Journal
NEUROLOGY
Volume 77, Issue 3, Pages 269-275Publisher
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1212/WNL.0b013e318225ab07
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Funding
- German Ministry of Education and Research at the University of Munich, Campus Grosshadern [01EO0901]
- Bundesministerium fur Bildung und Forschung (BMBF)
- Deutsche Forschungsgemeinschaft (DFG)
- German Ministry of Technology and Research (BMFT)
- Bavarian Ministry of Health
- Santhera Pharmaceuticals
- NIH
- Ludwig-Maximilians-Universitat Munchen
- Forderung fur Forschung und Lehre (FoFoLe)
- Wellcome Trust
- Foundation for Vascular Dementia Research
- TaroPharma
- Else Kroener Fresenius Foundation
- Eva Luise Koehler Foundation
- NIH (NEI/NINDS)
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Objective: The therapeutic effects of 4-aminopyridine (4AP) were investigated in a randomized, double-blind, crossover trial in 10 subjects with familial episodic ataxia with nystagmus. Methods: After randomization, placebo or 4AP (5 mg 3 times daily) was administered for 2 3-month-long treatment periods separated by a 1-month-long washout period. The primary outcome measure was the number of ataxia attacks per month; the secondary outcome measures were the attack duration and patient-reported quality of life (Vestibular Disorders Activities of Daily Living Scale [VDADL]). Nonparametric tests and a random-effects model were used for statistical analysis. Results: The diagnosis of episodic ataxia type 2 (EA2) was genetically confirmed in 7 subjects. Patients receiving placebo had a median monthly attack frequency of 6.50, whereas patients taking 4AP had a frequency of 1.65 (p = 0.03). Median monthly attack duration decreased from 13.65 hours with placebo to 4.45 hours with 4AP (p = 0.08). The VDADL score decreased from 6.00 to 1.50 (p = 0.02). 4AP was well-tolerated. Conclusions: This controlled trial on EA2 and familial episodic ataxia with nystagmus demonstrated that 4AP decreases attack frequency and improves quality of life. Level of evidence: This crossover study provides Class II evidence that 4AP decreases attack frequency and improves the patient-reported quality of life in patients with episodic ataxia and related familial ataxias. Neurology (R) 2011;77:269-275
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