{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T21:12:15Z","timestamp":1791925935850,"version":"4.3.4"},"reference-count":28,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,11,27]],"date-time":"2024-11-27T00:00:00Z","timestamp":1732665600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Platelet transfusion is considered the standard treatment for preventing or controlling severe haemorrhage in Glanzmann thrombasthenia (GT). However, platelet transfusion can have detrimental effects, including the production of anti\u2010GPIIb\/IIIa isoantibodies or anti\u2010HLA antibodies (Ab) and platelet transfusion refractoriness. Recombinant activated factor VII (rFVIIa) has been proposed as an alternative treatment to platelet transfusion.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We analyzed data from 77 case reports including 100 subjects to investigate the effectiveness and safety of rFVIIa in combination with platelets or antifibrinolytics for preventing or treating non\u2010surgical bleeds, as well as surgical and obstetrical procedures in GT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The dosage of rFVIIa was consistent with previous recommendations (90\u00a0\u00b5g\/kg per infusion). In subjects without Ab\/refractoriness (<jats:italic>n<\/jats:italic>\u00a0=\u00a056), rFVIIa was effective in managing 93% of non\u2010surgical bleeds (<jats:italic>n<\/jats:italic>\u00a0=\u00a042), 91% of minor (<jats:italic>n<\/jats:italic>\u00a0=\u00a011) and 92% of major (<jats:italic>n<\/jats:italic>\u00a0=\u00a026) surgical procedures and 89% of obstetrical procedures (<jats:italic>n<\/jats:italic>\u00a0=\u00a09). In subjects with Ab\/refractoriness (<jats:italic>n<\/jats:italic>\u00a0=\u00a044), rFVIIa was effective in managing 90% of non\u2010surgical bleeds (<jats:italic>n<\/jats:italic>\u00a0=\u00a039), and 75% of minor (<jats:italic>n<\/jats:italic>\u00a0=\u00a012) and 100% of major (<jats:italic>n<\/jats:italic>\u00a0=\u00a017) surgical procedures. The use of rFVIIa was safe, with 4 (2.7%) serious adverse events associated with rFVIIa.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Although the use of rFVIIa is currently restricted to subjects with Ab\/refractoriness or when platelets are not available, our findings suggest expanding the indications for rFVIIa to encompass GT without Ab\/refractoriness. Frontline use of rFVIIa may be proposed when clinically possible to mitigate the risks associated with platelet transfusion.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.15130","type":"journal-article","created":{"date-parts":[[2024,11,28]],"date-time":"2024-11-28T05:34:29Z","timestamp":1732772069000},"page":"7-15","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Efficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review"],"prefix":"10.1111","volume":"31","author":[{"given":"Paul","family":"Saultier","sequence":"first","affiliation":[{"name":"Department of Pediatric Hematology Immunology and Oncology Aix Marseille Univ, APHM, INSERM, INRAe, C2VN, La Timone Children's Hospital  Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michel","family":"Grino","sequence":"additional","affiliation":[{"name":"Reference Center for Inherited Platelet Disorders Aix Marseille Univ, APHM, INSERM, INRAe, C2VN  Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C\u00e9line","family":"Falaise","sequence":"additional","affiliation":[{"name":"Reference Center of Platelet Disorders Department of Pediatric Hematology Immunology and Oncology APHM  Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Voisin","sequence":"additional","affiliation":[{"name":"Hematology Laboratory Reference Center for Inherited Platelet Disorders Toulouse University Hospital  Toulouse France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C\u00e9cile","family":"Lavenu\u2010Bombled","sequence":"additional","affiliation":[{"name":"Department of Hematology APHP, INSERM, Hemostasis, HITh, U1176 Resource and Competence Center for Constitutional Hemorrhagic Disorders University of Paris\u2010Saclay Bic\u00eatre Hospital  Le Kremlin\u2010Bic\u00eatre 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