{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,27]],"date-time":"2026-03-27T21:08:19Z","timestamp":1774645699321,"version":"3.50.1"},"reference-count":51,"publisher":"The Endocrine Society","issue":"12","license":[{"start":{"date-parts":[[2024,10,24]],"date-time":"2024-10-24T00:00:00Z","timestamp":1729728000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004095","name":"Kyowa Kirin","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004095","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,10,29]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Objective<\/jats:title>\n                  <jats:p>To examine the real-world clinical and healthcare resource burden of familial hypophosphatemia (FH).<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>In a retrospective, observational cohort study using MarketScan claims data from 2017 to 2021, clinical characteristics and healthcare resource utilization (HCRU) and costs were compared between burosumab-na\u00efve pediatric and adult patients with \u2265 1 FH diagnosis code and matched controls without FH. Patient characteristics were evaluated at baseline, and disease characteristics, HCRU, and costs were evaluated over a 12-month follow-up period. Outcomes were analyzed descriptively. Costs were additionally analyzed using multivariate regression models.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>Overall, 570 patients with FH and 1710 non-FH matched controls were included. Approximately 10% of study participants were aged &amp;lt;\u202f18 years. Patients with FH had 7.8-fold higher mean baseline comorbidity (Charlson Comorbidity Index). The prevalence of morbidities over the 12-month follow-up period was higher in patients with FH than controls, including renal disease (33% vs 3%), arthralgia (25% vs 10%), osteoarthritis (17% vs 6%), and delayed growth\/walking difficulty (16% vs 2%; all P &amp;lt; .001). All-cause HCRU was significantly greater for patients with FH than controls over follow-up, including the proportion of patients with at least one inpatient admission (60% vs 4%), outpatient emergency room visit (52% vs 16%), and outpatient pharmacy prescription (96% vs 71%; all P &amp;lt; .001). The mean annual total healthcare cost per patient was also 22.6-fold higher for patients with FH than controls (adjusted cost difference = $129 643; P &amp;lt; .001). Differences were apparent across all age groups.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>Compared with non-FH matched controls, burosumab-na\u00efve patients with FH experienced multiple morbidities and had substantially higher HCRU and costs.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1210\/jendso\/bvae185","type":"journal-article","created":{"date-parts":[[2024,10,24]],"date-time":"2024-10-24T18:33:46Z","timestamp":1729794826000},"source":"Crossref","is-referenced-by-count":6,"title":["Real-World Clinical and Healthcare Resource Burden Among Burosumab-Na\u00efve Patients With Familial Hypophosphatemia"],"prefix":"10.1210","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7284-3467","authenticated-orcid":false,"given":"Erik A","family":"Imel","sequence":"first","affiliation":[{"name":"Departments of Medicine and Pediatrics, Indiana University School of Medicine , Indianapolis, IN 46202 ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-0779-9720","authenticated-orcid":false,"given":"Zhiyi","family":"Li","sequence":"additional","affiliation":[{"name":"Kyowa Kirin, Inc. , Princeton, NJ 08540 ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-3088-2883","authenticated-orcid":false,"given":"Heather M","family":"Heerssen","sequence":"additional","affiliation":[{"name":"Kyowa Kirin, Inc. , Princeton, NJ 08540 ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nicole","family":"Princic","sequence":"additional","affiliation":[{"name":"Merative , Ann Arbor, MI 48108 ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hana","family":"Schwartz","sequence":"additional","affiliation":[{"name":"Merative , Ann Arbor, MI 48108 ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yang","family":"Zhao","sequence":"additional","affiliation":[{"name":"Kyowa Kirin, Inc. , Princeton, NJ 08540 ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9980-9138","authenticated-orcid":false,"given":"Kathryn M","family":"Dahir","sequence":"additional","affiliation":[{"name":"Department of Endocrinology, Vanderbilt University Medical Center , Nashville, TN 37232 ,","place":["USA"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"80","published-online":{"date-parts":[[2024,10,24]]},"reference":[{"issue":"1","key":"2025021210520975100_bvae185-B1","doi-asserted-by":"crossref","first-page":"172","DOI":"10.1186\/s13023-020-01434-4","article-title":"The international X-linked hypophosphataemia (XLH) registry (NCT03193476): rationale for and description of an international, observational study","volume":"15","author":"Padidela","year":"2020","journal-title":"Orphanet J Rare Dis"},{"key":"2025021210520975100_bvae185-B2","author":"National Organization for Rare Disorders (NORD)"},{"issue":"6","key":"2025021210520975100_bvae185-B3","doi-asserted-by":"crossref","first-page":"366","DOI":"10.1038\/s41574-022-00662-x","article-title":"Interdisciplinary management of FGF23-related phosphate wasting syndromes: a consensus statement on the evaluation, diagnosis and care of patients with X-linked hypophosphataemia","volume":"18","author":"Trombetti","year":"2022","journal-title":"Nat Rev Endocrinol"},{"issue":"12","key":"2025021210520975100_bvae185-B4","doi-asserted-by":"crossref","first-page":"bvaa151","DOI":"10.1210\/jendso\/bvaa151","article-title":"X-linked hypophosphatemia: a new era in management","volume":"4","author":"Dahir","year":"2020","journal-title":"J Endocr Soc"},{"key":"2025021210520975100_bvae185-B5","article-title":"A literature review of the healthcare resource use and productivity burden of X-linked hypophosphatemia","author":"Pinedo-Villanueva"},{"issue":"7","key":"2025021210520975100_bvae185-B6","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1038\/s41581-019-0152-5","article-title":"Clinical practice recommendations for the diagnosis and management of x-linked hypophosphataemia","volume":"15","author":"Haffner","year":"2019","journal-title":"Nat Rev Nephrol"},{"issue":"2","key":"2025021210520975100_bvae185-B7","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1007\/s40272-020-00381-8","article-title":"New developments in the treatment of X-linked hypophosphataemia: implications for clinical management","volume":"22","author":"Saraff","year":"2020","journal-title":"Paediatr Drugs"},{"issue":"10","key":"2025021210520975100_bvae185-B8","doi-asserted-by":"crossref","first-page":"1937","DOI":"10.1007\/s00198-021-05997-1","article-title":"Burden of disease and clinical targets in adult patients with X-linked hypophosphatemia. 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