{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T12:40:05Z","timestamp":1788871205310,"version":"build-2803163510"},"reference-count":69,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2014,5,9]],"date-time":"2014-05-09T00:00:00Z","timestamp":1399593600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"National Council for Scientific Research Lebanon"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Fundamemntal Clinical Pharma"],"published-print":{"date-parts":[[2014,12]]},"abstract":"Abstract<\/jats:title>\n \n Huntington's disease (\n HD<\/jats:styled-content>\n ) is a rare multifactorial neurodegenerative disease. Both its natural course and any placebo effect are poorly known. All are obstacles to design randomized controlled trials (\n RCT<\/jats:styled-content>\n s). We conducted meta\u2010analyses of\n RCT<\/jats:styled-content>\n s and cohorts on all parameters of the\n U<\/jats:styled-content>\n nified\n H<\/jats:styled-content>\n untington's\n D<\/jats:styled-content>\n isease\n R<\/jats:styled-content>\n ating\n S<\/jats:styled-content>\n cale to determine the most appropriate outcomes and to minimize the number of patients required to design\n RCT<\/jats:styled-content>\n s in\n HD<\/jats:styled-content>\n . Twenty\u2010four\n RCT<\/jats:styled-content>\n s were included, involving 838 patients with a mean age of 50.0\u00a0\u00b1\u00a02.3\u00a0years and a mean total functional capacity (\n TFC<\/jats:styled-content>\n ) score of 9.8\u00a0\u00b1\u00a00.6. Nineteen cohorts were included involving 1939 patients with a mean age of 48.9\u00a0\u00b1\u00a02.3\u00a0years and a mean\n TFC<\/jats:styled-content>\n of 10.1\u00a0\u00b1\u00a00.7. Significant deterioration was observed in\n RCT<\/jats:styled-content>\n s for all scores except behavioral score. Effect sizes were comparable between\n RCT<\/jats:styled-content>\n s and cohorts for each test except that there was a significant difference for\n TFC<\/jats:styled-content>\n . The weighted mean deterioration per year on the\n TFC<\/jats:styled-content>\n scale was \u22120.5 (0.2) in\n RCT<\/jats:styled-content>\n s and \u22120.8 (0.2) in cohorts. The lowest number of patients required per group in a\n RCT<\/jats:styled-content>\n was for\n TFC<\/jats:styled-content>\n (19 per group), whereas 30 patients would be required per group for the total motor score (\n TMS<\/jats:styled-content>\n ). For cognition, the verbal fluency test required the smallest number of patients: 104 per group. In conclusion,\n TMS<\/jats:styled-content>\n and\n TFC<\/jats:styled-content>\n are the most appropriate outcomes to design\n RCT<\/jats:styled-content>\n s on\n HD<\/jats:styled-content>\n likewise the verbal fluency test for cognition. Our results suggest an effect of placebo administration on the total functional capacity.\n <\/jats:p>","DOI":"10.1111\/fcp.12077","type":"journal-article","created":{"date-parts":[[2014,4,5]],"date-time":"2014-04-05T03:28:02Z","timestamp":1396668482000},"page":"700-710","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["The most appropriate primary outcomes to design clinical trials on\n H<\/scp>\n untington's disease: meta\u2010analyses of cohort studies and randomized placebo\u2010controlled trials"],"prefix":"10.1111","volume":"28","author":[{"given":"Linda","family":"Salem","sequence":"first","affiliation":[{"name":"Inserm U955 Equipe 01 Cr\u00e9teil 94010 France"},{"name":"Universit\u00e9 Paris Est Facult\u00e9 de m\u00e9decine Cr\u00e9teil 94010 France"},{"name":"AP\u2010HP H\u00f4pital H. Mondor\u2010 A. 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Chenevier Centre de r\u00e9f\u00e9rence maladie de Huntington Cr\u00e9teil 94010 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Audrey","family":"Olivier","sequence":"additional","affiliation":[{"name":"CHU Angers D\u00e9partement de Neurologie Angers 49000 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Perrine","family":"Charles","sequence":"additional","affiliation":[{"name":"AP\u2010HP H\u00f4pital H. Mondor\u2010 A. Chenevier Centre de r\u00e9f\u00e9rence maladie de Huntington Cr\u00e9teil 94010 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Clarisse","family":"Scherer","sequence":"additional","affiliation":[{"name":"CHU Angers D\u00e9partement de Neurologie Angers 49000 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christophe","family":"Verny","sequence":"additional","affiliation":[{"name":"CHU Angers D\u00e9partement de Neurologie Angers 49000 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne\u2010Catherine","family":"Bachoud\u2010L\u00e9vi","sequence":"additional","affiliation":[{"name":"Inserm U955 Equipe 01 Cr\u00e9teil 94010 France"},{"name":"Universit\u00e9 Paris Est Facult\u00e9 de m\u00e9decine Cr\u00e9teil 94010 France"},{"name":"Ecole Normale Sup\u00e9rieure D\u00e9partement d'\u00e9tudes cognitives Paris 75005 France"},{"name":"AP\u2010HP H\u00f4pital H. Mondor\u2010 A. 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