Evidence map›Paper›PMID 40922023›Full record

Observational studyOrphanet journal of rare diseases2025

Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study.

Claudia D Wurster, Benjamin Stolte, Tobias Kessler, Maren Freigang, Bogdan Bjelica, Benjamin Ilse, Jan C Koch, Isabell Cordts, Alexander Mensch, Daniel Zeller and 27 more

Abstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

37 authors.

Claudia D WursterInstitute of Human Genetics, Ulm University and Ulm University Medical Center, Albert-Einstein-Allee 11, 89081, Ulm, Germany. claudia.wurster@uni-ulm.de.
Benjamin StolteDepartment of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, 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.
Bogdan BjelicaDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Benjamin IlseDepartment of Neurology, Jena University Hospital, Jena, Germany.
Jan C KochDepartment of Neurology, University Medicine Göttingen, Göttingen, Germany.
Isabell CordtsDepartment of Neurology, Klinikum rechts der Isar, School of Medicine and Health, Technical University of Munich, Munich, Germany.
Alexander MenschDepartment of Neurology, University Medicine Halle, Halle (Saale), Germany.
Daniel ZellerDepartment of Neurology, University Hospital Würzburg, Würzburg, Germany.
Zeljko UzelacDepartment of Neurology, Ulm University Medical Center, Ulm, Germany.
Georges SamDepartment of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
Hanna Sophie LappDepartment of Neurology, University Hospital Carl Gustav Carus at Technische Universität Dresden, Dresden, Germany.
Camilla WohnradeDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Annekathrin RödigerDepartment of Neurology, Jena University Hospital, Jena, Germany.
Mohamad Tareq MuhandesDepartment of Neurology, University Medicine Göttingen, Göttingen, Germany.
Ilka SchneiderDepartment of Neurology, University Medicine Halle, Halle (Saale), Germany.
Julia BellutDepartment of Neurology, University Hospital Würzburg, Würzburg, Germany.
Julia NentwichDepartment of Neurology, University Hospital Würzburg, Würzburg, Germany.
Johannes DorstDepartment of Neurology, Ulm University Medical Center, Ulm, Germany.
Joachim SchusterDepartment of Neurology, Ulm University Medical Center, Ulm, Germany.
Olivia Schreiber-KatzDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Alma OsmanovicDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Andreas TotzeckDepartment of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, Germany.
Andreas ThimmDepartment of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, Germany.
Robert SteinbachDepartment of Neurology, Jena University Hospital, Jena, Germany.
Julian GrosskreutzDepartment of Neurology, Jena University Hospital, Jena, Germany.
Christoph KleinschnitzDepartment of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, Germany.
Albert C LudolphDepartment of Neurology, Ulm University Medical Center, Ulm, Germany.
Marcus DeschauerDepartment of Neurology, Klinikum rechts der Isar, School of Medicine and Health, Technical University of Munich, Munich, Germany.
Janbernd KirschnerDepartment of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center - University of Freiburg, University of Freiburg, Freiburg, Germany.
Jens DreyhauptInstitute of Epidemiology and Medical Biometry, Ulm University, Ulm, Germany.
Kurt WollinskyDepartment of Anesthesiology, RKU, Ulm University, Ulm, Germany.
Susanne PetriDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Markus WeilerDepartment of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
Tim HagenackerDepartment of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, Germany.
René GüntherDepartment of Neurology, University Hospital Carl Gustav Carus at Technische Universität Dresden, Dresden, Germany.

Funding

Clinician Scientist Program of Ulm University
6 · The paper itself

Abstract

backgroundNatural history data show that respiratory function is impaired in SMA patients. Observational studies have shown stabilization of respiratory function in adult SMA patients treated with nusinersen. However, long-term studies investigating the effect of nusinersen on respiratory function in adult SMA patients are rare.

methodsWe examined respiratory function using forced vital capacity of predicted normal (FVC%), FVC in liters, capacity per second (FEV1) and peak expiratory flow (PEF) in 192 adult SMA patients treated with nusinersen for a median of 3.2 years (IQR: 2.1–4.0, range: 0.2–5.2). Changes in spirometric parameters were analyzed using individual linear regression models separate in each patient to estimate the slope. Additionally, three multivariable models were performed to assess the effect of age, sex, treatment duration, baseline FVC% and each one of the variables of interest (1) SMA type, (2) ambulation status, (3) spondylodesis on follow-up FVC%. Associations between respiratory parameters and motor function (HFMSE) were investigated via Scatter plots and Spearman’s rank correlation.

resultsSpirometric parameters remained stable during treatment (median annual rate of change of FVC% 0.17% (p = 0.40), FVC in liters  -0.002 (p = 0.59), FEV1 -0.014 l (p = 0.06) and PEF 0.025 l/s (p = 0.65)). In all multivariable models, age, sex, treatment duration, SMA type, ambulation status, and spondylodesis showed no significant association with follow-up FVC%. No significant correlations were observed between respiratory and motor function.

conclusionRespiratory parameters remained stable during treatment with nusinersen in adult SMA patients over several years. CLINICAL TRIAL NUMBER: Not applicable.

Indexed as

Muscular Atrophy, SpinalOligonucleotidesAdultFemaleHumansMaleMiddle AgedRespiratory Function TestsVital CapacityYoung AdultnusinersenOligonucleotidesAdult patientsForced vital capacity (FVC)NusinersenRespiratory functionSpinal muscular atrophy (SMA)

Identifiers

PMID40922023
PMCPMC12418672

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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.