Efficacy and safety of a new human fibrinogen concentrate in patients with congenital fibrinogen deficiency: an interim analysis of a Phase III trial

Basit Öğe Kaydı
dc.contributor.authorLissitchkov, Toshko
dc.contributor.authorMadan, Bella
dc.contributor.authorKhayat, Claudia Djambas
dc.contributor.authorZozulya, Nadezhda
dc.contributor.authorRoss, Cecil
dc.contributor.authorKarimi, Mehran
dc.contributor.authorKavakli, Kaan
dc.contributor.authorDe Angulo, Guillermo R.
dc.contributor.authorAlmomen, Abdulkareem
dc.contributor.authorSchwartz, Bruce A.
dc.contributor.authorSolomon, Cristina
dc.contributor.authorKnaub, Sigurd
dc.contributor.authorPeyvandi, Flora
dc.date.accessioned2019-10-27T10:43:11Z
dc.date.available2019-10-27T10:43:11Z
dc.date.issued2018
dc.departmentEge Üniversitesien_US
dc.description.abstractBACKGROUNDFibrinogen concentrate is the preferred choice for fibrinogen replacement in congenital fibrinogen deficiency. This study investigated hemostatic efficacy of a new plasma-derived, double virus-inactivated (using two dedicated virus inactivation/elimination steps) human fibrinogen concentrate for on-demand treatment of bleeding episodes (BEs) and surgical prophylaxis. STUDY DESIGN AND METHODSIn this planned interim analysis of a prospective, multinational Phase III study (NCT02267226), 13 patients with afibrinogenemia (12 years) received fibrinogen concentrate (FIBRYGA, Octapharma AG). Hemostatic efficacy was assessed by investigators and an independent data monitoring and endpoint adjudication committee (IDMEAC) using objective four-point criteria and by thromboelastometry maximum clot firmness (MCF). RESULTSFibrinogen concentrate was used on-demand to treat 23 BEs in 11 patients, with 21 (91.3%) requiring a single infusion only. Treatment success was 95.7% (90% confidence interval [CI], 0.81-1.00; assessment missing for one BE) by investigators and 100% (90% CI, 0.88-1.00) by IDMEAC. Mean MCF increased significantly from 0.0 to 6.5 mm (95% CI, 5.65-7.40; p<0.0001) at 1 hour postinfusion of a median (range) dose of 58.8 (33.9-101.7) mg/kg per BE. Four patients received fibrinogen concentrate as surgical prophylaxis, with intraoperative and postoperative treatment success rated 100% (90% CI, 0.50-1.00) by investigators and IDMEAC (median [range] dose per surgery 93.5 [34.1-225.4] mg/kg). No additional hemostatic interventions were required. No deaths, thromboses, or seroconversions were reported. CONCLUSIONThese data showed that the new fibrinogen concentrate was efficacious for on-demand treatment of acute bleeding and surgical prophylaxis in congenital afibrinogenemia patients.en_US
dc.identifier.doi10.1111/trf.14421en_US
dc.identifier.endpage422en_US
dc.identifier.issn0041-1132
dc.identifier.issn1537-2995
dc.identifier.issue2en_US
dc.identifier.pmid29194665en_US
dc.identifier.scopusqualityQ2en_US
dc.identifier.startpage413en_US
dc.identifier.urihttps://doi.org/10.1111/trf.14421
dc.identifier.urihttps://hdl.handle.net/11454/30731
dc.identifier.volume58en_US
dc.identifier.wosWOS:000424395000021en_US
dc.identifier.wosqualityQ2en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherWileyen_US
dc.relation.ispartofTransfusionen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.titleEfficacy and safety of a new human fibrinogen concentrate in patients with congenital fibrinogen deficiency: an interim analysis of a Phase III trialen_US
dc.typeArticleen_US

Dosyalar

Koleksiyon

WoS İndeksli Yayınlar Koleksiyonu
PubMed İndeksli Yayın Koleksiyonu
Scopus İndeksli Yayınlar Koleksiyonu