Evidence map›Paper›PMID 38860231›Full record

ArticleBMJ neurology open2024

Evidence for sodium valproate toxicity in mitochondrial diseases: a systematic analysis.

Thiloka E Ratnaike, Nour Elkhateeb, Angela Lochmüller, Christopher Gilmartin, Katherine Schon, Rita Horváth, Patrick F Chinnery

Abstract read
In one paragraph

Article in BMJ neurology open, 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. Drug safety in patients with mitochondrial disease: an observational cohort study.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026
    Observational
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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

7 authors.

Thiloka E RatnaikeDepartment of Paediatrics, University of Cambridge, Cambridge, UK.ORCID 0000-0001-5400-104X
Nour ElkhateebDepartment of Clinical Genetics, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.ORCID 0000-0002-3076-3178
Angela LochmüllerDepartment of Medical Genetics, Cambridge Biomedical Campus, Cambridge, UK.ORCID 0009-0001-3763-5881
Christopher GilmartinDepartment of Medical Genetics, Cambridge Biomedical Campus, Cambridge, UK.ORCID 0000-0001-5845-9429
Katherine SchonDepartment of Clinical Genetics, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.ORCID https://orcid.org/0000-0001-8054-8954
Rita HorváthCambridge Biomedical Campus Department of Clinical Neurosciences, University of Cambridge, Cambridge, Cambridgeshire, UK.ORCID https://orcid.org/0000-0002-9841-170X
Patrick F ChinneryCambridge Biomedical Campus Department of Clinical Neurosciences, University of Cambridge, Cambridge, Cambridgeshire, UK.ORCID 0000-0002-7065-6617

Funding

Medical Research Council MR/S035699/1
6 · The paper itself

Abstract

Background: We aimed to determine whether sodium valproate (VPA) should be contraindicated in all mitochondrial diseases, due to known VPA-induced severe hepatotoxicity in some mitochondrial diseases. Methods: We systematically reviewed the published literature for mitochondrial DNA (mtDNA) and common nuclear genotypes of mitochondrial diseases using PubMed, Ovid Embase, Ovid Medline and MitoPhen databases. We extracted patient-level data from peer-reviewed articles, published until July 2022, using the Human Phenotype Ontology to manually code clinical presentations for 156 patients with genetic diagnoses from 90 publications. Results: There were no fatal adverse drug reactions (ADRs) in the mtDNA disease group (35 patients), and only 1 out of 54 patients with a non- Conclusions: Our findings confirm the high risk of severe ADRs in any patient with recessive

Indexed as

EPILEPSYGENETICSMITOCHONDRIAL DISORDERSNEUROGENETICSNEUROPHARMACOLOGY

Identifiers

PMID38860231
PMCPMC11163645

What OpenQuestion holds

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Registered trials

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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.