title: Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER).
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 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 μg/kg total weekly dose were effective. These data provide a rationale for long-term, safe prophylaxis in factor VII deficiency.
authors: University of L'Aquila, Dipartimento di Medicina Interna e Sanità Pubblica, L'Aquila, Italy. marysanta@libero.it
authors: Mariasanta
authors: M
authors: Napolitano
authors: Muriel
authors: M
authors: Giansily-Blaizot
authors: Alberto
authors: A
authors: Dolce
authors: Jean F
authors: JF
authors: Schved
authors: Guenter
authors: G
authors: Auerswald
authors: Jørgen
authors: J
authors: Ingerslev
authors: Jens
authors: J
authors: Bjerre
authors: Carmen
authors: C
authors: Altisent
authors: Pimlak
authors: P
authors: Charoenkwan
authors: Lisa
authors: L
authors: Michaels
authors: Ampaiwan
authors: A
authors: Chuansumrit
authors: Giovanni
authors: G
authors: Di Minno
authors: Umran
authors: U
authors: Caliskan
authors: Guglielmo
authors: G
authors: Mariani
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005167:Factor VII / Q000627:therapeutic use*; D005168:Factor VII Deficiency / Q000517:prevention & control*; D015942:Factor VIIa / Q000235:genetics / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D010949:Plasma; D011994:Recombinant Proteins / Q000627:therapeutic use; D012042:Registries / Q000706:statistics & numerical data; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; D005167:Factor VII; D015942:Factor VIIa
references: Haemophilia. 2008 Jul;14(4):851-2
references: 18399984
references: J Thromb Haemost. 2009 May;7(5):774-9
references: 19245420
references: Orphanet J Rare Dis. 2010 Sep 07;5:23
references: 20822539
references: Am J Hematol. 1995 Dec;50(4):269-76
references: 7485101
references: Am J Hematol. 1981;10(1):79-88
references: 7258184
references: Clin Appl Thromb Hemost. 2012 Nov;18(6):588-93
references: 22327826
references: Blood. 2009 Aug 27;114(9):1974-86
references: 19587380
references: Thromb Haemost. 2005 Mar;93(3):481-7
references: 15735798
references: J Med Assoc Thai. 2002 Nov;85 Suppl 4:S1059-64
references: 12549776
references: J Thromb Haemost. 2005 Apr;3(4):799-800
references: 15842376
references: Haemostasis. 1996;26 Suppl 1:98-101
references: 8904182
references: J Thromb Haemost. 2011 Apr;9(4):700-10
references: 21255253
references: Southeast Asian J Trop Med Public Health. 2000 Dec;31(4):795-800
references: 11414431
references: Pediatr Hematol Oncol. 2006 Mar;23(2):129-33
references: 16651241
references: Pediatr Blood Cancer. 2007 Oct 15;49(5):736-9
references: 16317734
references: Br J Haematol. 2004 May;125(4):494-9
references: 15142120
references: Thromb Haemost. 2013 Feb;109(2):238-47
references: 23238632
references: Blood Coagul Fibrinolysis. 2003 Feb;14(2):217-20
references: 12632035
references: N Engl J Med. 2007 Aug 9;357(6):535-44
references: 17687129
references: Br J Haematol. 2011 Feb;152(3):340-6
references: 21158750
references: Haematologica. 2001 Jun;86(6):640-5
references: 11418374
references: Semin Thromb Hemost. 2009 Jun;35(4):400-6
references: 19598068
references: Haemophilia. 2006 Jan;12(1):19-27
references: 16409171
references: Haemophilia. 2011 Jul;17(4):713-5
references: 21299748
references: Haemophilia. 2010 Jul 1;16(4):569-83
references: 19906159
references: Blood. 2011 Mar 17;117(11):3199-208
references: 21252088
references: Haemophilia. 2012 Jan;18(1):e25-7
references: 21973016
references: J Thromb Haemost. 2007 Jul;5(7):1403-8
references: 17425666
references: Haemophilia. 2004 May;10(3):295-8
references: 15086331
references: J Thromb Haemost. 2003 Oct;1(10):2153-8
references: 14521598
references: J Thromb Haemost. 2007 Feb;5(2):433-4
references: 17109737
references: Eur J Haematol. 2007 Oct;79(4):354-9
references: 17692102
references: Haemophilia. 2007 Sep;13 Suppl 2:4-9
references: 17685917