{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T03:56:11Z","timestamp":1761796571634,"version":"build-2065373602"},"reference-count":12,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T00:00:00Z","timestamp":1761782400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmaceuticals"],"abstract":"<jats:p>Background: Eptacog beta is a novel recombinant activated factor VII (rFVIIa). Preclinical studies have shown the product has a similar profile to eptacog alfa. The PERSEPT 1 and PERSEPT 3 trials proved the efficacy and safety of eptacog beta for surgical prophylaxis and treatment of bleed in patients with haemophilia A or B with inhibitors. This led to approval of the product by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Recently, real-world data have been published on the use of eptacog beta on this population which has provided positive outcomes. Given the similar in vitro and in vivo profile of eptacog beta and eptacog alfa positive outcomes could be expected not only in patients with haemophilia and inhibitors, but also in other situations where eptacog alfa is a therapeutic option. Methods: We report on the use of this product for on-label and off-label populations in two Spanish hospitals. Results: We describe four cases in an on-label population (haemophilia A with inhibitors) and four cases in another population (one with acquired haemophilia and three with mild factor VII deficiency). Conclusions: Our experience provides further evidence of the efficacy and safety of eptacog beta for surgical prophylaxis and treatment of bleeding in patients with haemophilia A with inhibitors, but also in those with factor VII deficiency. To our knowledge, this is the first report to describe the use of eptacog beta for factor VII deficiency and acquired haemophilia.<\/jats:p>","DOI":"10.3390\/ph18111640","type":"journal-article","created":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T03:44:39Z","timestamp":1761795879000},"page":"1640","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real-World Experience with Eptacog Beta for On-Label and Off-Label Indications: The Spanish Experience"],"prefix":"10.3390","volume":"18","author":[{"ORCID":"https:\/\/orcid.org\/0009-0002-0342-3677","authenticated-orcid":false,"given":"Jose Manuel","family":"Martin de Bustamante","sequence":"first","affiliation":[{"name":"Department of Haematology, Hospital Universitario la Paz, 28046 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-6322-0659","authenticated-orcid":false,"given":"Mar\u00eda Isabel","family":"Rivas-Pollmar","sequence":"additional","affiliation":[{"name":"Department of Haematology, Hospital Universitario la Paz, 28046 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Natalia","family":"Acedo","sequence":"additional","affiliation":[{"name":"Department of Haematology, Hospital Universitario la Princesa, 28006 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4601-7150","authenticated-orcid":false,"given":"Nora","family":"Butta","sequence":"additional","affiliation":[{"name":"Department of Haematology, Hospital Universitario la Paz, 28046 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3937-3499","authenticated-orcid":false,"given":"Victor","family":"Jimenez-Yuste","sequence":"additional","affiliation":[{"name":"Department of Haematology, Hospital Universitario la Paz, 28046 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria Teresa","family":"\u00c1lvarez-Rom\u00e1n","sequence":"additional","affiliation":[{"name":"Department of Haematology, Hospital Universitario la Paz, 28046 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2025,10,30]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"736","DOI":"10.1016\/S0140-6736(24)02139-1","article-title":"Haemophilia","volume":"405","author":"Chowdary","year":"2025","journal-title":"Lancet"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1111\/hae.14046","article-title":"WFH Guidelines for the Management of Hemophilia, 3rd edition","volume":"26","author":"Srivastava","year":"2020","journal-title":"Haemophilia"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"102670","DOI":"10.1016\/j.rpth.2024.102670","article-title":"Recombinant factor VIIa: New insights into the mechanism of action through product innovation","volume":"9","author":"Escobar","year":"2024","journal-title":"Res. Pract. Thromb. Haemost."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1111\/hae.14944","article-title":"Eptacog beta for the management of patients with haemophilia A and B with inhibitors: A European perspective","volume":"30","author":"Miesbach","year":"2024","journal-title":"Haemophilia"},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1111\/hae.13110","article-title":"Kinetic analysis and binding studies of a new recombinant human factor VIIa for treatment of haemophilia","volume":"23","author":"Grandoni","year":"2017","journal-title":"Haemophilia"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"832","DOI":"10.1111\/hae.13301","article-title":"PERSEPT 1: A phase 3 trial of activated eptacog beta for on-demand treatment of haemophilia inhibitor-related bleeding","volume":"23","author":"Wang","year":"2017","journal-title":"Haemophilia"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"911","DOI":"10.1111\/hae.14418","article-title":"PERSEPT 3: A phase 3 clinical trial to evaluate the haemostatic efficacy of eptacog beta (recombinant human FVIIa) in perioperative care in subjects with haemophilia A or B with inhibitors","volume":"27","author":"Escobar","year":"2021","journal-title":"Haemophilia"},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"1321","DOI":"10.1111\/hae.15094","article-title":"Real-world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi-institutional case series","volume":"30","author":"Youkhana","year":"2024","journal-title":"Haemophilia"},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"715","DOI":"10.1080\/17474086.2023.2248385","article-title":"Efficacy and safety evaluation of eptacog beta (coagulation factor VIIa [recombinant]-jncw) for the treatment of hemophilia A and B with inhibitors","volume":"16","author":"Pipe","year":"2023","journal-title":"Expert Rev. Hematol."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"211","DOI":"10.1111\/hae.14247","article-title":"Outcomes for studies assessing the efficacy of hemostatic therapies in persons with congenital bleeding disorders","volume":"27","author":"Aquino","year":"2021","journal-title":"Haemophilia"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1177\/10600280211049394","article-title":"Eptacog Beta for Bleeding Treatment and Prevention in Congenital Hemophilia A and B With Inhibitors: A Review of Clinical Data and Implications for Clinical Practice","volume":"56","author":"Ciolek","year":"2022","journal-title":"Ann. Pharmacother."},{"key":"ref_12","first-page":"518","article-title":"Bleeding in patients with hemophilia who have inhibitors: Modeling US medical system utilization and cost avoidance between recombinant factor VIIa products with different clinical dosing requirements","volume":"28","author":"Alexander","year":"2022","journal-title":"J. Manag. Care Spec Pharm."}],"container-title":["Pharmaceuticals"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1424-8247\/18\/11\/1640\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T03:53:04Z","timestamp":1761796384000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1424-8247\/18\/11\/1640"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,10,30]]},"references-count":12,"journal-issue":{"issue":"11","published-online":{"date-parts":[[2025,11]]}},"alternative-id":["ph18111640"],"URL":"https:\/\/doi.org\/10.3390\/ph18111640","relation":{},"ISSN":["1424-8247"],"issn-type":[{"value":"1424-8247","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,10,30]]}}}