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Peer-reviewedCC BY-NC-ND 4.0Annals of Clinical and Translational Neurology

Efficacy of Fampridine on Refractory Optic Neuritis in Neuromyelitis Optica Spectrum Disorders

  1. 1Department of Neurology Sun Yat‐sen Memorial Hospital, Sun Yat‐sen University Guangzhou China
  2. 2Department of Neurology Shenshan Medical Center of Sun Yat‐sen Memorial Hospital Shanwei China
  3. 3State Key Laboratory of Ophthalmology, Zhongshan Ophthalmic Center Sun Yat‐sen University Guangzhou China

Abstract

Background: Neuromyelitis optica spectrum disorders (NMOSD) usually occur with acute refractory optic neuritis (ON). However, there is a lack of efficacious treatments to improve recovery. Since fampridine has been found to improve the prognosis of multiple sclerosis (MS) and MS‐related ON, it is necessary to explore the efficacy of fampridine on visual functions in NMOSD‐ON.

Methods: Patients aged 14–80 who suffered from acute refractory AQP4‐IgG positive ON within 2 weeks onset and with high‐dose corticosteroid treatment were included. Patients treated with sustained‐release fampridine (4‐aminopyridine, 4‐AP) as add‐on treatment for 3 months were compared with patients without 4‐AP (non‐4‐AP). Visual functional scores (VFS), Opticospinal Impairment Scale (OSIS), the logarithm of the minimum angle of resolution (LogMAR) of visual acuity (VA), the mean deviation (MD) value of the static visual field, and retina nerve fiber layer (RNFL) thickness were measured and followed up from baseline to 6 months.

Results: Patients with 4‐AP add‐on treatment improved VFS or OSIS greater than that with control (non‐4‐AP) at 3 and 6 months compared with baseline (all p < 0.05). 4‐AP add‐on treatment also increased the proportion of better improvement of ON (VFS ≤ 2) at 3 months (36.4% vs. 7.1% in control, p < 0.05 adjusted for plasma exchange), and 4‐AP improved visual field at 6 months (p < 0.05 adjusted for plasma exchange).

Conclusion: Fampridine as an add‐on treatment has potential functional benefit on refractory NMOSD‐ON and improves visual functions after the attack of ON.

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BibTeX
@article{wang2026efficacy,
  title = {{Efficacy of Fampridine on Refractory Optic Neuritis in Neuromyelitis Optica Spectrum Disorders}},
  author = {Hongxuan Wang and Wanru Chen and Xiangpen Li and Hengjian Lu and Chenguang Li and Xiaoni Zhang and Lulu Bu and Xiaoyu Xu and Qingyu Shen and Ying Peng},
  journal = {Annals of Clinical and Translational Neurology},
  year = {2026},
  month = oct,
  doi = {10.1002/acn3.70557},
  url = {https://doi.org/10.1002/acn3.70557},
  note = {CC BY-NC-ND 4.0},
}
RIS
TY  - JOUR
TI  - Efficacy of Fampridine on Refractory Optic Neuritis in Neuromyelitis Optica Spectrum Disorders
AU  - Hongxuan Wang
AU  - Wanru Chen
AU  - Xiangpen Li
AU  - Hengjian Lu
AU  - Chenguang Li
AU  - Xiaoni Zhang
AU  - Lulu Bu
AU  - Xiaoyu Xu
AU  - Qingyu Shen
AU  - Ying Peng
PY  - 2026
DA  - 2026/10/27
JO  - Annals of Clinical and Translational Neurology
DO  - 10.1002/acn3.70557
UR  - https://doi.org/10.1002/acn3.70557
AB  - Background: Neuromyelitis optica spectrum disorders (NMOSD) usually occur with acute refractory optic neuritis (ON). However, there is a lack of efficacious treatments to improve recovery. Since fampridine has been found to improve the prognosis of multiple sclerosis (MS) and MS‐related ON, it is necessary to explore the efficacy of fampridine on visual functions in NMOSD‐ON.  Methods: Patients aged 14–80 who suffered from acute refractory AQP4‐IgG positive ON within 2 weeks onset and with high‐dose corticosteroid treatment were included. Patients treated with sustained‐release fampridine (4‐aminopyridine, 4‐AP) as add‐on treatment for 3 months were compared with patients without 4‐AP (non‐4‐AP). Visual functional scores (VFS), Opticospinal Impairment Scale (OSIS), the logarithm of the minimum angle of resolution (LogMAR) of visual acuity (VA), the mean deviation (MD) value of the static visual field, and retina nerve fiber layer (RNFL) thickness were measured and followed up from baseline to 6 months.  Results: Patients with 4‐AP add‐on treatment improved VFS or OSIS greater than that with control (non‐4‐AP) at 3 and 6 months compared with baseline (all p < 0.05). 4‐AP add‐on treatment also increased the proportion of better improvement of ON (VFS ≤ 2) at 3 months (36.4% vs. 7.1% in control, p < 0.05 adjusted for plasma exchange), and 4‐AP improved visual field at 6 months (p < 0.05 adjusted for plasma exchange).  Conclusion: Fampridine as an add‐on treatment has potential functional benefit on refractory NMOSD‐ON and improves visual functions after the attack of ON.
SN  - 2328-9503
ER  -