Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER)

Basit Öğe Kaydı
dc.contributor.authorNapolitano, Mariasanta
dc.contributor.authorGiansily-Blaizot, Muriel
dc.contributor.authorDolce, Alberto
dc.contributor.authorSchved, Jean F.
dc.contributor.authorAuerswald, Guenter
dc.contributor.authorIngerslev, Jorgen
dc.contributor.authorBjerre, Jens
dc.date.accessioned2024-02-23T14:34:51Z
dc.date.available2024-02-23T14:34:51Z
dc.date.issued2013
dc.departmentNEÜen_US
dc.description.abstractBecause of the very short half-life of factor VII, prophylaxis in factor VII deficiency is considered a difficult endeavor. The clinical efficacy and safety of prophylactic regimens, and indications for their use, were evaluated in factor VII-deficient patients in the Seven Treatment Evaluation Registry. Prophylaxis data (38 courses) were analyzed from 34 patients with severe factor VII deficiency (<1-45 years of age, 21 female). Severest phenotypes (central nervous system, gastrointestinal, joint bleeding episodes) were highly prevalent. Twenty-one patients received recombinant activated factor VII (24 courses), four received plasma-derived factor VII, and ten received fresh-frozen plasma. Prophylactic schedules clustered into frequent courses (three times weekly, n=23) and infrequent courses (<= 2 times weekly, n=15). Excluding courses for menorrhagia, frequent and infrequent courses produced 18/23 (78%) and 5/12 (41%) excellent outcomes, respectively; relative risk, 1.88; 95% confidence interval, 0.93-3.79; P=0.079. Long-term prophylaxis lasted from 1 to >10 years. No thrombosis or new inhibitors occurred. In conclusion, a subset of patients with factor VII deficiency needed prophylaxis because of severe bleeding. Recombinant activated factor VII schedules based on frequent administrations (three times weekly) and a 90 mu g/kg total weekly dose were effective. These data provide a rationale for long-term, safe prophylaxis in factor VII deficiency.en_US
dc.description.sponsorshipNovo Nordisk and charities; Novo Nordisk A/Sen_US
dc.description.sponsorshipThis work was supported by institutional research organizations and unrestricted funding from Novo Nordisk and charities, administered by the Internal Medicine Department of the University of L'Aquila. GM, as coordinator of the International FVII Study Group and the STER, collected the financial support. Editorial assistance to the authors during the preparation of this manuscript was provided by Sharon Eastwood (medical writer, PAREXEL) and financially supported by Novo Nordisk A/S, in compliance with international guidelines for good publication practice.en_US
dc.identifier.doi10.3324/haematol.2012.074039
dc.identifier.endpage544en_US
dc.identifier.issn0390-6078
dc.identifier.issue4en_US
dc.identifier.pmid23403322en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage538en_US
dc.identifier.urihttps://doi.org/10.3324/haematol.2012.074039
dc.identifier.urihttps://hdl.handle.net/20.500.12452/15758
dc.identifier.volume98en_US
dc.identifier.wosWOS:000319897700018en_US
dc.identifier.wosqualityQ1en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherFerrata Storti Foundationen_US
dc.relation.ispartofHaematologicaen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subject[Keyword Not Available]en_US
dc.titleProphylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER)en_US
dc.typeArticleen_US

Dosyalar

Koleksiyon

WoS İndeksli Yayınlar Koleksiyonu
PubMed İndeksli Yayın Koleksiyonu