{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,30]],"date-time":"2026-07-30T02:19:03Z","timestamp":1785377943973,"version":"3.55.0"},"reference-count":44,"publisher":"American Society of Hematology","issue":"7","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2021,4,13]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia B range from mild to severe forms of the disease. The objective of this study was to characterize the economic burden associated with differing clinical profiles of hemophilia B from a US health system perspective. Using the IBM MarketScan database (June 2011-February 2019), a claims-based algorithm was developed to identify 4 distinct profiles (mild, moderate, moderate-severe, and severe) in adult males with hemophilia B based on the frequency of hemorrhage events and factor IX replacement claims. Mean annual health care resource use (HRU) and costs were statistically compared between patients with hemophilia B (N = 454) and 1:1 demographic-matched controls (N = 454), both overall and with stratification by clinical profile. Compared with matched controls, patients with hemophilia B had a significantly higher comorbidity burden (Charlson Comorbidity Index, mean \u00b1 standard deviation [SD]: 0.9 \u00b1 1.7 vs 0.3 \u00b1 0.9, P &amp;lt; .001). Across all clinical profiles, patients with hemophilia B had significantly higher HRU vs matched controls (mean \u00b1 SD: 0.3 \u00b1 0.6 vs 0.1 \u00b1 0.3 inpatient admissions; 0.6 \u00b1 1.2 vs 0.2 \u00b1 0.6 emergency department visits; 17.7 \u00b1 22.9 vs 8.0 \u00b1 11.0 outpatient visits; all P &amp;lt; .001). Annual total health care costs per patient among patients with hemophilia B were more than 25-fold higher vs matched controls (mean \u00b1 SD: $201\u2009635 \u00b1 $411\u2009530 vs $7879 \u00b1 $29\u2009040, respectively, P &amp;lt; .001). Annual total health care costs per patient increased with increasing severity (mean \u00b1 SD: mild, $80\u2009811 \u00b1 $284\u2009313; moderate, $137\u2009455 \u00b1 $222\u2009021; moderate-severe, $251\u2009619 \u00b1 $576\u2009886; severe, $632\u2009088 \u00b1 $501\u2009270). The findings of this study highlight the substantial burden of illness associated with hemophilia B.<\/jats:p>","DOI":"10.1182\/bloodadvances.2020003424","type":"journal-article","created":{"date-parts":[[2021,4,8]],"date-time":"2021-04-08T22:46:11Z","timestamp":1617921971000},"page":"1954-1962","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":31,"title":["Health care resource utilization and cost burden of hemophilia B in the United States"],"prefix":"10.1182","volume":"5","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1954-4385","authenticated-orcid":false,"given":"Tyler W.","family":"Buckner","sequence":"first","affiliation":[{"name":"Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0405-915X","authenticated-orcid":false,"given":"Iryna","family":"Bocharova","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kaitlin","family":"Hagan","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arielle G.","family":"Bensimon","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2741-8418","authenticated-orcid":false,"given":"Hongbo","family":"Yang","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric Q.","family":"Wu","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eileen K.","family":"Sawyer","sequence":"additional","affiliation":[{"name":"uniQure, Inc., Lexington, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nanxin","family":"Li","sequence":"additional","affiliation":[{"name":"uniQure, Inc., Lexington, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","published-online":{"date-parts":[[2021,4,8]]},"reference":[{"key":"2021040815315834500_B1","article-title":"Annual global survey","author":"World Federation of Hemophilia","year":"2017"},{"key":"2021040815315834500_B2","author":"Centers for Disease Control and Prevention"},{"issue":"8","key":"2021040815315834500_B3","doi-asserted-by":"crossref","first-page":"540","DOI":"10.7326\/M19-1208","article-title":"Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries","volume":"171","author":"Iorio","year":"2019","journal-title":"Ann Intern Med"},{"key":"2021040815315834500_B4","author":"National Organization for Rare Disorders"},{"issue":"1","key":"2021040815315834500_B5","doi-asserted-by":"crossref","first-page":"e1","DOI":"10.1111\/j.1365-2516.2012.02909.x","article-title":"Guidelines for the management of hemophilia","volume":"19","author":"Srivastava","year":"2013","journal-title":"Haemophilia"},{"key":"2021040815315834500_B6","volume-title":"GeneReviews","author":"Konkle","year":"2017"},{"issue":"6","key":"2021040815315834500_B7","doi-asserted-by":"crossref","first-page":"592","DOI":"10.1111\/ejh.13055","article-title":"Impact of hemophilia B on quality of life in affected men, women, and caregivers\u2014assessment of patient-reported outcomes in the B-HERO-S study","volume":"100","author":"Buckner","year":"2018","journal-title":"Eur J 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