{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T23:42:49Z","timestamp":1782949369519,"version":"3.54.5"},"reference-count":29,"publisher":"Georg Thieme Verlag KG","issue":"03","license":[{"start":{"date-parts":[[2020,10,29]],"date-time":"2020-10-29T00:00:00Z","timestamp":1603929600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004191","name":"Novo Nordisk","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100004191","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Thromb Haemost"],"published-print":{"date-parts":[[2021,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Platelet transfusion is the standard treatment to control or prevent bleeding in patients with Glanzmann's thrombasthenia (GT), but platelets are often unavailable. Recombinant activated factor VII (rFVIIa) is an effective alternative to platelets in patients with GT with past\/present refractoriness to platelet transfusions and antibodies to platelets. However, there is an unmet need for an alternative to platelets in patients without antibodies. This report summarizes evidence of efficacy and safety of rFVIIa in patients with GT without refractoriness or antibodies to platelets from three different sources: the Glanzmann's Thrombasthenia Registry (GTR), published literature (January 01, 1999 to December 01, 2017), and the Novo Nordisk safety surveillance database. In the GTR, 133 patients received rFVIIa for the treatment of 333 bleeding episodes and prevention of bleeding in 157 surgical procedures. Overall efficacy rates were 79 and 88%, respectively, in patients treated for bleeding episodes or for the prevention of bleeding during surgery; effectiveness was generally similar across refractoriness\/antibody status categories. Median dose per infusion of rFVIIa was close to that recommended for patients with GT (90 \u00b5g\/kg). Data from 14 published case reports also demonstrated that rFVIIa is effective with an acceptable safety profile in patients with GT without antibodies to platelets. Analysis of adverse events reported in GTR and in Novo Nordisk safety surveillance database did not raise any new safety concerns. These data supported the label extension of rFVIIa to include cases where platelets are not readily available, which was approved by the European Medicines Agency in December 2018.<\/jats:p>","DOI":"10.1055\/s-0040-1718373","type":"journal-article","created":{"date-parts":[[2020,10,30]],"date-time":"2020-10-30T00:32:26Z","timestamp":1604017946000},"page":"332-340","source":"Crossref","is-referenced-by-count":33,"title":["The Use of Recombinant Activated Factor VII in Patients with Glanzmann's Thrombasthenia"],"prefix":"10.1055","volume":"121","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7012-2420","authenticated-orcid":false,"given":"Man-Chiu","family":"Poon","sequence":"additional","affiliation":[{"name":"Department of Medicine, Pediatrics and Oncology, University of Calgary Cumming School of Medicine, Calgary, Alberta, Canada"},{"name":"Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Medical Centre, Calgary, Alberta, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2020,10,29]]},"reference":[{"issue":"07","key":"ref1","doi-asserted-by":"crossref","first-page":"1383","DOI":"10.1182\/blood.V75.7.1383.1383","article-title":"Glanzmann's thrombasthenia: the spectrum of clinical disease","volume":"75","author":"J N George","year":"1990","journal-title":"Blood"},{"issue":"05","key":"ref2","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1016\/j.blre.2017.03.005","article-title":"Should studies on Glanzmann thrombasthenia not be telling us more about cardiovascular disease and other major illnesses?","volume":"31","author":"A T Nurden","year":"2017","journal-title":"Blood Rev"},{"key":"ref3","doi-asserted-by":"crossref","first-page":"219","DOI":"10.2147\/JBM.S71319","article-title":"Glanzmann's thrombasthenia: pathogenesis, diagnosis, and current and emerging treatment options","volume":"6","author":"T Solh","year":"2015","journal-title":"J Blood Med"},{"issue":"02","key":"ref4","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1016\/j.tmrv.2016.01.001","article-title":"New insights into the treatment of Glanzmann thrombasthenia","volume":"30","author":"M C Poon","year":"2016","journal-title":"Transfus Med Rev"},{"issue":"06","key":"ref5","doi-asserted-by":"crossref","first-page":"604","DOI":"10.1055\/s-0038-1648233","article-title":"Alloimmunization in congenital deficiencies of platelet surface glycoproteins: focus on Glanzmann's thrombasthenia and Bernard-Soulier's Syndrome","volume":"44","author":"M C Poon","year":"2018","journal-title":"Semin Thromb Hemost"},{"issue":"05","key":"ref6","doi-asserted-by":"crossref","first-page":"e858","DOI":"10.1111\/j.1365-2516.2011.02516.x","article-title":"A systematic review of the management and outcomes of pregnancy in Glanzmann thrombasthenia","volume":"17","author":"S Siddiq","year":"2011","journal-title":"Haemophilia"},{"issue":"03","key":"ref7","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1016\/S0268-960X(02)00030-9","article-title":"Molecular basis of Glanzmann's thrombasthenia and current strategies in treatment","volume":"16","author":"S Bellucci","year":"2002","journal-title":"Blood Rev"},{"issue":"02","key":"ref8","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/j.tmrv.2009.11.001","article-title":"Blood still kills: six strategies to further reduce allogeneic blood transfusion-related mortality","volume":"24","author":"E C Vamvakas","year":"2010","journal-title":"Transfus Med Rev"},{"issue":"19","key":"ref9","doi-asserted-by":"crossref","first-page":"2025","DOI":"10.1001\/jama.2016.9185","article-title":"Clinical practice guidelines from the AABB: red blood cell transfusion thresholds and storage","volume":"316","author":"J L Carson","year":"2016","journal-title":"JAMA"},{"issue":"08","key":"ref11","first-page":"1038","article-title":"The international, prospective Glanzmann Thrombasthenia Registry: treatment and outcomes in surgical intervention","volume":"100","author":"M C Poon","year":"2015","journal-title":"Haematologica"},{"issue":"08","key":"ref12","first-page":"1031","article-title":"The international, prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes of non-surgical bleeding episodes in patients with Glanzmann thrombasthenia","volume":"100","author":"G Di Minno","year":"2015","journal-title":"Haematologica"},{"issue":"07","key":"ref13","doi-asserted-by":"crossref","first-page":"1096","DOI":"10.1111\/j.1538-7836.2004.00767.x","article-title":"Prophylactic and therapeutic recombinant factor VIIa administration to patients with Glanzmann's thrombasthenia: results of an international survey","volume":"2","author":"M C Poon","year":"2004","journal-title":"J Thromb Haemost"},{"key":"ref14","first-page":"340","article-title":"Recombinant factor VIIa (rFVIIa) use in glanzmann's thrombasthenia (GT) and other platelet disorders (OPDS): hemophilia and thrombosis research society (HTRS) registry data (abstract)","volume":"9","author":"M EN Chitlur","year":"2011","journal-title":"J Thromb Haemost"},{"key":"ref15","first-page":"A26","article-title":"Platelet-free hemotherapy in patients with Bernard Soulier syndrome (BSS) and Glanzmann's thrombasthenia (GT): successful bleeding prophylaxis with recombinant Factor VIIa (rFVIIa) for elective surgery (abstract)","volume":"30","author":"B HT Bomke","year":"2010","journal-title":"Hamostaseologie"},{"key":"ref16","first-page":"203","article-title":"Ovarian endometrioma in two women with Glanzmann's thrombasthenia (abstract)","volume":"18","author":"L Bowles","year":"2012","journal-title":"Haemophilia"},{"key":"ref17","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1016\/j.achaem.2015.07.119","article-title":"Effective therapy with recombinant factor VIIa in female patient suffering from Glanzmann thrombasthenia \u2013 the own experience (abstract)","volume":"46S","author":"B Ceglarek","year":"2015","journal-title":"Acta Haematol Pol"},{"issue":"S 02","key":"ref18","first-page":"839","article-title":"Coexistence of Glanzmann's thrombasthenia and maple syrup urine disease (MSUD): Implications for hemostatic management (abstract)","volume":"102","author":"M Elshinawy","year":"2017","journal-title":"Haematologica"},{"key":"ref19","first-page":"148","article-title":"The use of recombinant FVIIa in two children with Glanzmann's thrombasthenia with severe bleeding after trauma: po-tu-162 (abstract)","volume":"18","author":"A M 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Buuren","year":"2002","journal-title":"Dig Dis Sci"},{"issue":"05","key":"ref27","doi-asserted-by":"crossref","first-page":"644","DOI":"10.1055\/s-0037-1613884","article-title":"Use of recombinant factor VIIa in 3 patients with inherited type I Glanzmann's thrombasthenia undergoing invasive procedures","volume":"83","author":"R d'Oiron","year":"2000","journal-title":"Thromb Haemost"},{"issue":"08","key":"ref28","doi-asserted-by":"crossref","first-page":"641","DOI":"10.1080\/21678707.2017.1341306","article-title":"Glanzmann's thrombasthenia: strategies for identification and management","volume":"5","author":"M-C Poon","year":"2017","journal-title":"Expert Opin Orphan Drugs"},{"issue":"03","key":"ref30","doi-asserted-by":"crossref","first-page":"e286","DOI":"10.1055\/s-0039-1696657","article-title":"The international prospective Glanzmann Thrombasthenia Registry: pediatric treatment and outcomes","volume":"3","author":"R B Zotz","year":"2019","journal-title":"TH Open"},{"issue":"04","key":"ref31","doi-asserted-by":"crossref","first-page":"e275","DOI":"10.1111\/hae.13547","article-title":"Recombinant activated factor VII in approved indications: Update on safety","volume":"24","author":"E J Neufeld","year":"2018","journal-title":"Haemophilia"}],"container-title":["Thrombosis and 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