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Title: Prophylaxis in congenital factor VII deficiency: Indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER)
Authors: Mariasanta Napolitano
Muriel Giansily-Blaizot
Alberto Dolce
Jean F. Schved
Guenter Auerswald
Jørgen Ingerslev
Jens Bjerre
Carmen Altisent
Pimlak Charoenkwan
Lisa Michaels
Ampaiwan Chuansumrit
Giovanni Di Minno
Ümran Caliskan
Guglielmo Mariani
Universita degli Studi dell'Aquila
CHU Montpellier
Istituto Nazionale Di Statistica, Rome
Klinikum Bremen-Mitte
Skejby Sygehus, Aarhus University Hospital
Novo Nordisk AS
Hospital Universitari Vall d'Hebron
Chiang Mai University
Robert Wood Johnson University
Mahidol University
Universita degli Studi di Napoli Federico II
Selcuk Universitesi
University of Ferrara
Keywords: Medicine
Issue Date: 1-Apr-2013
Citation: Haematologica. Vol.98, No.4 (2013), 538-544
Abstract: Because 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 freshfrozen 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 μg/kg total weekly dose were effective. These data provide a rationale for long-term, safe prophylaxis in factor VII deficiency ( NCT01269138). © 2013 Ferrata Storti Foundation.
ISSN: 15928721
Appears in Collections:Scopus 2011-2015

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