1. Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER)
- Author
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Ampaiwan Chuansumrit, Jens Bjerre, Lisa Michaels, M. Napolitano, Pimlak Charoenkwan, G. Mariani, Muriel Giansily-Blaizot, Umran Caliskan, Guenter Auerswald, Carmen Altisent, Jørgen Ingerslev, Giovanni Di Minno, Alberto Dolce, Jean-François Schved, Selçuk Üniversitesi, Napolitano,M, Giansily-Bliazot,M, Dolce, A, Schved,JF, Auerswald,G, Ingerrslev,J, Bjerre,J, Altisent,C, Charoenkwan,P, Micheals,L, Chuansumrit,A, Di Minno,G, Caliskan, U, Mariani, G, Napolitano, Mariasanta, Giansily Blaizot, Muriel, Dolce, Alberto, Schved, Jean F., Auerswald, Guenter, Ingerslev, Jørgen, Bjerre, Jen, Altisent, Carmen, Charoenkwan, Pimlak, Michaels, Lisa, Chuansumrit, Ampaiwan, DI MINNO, Giovanni, Caliskan, Ümran, and Mariani, Guglielmo
- Subjects
Registrie ,Severe bleeding ,Adult ,Male ,Pediatrics ,medicine.medical_specialty ,Time Factors ,Time Factor ,Adolescent ,Factor VII Deficiency ,PROPHYLAXIS, FACTOR VII DEFICIENCY ,Factor VIIa ,chemistry.chemical_compound ,Plasma ,Young Adult ,Medicine ,Humans ,Registries ,Young adult ,Factor VII deficiency ,Child ,Factor VII ,business.industry ,Infant ,Hematology ,Recombinant Protein ,Middle Aged ,medicine.disease ,Thrombosis ,Recombinant Proteins ,Clinical trial ,Treatment Outcome ,chemistry ,Treatment evaluation ,Weekly dose ,Child, Preschool ,Female ,Original Articles and Brief Reports ,business ,Human - Abstract
WOS: 000319897700018, PubMed: 23403322, 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 (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., Novo Nordisk and charities; Novo Nordisk A/SNovo Nordisk, This 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.
- Published
- 2013