Evidence map›Paper›PMID 35861914›Full record

SynthesisActa neurologica Belgica2023

Biomarkers of therapeutic efficacy in adolescents and adults with 5q spinal muscular atrophy: a systematic review.

Maria Gavriilaki, Vasileios Papaliagkas, Alexandra Stamperna, Maria Moschou, Konstantinos Notas, Sotirios Papagiannopoulos, Marianthi Arnaoutoglou, Vasilios K Kimiskidis

Abstract readSystematic Review
PubMed Publisher
In one paragraph

Synthesis in Acta neurologica Belgica, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
2.6field-weighted citation impact, top 9% 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

11 citing papers in PubMed, 17 citations in OpenAlex.

  1. Article
  2. Observational
  3. Biomarkers in spinal muscular atrophy.Frontiers in neurology · 2025
    Review
  4. Review
  5. Review
  6. Article
  7. Article
  8. Review
  9. Article
  10. Article
  11. 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.

Maria Gavriilaki1st Department of Neurology, School of Medicine, AHEPA University Hospital, Aristotle University of Thessaloniki, S. Kyriakidi Str. 1, 546 36, Thessaloniki, Greece. mariagavri6@yahoo.gr.ORCID http://orcid.org/0000-0002-1958-0063
Vasileios PapaliagkasDepartment of Biomedical Sciences, School of Health Sciences, International Hellenic University, Thessaloniki, Greece.
Alexandra Stamperna2nd Department of Pediatrics, AHEPA University Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Maria Moschou1st Department of Neurology, School of Medicine, AHEPA University Hospital, Aristotle University of Thessaloniki, S. Kyriakidi Str. 1, 546 36, Thessaloniki, Greece.
Konstantinos NotasLaboratory of Clinical Neurophysiology, School of Medicine, AHEPA University Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Sotirios Papagiannopoulos3rd Department of Neurology, School of Medicine, G. Papanicolaou Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Marianthi ArnaoutoglouLaboratory of Clinical Neurophysiology, School of Medicine, AHEPA University Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Vasilios K Kimiskidis1st Department of Neurology, School of Medicine, AHEPA University Hospital, Aristotle University of Thessaloniki, S. Kyriakidi Str. 1, 546 36, Thessaloniki, Greece.
Aristotle University of Thessaloniki · GRInternational Hellenic University · GR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThe therapeutic landscape of spinal muscular atrophy (SMA) was dramatically transformed with the introduction of three disease-modifying therapies (DMTs). A systematic review was performed to assess available evidence regarding quantitative therapeutic biomarkers used in SMA patients older than 11 years under treatment with DMTs.

methodsLatest literature search in MEDLINE, EMBASE, Cochrane databases and gray literature resources was performed in June 2021. Studies reporting only motor function or muscle strength scales or pulmonary function tests were excluded. Primary outcome was the change from baseline score of any serum, cerebrospinal fluid (CSF) or neurophysiologic biomarker examined.

resultsDatabase and gray literature search yielded a total of 8050 records. We identified 14 records published from 2019 until 2021 examining 18 putative serum, CSF or neurophysiologic biomarkers along with routine CSF parameters in 295 SMA nusinersen-treated type 2-4 patients older than 11 years of age. There is evidence based on real-world observational studies suggesting that serum creatinine, creatine kinase activity levels along with CSF Αβ42, glial fibrillary acidic protein concentration as well as ulnar compound motor action potential amplitude and single motor unit potential amplitude changes may depict therapeutic response in this population.

conclusionThis systematic review explored for the first-time biomarkers used to monitor therapeutic efficacy in SMA adolescents and adults treated with DMTs. Research in this area is in its early stages, and our systematic review can facilitate selection of quantitative therapeutic biomarkers that may be used as surrogate measures of treatment efficacy in future trials. PROTOCOL REGISTRATION: PROSPERO CRD42021245516.

Indexed as

Muscular Atrophy, SpinalSpinal Muscular Atrophies of ChildhoodAdolescentAdultBiomarkersHumansTreatment OutcomeBiomarkersAdult spinal muscular atrophyBiomarkersNervous system diseasesSystematic reviewTherapeutics

Identifiers

PMID35861914
OpenAlexW4286498762

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.