ArticleJournal of inherited metabolic disease2018
Outcome measures for children with mitochondrial disease: consensus recommendations for future studies from a Delphi-based international workshop.
Article in Journal of inherited metabolic disease, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
What it found
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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.
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Who cites it
15 citing papers in PubMed, 32 citations in OpenAlex.
- Trial
- Perianesthetic Complications in Genetic Mitochondrial Disease: A Review of Case Reports.Paediatric anaesthesia · 2026Review
- Advances in Primary Mitochondrial Diseases: Diagnosis, Natural History Studies and Clinical Trials.Genes · 2026Review
- Diagnostic Criteria and Management of MELAS and Stroke-Like Episodes: Consensus-Based Statements.European journal of neurology · 2026Article
- Management of seizures in patients with primary mitochondrial diseases: consensus statement from the InterERNs Mitochondrial Working Group.European journal of neurology · 2024Article
- Use of genotypic HIV DNA testing: a DELPHI-type consensus.The Journal of antimicrobial chemotherapy · 2024Article
- Understanding the impact of pediatric single large-scale mtDNA deletion syndromes on caregivers: Burdens and challenges.JIMD reports · 2023Article
- Moderate Psoriasis in Clinical Practice: French Expert Consensus Using a Modified Delphi Method.Advances in therapy · 2022Article
- Natural History of Leigh Syndrome: A Study of Disease Burden and Progression.Annals of neurology · 2022Observational
- Development of a Mitochondrial Myopathy-Composite Assessment Tool.JCSM clinical reports · 2021Article
- Movement Disorders in Children with a Mitochondrial Disease: A Cross-Sectional Survey from the Nationwide Italian Collaborative Network of Mitochondrial Diseases.Journal of clinical medicine · 2021Article
- Moving towards clinical trials for mitochondrial diseases.Journal of inherited metabolic disease · 2021Review
- Primary Coenzyme Q10 Deficiency-7 and PathogenicFrontiers in genetics · 2021Review
- Genes, the brain, and artificial intelligence in evolution.Journal of human genetics · 2021Review
- Mitochondrial disorders and drugs: what every physician should know.Drugs in context · 2019Review
Corrections and comments
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Authors and funding
13 authors at 7 institutions in 5 countries.
Funding
Abstract
Although there are no effective disease-modifying therapies for mitochondrial diseases, an increasing number of trials are being conducted in this rare disease group. The use of sensitive and valid endpoints is essential to test the effectiveness of potential treatments. There is no consensus on which outcome measures to use in children with mitochondrial disease. The aims of this two-day Delphi-based workshop were to (i) define the protocol for an international, multi-centre natural history study in children with mitochondrial myopathy and (ii) to select appropriate outcome measures for a validation study in children with mitochondrial encephalopathy. We suggest two sets of outcome measures for a natural history study in children with mitochondrial myopathy and for a proposed validation study in children with mitochondrial encephalopathy.
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Registered trials
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.