biotin group versus 4.62 in the placebo group, and the corresponding subject global impression scores were 4.27 versus 4.76, with both differences being statistically significant.
Presenter Ayman Tourbah (CHU de Reims, France) noted that physicians mostly scored patients receiving biotin (300 mg/day) between no change and much improved, whereas they scored those in the placebo group between no change and much or very much worse.
This confirms the clinical relevance of the trial’s previously reported primary finding, he told delegates at the European Academy of Neurology congress in Berlin, Germany.
The primary endpoint was either improvement on the Expanded Disability Status Scale (EDSS), defined as a 1-point improvement for patients with a baseline score of 4.5 to 5.5 or of 0.5 points for patients with a score of 6 or 7, or at least a 20% improvement on the timed 25-metre walk (TW25).
The proportion with this outcome at 9 months, and confirmed at 12 months, was 12.62% in the 103 patients in the biotin group versus 0.0% of the 51 in the placebo group. More patients met the primary endpoint with the EDSS than the TW25, Tourbah noted.
Biotin is a co-enzyme for several carboxylases, one of which is thought to have a key role in myelin synthesis. Thus, it could potentially alleviate MS symptoms in two ways: by increasing ATP levels and reversing the “virtual hypoxia” found in MS muscles; and by promoting myelin repair via fatty acid synthesis.
Patients taking biotin improved on the EDSS by 3 months and remained improved for the duration of the study, whereas the placebo group had worse scores than at baseline except for a slight improvement at month 3, which Tourbah attributed to the placebo effect.
The relapse rate was 3.9% in the biotin group, compared with 7.8% among patients taking placebo. However, among patients who underwent magnetic resonance imaging, rates of new and enlarging T2 lesions were higher in the biotin group than the placebo group (23.4 vs 13.0% and 8.5 vs 0.0%, respectively), although this was not significant.
Adverse effects were similar in the two groups. Five patients in the biotin group had apparent hypothyroidism by the end of the study, but Tourbah said this was a consequence of high plasma biotin levels interfering with immunoassays, producing misleading test results.
The same could be true for other tests involving biotinylated antibodies and substrates, said Tourbah, adding that “this requires careful information for patients, families and physicians.”
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