Evidence map›Paper›PMID 34165911›Full record

ArticleAnnals of clinical and translational neurology2021

Nusinersen in pediatric and adult patients with type III spinal muscular atrophy.

Maria Carmela Pera, Giorgia Coratti, Francesca Bovis, Marika Pane, Amy Pasternak, Jacqueline Montes, Valeria A Sansone, Sally Dunaway Young, Tina Duong, Sonia Messina and 23 more

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

Article in Annals of clinical and translational neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 2 pooled it
4.6field-weighted citation impact, top 4% 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

30 citing papers in PubMed, 2 syntheses or guidelines pooled it, 49 citations in OpenAlex.

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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

33 authors at 14 institutions in 3 countries.

Maria Carmela PeraPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Giorgia CorattiPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.ORCID 0000-0001-6666-5628
Francesca BovisBiostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy.ORCID 0000-0002-3823-4000
Marika PanePediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Amy PasternakDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Jacqueline MontesDepartments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, New York, USA.ORCID 0000-0002-7357-0819
Valeria A SansoneNeurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan, Italy.
Sally Dunaway YoungDepartment of Neurology, Stanford University, Palo Alto, California, USA.
Tina DuongDepartment of Neurology, Stanford University, Palo Alto, California, USA.
Sonia MessinaDepartment of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy.
Irene MizzoniUnit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Adele D'AmicoUnit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Matthew CivitelloNemours Children's Hospital, University of Central Florida College of Medicine, Orlando, USA.
Allan M GlanzmanDepartment of Physical Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Claudio BrunoCenter of Experimental and Translational Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Francesca SalminNeurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan, Italy.
Simone MorandoCenter of Experimental and Translational Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Roberto De SanctisCentro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Maria SframeliDepartment of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy.
Laura AntonaciPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Anna Lia FrongiaPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
Annemarie RohwerDubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
Mariacristina ScotoDubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
Darryl C De VivoDepartments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, New York, USA.
Basil T DarrasDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
John DayDepartment of Neurology, Stanford University, Palo Alto, California, USA.
William MartensUniversity of Rochester Medical Center, Rochester, New York, USA.
Katia A PatanellaDepartment of Neurosciences, Institute of Neurology, Catholic University, Rome, Italy.
Enrico BertiniUnit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Francesco MuntoniDubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
Richard FinkelNemours Children's Hospital, University of Central Florida College of Medicine, Orlando, USA.
Eugenio MercuriPediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.ORCID 0000-0002-9851-5365
iSMAC group
Università Cattolica del Sacro Cuore · ITBambino Gesù Children's Hospital · ITGreat Ormond Street Hospital · GBPalo Alto University · USBoston Children's Hospital · USColumbia University Irving Medical Center · USIstituto Giannina Gaslini · ITUniversity of Central Florida · USUniversity of Messina · ITUniversity of Milan · ITAgostino Gemelli University Polyclinic · ITChildren's Hospital of Philadelphia · USUniversity of Genoa · ITUniversity of Rochester Medical Center · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveWe report longitudinal data from 144 type III SMA pediatric and adult patients treated with nusinersen as part of an international effort.

methodsPatients were assessed using Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6-Minute Walk Test (6MWT) with a mean follow-up of 1.83 years after nusinersen treatment.

resultsOver 75% of the 144 patients had a 12-month follow-up. There was an increase in the mean scores from baseline to 12 months on both HFMSE (1.18 points, p = 0.004) and RULM scores (0.58 points, p = 0.014) but not on the 6MWT (mean difference = 6.65 m, p = 0.33). When the 12-month HFMSE changes in the treated cohort were compared to an external cohort of untreated patients, in all untreated patients older than 7 years, the mean changes were always negative, while always positive in the treated ones. To reduce a selection bias, we also used a multivariable analysis. On the HFMSE scale, age, gender, baseline value, and functional status contributed significantly to the changes, while the number of SMN2 copies did not contribute. The effect of these variables was less obvious on the RULM and 6MWT.

interpretationOur results expand the available data on the effect of Nusinersen on type III patients, so far mostly limited to data from adult type III patients.

Indexed as

Outcome Assessment, Health CareRegistriesAdolescentAdultAgedChildChild, PreschoolFemaleHumansLongitudinal StudiesMaleMiddle AgedOligonucleotidesSeverity of Illness IndexSpinal Muscular Atrophies of ChildhoodYoung AdultnusinersenOligonucleotides

Identifiers

PMID34165911
PMCPMC8351459
OpenAlexW3176827893

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.