@article{bb73fe6397584b63a1f8420f7d845918,
title = "Change in MRI striatal volumes as a biomarker in preclinical Huntington's disease",
abstract = "This article discusses the need for biomarkers and surrogate endpoints for future clinical trials in individuals at risk for Huntington's disease. Definitions and criteria are presented for biomarkers and surrogate endpoints, and data are presented suggesting that striatal volumes, as measured on MRI scans, meet the criteria for a biomarker. Biomarkers can be used in lieu of clinical endpoints in treatment trials if there is evidence that treatment affects the biomarker in a way that is predictive of endpoint status. Because there are currently no effective treatments for Huntington's disease, it is not yet possible to validate whether change in MRI striatal volumes can serve as an effective surrogate endpoint. It is recommended that future clinical trials be designed using MRI striatal volumes to {"}screen{"} potential treatments. Those treatments that reduce the rate of striatal atrophy can then be tested with delay of symptom onset as the clinical endpoint. This strategy is essential if efficient and cost-effective clinical trials are to be conducted in the preclinical stage of Huntington's disease.",
keywords = "Biomarker, Clinical trial, Huntington's disease, Preclinical, Surrogate endpoint",
author = "Aylward, {Elizabeth H.}",
note = "Funding Information: The studies described in this manuscript would have been impossible without my co-investigators at Johns Hopkins HD Research Center, Baltimore, MD (Christopher Ross, M.D., Ph.D., Director) including: Adam Rosenblatt, M.D., Jason Brandt, Ph.D., and Barnett Shpritz; colleagues at University of Washington, Seattle, WA, including Research Technicians Katherine Field, B.S. and Venugopal Yallapragada, M.A., Nurse Coordinator Hillary Lipe, R.N., Clinic Director Thomas Bird, M.D.; and PI of the PREDICT-HD study, Jane Paulsen, University of Iowa. Funding for the studies described in this manuscript came from the National Institutes of Health NINDS, including 5R01NS040068-02 (PI: Paulsen, J. Neurobiological Predictors of Huntington's Disease) and 5P01NS016375-25 (PI: Ross, C. Research Center without Walls); from the University of Washington Center for Human Development and Disabilities (Michael Guralnick, Director), and from the Huntington's Disease Society of America. This manuscript summarizes Dr. Aylward's talk, “Early Markers of Huntington's Disease,” which was presented at the World Congress on Huntington's Disease in Manchester, UK, on September 11, 2005. Copyright: Copyright 2008 Elsevier B.V., All rights reserved.",
year = "2007",
month = apr,
day = "30",
doi = "10.1016/j.brainresbull.2006.10.028",
language = "English (US)",
volume = "72",
pages = "152--158",
journal = "Brain Research Bulletin",
issn = "0361-9230",
publisher = "Elsevier Inc.",
number = "2-3 SPEC. ISS.",
}