Evidence map›Paper›PMID 38361750›Full record

ArticleThe Lancet regional health. Europe2024

Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study.

René Günther, Claudia Diana Wurster, Svenja Brakemeier, Alma Osmanovic, Olivia Schreiber-Katz, Susanne Petri, Zeljko Uzelac, Miriam Hiebeler, Simone Thiele, Maggie C Walter and 54 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in The Lancet regional health. Europe, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
42citing papers in PubMed, 3 pooled it
24.5field-weighted citation impact, top 1% 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

42 citing papers in PubMed, 3 syntheses or guidelines pooled it, 56 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Trial
  5. Review
  6. Review
  7. Observational
  8. Review
  9. Article
  10. Review
  11. Article
  12. Observational
  13. Article
  14. Article
  15. Article
  16. Article
  17. Observational
  18. Cerebrospinal fluid metabolomics reveals predictive biomarkers of nusinersen therapy efficacy in type II and type III spinal muscular atrophy patients.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
    Article
  19. Observational
  20. 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

64 authors at 20 institutions in 4 countries.

René GüntherDepartment of Neurology, University Hospital Carl Gustav Carus at Technische Universität Dresden, Dresden, Germany.
Claudia Diana WursterDepartment of Neurology, Ulm University, Ulm, Germany.
Svenja BrakemeierDepartment of Neurology, and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Medicine Essen, Essen, Germany.
Alma OsmanovicDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Olivia Schreiber-KatzDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Susanne PetriDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Zeljko UzelacDepartment of Neurology, Ulm University, Ulm, Germany.
Miriam HiebelerFriedrich Baur Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.
Simone ThieleFriedrich Baur Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.
Maggie C WalterFriedrich Baur Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.
Markus WeilerDepartment of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
Tobias KesslerDepartment of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
Maren FreigangDepartment of Neurology, University Hospital Carl Gustav Carus at Technische Universität Dresden, Dresden, Germany.
Hanna Sophie LappDepartment of Neurology, University Hospital Carl Gustav Carus at Technische Universität Dresden, Dresden, Germany.
Isabell CordtsDepartment of Neurology, Technical University of Munich, School of Medicine, Munich, Germany.
Paul LingorDepartment of Neurology, Technical University of Munich, School of Medicine, Munich, Germany.
Marcus DeschauerDepartment of Neurology, Technical University of Munich, School of Medicine, Munich, Germany.
Andreas HahnDepartment of Child Neurology, Justus-Liebig University Gießen, Gießen, Germany.
Kyriakos MartakisDepartment of Child Neurology, Justus-Liebig University Gießen, Gießen, Germany.
Robert SteinbachDepartment of Neurology, Jena University Hospital, Jena, Germany.
Benjamin IlseDepartment of Neurology, Jena University Hospital, Jena, Germany.
Annekathrin RödigerDepartment of Neurology, Jena University Hospital, Jena, Germany.
Julia BellutDepartment of Neurology, University Hospital Würzburg, Würzburg, Germany.
Julia NentwichDepartment of Neurology, University Hospital Würzburg, Würzburg, Germany.
Daniel ZellerDepartment of Neurology, University Hospital Würzburg, Würzburg, Germany.
Mohamad Tareq MuhandesDepartment of Neurology, University Medicine Göttingen, Göttingen, Germany.
Tobias BaumDepartment of Neurology, University Medicine Göttingen, Göttingen, Germany.
Jan Christoph KochDepartment of Neurology, University Medicine Göttingen, Göttingen, Germany.
Bertold SchrankDepartment of Neurology, Deutsche Klinik für Diagnostik HELIOS Clinic of Wiesbaden, Wiesbaden, Germany.
Sophie FischerTranslational Neurodegeneration Section "Albrecht Kossel", Department of Neurology, University Medical Center Rostock, 18147, Rostock, Germany.
Andreas HermannTranslational Neurodegeneration Section "Albrecht Kossel", Department of Neurology, University Medical Center Rostock, 18147, Rostock, Germany.
Christoph KammDepartment of Neurology, University of Rostock, Rostock, Germany.
Steffen NaegelDepartment of Neurology, University Medicine Halle, Halle (Saale), Germany.
Alexander MenschDepartment of Neurology, University Medicine Halle, Halle (Saale), Germany.
Markus WeberNeuromuscular Diseases Unit/ALS Clinic, Cantonal Hospital St. Gallen, St. Gallen, Switzerland.
Christoph NeuwirthNeuromuscular Diseases Unit/ALS Clinic, Cantonal Hospital St. Gallen, St. Gallen, Switzerland.
Helmar C LehmannDepartment of Neurology and Center for Rare Diseases, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany.
Gilbert WunderlichDepartment of Neurology and Center for Rare Diseases, Faculty of Medicine and University Hospital Cologne, University of Cologne, Cologne, Germany.
Christian StadlerDepartment of Neurology, Klinikum Klagenfurt am Wörthersee, Klagenfurt am Wörthersee, Austria.
Maike TomfordeDepartment of Neurology, University Hospital Kiel, Kiel, Germany.
Annette GeorgeDepartment of Pediatric Neurology, Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Martin GroßFaculty of Medicine and Health Sciences, Carl von Ossietzky University Oldenburg, Oldenburg, Germany.
Astrid PechmannDepartment of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Janbernd KirschnerDepartment of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Matthias TürkDepartment of Neurology, University Hospital Erlangen, Friedrich-Alexander University Erlangen-Nuremberg (FAU), Germany.
Mareike SchimmelPediatrics and Adolescent Medicine, Faculty of Medicine University Hospital Augsburg, Augsburg, Germany.
Günther BernertDepartment of Pediatrics and Pediatric Neurology, Clinic Favoriten, Vienna, Austria.
Pascal MartinDepartment of Neurology and Epileptology, Hertie Institute for Clinical Brain Research, University Hospitals Tubingen, Tubingen, Germany.
Christian RauscherDepartment of Pediatrics, Paracelsus Medical University, Salzburg, Austria.
Gerd Meyer Zu HörsteDepartment of Neurology, University Hospital Münster, Münster, Germany.
Petra BaumDepartment of Neurology, University of Leipzig Medical Centre, Leipzig, Germany.
Wolfgang LöscherDivision of Neurology, Medical University Innsbruck, Innsbruck, Austria.
Marina Flotats-BastardasDepartment of Pediatric Neurology, Saarland University Hosptial, Homburg, Germany.
Cornelia KöhlerDepartment of Neuropaediatrics, University Children's Hospital, Ruhr-University Bochum, Bochum, Germany.
Kristina Probst-SchendzielorzInitiative SMA der Deutschen Gesellschaft für Muskelkranke, Freiburg, Germany.
Susanne GoldbachInitiative SMA der Deutschen Gesellschaft für Muskelkranke, Freiburg, Germany.
Ulrike Schara-SchmidtDepartment of Paediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, Center for Translational Neuro- and Behavioral Sciences, University Hospital, University of Duisburg-Essen, Essen, Germany.
Wolfgang Müller-FelberDepartment of Neuropediatrics, Dr. v. Haunersche Kinderklinik, University Children's Hospital, Ludwig-Maximilians-Universität München, München, Germany.
Hanns LochmüllerDepartment of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Otgonzul von VelsenInstitute of Medical Informatics, Biometrics, and Epidemiology, University Hospital Essen, Essen, Germany.
SMArtCARE Study Group
Christoph KleinschnitzDepartment of Neurology, and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Medicine Essen, Essen, Germany.
Albert C LudolphDepartment of Neurology, Ulm University, Ulm, Germany.
Tim HagenackerDepartment of Neurology, and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Medicine Essen, Essen, Germany.
German Cancer Research Center · DEEssen University Hospital · DEMedizinische Hochschule Hannover · DEFriedrich Baur Stiftung · DEGerman Center for Neurodegenerative Diseases · DEJena University Hospital · DETechnical University of Munich · DEUniversität Ulm · DEUniversity Hospital Carl Gustav Carus · DEHeidelberg University · DEInnsbruck Medical University · ATJustus-Liebig-Universität Gießen · DEUniversity of Cologne · DEUniversity of Freiburg · DECarl von Ossietzky Universität Oldenburg · DEChristian-Albrechts-Universität zu Kiel · DEDeutsche Gesellschaft für Muskelkranke · DEFriedrich-Alexander-Universität Erlangen-Nürnberg · DEKantonsspital St. Gallen · CHLudwig-Maximilians-Universität München · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Evidence for the efficacy of nusinersen in adults with 5q-associated spinal muscular atrophy (SMA) has been demonstrated up to a period of 16 months in relatively large cohorts but whereas patients reach a plateau over time is still to be demonstrated. We investigated the efficacy and safety of nusinersen in adults with SMA over 38 months, the longest time period to date in a large cohort of patients from multiple clinical sites. Methods: Our prospective, observational study included adult patients with SMA from Germany, Switzerland, and Austria (July 2017 to May 2022). All participants had genetically-confirmed, 5q-associated SMA and were treated with nusinersen according to the label. The total Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM) scores, and 6-min walk test (6 MWT; metres), were recorded at baseline and 14, 26, and 38 months after treatment initiation, and pre and post values were compared. Adverse events were also recorded. Findings: Overall, 389 patients were screened for eligibility and 237 were included. There were significant increases in all outcome measures compared with baseline, including mean HFMSE scores at 14 months (mean difference 1.72 [95% CI 1.19-2.25]), 26 months (1.20 [95% CI 0.48-1.91]), and 38 months (1.52 [95% CI 0.74-2.30]); mean RULM scores at 14 months (mean difference 0.75 [95% CI 0.43-1.07]), 26 months (mean difference 0.65 [95% CI 0.27-1.03]), and 38 months (mean difference 0.72 [95% CI 0.25-1.18]), and 6 MWT at 14 months (mean difference 30.86 m [95% CI 18.34-43.38]), 26 months (mean difference 29.26 m [95% CI 14.87-43.65]), and 38 months (mean difference 32.20 m [95% CI 10.32-54.09]). No new safety signals were identified. Interpretation: Our prospective, observational, long-term (38 months) data provides further real-world evidence for the continuous efficacy and safety of nusinersen in a large proportion of adult patients with SMA. Funding: Financial support for the registry from Biogen, Novartis and Roche.

Indexed as

Antisense oligonucleotideIntrathecal therapyMotor neuron diseaseNusinersenSpinal muscular atrophy

Identifiers

PMID38361750
PMCPMC10864329
OpenAlexW4391589874

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.