{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T11:34:27Z","timestamp":1792668867701,"version":"4.5.0"},"reference-count":26,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Hematology"],"published-print":{"date-parts":[[2023,1]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>The bypassing agent, activated prothrombin complex concentrate [aPCC, FEIBA (factor VIII inhibitor bypass activity); Baxalta US Inc, a Takeda company, Lexington, MA, USA], is indicated for the treatment of bleeding episodes, perioperative management, and routine prophylaxis in patients with hemophilia A or B with inhibitors. In certain countries, aPCC is also indicated for the treatment of bleeding episodes and perioperative management in patients with acquired hemophilia A.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives:<\/jats:title>\n                    <jats:p>To describe long-term, real-world effectiveness, safety, and quality-of-life outcomes for patients with congenital hemophilia A or B and high-responding inhibitors receiving aPCC treatment in routine clinical practice.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design:<\/jats:title>\n                    <jats:p>FEIBA Global Outcome (FEIBA GO; EUPAS6691) was a prospective, observational study.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Investigators determined the treatment regimen and clinical monitoring frequency. The planned patient observation period was 4 years. Data are from the safety analysis set (patients who received \u2a7e1 aPCC infusion).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Overall, 50 patients received either aPCC prophylaxis ( n\u2009=\u200937) or on-demand therapy ( n\u2009=\u200913) at screening [hemophilia A, n\u2009= 49; hemophilia B, n\u2009=\u20091; median (range) age, 16.5 [2\u201371] years). Mean\u2004\u00b1\u2004standard deviation overall annualized bleeding rate and annualized joint bleeding rate for patients receiving prophylaxis were 6.82\u2009\u00b1\u200911.52 and 3.77\u2009\u00b1\u20095.71, respectively, and for patients receiving on-demand therapy were 10.94\u2009\u00b1\u200911.27 and 6.94\u2009\u00b1 7.39, respectively. Overall, 177 and 31 adverse events (AEs) were reported in 28 of 40 and 10 of 13 patients receiving prophylaxis or on-demand therapy, respectively. Two serious AEs were considered possibly related to aPCC: acute myocardial infarction due to coronary artery embolism in one patient receiving prophylaxis. No thrombotic microangiopathy was reported. No AEs resulted in death.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>This study demonstrated the long-term, real-world effectiveness and consistent safety profile of aPCC as on-demand therapy and prophylactic treatment in patients with hemophilia and high-responding inhibitors.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial registry:<\/jats:title>\n                    <jats:p>FEIBA Global Outcome Study; EUPAS6691 https:\/\/www.encepp.eu\/encepp\/viewResource.htm?id=32774<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/20406207231184323","type":"journal-article","created":{"date-parts":[[2023,7,24]],"date-time":"2023-07-24T06:27:27Z","timestamp":1690180047000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":9,"title":["Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study"],"prefix":"10.1177","volume":"14","author":[{"given":"Carmen Escuriola","family":"Ettingshausen","sequence":"first","affiliation":[{"name":"H\u00e4mophilie-Zentrum Rhein Main \u2013 HZRM GmbH, M\u00f6rfelden-Walldorf, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5429-8437","authenticated-orcid":false,"given":"Cedric","family":"Hermans","sequence":"additional","affiliation":[{"name":"Hemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, UCLouvain, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P\u00e5l A.","family":"Holme","sequence":"additional","affiliation":[{"name":"Department of Haematology, Oslo University Hospital and Institute for Clinical Medicine, University of Oslo, Oslo, Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana R.","family":"Cid","sequence":"additional","affiliation":[{"name":"Unidad de Hemostasia y Trombosis, Hospital Universitario y Polit\u00e9cnico La Fe, Val\u00e8ncia, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kate","family":"Khair","sequence":"additional","affiliation":[{"name":"Centre for Outcomes and Experience Research in Children\u2019s Health, Illness and Disability (ORCHID), Great Ormond Street Hospital for Children, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannes","family":"Oldenburg","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine, Bonn University Clinic, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claude","family":"N\u00e9grier","sequence":"additional","affiliation":[{"name":"University Claude Bernard Lyon 1, Lyon, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaco","family":"Botha","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals International AG, Z\u00fcrich, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aurelia","family":"Lelli","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals International AG, Z\u00fcrich, Switzerland"},{"name":"R&D, Plasma Derived Therapy, Takeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Glattpark-Opfikon, Z\u00fcrich, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerzy","family":"Windyga","sequence":"additional","affiliation":[{"name":"Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2023,7,24]]},"reference":[{"key":"bibr1-20406207231184323","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"bibr2-20406207231184323","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-09-457036"},{"key":"bibr3-20406207231184323","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019000658"},{"key":"bibr4-20406207231184323","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.239160"},{"key":"bibr5-20406207231184323","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.13193"},{"key":"bibr6-20406207231184323","doi-asserted-by":"publisher","DOI":"10.1111\/jth.12990"},{"key":"bibr7-20406207231184323","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2011.02692.x"},{"key":"bibr8-20406207231184323","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.24856"},{"key":"bibr9-20406207231184323","unstructured":"Baxalta US Inc. 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