PMID- 37529276 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20230803 IS - 2040-6207 (Print) IS - 2040-6215 (Electronic) IS - 2040-6207 (Linking) VI - 14 DP - 2023 TI - Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study. PG - 20406207231184323 LID - 10.1177/20406207231184323 [doi] LID - 20406207231184323 AB - BACKGROUND: 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. OBJECTIVES: 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. DESIGN: FEIBA Global Outcome (FEIBA GO; EUPAS6691) was a prospective, observational study. METHODS: 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 ⩾1 aPCC infusion). RESULTS: Overall, 50 patients received either aPCC prophylaxis (n = 37) or on-demand therapy (n = 13) at screening [hemophilia A, n = 49; hemophilia B, n = 1; median (range) age, 16.5 [2-71] years). Mean +/- standard deviation overall annualized bleeding rate and annualized joint bleeding rate for patients receiving prophylaxis were 6.82 +/- 11.52 and 3.77 +/- 5.71, respectively, and for patients receiving on-demand therapy were 10.94 +/- 11.27 and 6.94 +/- 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. CONCLUSION: 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. TRIAL REGISTRY: FEIBA Global Outcome Study; EUPAS6691 https://www.encepp.eu/encepp/viewResource.htm?id=32774. CI - (c) The Author(s), 2023. FAU - Ettingshausen, Carmen Escuriola AU - Ettingshausen CE AD - Hamophilie-Zentrum Rhein Main - HZRM GmbH, Morfelden-Walldorf, Germany. FAU - Hermans, Cedric AU - Hermans C AUID- ORCID: 0000-0001-5429-8437 AD - Hemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, UCLouvain, Brussels, Belgium. FAU - Holme, Pal A AU - Holme PA AD - Department of Haematology, Oslo University Hospital and Institute for Clinical Medicine, University of Oslo, Oslo, Norway. FAU - Cid, Ana R AU - Cid AR AD - Unidad de Hemostasia y Trombosis, Hospital Universitario y Politecnico La Fe, Valencia, Spain. FAU - Khair, Kate AU - Khair K AD - Centre for Outcomes and Experience Research in Children's Health, Illness and Disability (ORCHID), Great Ormond Street Hospital for Children, London, UK. FAU - Oldenburg, Johannes AU - Oldenburg J AD - Institute of Experimental Hematology and Transfusion Medicine, Bonn University Clinic, Bonn, Germany. FAU - Negrier, Claude AU - Negrier C AD - University Claude Bernard Lyon 1, Lyon, France. FAU - Botha, Jaco AU - Botha J AD - Takeda Pharmaceuticals International AG, Zurich, Switzerland. FAU - Lelli, Aurelia AU - Lelli A AD - Takeda Pharmaceuticals International AG, Zurich, Switzerland. AD - R&D, Plasma Derived Therapy, Takeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Glattpark-Opfikon, Zurich, Switzerland. FAU - Windyga, Jerzy AU - Windyga J AD - Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland. LA - eng PT - Journal Article DEP - 20230724 PL - England TA - Ther Adv Hematol JT - Therapeutic advances in hematology JID - 101549589 PMC - PMC10387704 OTO - NOTNLM OT - factor VIII inhibitor bypassing activity OT - hemophilia A OT - hemophilia B OT - inhibitors OT - observational study COIS- The authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Carmen Escuriola Ettingshausen: grant/research support [Biotest, CSL Behring, Octapharma, Shire (a Takeda company), Sobi]; honoraria for lectures/advisory boards [Bayer, Biotest, CSL Behring, Grifols, Kedrion, LFB, Novo Nordisk, Octapharma, Pfizer, Roche/Chugai, Shire (a Takeda company), Sobi]; and consultancy [BioMarin, Biotest, CSL Behring, Grifols, Novo Nordisk, Octapharma, Roche/Chugai, Shire (a Takeda company), Sobi]. Cedric Hermans: grant/research support [Bayer, Pfizer, Shire (a Takeda company), Sobi]; honoraria for lectures/advisory boards [Bayer, Biogen, CAF-DCF, CSL Behring, Kedrion, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Shire (a Takeda company), Sobi]; and consultancy [Bayer, Biogen, CAF-DCF, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Shire (a Takeda company), Sobi]. Pal A. Holme: grant/research support [Bayer, Octapharma, Pfizer, Shire (a Takeda company), Sobi]; and honoraria for lectures/advisory boards [Bayer, CSL Behring, Novo Nordisk, Pfizer, Shire (a Takeda company), Sobi]. Ana R. Cid: honoraria for lectures/advisory boards [Novo Nordisk, Roche, Shire (a Takeda company), Sobi]; and consultancy (Roche, Sobi). Kate Khair: grant/research support [Baxalta/Shire (a Takeda company), CSL Behring, Novo Nordisk, Pfizer, Roche, Sobi, uniQure]; honoraria for lectures/advisory boards [Bayer, Novo Nordisk, Pfizer, Roche, Shire (a Takeda company), Sobi]; consultancy [Bayer, Novo Nordisk, Roche, Shire (a Takeda company), Sobi]; support attending meetings and/or travel (Bayer, Novo Nordisk); leadership or fiduciary role in other board, society, committee or advocacy group (Vice President board of Trustees The Haemophilia Society); and stock or stock options (Haemnet Ltd, Medikhair). Johannes Oldenburg: grant/research support [Bayer, Biotest, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Shire (a Takeda company)]; honoraria for lectures/advisory boards [Bayer, Biogen, Biotest, Chugai, CSL Behring, Grifols, Novo Nordisk, Octapharma, Pfizer, Roche, Shire (a Takeda company), Sobi]; and consultancy [Bayer, Biogen, Biotest, Chugai, CSL Behring, Grifols, Novo Nordisk, Octapharma, Pfizer, Roche, Shire (a Takeda company), Sobi]. Claude Negrier: grant/research support [CSL Behring, Octapharma, Shire (a Takeda company), Sobi]; and consultancy [Bayer, BioMarin, CSL Behring, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Shire (a Takeda company), Sobi, Spark]. Jaco Botha: employee of Takeda Pharmaceuticals International AG, and Takeda stock owner. Aurelia Lelli: employee of Takeda Pharmaceuticals International AG, and Takeda stock owner. Jerzy Windyga: grant/research support and honoraria for lectures [Amgen, Alnylam, Baxalta/Shire (a Takeda company), Bayer, CSL Behring, LFB, Novartis, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Siemens, Sobi, Swixx Biopharma]. EDAT- 2023/08/02 06:43 MHDA- 2023/08/02 06:44 PMCR- 2023/07/24 CRDT- 2023/08/02 03:53 PHST- 2022/08/31 00:00 [received] PHST- 2023/06/08 00:00 [accepted] PHST- 2023/08/02 06:44 [medline] PHST- 2023/08/02 06:43 [pubmed] PHST- 2023/08/02 03:53 [entrez] PHST- 2023/07/24 00:00 [pmc-release] AID - 10.1177_20406207231184323 [pii] AID - 10.1177/20406207231184323 [doi] PST - epublish SO - Ther Adv Hematol. 2023 Jul 24;14:20406207231184323. doi: 10.1177/20406207231184323. eCollection 2023.