Motor, cognitive, and functional declines contribute to a single progressive factor in early HD

Scott A Schobel; Giuseppe Palermo; Peggy Auinger; Jeffrey D Long; Shiyang Ma; Omar S Khwaja; Dylan Trundell; Merit Cudkowicz; Steven Hersch; Cristina Sampaio; E Ray Dorsey; Blair R Leavitt; Karl D Kieburtz; Jeffrey J Sevigny; Douglas R Langbehn; Sarah J Tabrizi

doi:10.1212/WNL.0000000000004743

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Motor, cognitive, and functional declines contribute to a single progressive factor in early HD

Journal article

Open access

Peer reviewed

Motor, cognitive, and functional declines contribute to a single progressive factor in early HD

Scott A Schobel, Giuseppe Palermo, Peggy Auinger, Jeffrey D Long, Shiyang Ma, Omar S Khwaja, Dylan Trundell, Merit Cudkowicz, Steven Hersch, Cristina Sampaio, …

Neurology, Vol.89(24), pp.2495-2502

12/12/2017

DOI: 10.1212/WNL.0000000000004743

PMCID: PMC5729794

PMID: 29142089

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Abstract

To identify an improved measure of clinical progression in early Huntington disease (HD) using data from prospective observational cohort studies and placebo group data from randomized double-blind clinical trials.\nWe studied Unified Huntington Disease Rating Scale (UHDRS) and non-UHDRS clinical measures and brain measures of progressive atrophy in 1,668 individuals with early HD followed up prospectively for up to 30 to 36 months of longitudinal clinical follow-up.\nThe results demonstrated that a composite measure of motor, cognitive, and global functional decline best characterized clinical progression and was most strongly associated with brain measures of progressive corticostriatal atrophy.\nUse of a composite motor, cognitive, and global functional clinical outcome measure in HD provides an improved measure of clinical progression more related to measures of progressive brain atrophy and provides an opportunity for enhanced clinical trial efficiency relative to currently used individual motor, cognitive, and functional outcome measures.

Prospective Studies

Humans

Middle Aged

Huntington Disease - psychology

Male

Apathy

Cognitive Dysfunction - psychology

Ubiquinone - therapeutic use

Adult

Female

Huntington Disease - drug therapy

Motor Skills

Huntington Disease - physiopathology

Reproducibility of Results

Signal-To-Noise Ratio

Ubiquinone - analogs & derivatives

Cognitive Dysfunction - physiopathology

Vitamins - therapeutic use

Disease Progression

Emotions

Randomized Controlled Trials as Topic

Neuropsychological Tests

Facial Recognition

Social Perception

Stroop Test

Longitudinal Studies

Details

Title: Subtitle: Motor, cognitive, and functional declines contribute to a single progressive factor in early HD
Creators: Scott A Schobel - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK. scott.schobel@roche.com
Giuseppe Palermo - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Peggy Auinger - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Jeffrey D Long - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Shiyang Ma - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Omar S Khwaja - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Dylan Trundell - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Merit Cudkowicz - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Steven Hersch - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Cristina Sampaio - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
E Ray Dorsey - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Blair R Leavitt - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Karl D Kieburtz - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Jeffrey J Sevigny - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Douglas R Langbehn - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Sarah J Tabrizi - From F. Hoffman-La Roche, Ltd (S.A.S., G.P., O.S.K., D.T., J.J.S.), Roche Innovation Center, Basel, Switzerland; University of Rochester (P.A., S.M., E.R.D., K.D.K.), NY; University of Iowa (J.D.L., D.R.L.), Iowa City; Massachusetts General Hospital and Harvard Medical School (M.C., S.H.), Boston; CHDI Management/Foundation (C.S.), Princeton, NJ; University of British Columbia, Vancouver, Canada (B.R.L.); and University College London (S.J.T.), UK
Resource Type: Journal article
Publication Details: Neurology, Vol.89(24), pp.2495-2502
DOI: 10.1212/WNL.0000000000004743
PMID: 29142089
PMCID: PMC5729794
NLM abbreviation: Neurology
ISSN: 0028-3878
eISSN: 1526-632X
Publisher: United States
Grant note: U01 NS052592 / NINDS NIH HHS\n200181/Z/15/Z / Wellcome Trust\nR01 NS091161 / NINDS NIH HHS\nR01 NS052592 / NINDS NIH HHS
Language: English
Date published: 12/12/2017
Academic Unit: Psychiatry; Iowa Neuroscience Institute; Biostatistics
Record Identifier: 9984066390802771

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