{
  "abstract": "Background and Importance Cerebellar ataxias are a heterogeneous group of neurological disorders characterised by impaired coordination and balance. Pharmacological options are limited. Fampridine, a potassium channel blocker approved for gait improvement in multiple sclerosis, is being used off-label in ataxia despite limited clinical evidence. Real-world data are needed to clarify its therapeutic value and safety profile.Aim and Objectives To evaluate the real-world effectiveness and safety of off-label fampridine in patients with cerebellar ataxia, describe their baseline characteristics, and identify potential predictors of treatment response.Material and Methods A retrospective observational study included 30 patients with different forms of cerebellar ataxia who initiated off-label fampridine. Inclusion criteria were estimated glomerular filtration rate (eGFR) >50 mL/min and prescription made between October 2021 and October 2025, following internal committee approval. Follow-up mean time was 14 ± 12 months. Effectiveness was assessed using the Timed 25-Foot Walk Test (T25-FWT) at baseline and after 2 weeks of treatment. Safety outcomes included treatment discontinuation rate, duration of therapy, and adverse drug reactions (ADRs).Abstract 4CPS-315 Table 1Baseline characteristics Characteristics N= 30 Age 64 +- 12,15 (SD) Sex (male) 19 (63%) Type of ataxia - Idiopathic 11 (36,7%) - CANVAS (RFC1 mutation) 5 (16,7%) - SCA27B (FGF14 expansion) 9 (30%) - Episodic type 2 1 (3,3%) - MSA-cerebellar 1 (3,3%) - SPG7-related paraparesis 3 (10%) Alcohol consumption 9 (30%) Nystagmus 20 (66,67%) Results Among 30 patients (mean age 64 ± 12 years; 63% male), 16 (53.3%) showed improvement in T25-FWT at 2-weeks, with a mean reduction of 2.4 seconds (range –0.7 to –9.0). At data cut-off, treatment was discontinued in 21 patients (70%), with a median (IQR) duration of 90 (30-271) days. ADRs occurred in 50% of patients, mainly insomnia, gastrointestinal discomfort and dizziness.Conclusion and Relevance Fampridine may provide functional benefits in cerebellar ataxia, but its real-world use is limited by high discontinuation rates and frequent ADRs. Close monitoring and pharmacy-led review of off-label prescriptions are essential to ensure safe and effective therapy. Further controlled studies are needed to confirm these findings.Conflict of Interest No conflict of interest",
  "authors": [
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "C Saiz Martinez"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Neurology, Donostia, Spain"
      ],
      "name": "G Nuñez Manjarres"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "A Latasa Berasategui"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "I Zipitria Sinde"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "I Sanchez Monasterio"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "N Ugartetxea Arakistain"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "M Salegi Ansa"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "L Simon Castelruiz"
    },
    {
      "affiliations": [
        "Hospital Universitario Donostia, Hospital Pharmacy, Donostia, Spain"
      ],
      "name": "L Murua Etxarri"
    }
  ],
  "title": "4CPS-315 Real-world evaluation of off-label fampridine use in cerebellar ataxia: safety and effectiveness in a tertiary hospital",
  "uid": "69fc6903-6346-5252-b548-fb1a754262f8"
}