Session Information
Date: Monday, September 23, 2019
Session Title: Clinical Trials, Pharmacology and Treatment
Session Time: 1:45pm-3:15pm
Location: Agora 3 West, Level 3
Objective: Study the effect of fampridine in gait outcomes in patients with hereditary spastic paraplegia (HSP).
Background: Fampridine acts through blockade of voltage-gated potassium channels, improving action potential conduction in demyelinated axons. It is approved for treatment of gait disturbances in patients with multiple sclerosis (MS). Hereditary spastic paraplegias are a heterogenous group of disorders with corticospinal axonal degeneration leading to gait disturbances. Scarce published data suggest fampridine might be beneficial in HSP.
Method: Prospective, uncontrolled, open trial. Patients were prescribed oral fampridine 10mg bid for 6 months. Gait evaluation with the Timed-25-Foot-Walk (T25FW) test and a 7-point Likert scale for gait improvement were done. The first examination was done at inclusion date and then at day 15, 1 and 6 months after treatment was started. Fampridine safety was assessed clinically and through routine lab tests. All patients provided informed consent.
Results: 11 patients were included between March 2017 and January 2019, and 7 completed the study. Four (57,1%) were male, with mean age of 43 years. Five patients had a genetic mutation identified (SPG4, SPG11, SPG28) and 71% were on anti-spastic medication. There was an improvement in the T25FW after introduction of fampridine: from mean 9,82 seconds (s) at first evaluation to 8,62s (12,2% improvement), 8,05s (18%) and 6,92s (29,5%) at day 15, 1 month and 6 moths, respectively. Median Likert scale at 6 months showed moderate improvement (ranging from slight to significant improvement). Four patients interrupted the trial due to feeling of inefficacy (2) or comorbidities (2). Seven adverse events were reported.
Conclusion: Despite study limitations, our results suggest a possible beneficial effect of this drug. Fampridine may be a new option for symptomatic treatment in HSP, but controlled, double blind clinical trials are necessary.
To cite this abstract in AMA style:
M. Rosário, J. Ferreira, J. Ferro, L. Guedes. Symptomatic treatment of hereditary spastic paraplegias with fampridine: a pilot study [abstract]. Mov Disord. 2019; 34 (suppl 2). https://www.mdsabstracts.org/abstract/symptomatic-treatment-of-hereditary-spastic-paraplegias-with-fampridine-a-pilot-study/. Accessed May 15, 2025.« Back to 2019 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/symptomatic-treatment-of-hereditary-spastic-paraplegias-with-fampridine-a-pilot-study/