Prophylaxis and therapy with factor VII concentrate (human) immuno, vapor heated in patients with congenital factor VII deficiency: A summary of case reports

Abstract

Hereditary factor VII deficiency is a rare autosomal recessive condition, usually associated with normal or reduced levels of a functionally defective molecule. The available means of treating this condition in North America presents serious health risks to the patient. Transfusion with fresh frozen plasma carries a risk of volume overload and a significant risk for viral transmission. Sustained prothrombin complex therapy is associated with a high risk for thrombogenic complications. This communication describes the use of Factor VII Concentrate (Human) Immuno, Vapor Heated--an intermediate purity factor VII concentrate from Immuno A.G.--for the treatment of 13 patients with factor VII deficiency. Treatment regimens described include those for long-term prophylaxis (three children), acute hemorrhages (two children, one adult), peripartum prophylaxis (one patient), and surgical coverage (two children, four adults). Prophylaxis and therapy were successful in all cases, the medication was well-tolerated, and there were no complications. In the three cases of long-term prophylaxis in children, doses of 10-50 IU/kg were given one to three times a week; one patient has undergone long-term prophylaxis for approximately 8 years, one patient for 1 year, and one patient for 1 1/2 years. Three cases in which Factor VII Concentrate was principally used for treatment of acute episodes of bleeding are described. One infant received Factor VII Concentrate on about 50 occasions for treatment of mucosal bleeding; a correction to 40-100% resulted in cessation of bleeding within 15 min in all cases. For treatment of an episode of intracranial bleeding, an 8-year-old boy received a dose of 37 IU/kg Factor VII Concentrate every 6 hr for peak factor VII levels of approximately 100% and troughs as low as 4% over the 11-day treatment period. A 37-year-old adult male with intracranial bleeding received alternating doses of 16 IU/kg and 8 IU/kg every 6 hr for 10 days with peak factor VII levels in the upper thirties (%). The peak favor VII level during surgical coverage with Factor VII Concentrate (neurosurgery, open reduction of ankle bones, dental surgery, pituitary adenoma surgery, closed liver biopsy) was approximately 100% in all cases, with trough levels ranging from 8 to 65% over treatment periods of 24 hr to 16 days using treatment intervals of 6-12 hr.

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

“…Importantly, central nervous system bleeding was the most frequent indication for prophylaxis (16/34 individuals, 47%) followed by hemarthrosis [12/34 (35%)], a finding consistent with the literature. [13][14][15][16][18][19][20][21][22][23]26 The severity of the clinical picture is also highlighted by the age at disease presentation [within the first year of life in 18/34 (53%) cases] and the high number of different bleeding symptoms reported in many of the patients. Clotting phenotypes were consistent with the clinical phenotypes, as indicated by the low to very low residual FVIIc levels.…”

“…[8][9][10][11] Clinical experience with prophylaxis in FVII deficiency has been limited to individual case reports, mostly not published in full. [12][13][14][15][16][17][18][19][20][21][22][23][24][25][26][27][28][29] The Seven Treatment Evaluation Registry (STER, www.targetseven.org) has collected information on prophylaxis from a worldwide series of well-characterized FVII-deficient patients. Here we report an analysis of prophylaxis in these patients, focusing…”

The rare coagulation disorders – review with guidelines for management from the United Kingdom Haemophilia Centre Doctors' Organisation

“…one to three times a week (grade B recommendations based on level III evidence). Although illogical when once considers the short half-life of FVII, in practice it appears successful [103]. rFVIIa -In the study of Mariani et al, rFVIIa was used to treat 17 patients (FVII:C <1% in nine patients and <5% in an additional five patients) with 27 spontaneous bleeding episodes who in addition also underwent seven major and 13 minor surgical procedures [104].…”

Recessively inherited coagulation disorders

“…Two commercial manufacturers produce virusinactivated FVII concentrates, used successfully in small series of patients. 86,87 The recommended starting dose is 30 to 40 U/kg, repeated every 12 hours as necessary (Table 6).…”