{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,14]],"date-time":"2026-02-14T02:45:52Z","timestamp":1771037152589,"version":"3.50.1"},"reference-count":18,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,5,29]],"date-time":"2021-05-29T00:00:00Z","timestamp":1622246400000},"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":["Haemophilia"],"published-print":{"date-parts":[[2021,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Emicizumab is the first approved non\u2010factor therapy for haemophilia A. It provides superior prophylactic bleeding control compared to other products in both patients with and patients without inhibitors. However, there is no real\u2010world data about the monetary consequences of starting emicizumab.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To examine the estimated costs of starting emicizumab in a cohort of real\u2010world haemophilia A patients with and without inhibitors.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The cost of haemostatic therapy for 6\u00a0months before and after initiating emicizumab for participants in a multicentre observational study was calculated based on the type of product and dosing that was used for prophylaxis and treating breakthrough bleeds, the number of treated bleeds and the participant weight.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Ninety\u2010two patients were included, 18 with an active inhibitor. The median age was 8.7\u00a0years. The median total cost for all patients decreased from $176,720 to $128,099 (<jats:italic>p\u00a0=<\/jats:italic>\u00a0.04) after initiating emicizumab, largely because of decrease in the total cost of high\u2010cost outliers. The cost of prophylaxis and the total cost of bleeds also significantly decreased after starting emicizumab, both for patient with and patients without inhibitors.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Starting or switching to prophylaxis with emicizumab results in decreased costs for the treatment of patients with haemophilia A. This real\u2010world data could inform on payer decisions as well as future cost\u2010effective analysis.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14347","type":"journal-article","created":{"date-parts":[[2021,6,3]],"date-time":"2021-06-03T11:19:18Z","timestamp":1622719158000},"page":"591-598","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":21,"title":["Real\u2010world cost estimates of initiating emicizumab in US patients with haemophilia A"],"prefix":"10.1111","volume":"27","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6772-4140","authenticated-orcid":false,"given":"Benjamin J.","family":"Samelson\u2010Jones","sequence":"first","affiliation":[{"name":"The Children\u2019s Hospital of Philadelphia University of Pennsylvania Philadelphia PA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9226-2347","authenticated-orcid":false,"given":"Christine","family":"Guelcher","sequence":"additional","affiliation":[{"name":"Children\u2019s National Hospital George Washington University Washington DC USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5067-2943","authenticated-orcid":false,"given":"Jan","family":"Kuhn","sequence":"additional","affiliation":[{"name":"Virginia Commonwealth University Richmond VA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Regina","family":"Butler","sequence":"additional","affiliation":[{"name":"The Children\u2019s Hospital of Philadelphia University of Pennsylvania Philadelphia PA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gita","family":"Massey","sequence":"additional","affiliation":[{"name":"Virginia Commonwealth University Richmond VA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael F.","family":"Guerrera","sequence":"additional","affiliation":[{"name":"Children\u2019s National Hospital George Washington University Washington DC USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8152-1040","authenticated-orcid":false,"given":"Leslie","family":"Raffini","sequence":"additional","affiliation":[{"name":"The Children\u2019s Hospital of Philadelphia University of Pennsylvania Philadelphia PA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2021,5,29]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-08-742312"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703068"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1803550"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019001869"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14005"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.27886"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13618"},{"key":"e_1_2_7_9_1","first-page":"2020","article-title":"Valoctocogene roxaparvovec and emicizumab for hemophilia A: effectiveness and value; 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