Evidence map›Paper›PMID 34484942›Full record

ArticleNeurology. Clinical practice2021

Fampridine and Acetazolamide in EA2 and Related Familial EA: A Prospective Randomized Placebo-Controlled Trial.

Carolin Muth, Julian Teufel, Ludger Schöls, Matthis Synofzik, Christiana Franke, Dagmar Timmann, Ulrich Mansmann, Michael Strupp

Open access · greenAbstract read
In one paragraph

Article in Neurology. Clinical practice, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed, 1 pooled it
2.8field-weighted citation impact, top 8% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

15 citing papers in PubMed, 1 synthesis or guideline pooled it, 49 citations in OpenAlex.

  1. Episodic Ataxias: Primary and Secondary Etiologies, Treatment, and Classification Approaches.Tremor and other hyperkinetic movements (New York, N.Y.) · 2023
    Pooled it
  2. Drug repurposing in hereditary ataxia.npj drug discovery · 2026
    Review
  3. Review
  4. Article
  5. Treatment of primary adult-onset neurodegenerative cerebellar ataxias.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026
    Review
  6. Article
  7. Article
  8. Article
  9. Article
  10. Review
  11. Article
  12. Review
  13. Review
  14. Article
  15. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 2 institutions in 1 country.

Carolin MuthDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Julian TeufelDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Ludger SchölsDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Matthis SynofzikDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Christiana FrankeDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Dagmar TimmannDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Ulrich MansmannDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Michael StruppDepartment of Neurology and German Center for Vertigo and Balance Disorders (DSGZ) (CM, JT, M. Strupp), Ludwig Maximilians University, Munich, LMU University Hospital, Campus Grosshadern; Department of Neurology and Hertie-Institute for Clinical Brain Research (LS, M. Synofzik), Eberhard Karls University and German Center for Neurodegenerative Diseases (DZNE), Tübingen; Department of Neurology (CF), Charité-Universitätsmedizin Berlin, Berlin, Germany, Formerly Department of Neurology, University of Dresden; Department of Neurology (DT), Essen University Hospital, University of Duisburg-Essen; and Department of Medical Information Sciences (UM), Biometry, and Epidemiology (IBE), Ludwig Maximilian University, Munich, Germany.
Essen University Hospital · DEUniversity of Duisburg-Essen · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo determine the efficacy and safety of the treatment with prolonged-release 4-aminopyridine (fampridine) and acetazolamide for patients with episodic ataxia type 2 (EA2), patients with EA2 were treated with a random sequence of fampridine, acetazolamide, and placebo in a 3-period crossover trial.

methodsA total of 30 patients with EA2 (8 female; aged 20-71 years; 18 genetically confirmed, 4 with a positive family history, 8 with the clinical diagnosis) were enrolled in this phase III, randomized, double-blind, placebo-controlled, 3-period crossover trial. Each period lasted 12 weeks with a 4-week washout period. Each patient received a random sequence of 20 mg/d fampridine, 750 mg/d acetazolamide, and placebo. The primary end point was the number of attacks during the last 30 days within the 12-week treatment period. Participants, caregivers, and those assessing the outcomes were blinded to the intervention.

resultsCompared with placebo, fampridine reduced the number of attacks to 63% (95% CI 54%-74%) and acetazolamide to 52% (95% CI 46%-60%). A total of 39 (26.5%) adverse events were observed under treatment with fampridine (mostly tingling paresthesia and fatigue), 66 (44.9%) happened under acetazolamide (mostly taste disturbance and gastrointestinal complaints), and 42 (28.6%) under placebo (mostly gastrointestinal complaints).

conclusionBoth fampridine and acetazolamide significantly reduce the number of attacks in patients with EA2 and related EA in comparison to placebo. Fampridine 10 mg twice daily had fewer side effects than acetazolamide 250 mg 3 times daily. The trial was registered with DRKS.de (DRKS00005258) and EudraCT (2013-000107-17). This study was supported by the Federal Ministry of Education and Research (BMBF) (grant number 01EO0901). Fampridine (study medication) was provided by Biogen Idec. CLASSIFICATION OF EVIDENCE: Class II evidence.

Identifiers

PMID34484942
PMCPMC8382428
OpenAlexW3106641367

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.