{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,5]],"date-time":"2026-05-05T16:48:20Z","timestamp":1777999700811,"version":"3.51.4"},"reference-count":14,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2021,11,2]],"date-time":"2021-11-02T00:00:00Z","timestamp":1635811200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2022,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>Data regarding the comparative effectiveness of myasthenia gravis (MG) treatments is not available. We used patient input to identify a patient\u2010centered outcome measure (PCOM) for PROMISE\u2010MG, a comparative effectiveness trial of MG treatments.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>First, a questionnaire survey was administered to 58 people with MG at the patient meeting of the Myasthenia Gravis Foundation of America (MGFA), evaluating the impact of MG\u2010related symptoms and MG treatments on patients' lives. Second, an online focus group of 13 patients with MG was conducted. Third, a potential outcome measure was selected. Fourth, the selected PCOM was evaluated by patients to assess how completely and accurately it captured their experiences with MG.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The patient survey showed that limb weakness had the most impact on patients' lives. Weight gain, mood swings, insomnia, and diarrhea were the most bothersome treatment side effects. Avoiding hospitalization was very important. Focus group participants reported fatigue as one of the most bothersome symptoms and differentiated it from myasthenic weakness. They defined an ideal treatment as having minimal or no side effects and an 80% improvement in symptoms.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>Based on patient input, the 15\u2010item Myasthenia Gravis Quality of Life\u2014Revised (MG\u2010QOL15R) scale, a validated patient\u2010reported outcome measure (PRO), was selected as the primary PCOM for PROMISE\u2010MG. Avoiding hospitalization and having minimal to no treatment adverse effects were selected as additional outcome measures. The patient\u2010centeredness of a PRO depends on the context of a study: PROs should be evaluated for appropriateness as a PCOM for every study.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27443","type":"journal-article","created":{"date-parts":[[2021,10,23]],"date-time":"2021-10-23T09:37:58Z","timestamp":1634981878000},"page":"75-81","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Identifying a patient\u2010centered outcome measure for a comparative effectiveness treatment trial in myasthenia gravis"],"prefix":"10.1002","volume":"65","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9412-7349","authenticated-orcid":false,"given":"Pushpa","family":"Narayanaswami","sequence":"first","affiliation":[{"name":"Department of Neurology Beth Israel Deaconess Medical Center\/Harvard Medical School  Boston Massachusetts USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Donald B.","family":"Sanders","sequence":"additional","affiliation":[{"name":"Department of Neurology Duke University Medical Center  Durham North Carolina USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kathie","family":"Bibeau","sequence":"additional","affiliation":[{"name":"Myasthenia Gravis Foundation of America  Seattle Washington USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andrew","family":"Krueger","sequence":"additional","affiliation":[{"name":"Accordant Health Services  Greensboro North Carolina USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jurgen","family":"Venitz","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutics Virginia Commonwealth University School of Pharmacy  Richmond Virginia USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeffrey T.","family":"Guptill","sequence":"additional","affiliation":[{"name":"Department of Neurology Duke University Medical Center  Durham North Carolina USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2021,11,2]]},"reference":[{"key":"e_1_2_10_2_1","volume-title":"BEST (Biomarkers, EndpointS, and other Tools) Resource [Internet]","author":"FDA\u2010NIH Biomarker Working Group","year":"2016"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1212\/CPJ.0000000000000438"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1097\/CND.0000000000000156"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdv181"},{"key":"e_1_2_10_6_1","unstructured":"US Food and Drug Administration. Value and use of patient reported outcomes (PROs) in assessing effects of medical devices. CDRH strategic priorities 2016\u20132017.https:\/\/www.fda.gov\/files\/about%20fda\/published\/value-and-use-of-patient-reported-outcomes-%28pros%29-in-assessing-effects-of-medical-devices.pdf. Accessed September 6 2021."},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25198"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.52.7.1487"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002790"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/1475-6773.12275"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/S40037-019-0509-2"},{"key":"e_1_2_10_12_1","unstructured":"Common Terminology Criteria for Adverse Effects. U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES National Institutes of Health National Cancer Institute; 2010.https:\/\/evs.nci.nih.gov\/ftp1\/ctcae\/ctcae_4.03\/ctcae_4.03_2010-06-14_quickreference_5x7.pdf. Accessed September 6 2021."},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21016"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21883"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.21609"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27443","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27443","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27443","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,25]],"date-time":"2023-08-25T16:59:41Z","timestamp":1692982781000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27443"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,11,2]]},"references-count":14,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2022,1]]}},"alternative-id":["10.1002\/mus.27443"],"URL":"https:\/\/doi.org\/10.1002\/mus.27443","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,11,2]]},"assertion":[{"value":"2021-06-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-10-18","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-11-02","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}