{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T12:38:17Z","timestamp":1753879097509,"version":"3.41.2"},"reference-count":19,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2017,12,5]],"date-time":"2017-12-05T00:00:00Z","timestamp":1512432000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"Orphan Medical, Inc."}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Journal of Sleep Research"],"published-print":{"date-parts":[[2018,8]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>Our objective was to define responder criteria using an anchor\u2010based approach for frequency of cataplexy attacks and excessive daytime sleepiness in patients with narcolepsy undergoing sodium oxybate treatment. We used pooled data from two randomized, placebo\u2010controlled, double\u2010blind, multicentre 4\u2010 and 8\u2010week trials of sodium oxybate for narcolepsy with cataplexy and analysed using receiver operator characteristics analysis. The percentage change in frequency of weekly cataplexy attacks and the Epworth Sleepiness Scale outcomes were compared with Clinical Global Impression of Change ratings, used as the anchor to define true response. Participants (<jats:italic>n<\/jats:italic>\u00a0=\u00a0336) were 39% male, 89% white, with a mean age of 41.5 (15.3)\u00a0years, reporting a median of 20.5 cataplexy attacks per week and a mean Epworth Sleepiness score of 17.5 at baseline. A majority (51%) were Much Improved or Very Much Improved based on Clinical Global Impression of Change ratings, considered a true response to treatment. Area under the curve values for % reduction in cataplexy attacks (77%) and % change in sleepiness score (78%) supported response definition thresholds of 46% and 12%, respectively. Classification using either response definition agreed with the anchor for approximately 71% of participants. Cataplexy response definition was more sensitive (cataplexy\u00a0=\u00a00.77, Epworth Sleepiness Scale\u00a0=\u00a00.69), while sleepiness was more specific (cataplexy\u00a0=\u00a00.66, Epworth Sleepiness Scale\u00a0=\u00a00.75). Both responder definitions showed a dose\u2013response relationship with sodium oxybate, demonstrating their validity using an external criterion. Weekly cataplexy attacks and Epworth Sleepiness Scale can be used to help document clinical response to narcolepsy treatment using criteria of 46% and 12% reductions, respectively.<\/jats:p>","DOI":"10.1111\/jsr.12628","type":"journal-article","created":{"date-parts":[[2017,12,5]],"date-time":"2017-12-05T11:56:05Z","timestamp":1512474965000},"source":"Crossref","is-referenced-by-count":5,"title":["Criteria for gauging response to sodium oxybate for narcolepsy"],"prefix":"10.1111","volume":"27","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4292-6138","authenticated-orcid":false,"given":"Alana D.","family":"Steffen","sequence":"first","affiliation":[{"name":"College of Nursing University of Illinois at Chicago Chicago IL USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chinglin","family":"Lai","sequence":"additional","affiliation":[{"name":"Jazz Pharmaceuticals Palo Alto CA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Terri E.","family":"Weaver","sequence":"additional","affiliation":[{"name":"Center for Narcolepsy Sleep and Health, and Department of Biobehavioral Health Science College of Nursing University of Illinois at Chicago"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2017,12,5]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1177\/0962280213476377"},{"key":"e_1_2_8_3_1","unstructured":"Clayton P.cutpt\u2010Epirical estimation of cutpoint for a diagnostic test.2013.http:\/\/fmwww.bc.edu\/RePEc\/bocode\/c\/cutpt.sthlp."},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.2307\/2531595"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/bimj.200410135"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.5665\/sleep.4810"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1093\/sleep\/14.6.540"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2869.2000.00177.x"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.4509"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1586\/erp.11.12"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1186\/1745-6215-8-31"},{"volume-title":"Statistical Methods","year":"1989","author":"Snedecor G.","key":"e_1_2_8_12_1"},{"volume-title":"Stata Statistical Software: Release 12","year":"2011","author":"StataCorp","key":"e_1_2_8_13_1"},{"key":"e_1_2_8_14_1","first-page":"42","article-title":"A randomized, double blind, placebo\u2010controlled multicenter trial comparing the effects of three doses of orally administered sodium oxybate with placebo for the treatment of narcolepsy","volume":"25","author":"The U.S. Xyrem\u00ae Multicenter Study Group","year":"2002","journal-title":"Sleep"},{"key":"e_1_2_8_15_1","unstructured":"US Department of Health and Human Services.Guidance for industry on patient\u2010reported outcome measures: use in medical product development to support labeling claims. 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