Abstract
With many viable strategies in the therapeutic pipeline, upcoming clinical trials in hereditary and sporadic degenerative ataxias will benefit from non-invasive MRI biomarkers for patient stratification and the evaluation of therapies. The MRI Biomarkers Working Group of the Ataxia Global Initiative therefore devised guidelines to facilitate harmonized MRI data acquisition in clinical research and trials in ataxias. Recommendations are provided for a basic structural MRI protocol that can be used for clinical care and for an advanced multi-modal MRI protocol relevant for research and trial settings. The advanced protocol consists of modalities with demonstrated utility for tracking brain changes in degenerative ataxias and includes structural MRI, magnetic resonance spectroscopy, diffusion MRI, quantitative susceptibility mapping, and resting-state functional MRI. Acceptable ranges of acquisition parameters are provided to accommodate diverse scanner hardware in research and clinical contexts while maintaining a minimum standard of data quality. Important technical considerations in setting up an advanced multi-modal protocol are outlined, including the order of pulse sequences, and example software packages commonly used for data analysis are provided. Outcome measures most relevant for ataxias are highlighted with use cases from recent ataxia literature. Finally, to facilitate access to the recommendations by the ataxia clinical and research community, examples of datasets collected with the recommended parameters are provided and platform-specific protocols are shared via the Open Science Framework.
| Original language | English (US) |
|---|---|
| Journal | Cerebellum |
| DOIs | |
| State | Accepted/In press - 2023 |
Bibliographical note
Funding Information:Open Access funding enabled and organized by CAUL and its Member Institutions. The preparation of the manuscript and example protocols in OSF and the collection of the MRI data presented in this manuscript were supported by the National Institute of Neurological Disorders and Stroke (NINDS) grants U01 NS104326 and R01 NS080816, Friedreich’s Ataxia Research Alliance, the National Ataxia Foundation (NAF), the Hertie Network of Excellence in Clinical Neuroscience, German Research Foundation (DFG, DE 2516/1–1 and TI 239/17–1), ESMI, an EU Joint Programme—Neurodegenerative Disease Research (JPND) project ( www.jpnd.eu ), Australian NHMRC Ideas Grant #1184403, and an Academic Investment Research Program (AIRP) award at the University of Minnesota. The Center for Magnetic Resonance Research is supported by the National Institute of Biomedical Imaging and Bioengineering (NIBIB) grant P41 EB027061, the Institutional Center Cores for Advanced Neuroimaging award P30 NS076408 and S10 OD017974.
Funding Information:
G. Ö. consulted for IXICO Technologies Limited and uniQure biopharma, served in the Scientific Advisory Board of BrainSpec Inc, and receives research support from Biogen. P-G. H. discloses research support from Minoryx Therapeutics. C. L. discloses research support from Minoryx Therapeutics and Biogen, Inc. G. Ö. and C. L. are inventors of a patent pertaining to the AutoVOI method, which is freely available to academic investigators ( https://www.cmrr.umn.edu/autovoi/ ). A. J. S. is a full-time employee of Takeda Pharmaceuticals Ltd. K. R. A. V. D. is a full-time employee of Pfizer, Inc. S. C., A. D., J. F., D. T., and I. H. H. have no conflict of interests to declare with regard to the content of the manuscript.
Publisher Copyright:
© 2023, The Author(s).
Keywords
- Diffusion MRI
- Functional MRI
- MRI biomarkers
- Magnetic resonance spectroscopy
- Quantitative susceptibility mapping
- Structural MRI
PubMed: MeSH publication types
- Journal Article
- Review