Clinical-Genetic Associations in the Prospective Huntington at Risk Observational Study (PHAROS): Implications for Clinical Trials

IMPORTANCE: Identifying measures that are associated with the cytosine-adenine-guanine (CAG) expansion in individuals before diagnosis of Huntington disease (HD) has implications for designing clinical trials. OBJECTIVE: To identify the earliest features associated with the motor diagnosis of HD in...

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Veröffentlicht in:	JAMA neurology 2016-01, Vol.73 (1), p.1-9
Hauptverfasser:	Biglan, Kevin Michael, Shoulson, Ira, Kieburtz, Karl, Oakes, David, Kayson, Elise, Zhao, Hongwei, Romer, Megan, Young, Anne, Hersch, Steven, Penney, Jack, Marder, Karen, Paulsen, Jane, Quaid, Kimberly, Siemers, Eric, Tanner, Caroline, Mallonee, William, Suter, Greg, Dubinsky, Richard, Gray, Carolyn, Nance, Martha, Bundlie, Scott, Radtke, Dawn, Kostyk, Sandra, Baic, Corrine, Caress, James, Hunt, Victoria, O’Neill, Christine, Chouinard, Sylvain, Factor, Stewart, Greenamyre, Timothy, Wood-Siverio, Cathy, Corey-Bloom, Jody, Song, David, Peavy, Guerry, Moskowitz, Carol, Wesson, Melissa, Bird, Thomas, Lipe, Hillary, Blindauer, Karen, Marshall, Frederick, Zimmerman, Carol, Goldstein, Jody, Rosas, Diana, Novak, Peter, Caviness, John, Adler, Charles, Duffy, Amy, Tempkin, Teresa, Richman, David, Seeberger, Lauren, Albin, Roger, Chou, Kelvin L, Racette, Brad, Perlman, Susan, Bordelon, Yvette, Martin, Wayne, Wieler, Marguerite, Leavitt, Blair, Raymond, Lynn, Decolongon, Joji, Clarke, Lorne, Jankovic, Joseph, Hunter, Christine, Hauser, Robert A, Sanchez-Ramos, Juan, Furtado, Sarah, Suchowersky, Oksana, Klimek, Mary Lou, Guttman, Mark, Sethna, Rustom, Feigin, Andrew, Cox, Marie, Shannon, Barbara, Percy, Alan, Dure, Leon, Johnson, William, Higgins, Donald, Molho, Eric, Nickerson, Constance, Evans, Sharon, Hobson, Douglas, Singer, Carlos, Galvez-Jimenez, Nestor, Shannon, Kathleen, Comella, Cynthia, Ross, Christopher, Saint-Hilaire, Marie H, Testa, Claudia, Rosenblatt, Adam, Como, Peter, Kumar, Rajeev, Stout, Julie, Brocht, Alicia, Weaver, Christine, Foroud, Tatiana, Gusella, James, MacDonald, Marcy, Myers, Richard, Fahn, Stanley, Shults, Clifford
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Cohort Studies Female Genetic Association Studies - methods Humans Huntington Disease - diagnosis Huntington Disease - genetics Longitudinal Studies Male Middle Aged Mutation - genetics Prospective Studies Randomized Controlled Trials as Topic - methods Single-Blind Method Trinucleotide Repeat Expansion - genetics
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Zusammenfassung:	IMPORTANCE: Identifying measures that are associated with the cytosine-adenine-guanine (CAG) expansion in individuals before diagnosis of Huntington disease (HD) has implications for designing clinical trials. OBJECTIVE: To identify the earliest features associated with the motor diagnosis of HD in the Prospective Huntington at Risk Observational Study (PHAROS). DESIGN, SETTING, AND PARTICIPANTS: A prospective, multicenter, longitudinal cohort study was conducted at 43 US and Canadian Huntington Study Group research sites from July 9, 1999, through December 17, 2009. Participants included 983 unaffected adults at risk for HD who had chosen to remain unaware of their mutation status. Baseline comparability between CAG expansion (≥37 repeats) and nonexpansion (
ISSN:	2168-6149 2168-6157
DOI:	10.1001/jamaneurol.2015.2736