Robust markers and sample sizes for multicenter trials of Huntington disease

Journal article


Wijeratne, Peter A., Johnson, Eileanoir B., Eshaghi, Arman, Aksman, Leon, Gregory, Sarah, Johnson, Hans J., Poudel, Govinda R., Mohan, Amrita, Sampaio, Cristina, Georgiou-Karistianis, Nellie, Paulsen, Jane S., Tabrizi, Sarah J., Scahill, Rachael I., the Image-Hd, Predict-Hd Track-Hd investigators and Alexander, Daniel C.. (2020) Robust markers and sample sizes for multicenter trials of Huntington disease. Annals of Neurology. 87(5), pp. 751-762. https://doi.org/10.1002/ana.25709
AuthorsWijeratne, Peter A., Johnson, Eileanoir B., Eshaghi, Arman, Aksman, Leon, Gregory, Sarah, Johnson, Hans J., Poudel, Govinda R., Mohan, Amrita, Sampaio, Cristina, Georgiou-Karistianis, Nellie, Paulsen, Jane S., Tabrizi, Sarah J., Scahill, Rachael I., the Image-Hd, Predict-Hd Track-Hd investigators and Alexander, Daniel C.
Abstract

Objective
The identification of sensitive biomarkers is essential to validate therapeutics for Huntington disease (HD). We directly compare structural imaging markers across the largest collective imaging HD dataset to identify a set of imaging markers robust to multicenter variation and to derive upper estimates on sample sizes for clinical trials in HD.

Methods
We used 1 postprocessing pipeline to retrospectively analyze T1‐weighted magnetic resonance imaging (MRI) scans from 624 participants at 3 time points, from the PREDICT‐HD, TRACK‐HD, and IMAGE‐HD studies. We used mixed effects models to adjust regional brain volumes for covariates, calculate effect sizes, and simulate possible treatment effects in disease‐affected anatomical regions. We used our model to estimate the statistical power of possible treatment effects for anatomical regions and clinical markers.

Results
We identified a set of common anatomical regions that have similarly large standardized effect sizes (>0.5) between healthy control and premanifest HD (PreHD) groups. These included subcortical, white matter, and cortical regions and nonventricular cerebrospinal fluid (CSF). We also observed a consistent spatial distribution of effect size by region across the whole brain. We found that multicenter studies were necessary to capture treatment effect variance; for a 20% treatment effect, power of >80% was achieved for the caudate (n = 661), pallidum (n = 687), and nonventricular CSF (n = 939), and, crucially, these imaging markers provided greater power than standard clinical markers.

Interpretation
Our findings provide the first cross‐study validation of structural imaging markers in HD, supporting the use of these measurements as endpoints for both observational studies and clinical trials.

Year2020
JournalAnnals of Neurology
Journal citation87 (5), pp. 751-762
PublisherJohn Wiley & Sons
ISSN0364-5134
Digital Object Identifier (DOI)https://doi.org/10.1002/ana.25709
Scopus EID2-s2.0-85082581297
Open accessPublished as ‘gold’ (paid) open access
Research or scholarlyResearch
Page range751-762
FunderNational Health and Medical Research Council
Publisher's version
License
File Access Level
Open
Output statusPublished
Publication dates
Online14 Mar 2020
Publication process dates
Accepted21 Feb 2020
Deposited06 May 2021
Grant IDNHMRC/606650
Permalink -

https://acuresearchbank.acu.edu.au/item/8vz95/robust-markers-and-sample-sizes-for-multicenter-trials-of-huntington-disease

Download files


Publisher's version
OA_Wijeratne_2020_Robust_markers_and_sample_sizes_for.pdf
License: CC BY 4.0
File access level: Open

  • 2
    total views
  • 0
    total downloads
  • 0
    views this month
  • 0
    downloads this month
These values are for the period from 19th October 2020, when this repository was created.

Export as

Related outputs

Network diffusion modeling predicts neurodegeneration in traumatic brain injury
Poudel, Govinda R., Dominguez D, Juan F., Verhelst, Helena, Vander Linden, Catharine, Deblaere, Karel, Jones, Derek K., Cerin, Ester, Vingerhoets, Guy and Caeyenberghs, Karen. (2020) Network diffusion modeling predicts neurodegeneration in traumatic brain injury. Annals of Clinical and Translational Neurology. 7(3), pp. 270-279. https://doi.org/10.1002/acn3.50984
International Mind, Activities and Urban Places (iMAP) study: methods of a cohort study on environmental and lifestyle influences on brain and cognitive health
Ester Cerin, Anthony Barnett, Basile Chaix, Mark Nieuwenhuijsen, Karen Caeyenberghs, Bin Jalaludin, Takemi Sugiyama, James Sallis, Nicola T. Lautenschlager, Michael Y Ni, Govinda Poudel, David Donaire Gonzalez, Rachel Tham, Amanda Wheeler, Luke Knibbs, Linwei Tian, Kai Chan, David Dunstan, Alison Carver and Kaarin Anstey. (2020) International Mind, Activities and Urban Places (iMAP) study: methods of a cohort study on environmental and lifestyle influences on brain and cognitive health. BMJ Open. 10(3), pp. 1-15. https://doi.org/10.1136/bmjopen-2019-036607
Rate of torque development and striatal shape in individuals with prodromal Huntington's disease
Travis M. Cruickshank, Alvaro P. Reyes, Timothy S. Pulverenti, Tim J. Rankin, Danielle M. Bartlett, Anthony Blazevich, Govinda Poudel, Mel R. Ziman and Gabriel S Trajano. (2020) Rate of torque development and striatal shape in individuals with prodromal Huntington's disease. Scientific Reports. 10(1), pp. 1-8. https://doi.org/10.1038/s41598-020-72042-2
The effects of multidisciplinary rehabilitation on neuroimaging, biological, cognitive and motor outcomes in individuals with premanifest Huntington's disease
Danielle M. Bartlett, Andrew D. Govus, Tim J. Rankin, A. Lampit, Kirk W. Feindel, Govinda Poudel, Wei Peng Teo, Johnny Lo, Nellie Georgiou-Karistianis, Mel R. Ziman and Travis M. Cruickshank. (2020) The effects of multidisciplinary rehabilitation on neuroimaging, biological, cognitive and motor outcomes in individuals with premanifest Huntington's disease. Journal of the Neurological Sciences. 416, pp. 1-9. https://doi.org/10.1016/j.jns.2020.117022
Exploring the brain-body composition relationship in Huntington's disease
Mitchell Turner, Alvaro Reyes, Danielle M. Bartlett, Scott Culpin, Nicolas H. Hart, Luca Hardt, Kirk Feindel, Govinda R. Poudel, Mel Ziman and Travis M. Cruickshank. (2020) Exploring the brain-body composition relationship in Huntington's disease. The Journal of Musculoskeletal and Neuronal Interactions. 20(3), pp. 332-338.
Dual tasking impairments are associated with striatal pathology in Huntington’s disease
Johnny Lo, Alvaro Reyes, Timothy S. Pulverenti, Timothy J. Rankin, Danielle M. Bartlett, Pauline Zaenker, Grant Rowe, Kirk Feindel, Govinda Poudel, Nellie Georgiou-Karistianis, Mel R. Ziman and Travis M. Cruickshank. (2020) Dual tasking impairments are associated with striatal pathology in Huntington’s disease. Annals of Clinical and Translational Neurology. 7(9), pp. 1608-1619. https://doi.org/10.1002/acn3.51142
Oxytocin selectively modulates brain processing of disgust in Huntington's disease gene carriers
Izelle Labuschagne, Govinda Poudel, Catarina C. Kordsachia, Qizhu Wu, Hannah Thomson, Nellie Georgiou-Karistianis and Julie C Stout. (2018) Oxytocin selectively modulates brain processing of disgust in Huntington's disease gene carriers. Progress in Neuropsychopharmacology and Biological Psychiatry. 81, pp. 11-16. https://doi.org/10.1016/j.pnpbp.2017.09.023
A critical review of brain and cognitive reserve in Huntington's disease
Soloveva, Maria V., Jamadar, Sharna D., Poudel, Govinda and Georgiou-Karistianis, Nellie. (2018) A critical review of brain and cognitive reserve in Huntington's disease. Neuroscience and Biobehavioral Reviews. 88, pp. 155 - 169. https://doi.org/10.1016/j.neubiorev.2018.03.003
Temporal evolution of neutral activity and connectivity during microsleeps when rested and following sleep restriction
Poudel, Govinda, Innes, Carrie R. H. and Jones, Richard. (2018) Temporal evolution of neutral activity and connectivity during microsleeps when rested and following sleep restriction. NeuroImage. 174, pp. 263 - 273. https://doi.org/10.1016/j.neuroimage.2018.03.031
Neural correlates of decision-making during a Bayesian choice task
Poudel, Govinda, Bhattarai, Anjan, Dickinson, David L. and Drummond, Sean P. A.. (2017) Neural correlates of decision-making during a Bayesian choice task. NeuroReport. 28(4), pp. 193 - 199. https://doi.org/10.1097/WNR.0000000000000730
Emotion processing in persons who respond vicariously towards others in pain: Disinhibited left-lateralized neural activity for threatening expressions
Giummarra, Melita J., Poudel, Govinda, Niu, P. Amanda, Nicholls, Michael E. R., Fielding, Joanne, Verdejo-Garcia, Antonio and Labuschagne, Izelle. (2017) Emotion processing in persons who respond vicariously towards others in pain: Disinhibited left-lateralized neural activity for threatening expressions. Laterality: Assymetries of body, brain and cognition. 23(2), pp. 184 - 208. https://doi.org/10.1080/1357650X.2017.1349781
Longitudinal changes in the fronto-striatal network are associated with executive dysfunction and behavioral dysregulation in Huntington's disease: 30 months IMAGE-HD data
Dominguez, Juan F., Poudel, Govinda, Stout, Julie C., Gray, Marcus, Chua, Phyllis, Borowsky, Beth, Egan, Gary F. and Georgiou-Karistianis, Nellie. (2017) Longitudinal changes in the fronto-striatal network are associated with executive dysfunction and behavioral dysregulation in Huntington's disease: 30 months IMAGE-HD data. Cortex. 92, pp. 139 - 149. https://doi.org/10.1016/j.cortex.2017.04.001
Fronto-cerebellar dysfunction and dysconnectivity underlying cognition in Friedreich Ataxia: The IMAGE-FRDA Study
Harding, Ian H., Corben, Louise A., Storey, Elsdon, Egan, Gary F., Stagnitti, Monique R., Poudel, Govinda, Delatycki, Martin B. and Georgiou-Karistianis, Nellie. (2016) Fronto-cerebellar dysfunction and dysconnectivity underlying cognition in Friedreich Ataxia: The IMAGE-FRDA Study. Human Brain Mapping. 37(1), pp. 338 - 350. https://doi.org/10.1002/hbm.23034
Large-scale comparative visualisation of sets of multidimensional data
Vohl, Dany, Barnes, David G., Fluke, Christopher J., Poudel, Govinda, Georgiou-Karistianis, Nellie, Hassan, Amr H., Benovitski, Yuri, Wong, Tsz Ho, Kaluza, Owen L., Nguyen, Toan D. and Bonnington, C. Paul. (2016) Large-scale comparative visualisation of sets of multidimensional data. PeerJ. 2016(10), pp. 1 - 36. https://doi.org/10.7717/peerj-cs.88
Iron accumulation in the basal ganglia in Huntington's disease: Cross-sectional data from the IMAGE-HD study
Dominguez, Juan F., Ng, Amanda C. L., Poudel, Govinda, Stout, Julie C., Churchyard, A., Chua, Phyllis, Egan, Gary F. and Georgiou-Karistianis, Nellie. (2016) Iron accumulation in the basal ganglia in Huntington's disease: Cross-sectional data from the IMAGE-HD study. Journal of Neurology, Neurosurgery and Psychiatry. 87(5), pp. 545 - 549. https://doi.org/10.1136/jnnp-2014-310183
Subjective sleep problems in Huntington's disease: A pilot investigation of the relationship to brain structure, neurocognitive, and neuropsychiatric function
Baker, Chaya Rochel, Dominguez, Juan F., Stout, Julie C., Gabery, Sanaz, Churchyard, A., Chua, Phyllis, Egan, Gary F., Petersen, Asa, Georgiou-Karistianis, Nellie and Poudel, Govinda. (2016) Subjective sleep problems in Huntington's disease: A pilot investigation of the relationship to brain structure, neurocognitive, and neuropsychiatric function. Journal of the Neurological Sciences. 364, pp. 148 - 153. https://doi.org/10.1016/j.jns.2016.03.021
Microsleeps are associated with stage-2 sleep spindles from hippocampal-temporal network
Jonmohamadi, Yaqub, Poudel, Govinda, Innes, Carrie C. R. H. and Jones, Richard. (2016) Microsleeps are associated with stage-2 sleep spindles from hippocampal-temporal network. International Journal of Neural Systems. 26(4), pp. 1 - 12. https://doi.org/10.1142/S0129065716500155
Multimodal imaging biomarkers in premanifest and early Huntington's disease: 30-month IMAGE-HD data
Dominguez, Juan, Stout, Julie C., Poudel, Govinda, Churchyard, Andrew, Chua, Phyllis, Egan, Gary F. and Georgiou-Karistianis, Nellie. (2016) Multimodal imaging biomarkers in premanifest and early Huntington's disease: 30-month IMAGE-HD data. British Journal of Psychiatry. 208(6), pp. 571 - 578. https://doi.org/10.1192/bjp.bp.114.156588
Time-varying effective connectivity of the cortical neuroelectric activity associated with behavioural microsleeps
Toppi, Jlenia, Astolfi, Laura, Poudel, Govinda, H., Babiloni, Fabio, Jones, Richard and Innes, Carrie R. H.. (2016) Time-varying effective connectivity of the cortical neuroelectric activity associated with behavioural microsleeps. NeuroImage. 124, pp. 421 - 432. https://doi.org/10.1016/j.neuroimage.2015.08.059
Characterising upper limb movements in Huntington's Disease and the impact of restricted visual cues
Despard, Jessica, Ternes, Anne-Marie, Dimech-Betancourt, Bleydy, Poudel, Govinda, Churchyard, Andrew and Georgiou-Karistianis, Nellie. (2015) Characterising upper limb movements in Huntington's Disease and the impact of restricted visual cues. PLoS ONE. 10(8), pp. 1 - 18. https://doi.org/10.1371/journal.pone.0133709
Comparison of beamformers for EEG source signal reconstruction
Jonmohamadi, Yaqub, Poudel, Govinda, Innes, Carrie, Weiss, Daniel, Krueger, Rejko and Jones, Richard. (2014) Comparison of beamformers for EEG source signal reconstruction. Biomedical Signal Processing and Control. 14(1), pp. 175 - 188. https://doi.org/10.1016/j.bspc.2014.07.014
Source-space ICA for EEG source separation, localization, and time-course reconstruction
Jonmohamadi, Yaqub, Poudel, Govinda, Innes, Carrie and Jones, Richard. (2014) Source-space ICA for EEG source separation, localization, and time-course reconstruction. NeuroImage. 101(November), pp. 720 - 737. https://doi.org/10.1016/j.neuroimage.2014.07.052
The Multi-modal Australian ScienceS Imaging and Visualization Environment (MASSIVE) high performance computing infrastructure: Applications in neuroscience and neuroinformatics research
Goscinski, Wojtek J., McIntosh, Paul, Felzmann, Ulrich, Maksimenko, Anton, Hall, Christopher J., Gureyev, Timur, Thompson, Darren, Janke, Andrew, Galloway, Graham, Killeen, Neil E. B., Raniga, Parnesh, Kaluza, Owen, Ng, Amanda, Poudel, Govinda, Barnes, David G., Nguyen, Toan, Bonnington, Paul and Egan, Gary F.. (2014) The Multi-modal Australian ScienceS Imaging and Visualization Environment (MASSIVE) high performance computing infrastructure: Applications in neuroscience and neuroinformatics research. Frontiers in Neuroinformatics. 8(March), pp. 1 - 13. https://doi.org/10.3389/fninf.2014.00030
Functional magnetic resonance imaging of working memory in Huntington's disease: Cross-sectional data from the IMAGE-HD study
Georgiou-Karistianis, Nellie, Stout, Julie C., Domínguez, Juan F., Carron, Sarah P., Ando, Ayaka, Churchyard, Andrew, Chua, Phyllis, Bohanna, India, Dymowski, Alicia R., Poudel, Govinda and Egan, Gary F.. (2014) Functional magnetic resonance imaging of working memory in Huntington's disease: Cross-sectional data from the IMAGE-HD study. Human Brain Mapping. 35(5), pp. 1847 - 1864. https://doi.org/10.1002/hbm.22296
Voxel-ICA for reconstruction of source signal time-series and orientation in EEG and MEG
Jonmohamadi, Yaqub, Poudel, Govinda, Innes, Carrie and Jones, Richard. (2014) Voxel-ICA for reconstruction of source signal time-series and orientation in EEG and MEG. Australasian Physical and Engineering Sciences in Medicine. 37(2), pp. 457 - 464. https://doi.org/10.1007/s13246-014-0265-x
Multi-modal neuroimaging in premanifest and early Huntington's disease : 18 month longitudinal data from the IMAGE-HD study
Domínguez D, Juan F., Egan, Gary F., Gray, Marcus A., Poudel, Govinda R., Churchyard, Andrew, Chua, Phyllis, Stout, Julie C. and Georgiou-Karistianis, Nellie. (2013) Multi-modal neuroimaging in premanifest and early Huntington's disease : 18 month longitudinal data from the IMAGE-HD study. PLoS ONE. 8(9), pp. 1-10. https://doi.org/10.1371/journal.pone.0074131
Functional and connectivity changes during working memory in Huntington's disease : 18 month longitudinal data from the IMAGE-HD study
Georgiou-Karistianis, Nellie, Poudel, Govinda R., Domínguez D, Juan F., Langmaid, Rebecca, Gray, Marcus A., Churchyard, Andrew, Chua, Phyllis, Borowsky, Beth, Egan, Gary F. and Stout, Julie C.. (2013) Functional and connectivity changes during working memory in Huntington's disease : 18 month longitudinal data from the IMAGE-HD study. Brain and Cognition. 83(1), pp. 80-91. https://doi.org/10.1016/j.bandc.2013.07.004
Efficient and regular patterns of nighttime sleep are related to increased vulnerability to microsleeps following a single night of sleep restriction
Innes, Carrie R. H., Poudel, Govinda R. and Jones, Richard D.. (2013) Efficient and regular patterns of nighttime sleep are related to increased vulnerability to microsleeps following a single night of sleep restriction. Chronobiology International. 30(9), pp. 1187-1196. https://doi.org/10.3109/07420528.2013.810222
Distinct neural correlates of time-on-task and transient errors during a visuomotor tracking task after sleep restriction
Poudel, Govinda R., Innes, Carrie R. H. and Jones, Richard D.. (2013) Distinct neural correlates of time-on-task and transient errors during a visuomotor tracking task after sleep restriction. NeuroImage. 77, pp. 105-113. https://doi.org/10.1016/j.neuroimage.2013.03.054
Time-Varying Functional Connectivity for Understanding the Neural Basis of Behavioral Microsleeps
J Toppi, L Astolfi, GR Poudel, F Babiloni, L Macchiusi, D Mattia, S Salinari and RD Jones. (2012) Time-Varying Functional Connectivity for Understanding the Neural Basis of Behavioral Microsleeps. 34th Annual International Conference of the IEEE EMBS. IEEE, Institute of Electrical and Electronics Engineers. pp. 4708-4711 https://doi.org/10.1109/EMBC.2012.6347018
Performance of Beamformers on EEG Source Reconstruction
Yaqub Jon Mohamadi, Govinda Poudel, Carrie R H Innes and Richard Jones. (2012) Performance of Beamformers on EEG Source Reconstruction. 34th Annual International Conference of the IEEE EMBS. IEEE, Institute of Electrical and Electronics Engineers. https://doi.org/10.1109/EMBC.2012.6346476
Cerebral perfusion differences between drowsy and nondrowsy individuals after acute sleep restriction
Poudel, Govinda R., Innes, Carrie R. H. and Jones, Richard D.. (2012) Cerebral perfusion differences between drowsy and nondrowsy individuals after acute sleep restriction. Sleep. 35(8), pp. 1085-1096. https://doi.org/10.5665/sleep.1994