{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T12:43:35Z","timestamp":1790772215768,"version":"4.1.0"},"reference-count":61,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,5,27]],"date-time":"2023-05-27T00:00:00Z","timestamp":1685145600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["pericles.pericles-gcp.literatumonline.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,7]]},"abstract":"Abstract<\/jats:title>\n \n Introduction<\/jats:title>\n The real\u2010world effectiveness of the efmoroctocog alfa (recombinant FVIII Fc fusion protein, a rFVIIIFc) has been investigated in numerous studies, however, currently, there exists no comprehensive collection of the existing real\u2010world evidence (RWE) on the performance of prophylactic use of rFVIIIFc.<\/jats:p>\n <\/jats:sec>\n \n Aim<\/jats:title>\n The aims of this systematic literature study were to identify, review, evaluate and collate the RWE of prophylactic rFVIIIFc for patients with haemophilia A reported in Europe.<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n We searched Medline and Embase from 2014 to February 2022 to identify publications reporting the effectiveness of rFVIIIFc in patients with haemophilia A. The outcomes of interest were annualised bleeding rates (ABR, AjBR, AsBR), injection frequency, factor consumption, adherence, development of inhibitors and quality\u2010of\u2010life measures.<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n 46 eligible publications (eight full\u2010text articles) were included. rFVIIIFc showed a low ABR in patients with haemophilia A. Studies assessing treatment switching from a standard half\u2010life (SHL) treatment to rFVIIIFc found that the ABR and consumption were reduced in most patients. Studies assessing rFVIIIFc effectiveness reported a median ABR between 0.0 and 2.0 with median injections per week ranging between 1.8 and 2.4 and median doses between 60 and 105\u00a0IU\/kg\/week. Of the studies assessing inhibitor development, only one study reported an incidence of a low titre inhibitor, and no patients developed clinically significant inhibitors.<\/jats:p>\n <\/jats:sec>\n \n Conclusion<\/jats:title>\n rFVIIIFc prophylaxis treatment results in a low ABR across studies in patients with haemophilia A in a European real\u2010world setting, which correlates with findings from clinical trials assessing the efficacy of rFVIIIFc in patients with haemophilia A.<\/jats:p>\n <\/jats:sec>","DOI":"10.1111\/hae.14797","type":"journal-article","created":{"date-parts":[[2023,5,27]],"date-time":"2023-05-27T14:34:23Z","timestamp":1685198063000},"page":"963-974","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Real\u2010world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe"],"prefix":"10.1111","volume":"29","author":[{"given":"Jan","family":"Blatn\u00fd","sequence":"first","affiliation":[{"name":"Department of Paediatric Haematology and Biochemistry University Hospital Brno and Masaryk University Brno Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma Munk","family":"Nielsen","sequence":"additional","affiliation":[{"name":"EY Godkendt Revisionspartnerselskab Frederiksberg Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2839-4510","authenticated-orcid":false,"given":"Signe Baattrup","family":"Reitzel","sequence":"additional","affiliation":[{"name":"EY Godkendt Revisionspartnerselskab Frederiksberg Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annabell Cajus","family":"McMillan","sequence":"additional","affiliation":[{"name":"EY Godkendt Revisionspartnerselskab Frederiksberg Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne","family":"Dan\u00f8","sequence":"additional","affiliation":[{"name":"EY Godkendt Revisionspartnerselskab Frederiksberg Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda","family":"Bystrick\u00e1","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nana","family":"Kragh","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4194-8183","authenticated-orcid":false,"given":"Robert","family":"Klamroth","sequence":"additional","affiliation":[{"name":"Department for Internal Medicine Vivantes Hospital Friedrichshain, Haemophilia treatment centre Berlin Germany"},{"name":"Institute of Experimental Haematology and Transfusion Medicine University Hospital Bonn Medical Faculty, University of Bonn Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,5,27]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1177\/20406207221104591"},{"key":"e_1_2_12_3_1","unstructured":"World Federation of Hemophilia.Report on the Annual Global Survey 2016.2017."},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.4274\/tjh.galenos.2019.2018.0393"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-10-529974"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13953"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12911"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pmed.1000100"},{"key":"e_1_2_12_9_1","unstructured":"Healthcare Systems in the USA and Europe: Differences Challenges Trends \u2010 BEKEY [cited 2023 Mar 13]. Available from:https:\/\/bekey.io\/blog\/healthcare\u2010systems\u2010in\u2010the\u2010usa\u2010and\u2010europe\u2010differences\u2010challenges\u2010trends"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13643-016-0384-4"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijsu.2010.02.007"},{"key":"e_1_2_12_12_1","first-page":"3","article-title":"Postive impact of switching previously treated patients (PTPS) to recombinant factor VIII FC fusion protein (rFVIIIFc) in the annualized bleeding rate (ABR): real world experience (abstract)","volume":"27","author":"Benitez Hidalgo O","year":"2021","journal-title":"Haemophilia"},{"key":"e_1_2_12_13_1","first-page":"35","article-title":"Recombinant factor VIII Fc (rFVIIIFc) in real life: clinical and economic outcomes (abstract)","volume":"25","author":"Delmotte N","year":"2019","journal-title":"Haemophilia"},{"key":"e_1_2_12_14_1","first-page":"35","article-title":"Clinical impact and pharmacoeconomic analysis of efmoroctocog alfa treatment in patients with severe hemophilia A (abstract)","volume":"25","author":"Garcia MF","year":"2019","journal-title":"Haemophilia"},{"key":"e_1_2_12_15_1","first-page":"32","article-title":"Use of extended half life factor VIII products in children with severe hemophilia A on prophylaxis: a single centre experience (abstract)","volume":"24","author":"Komitopoulou A","year":"2018","journal-title":"Haemophilia"},{"key":"e_1_2_12_16_1","first-page":"27","article-title":"Surgical outcomes in patients with severe haemophilia receiving rFVIIIFc and rFIXFc: real\u2010world experience in the Nordic countries (abstract)","volume":"26","author":"Lehtinen A\u2010E","year":"2020","journal-title":"Haemophilia"},{"key":"e_1_2_12_17_1","first-page":"1","article-title":"Comparison of FVIII prophylaxis treatment regimen and associated clinical outcomes between rVIII\u2010singlechain and other rFVIII products commonly used in Italian hemophilia A patients (abstract)","volume":"4","author":"Mancuso ME","year":"2020","journal-title":"Res Pract Thromb Haemost"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2019-127946"},{"key":"e_1_2_12_19_1","first-page":"1","article-title":"Pharmacokinetic and clinical improvements after pk\u2010guided switch from standard half\u2010life to extended half\u2010life factor VIII products (abstract)","volume":"4","author":"Megias\u2010Vericat JE","year":"2020","journal-title":"Res Pract Thromb Haemost"},{"key":"e_1_2_12_20_1","first-page":"1","article-title":"Real world switching from conventional FVIII products to RFVIIIFC in France, Germany, Italy and the UK (poster)","author":"Myren K\u2010J","year":"2018","journal-title":"HemaSphere"},{"key":"e_1_2_12_21_1","first-page":"24","article-title":"Prophylactic FVIII treatment regimen and clinical outcomes in German haemophilia A patients: comparison of rVIII\u2010SingleChain and other rFVIII products (abstract)","volume":"26","author":"Olivieri M","year":"2020","journal-title":"Haemophilia"},{"key":"e_1_2_12_22_1","first-page":"24","article-title":"Adherence to prophylaxis with recombinant factor VIII Fc in haemophilia A patients (abstract)","volume":"26","author":"Pitance V","year":"2020","journal-title":"Haemophilia"},{"key":"e_1_2_12_23_1","first-page":"35","article-title":"Switching toward the use of recombinant factor VIII Fc fusion proteinStudy among 30 patients with severe hemophilia A (abstract)","volume":"78","author":"Sattler L","year":"2020","journal-title":"Ann Biol Clin (Paris)"},{"key":"e_1_2_12_24_1","article-title":"Within\u2010patient comparison of treatment patterns before and after switching to rFVIIIFc: a report from the UK national haemophilia database (abstract)","volume":"24","author":"Scott M","year":"2018","journal-title":"Haemophilia"},{"key":"e_1_2_12_25_1","article-title":"The effect of switching to rFVIIIFc on treatment patterns and annualised bleed rate before and after: a within\u2010patient comparison from the UK National haemophilia database (abstract)","volume":"24","author":"Scott M","year":"2018","journal-title":"Haemophilia"},{"key":"e_1_2_12_26_1","article-title":"The impact of switching previously treated patients (PTPs) to recombinant factor VIII FC fusion protein (rFVIIIFc) in reducing the annualized bleeding rate (ABR): a single center experience (abstract)","volume":"4","author":"Benitez O","year":"2020","journal-title":"Res Pract Thromb Haemost"},{"key":"e_1_2_12_27_1","article-title":"Perioperative management of patients with severe haemophilia a with recombinant factor VIII FC fusion protein (rFVIIIFc) (abstract)","volume":"27","author":"Bocchinfuso S","year":"2021","journal-title":"Haemophilia"},{"key":"e_1_2_12_28_1","article-title":"Years ofpprophylaxis with rFVIII\u2010Fc in haemophilia in a portuguese haemophilia center: did doctors and patients see a change? (abstract)","author":"Campanico S","year":"2021","journal-title":"Res Pract Thromb Haemost"},{"key":"e_1_2_12_29_1","article-title":"Scottish experience introducing extended half\u2010life factor concentrates in haemophilia A and haemophilia B patients (abstract)","author":"Chalmers E","year":"2018","journal-title":"Haemophilia"},{"key":"e_1_2_12_30_1","article-title":"Interim analysis of the prevent study: real world prospective data from children and adolescents with haemophilia A or B treated with recombinant factor VIII FC (rFVIIIFc) or recombinant factor IX FC (rFIXFc) (abstract)","author":"Heller C","year":"2021","journal-title":"Res Pract Thromb Haemost"},{"key":"e_1_2_12_31_1","article-title":"Real\u2010world usage of rFVIIIFc in Sweden: a report from the Swedish National Registry for bleeding disorders (abstract)","volume":"26","author":"Holmstrom M","year":"2020","journal-title":"Haemophilia"},{"key":"e_1_2_12_32_1","article-title":"Real\u2010world use of FVIII in people with severe hemophilia A without inhibitor on prophylaxis, comparing switched to rFVIII\u2010FC versus non\u2010switched patients: a multicentre, retrospective, non interventional, before\u2010after study with the French nationwide claims database (MOTHIF\u2010II) (abstract)","author":"Horvais V","year":"2021","journal-title":"Res Pract Thromb Haemost"},{"key":"e_1_2_12_33_1","article-title":"Rfviiifc: a good choice for children with hemophilia. a single center experience (abstract)","author":"Izzo T","year":"2017","journal-title":"Blood Transfus"},{"key":"e_1_2_12_34_1","article-title":"Switch to extended half\u2010life recombinant factor VIII FC in severe hemophilia A patients under prophylaxis. 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