Ascorbic Acid in Charcot-Marie-Tooth Disease Type 1A
Author Information
Author(s): Davide Pareyson, Mary M Reilly, Angelo Schenone, Gian Maria Fabrizi, Tiziana Cavallaro, Lucio Santoro, Giuseppe Vita, Aldo Quattrone, Luca Padua, Franco Gemignani, Francesco Visioli, Matilde Laurà, Davide Radice, Daniela Calabrese, Richard AC Hughes, Alessandra Solari
Primary Institution: Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Foundation, Carlo Besta Neurological Institute, Milan, Italy
Hypothesis
Does ascorbic acid improve neuropathy in patients with Charcot-Marie-Tooth disease type 1A?
Conclusion
Ascorbic acid supplementation had no significant effect on neuropathy compared with placebo after 2 years.
Supporting Evidence
- 277 patients were enrolled and randomly assigned to treatment.
- Treatment was well tolerated with 89% of patients completing the study.
- Mean CMT neuropathy score did not significantly change between groups.
Takeaway
The study tested if vitamin C could help people with a nerve disease called CMT, but it didn't work.
Methodology
Patients aged 18-70 with symptomatic CMT1A were randomly assigned to receive either 1.5 g/day of ascorbic acid or placebo for 24 months, with various clinical assessments conducted.
Potential Biases
Potential biases related to the randomization process and the masking of treatment allocation.
Limitations
The study may not have been long enough to detect small effects, and the chosen dose of ascorbic acid might not have been sufficient.
Participant Demographics
Adult patients aged 18-70 years with symptomatic CMT1A from nine centers in Italy and the UK.
Statistical Information
P-Value
0.93
Confidence Interval
95% CI −0.6 to 0.7
Statistical Significance
p=0.93
Digital Object Identifier (DOI)
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