{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T07:27:05Z","timestamp":1788852425924,"version":"build-2803163510"},"reference-count":31,"publisher":"Oxford University Press (OUP)","issue":"11","license":[{"start":{"date-parts":[[2024,2,22]],"date-time":"2024-02-22T00:00:00Z","timestamp":1708560000000},"content-version":"vor","delay-in-days":113,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2023,9,4]],"date-time":"2023-09-04T00:00:00Z","timestamp":1693785600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"Kyowa Kirin International"},{"DOI":"10.13039\/100013220","name":"Ultragenyx Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013220","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013220","name":"Ultragenyx Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013220","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["asbmr.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2023,11,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Tumor-induced osteomalacia (TIO) is an ultra-rare disease caused by tumors that secrete fibroblast growth factor 23, leading to chronic hypophosphatemia, poor skeletal health, and impaired physical function. In a phase 2 trial (UX023T-CL201; NCT02304367; n\u2009=\u200914), 48\u2009weeks of burosumab treatment restored phosphate homeostasis, with improvements in skeletal health, functional mobility, and patient-reported pain, fatigue, and health-related quality of life (HRQL) (SF-36 v2). Here, we report an exploratory mixed-methods analysis of change from baseline after 144\u2009weeks of burosumab treatment alongside qualitative data from exit interviews with 8 of 14 trial participants to evaluate meaningful treatment effects from a patient perspective. The interview subset (n\u2009=\u20098) reported pain and fatigue and compromised HRQL at baseline. In the interviews, participants reported that compromised HRQL and pain were the most important aspects of the disease to treat; both were considered more bothersome than fatigue and compromised physical function and activities of daily living. Improvements in pain and fatigue after treatment were reported, some of which achieved statistically and\/or clinically meaningful thresholds. Furthermore, improvements in SF-36 v2 scores were most pronounced in the Physical Component Score and its Physical Function and Bodily Pain domains. Overall, the interview subset provided descriptions of symptomatic improvement and its clinical meaningfulness, including physical function, participation in activities of daily living, and mental well-being. Thus, this exploratory mixed-methods analysis provides deeper understanding of patients' perception of clinical meaningfulness beyond that articulated in validated patient-reported outcome instruments. \u00a9 2023 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR).<\/jats:p>","DOI":"10.1002\/jbmr.4900","type":"journal-article","created":{"date-parts":[[2023,8,14]],"date-time":"2023-08-14T07:29:07Z","timestamp":1691998147000},"page":"1654-1664","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":21,"title":["Burosumab Improves Patient-Reported Outcomes in Adults With Tumor-Induced Osteomalacia: Mixed-Methods Analysis"],"prefix":"10.1093","volume":"38","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9386-7732","authenticated-orcid":false,"given":"Suzanne M","family":"Jan de Beur","sequence":"first","affiliation":[{"name":"John Hopkins University School of Medicine Baltimore MD USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tricia","family":"Cimms","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc Novato CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3454-9491","authenticated-orcid":false,"given":"Annabel","family":"Nixon","sequence":"additional","affiliation":[{"name":"Chilli Consultancy Salisbury UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Theodore-Oklota","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc Novato CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diana","family":"Luca","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc Novato CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary Scott","family":"Roberts","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc Novato CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shayna","family":"Egan","sequence":"additional","affiliation":[{"name":"Lumanity Bethesda MD 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