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Efficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review.

dc.rights.licenseopenen_US
dc.contributor.authorSAULTIER, Paul
dc.contributor.authorGRINO, Michel
dc.contributor.authorFALAISE, Céline
dc.contributor.authorVOISIN, Sophie
dc.contributor.authorLAVENU-BOMBLED, Cécile
dc.contributor.authorIBRAHIM-KOSTA, Manal
dc.contributor.authorPETIT, Audrey
dc.contributor.authorBOUTROUX, Hélène
dc.contributor.authorDESPREZ, Dominique
hal.structure.identifierBiologie des maladies cardiovasculaires = Biology of Cardiovascular Diseases
dc.contributor.authorFIORE, Mathieu
dc.contributor.authorD'OIRON, Roseline
dc.contributor.authorALESSI, Marie-Christine
dc.date.accessioned2024-12-09T10:54:45Z
dc.date.available2024-12-09T10:54:45Z
dc.date.issued2024-11-27
dc.identifier.issn1365-2516en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/203794
dc.description.abstractEnPlatelet 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-GPIIb/IIIa isoantibodies or anti-HLA antibodies (Ab) and platelet transfusion refractoriness. Recombinant activated factor VII (rFVIIa) has been proposed as an alternative treatment to platelet transfusion. 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-surgical bleeds, as well as surgical and obstetrical procedures in GT. The dosage of rFVIIa was consistent with previous recommendations (90 µg/kg per infusion). In subjects without Ab/refractoriness (n = 56), rFVIIa was effective in managing 93% of non-surgical bleeds (n = 42), 91% of minor (n = 11) and 92% of major (n = 26) surgical procedures and 89% of obstetrical procedures (n = 9). In subjects with Ab/refractoriness (n = 44), rFVIIa was effective in managing 90% of non-surgical bleeds (n = 39), and 75% of minor (n = 12) and 100% of major (n = 17) surgical procedures. The use of rFVIIa was safe, with 4 (2.7%) serious adverse events associated with rFVIIa. 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.
dc.language.isoENen_US
dc.subject.enGlanzmann thrombasthenia; antifibrinolytics; antiplatelet antibodies; platelet refractoriness; platelet transfusion; rFVIIa.
dc.title.enEfficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review.
dc.title.alternativeHaemophiliaen_US
dc.typeArticle de revueen_US
dc.identifier.doi10.1111/hae.15130en_US
dc.subject.halSciences du Vivant [q-bio]/Médecine humaine et pathologieen_US
dc.identifier.pubmed39604156en_US
bordeaux.journalHaemophiliaen_US
bordeaux.hal.laboratoriesBiologie des maladies cardiovasculaires (BMC) - UMR 1034en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.institutionINSERMen_US
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
bordeaux.import.sourcepubmed
hal.identifierhal-04826676
hal.version1
hal.date.transferred2024-12-09T10:54:47Z
hal.popularnonen_US
hal.audienceInternationaleen_US
hal.exporttrue
workflow.import.sourcepubmed
dc.rights.ccPas de Licence CCen_US
bordeaux.COinSctx_ver=Z39.88-2004&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.jtitle=Haemophilia&rft.date=2024-11-27&rft.eissn=1365-2516&rft.issn=1365-2516&rft.au=SAULTIER,%20Paul&GRINO,%20Michel&FALAISE,%20C%C3%A9line&VOISIN,%20Sophie&LAVENU-BOMBLED,%20C%C3%A9cile&rft.genre=article


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