Cognitive composites for genetic frontotemporal dementia: GENFI-Cog.
Jackie M PoosKatrina M MooreJennifer NicholasLucy L RussellGeorgia PeakmanRhian S ConveryLize C JiskootEmma van der EndeEsther van den BergJanne M PapmaHarro SeelaarYolande A L PijnenburgFermin MorenoRaquel Sanchez-ValleBarbara BorroniRobert LaforceMario MasellisCarmela TartagliaCaroline GraffDaniela GalimbertiJames B RoweElizabeth FingerMatthis SynofzikRik VandenbergheAlexandre de MendonçaPietro TiraboschiIsabel SantanaSimon DucharmeChris ButlerAlexander GerhardJohannes LevinAdrian DanekMarkus OttoIsabel Le BerFlorence PasquierJohn C van SwietenJonathan Daniel Rohrernull nullPublished in: Alzheimer's research & therapy (2022)
We created gene-specific cognitive composite scores for C9orf72, GRN, and MAPT mutation carriers, which resulted in substantially lower estimated sample sizes to detect a treatment effect than the individual cognitive tests. The GENFI-Cog composites have potential as cognitive endpoints for upcoming clinical trials. The results from this study provide recommendations for estimating sample size and trial duration.