{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,11]],"date-time":"2025-12-11T20:47:29Z","timestamp":1765486049573},"reference-count":56,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2016,8,31]],"date-time":"2016-08-31T00:00:00Z","timestamp":1472601600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100009431","name":"Baxalta","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100009431","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2016,11]]},"abstract":"<jats:p>The safety and efficacy of treatment options for patients with haemophilia have significantly improved over the last two decades, particularly with greater utilization of prophylactic approaches. Consequently, it is becoming increasingly difficult to differentiate the treatment benefits of available choices based on standard endpoints such as annualized bleeding rates and joint health scores. Patient\u2010reported outcomes (<jats:styled-content style=\"fixed-case\">PRO<\/jats:styled-content>s) have shown limited ability to discriminate between treatment outcomes, in part because of their comprehensive nature; i.e. differences in specific outcomes meaningful to individual patients are masked by a global scoring system based on a fixed set of items, many of which may be unimportant for any given patient. There is a clear need for new outcome measures. Initiatives to develop patient\u2010centric outcomes that capture clinically meaningful change are ongoing. One such approach, goal attainment scaling (<jats:styled-content style=\"fixed-case\">GAS<\/jats:styled-content>), allows patients, in collaboration with a trained clinician, to select goals from a medical condition\u2010specific menu of options and subsequently facilitates quantitative assessment of goal realization. Thus, it is fully personalized and sensitive to small, often idiosyncratic, treatment benefits, such as improvements in functional capacity. In this paper, we present the underlying rationale for <jats:styled-content style=\"fixed-case\">GAS<\/jats:styled-content> and one other novel approach to <jats:styled-content style=\"fixed-case\">PRO<\/jats:styled-content> personalization, and discuss their potential to augment current outcome measures by reliably detecting and quantifying treatment effects in individuals with haemophilia on prophylaxis.<\/jats:p>","DOI":"10.1111\/hae.13066","type":"journal-article","created":{"date-parts":[[2016,9,1]],"date-time":"2016-09-01T03:16:43Z","timestamp":1472699803000},"page":"825-832","source":"Crossref","is-referenced-by-count":39,"title":["Recognizing the need for personalization of haemophilia patient\u2010reported outcomes in the prophylaxis era"],"prefix":"10.1111","volume":"22","author":[{"given":"M.","family":"Recht","sequence":"first","affiliation":[{"name":"The Hemophilia Center Oregon Health &amp; Science University Portland OR USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"B. 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