Evidence map›Paper›PMID 39198371›Full record

ReviewPaediatric drugs2024

Targeted Treatments for Myasthenia Gravis in Children and Adolescents.

Sithara Ramdas, Teresa Painho, Maria I Vanegas, Dennis T Famili, Ming J Lim, Heinz Jungbluth

Abstract readReview
PubMed Publisher
In one paragraph

Review in Paediatric drugs, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
–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

8 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
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

6 authors.

Sithara RamdasDepartment of Paediatrics, MDUK Neuromuscular Centre, University of Oxford, Oxford, UK.
Teresa PainhoDepartment of Paediatrics, MDUK Neuromuscular Centre, University of Oxford, Oxford, UK.
Maria I VanegasDepartment of Paediatric Neurology, Evelina London Children's Hospital, Guy's & St. Thomas' Hospital NHS Foundation Trust, Children's Neurosciences Centre, F02-Becket House, Lambeth Palace Road, London, SE1 7EU, UK.
Dennis T FamiliDepartment of Paediatric Neurology, Evelina London Children's Hospital, Guy's & St. Thomas' Hospital NHS Foundation Trust, Children's Neurosciences Centre, F02-Becket House, Lambeth Palace Road, London, SE1 7EU, UK.
Ming J LimDepartment of Paediatric Neurology, Evelina London Children's Hospital, Guy's & St. Thomas' Hospital NHS Foundation Trust, Children's Neurosciences Centre, F02-Becket House, Lambeth Palace Road, London, SE1 7EU, UK.
Heinz JungbluthDepartment of Paediatric Neurology, Evelina London Children's Hospital, Guy's & St. Thomas' Hospital NHS Foundation Trust, Children's Neurosciences Centre, F02-Becket House, Lambeth Palace Road, London, SE1 7EU, UK. Heinz.Jungbluth@gstt.nhs.uk.ORCID http://orcid.org/0000-0002-7159-3427

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Myasthenia gravis (MG) is an antibody-mediated disorder of the neuromuscular junction affecting children and adults. MG is a treatable condition with most patients requiring immunosuppression for disease control and/or remission. Juvenile myasthenia gravis (JMG) is rare in comparison with adult-onset MG but given the same underlying pathophysiology, treatment strategies are similar to those in adults. Until recently, there were only a few randomised controlled trials (RCTs) for MG treatments in adults and none in children, and management strategies were primarily based on expert consensus. In addition, treatment options for refractory MG cases have been severely limited, resulting in poor long-term quality of life in such patients due to the significant disease burden. Recently, there have been several RCTs focussing on novel therapeutic strategies with potentially promising outcomes, suggesting a change in MG management over the coming years and access to more effective and faster-acting drugs for MG patients. This paper will review current and new MG treatments including efgartigimod, eculizumab, rozanolixizumab, ravulizumab, and zilucoplan, with a focus on juvenile myasthenia gravis.

Indexed as

Myasthenia GravisAdolescentChildHumansQuality of Life

Identifiers

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.