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Record W3005022595 · doi:10.3233/jnd-190423

Improved Criteria for the Classification of Titin Variants in Inherited Skeletal Myopathies

2020· article· en· W3005022595 on OpenAlexaff
Marco Savarese, Mridul Johari, Katherine Johnson, Meharji Arumilli, Annalaura Torella, Ana Töpf, Anna Rubegni, Marius Kuhn, Teresa Giugliano, Dieter Gläser, Fabiana Fattori, Rachel Thompson, Sini Penttilä, Sara Lehtinen, Sara Gibertini, Alessandra Ruggieri, Marina Mora, Aleš Maver, Borut Peterlin, Ami Mankodi, Hanns Lochmüller, Filippo M. Santorelli, Benedikt Schoser, Lenka Fajkusová, Volker Straub, Vincenzo Nigro, Peter Hackman, Bjarne Udd

Bibliographic record

VenueJournal of Neuromuscular Diseases · 2020
Typearticle
Languageen
FieldMedicine
TopicCardiomyopathy and Myosin Studies
Canadian institutionsOttawa HospitalChildren's Hospital of Eastern OntarioUniversity of Ottawa
Fundersnot available
KeywordsTitinSkeletal muscleMedicineMedical geneticsGenomicsGenetic diagnosisComputational biologyBioinformaticsGeneticsBiologyGeneInternal medicineGenomeSarcomere

Abstract

fetched live from OpenAlex

BACKGROUND: Extensive genetic screening results in the identification of thousands of rare variants that are difficult to interpret. Because of its sheer size, rare variants in the titin gene (TTN) are detected frequently in any individual. Unambiguous interpretation of molecular findings is almost impossible in many patients with myopathies or cardiomyopathies. OBJECTIVE: To refine the current classification framework for TTN-associated skeletal muscle disorders and standardize the interpretation of TTN variants. METHODS: We used the guidelines issued by the American College of Medical Genetics and Genomics (ACMG) and the Association for Molecular Pathology (AMP) to re-analyze TTN genetic findings from our patient cohort. RESULTS: We identified in the classification guidelines three rules that are not applicable to titin-related skeletal muscle disorders; six rules that require disease-/gene-specific adjustments and four rules requiring quantitative thresholds for a proper use. In three cases, the rule strength need to be modified. CONCLUSIONS: We suggest adjustments are made to the guidelines. We provide frequency thresholds to facilitate filtering of candidate causative variants and guidance for the use and interpretation of functional data and co-segregation evidence. We expect that the variant classification framework for TTN-related skeletal muscle disorders will be further improved along with a better understanding of these diseases.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.055
metaresearch head score (Gemma)0.144
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: none
GenreCandidate signal: Methods · Consensus signal: Methods
Teacher disagreement score0.055
Threshold uncertainty score0.291

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0550.144
Meta-epidemiology (narrow)0.0020.001
Meta-epidemiology (broad)0.0020.003
Bibliometrics0.0120.004
Science and technology studies0.0020.002
Scholarly communication0.0040.002
Open science0.0060.003
Research integrity0.0040.004
Insufficient payload (model declined to judge)0.0020.002

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.046
GPT teacher head0.309
Teacher spread0.262 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designNot applicable
Domainnot available
GenreMethods

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations26
Published2020
Admission routes1
Has abstractyes

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Same venueJournal of Neuromuscular DiseasesSame topicCardiomyopathy and Myosin StudiesFrench-language works237,207