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Record W2944460071 · doi:10.1016/j.jalz.2019.01.012

Assessment of executive function declines in presymptomatic and mildly symptomatic familial frontotemporal dementia: NIH‐EXAMINER as a potential clinical trial endpoint

2019· article· en· W2944460071 on OpenAlexafffund
Adam M. Staffaroni, Lynn Bajorek, Kaitlin B. Casaletto, Yann Cobigo, Sheng‐Yang M. Goh, Amy Wolf, Hilary W. Heuer, Fanny M. Elahi, Peter A. Ljubenkov, Reilly Dever, John Kornak, Brian S. Appleby, Jessica Bove, Yvette Bordelon, Patrick Brannelly, Danielle Brushaber, Christina Caso, Giovanni Coppola, Christina Dheel, Bradford C. Dickerson, Susan Dickinson, S. Carbajal Domínguez, Kimiko Domoto‐Reilly, Kelly Faber, Jessica Ferrall, Julie A. Fields, Ann Fishman, Jamie Fong, Tatiana Foroud, Leah K. Forsberg, Ralitza H. Gavrilova, Debra Gearhart, Behnaz Ghazanfari, Nupur Ghoshal, Jill Goldman, Jonathan Graff‐Radford, Neill R. Graff‐Radford, Ian Grant, Murray Grossman, Dana Haley, Ging‐Yuek Robin Hsiung, Edward D. Huey, David J. Irwin, David T. Jones, Lynne C. Jones, Kejal Kantarci, Anna Karydas, Daniel Kaufer, Diana Kerwin, David S. Knopman, Ruth Kraft, Walter K. Kremers, Walter A. Kukull, Irene Litvan, Diane Lucente, Codrin Lungu, Ian R. Mackenzie, Miranda Maldonado, Masood Manoochehri, Scott M. McGinnis, Emily McKinley, Mario F. Mendez, Bruce L. Miller, Namita Multani, Chiadi U. Onyike, Jaya Padmanabhan, Alexander Pantelyat, Rodney Pearlman, Len Petrucelli, Madeline Potter, Rosa Rademakers, Eliana Marisa Ramos, Katherine P. Rankin, Katya Rascovsky, Erik D. Roberson, Emily Rogalskı, Pheth Sengdy, Leslie M. Shaw, Jeremy A. Syrjanen, Maria Carmela Tartaglia, Nadine Tatton, Joanne Taylor, Arthur W. Toga, John Q. Trojanowski, Sandra Weıntraub, Ping Wang, Bonnie Wong, Zbigniew K. Wszołek, Adam L. Boxer, Bradley F. Boeve, Joel H. Kramer, Howard J. Rosen

Bibliographic record

VenueAlzheimer s & Dementia · 2019
Typearticle
Languageen
FieldMedicine
TopicAmyotrophic Lateral Sclerosis Research
Canadian institutionsUniversity of British ColumbiaOccupational Cancer Research CentreUniversity of Toronto
FundersNational Institute of Neurological Disorders and StrokeNational Institute of Mental HealthNational Institute on AgingJanssen PharmaceuticalsAlzheimer Society of B.C.Canadian Institutes of Health ResearchAvid RadiopharmaceuticalsUniversity of California, San DiegoGE HealthcareEisaiParkinsonfondenUniversity of California, San FranciscoAlzheimer SocietyTauRx PharmaceuticalsPuma BiotechnologyAbbVieTeva Pharmaceutical IndustriesPfizerBiogenUniversity of PennsylvaniaNew York State Department of HealthWyethUniversity of Southern CaliforniaRocheCenters for Disease Control and PreventionNovartisNational Institutes of HealthAssociation for Frontotemporal DegenerationMerckAlzheimer's Drug Discovery FoundationU.S. Department of DefenseEli Lilly and CompanyBrightFocus FoundationAllerganAstraZenecaBristol-Myers SquibbAlzheimer's AssociationHuntington's Disease Society of AmericaGenentechLarry L. Hillblom FoundationMichael J. Fox Foundation for Parkinson's Research
KeywordsFrontotemporal dementiaFrontotemporal lobar degenerationC9orf72Clinical Dementia RatingDementiaRating scaleNeuropsychologyMedicinePsychologyClinical trialExecutive dysfunctionCognitionDiseasePsychiatryOncologyInternal medicineDevelopmental psychology

Abstract

fetched live from OpenAlex

INTRODUCTION: Identifying clinical measures that track disease in the earliest stages of frontotemporal lobar degeneration (FTLD) is important for clinical trials. Familial FTLD provides a unique paradigm to study early FTLD. Executive dysfunction is a clinically relevant hallmark of FTLD and may be a marker of disease progression. METHODS: Ninety-three mutation carriers with no symptoms or minimal/questionable symptoms (MAPT, n = 31; GRN, n = 28; C9orf72, n = 34; Clinical Dementia Rating scale plus NACC FTLD Module < 1) and 78 noncarriers enrolled through Advancing Research and Treatment in Frontotemporal Lobar Degeneration/Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects studies completed the Executive Abilities: Measures and Instruments for Neurobehavioral Evaluation and Research (NIH-EXAMINER) and the UDS neuropsychological battery. Linear mixed-effects models were used to identify group differences in cognition at baseline and longitudinally. We examined associations between cognition, clinical functioning, and magnetic resonance imaging volumes. RESULTS: NIH-EXAMINER scores detected baseline and differences in slopes between carriers and noncarriers, even in carriers with a baseline Clinical Dementia Rating scale plus NACC FTLD Module = 0. NIH-EXAMINER declines were associated with worsening clinical symptoms and brain volume loss. DISCUSSION: The NIH-EXAMINER is sensitive to cognitive changes in presymptomatic familial FTLD and is a promising surrogate endpoint.

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)0.013
metaresearch head score (Gemma)0.007
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.013
Threshold uncertainty score0.071

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0130.007
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0010.001
Open science0.0000.000
Research integrity0.0010.001
Insufficient payload (model declined to judge)0.0020.000

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 head0.044
GPT teacher head0.361
Teacher spread0.317 · 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 designObservational
Domainnot available
GenreEmpirical

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

Citations51
Published2019
Admission routes2
Has abstractyes

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