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Record W4225128244 · doi:10.1212/wnl.0000000000200384

Longitudinal Cognitive Changes in Genetic Frontotemporal Dementia Within the GENFI Cohort

2022· article· en· W4225128244 on OpenAlexfundno aff
Jackie M. Poos, Amy MacDougall, Esther van den Berg, Lize C. Jiskoot, Janne M. Papma, Emma L. van der Ende, Harro Seelaar, Lucy L. Russell, Georgia Peakman, Rhian S. Convery, Yolande A.L. Pijnenburg, Fermín Moreno, Raquel Sánchez‐Valle, Barbara Borroni, Robert Laforce, Marie-Claire Doré, Mario Masellis, Maria Carmela Tartaglia, Caroline Graff, Daniela Galimberti, James B. Rowe, Elizabeth Finger, Matthis Synofzik, Rik Vandenberghe, Alexandre de Mendonça, Pietro Tiraboschi, Isabel Santana, Simon Ducharme, Christopher Butler, Alexander Gerhard, Johannes Levin, Adrian Danek, Markus Otto, Isabelle Le Ber, Florence Pasquier, John C. van Swieten, Jonathan D. Rohrer, Martin N. Rossor, Nick C. Fox, Jason D. Warren, Ione Woollacott, Rachelle Shafei, Carolin Heller, Imogen J. Swift, Katrina Moore, Aitana Sogorb‐Esteve, Annabel Nelson, Arabella Bouzigues, Caroline Greaves, David L. Cash, Hanya Benotmane, Emily Todd, Henrik Zetterberg, Kiran Samra, Martina Bocchetta, Rita Guerreiro, José Brás, David L. Thomas, Jennifer Nicholas, Simon Mead, Lieke Meeter, Jessica Panman, Lucia Giannini, Rick van Minkelen, Myriam Barandiarán, Begoña Indakoetxea, Alazne Gabilondo, Mikel Tainta, María de Arriba, Miren Zulaica, Jorge Villanúa, Zigor Díaz, Marta Cañada, Patricia Alves, Sergi Borrego‐Écija, Jaume Olives, Albert Lladó, Mircea Balasa, Anna Antonell, Núria Bargalló, Enrico Premi, Maura Cosseddu, Stefano Gazzina, Alessandro Padovani, Alberto Benussi, Valentina Cantoni, Roberto Gasparotti, Silvana Archetti, Sandra E. Black, Sara Mitchell, Ekaterina Rogaeva, Morris Freedman, Ron Keren, David F. Tang‐Wai, Linn Öijerstedt, Christin Andersson, Vesna Jelić, Håkan Thonberg, Andrea Arighi, Chiara Fenoglio, Elio Scarpini, Giorgio Fumagalli, Thomas Cope, Carolyn Timberlake, Timothy Rittman, Christen Shoesmith, Rosa Rademakers, Carlo Wilke, Lisa Graf, H.‐O. Karnath, Benjamin Bender, Rose Bruffaerts, Philip Van Damme, Mathieu Vandenbulcke, Annick Vogels, Koen Poesen, Catarina B. Ferreira, Gabriel Miltenberger, Frederico Simões do Couto, Carolina Maruta, Ana Verdelho, Sònia Afonso, Ricardo Taipa, Paola Caroppo, Giuseppe Di Fede, Giorgio Giaccone, Sara Prioni, Veronica Redaelli, Giacomina Rossi, Diana Duro, Maria Rosário Almeida, Miguel Castelo‐Branco, Maria João Leitão, Miguel Tábuas‐Pereira, Beatriz Santiago, Serge Gauthier, Pedro Rosa‐Neto, Michele Veldsman, Paul Thompson, Tobias Langheinrich, Catharina Prix, Tobias Hoegen, Elisabeth Wlasich, Sandra Loosli, Sonja Schönecker, Elisa Semler, Sarah Anderl‐Straub, Jolina Lombardi, Benedetta Nacmias, Camilla Ferrari, Cristina Polito, Gemma Lombardi, Valentina Bessi, Agnès Camuzat, Alexis Brice, Anne Bertrand, Aurélie Funkiewiez, Daisy Rinaldi, Dario Saracino, Olivier Colliot, Sabrina Sayah, Adeline Rollin, Grégory Kuchcinski, Maxime Bertoux, Thibaud Lebouvier, Vincent Deramecourt

Bibliographic record

VenueNeurology · 2022
Typearticle
Languageen
FieldMedicine
TopicAmyotrophic Lateral Sclerosis Research
Canadian institutionsnot available
FundersInstituto de Salud Carlos IIIMedical Research CouncilFonds de Recherche du Québec - SantéNational Institutes of HealthJanssen PharmaceuticalsDementias Platform UKStichting DioraphteBayer VitalEvelyn TrustNovo NordiskKU LeuvenVetenskapsrådetEisaiMinistero della SaluteCanadian Institutes of Health ResearchFonds Wetenschappelijk OnderzoekFundação para a Ciência e a TecnologiaKarolinska InstitutetAlzheimer's Drug Discovery FoundationWeston Brain InstituteZonMwBundesministerium für Bildung und ForschungNational Institute for Health and Care ResearchFondation Brain CanadaDenali TherapeuticsEU Joint Programme – Neurodegenerative Disease ResearchAdvocacy for Neuroacanthocytosis PatientsAlzheimer's AssociationAstex PharmaceuticalsSunovionAlzheimer NederlandBiogenAstraZenecaBrain Research UKEli Lilly and CompanyNederlandse Organisatie voor Wetenschappelijk OnderzoekWellcome TrustDeutsche ForschungsgemeinschaftIonis Pharmaceuticals
KeywordsC9orf72Frontotemporal dementiaDementiaCohortCognitive declineNeuropsychologyClinical Dementia RatingExecutive dysfunctionDiseaseFrontotemporal lobar degenerationOncologyPsychologyExecutive functionsMedicineCognitionInternal medicinePsychiatry

Abstract

fetched live from OpenAlex

<h3>Background and Objectives</h3> Disease-modifying therapeutic trials for genetic frontotemporal dementia (FTD) are underway, but sensitive cognitive outcome measures are lacking. The aim of this study was to identify such cognitive tests in early stage FTD by investigating cognitive decline in a large cohort of genetic FTD pathogenic variant carriers and by investigating whether gene-specific differences are moderated by disease stage (asymptomatic, prodromal, and symptomatic). <h3>Methods</h3> <i>C9orf72</i>, <i>GRN</i>, and <i>MAPT</i> pathogenic variant carriers as well as controls underwent a yearly neuropsychological assessment covering 8 cognitive domains as part of the Genetic FTD Initiative, a prospective multicenter cohort study. Pathogenic variant carriers were stratified according to disease stage using the global Clinical Dementia Rating (CDR) plus National Alzheimer9s Coordinating Center (NACC) FTLD score (0, 0.5, or ≥1). Linear mixed-effects models were used to investigate differences between genetic groups and disease stages as well as the 3-way interaction between time, genetic group, and disease stage. <h3>Results</h3> A total of 207 <i>C9orf72</i>, 206 <i>GRN</i>, and 86 <i>MAPT</i> pathogenic variant carriers and 255 controls were included. <i>C9orf72</i> pathogenic variant carriers performed lower on attention, executive function, and verbal fluency from CDR plus NACC FTLD 0 onwards, with relatively minimal decline over time regardless of the CDR plus NACC FTLD score (i.e., disease progression). The cognitive profile in <i>MAPT</i> pathogenic variant carriers was characterized by lower memory performance at CDR plus NACC FTLD 0.5, with decline over time in language from the CDR plus NACC FTLD 0.5 stage onwards, and executive dysfunction rapidly developing at CDR plus NACC FTLD ≥1. <i>GRN</i> pathogenic variant carriers declined on verbal fluency and visuoconstruction in the CDR plus NACC FTLD 0.5 stage, with progressive decline in other cognitive domains starting at CDR plus NACC FTLD ≥1. <h3>Discussion</h3> We confirmed cognitive decline in the asymptomatic and prodromal stage of genetic FTD. Specifically, tests for attention, executive function, language, and memory showed clear differences between genetic groups and controls at baseline, but the speed of change over time differed depending on genetic group and disease stage. This confirms the value of neuropsychological assessment in tracking clinical onset and progression and could inform clinical trials in selecting sensitive end points for measuring treatment effects as well as characterizing the best time window for starting treatment.

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 distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesInsufficient payload (model declined to judge)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.008
Threshold uncertainty score0.999

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.001
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 head0.037
GPT teacher head0.295
Teacher spread0.258 · 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 teacher head, not a consensus.

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".

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Citations29
Published2022
Admission routes1
Has abstractyes

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