Treatment of Factor VII Deficiency with Recombinant Factor VIla

Bauer, Kenneth A.

doi:10.1159/000217259

Cited by 37 publications

(34 citation statements)

References 9 publications

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“…These data were confirmed by the ROC analysis, which indicated that haemostasis could be secured by single doses of at least 13 lg/kg and three or more administrations/ day. The result obtained through the ROC analysis was in agreement with previous observations stemming from compassionate use studies or clinical observations (Bauer, 1996;Mariani et al, 1999). Therefore, the low-dose RT schedule administered appears the most plausible explanation for the reported bleeding events.…”

Section: Discussionsupporting

confidence: 89%

Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation – the surgical STER

et al. 2010

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SummaryExcessive bleeding represents a major complication of surgical interventions and its control is especially relevant in patients with Congenital Bleeding Disorders (CBD). In factor VII (FVII) deficiency, scanty data on surgery is available to guide treatment strategies. The STER (Seven Treatment Evaluation Registry) is a multi-centre, prospective, observational, webbased study protocol providing the frame for a structured and detailed data collection. Inhibitor occurrence was checked in a centralized fashion. Fortyone surgical operations (24 'major' and 17 'minor') were performed in 34 subjects with a carefully characterized FVII deficiency under the coverage of recombinant activated Factor VII (rFVIIa). Bleeding occurred during three major interventions of orthopaedic surgery, but rFVIIa was given at very low dose in each case. An antibody to FVII was observed in one patient who underwent a multiple dental extraction. No thromboses were reported during the 30-d follow up period. Replacement therapy with rFVIIa proved effective when suitable doses were used, which, during the period of maximum bleeding risk (the day of operation), were calculated (Receiver Operated Characteristic analysis) to be of at least 13 lg/kg/body weight per single dose and no less than three administrations. This indication is important especially in the case of major surgery.

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Section: Discussionsupporting

confidence: 89%

Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation – the surgical STER

et al. 2010

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“…As the presence of rVIIa is thought to be required when haemostasis is challenged, prophylaxis with rVIIa would seem unlikely to succeed because of its short half-life in blood. On-demand therapy with rVIIa for FVII deficiency has been described before in small studies and case reports (Bauer, 1996;Ingerslev et al, 1997;Mariani et al, 1999;Hunault & Bauer, 2000;Berrettini et al, 2001;Charpiat et al, 2002;Eskandari et al, 2002). Furthermore, rVIIa has been administered for many indications, such as haemophilia A and B with inhibitors, for which the product was originally developed, surgical procedures or trauma in otherwise healthy patients (Hedner, 2003).…”

Section: Patient 1 Patient 2 Patientmentioning

confidence: 97%

Prophylactic effect of recombinant factor VIIa in factor VII deficient patients

Mathijssen¹,

Masereeuw

Verbeek³

et al. 2004

Br J Haematol

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Summary Inherited factor VII (FVII) deficiency is a rare autosomal recessive disorder associated with a bleeding tendency. We describe three patients with congenital FVII deficiency who have been treated with activated recombinant factor VII (rVIIa). Two patients had novel mutations and were treated prophylactically with 1·2 mg rVIIa two to three times a week. Patients 1 and 2 had a severe bleeding tendency. The frequency and severity of bleeding decreased by treatment with rVIIa compared with similar treatment with plasma‐derived FVII. The third patient with a moderate bleeding phenotype was treated on demand and showed no change in the frequency of bleeding upon treatment with rVIIa or plasma products. The beneficial effect of rVIIa cannot be explained by the rVIIa half‐lives. Pharmacokinetical analysis showed rVIIa activity half‐lives of 35, 50 and 54 min for patients 1, 2 and 3, respectively. In conclusion, prophylactic treatment of FVII deficient patients with rVIIa appears to be applicable, safe and successful, although the mechanism of action remains to be elucidated.

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“…Only one factor VII-deficient patient has developed anti-factor VII antibodies after treatment with rfVIIa. 45 …”

Section: Use Of Factor Viia In Patients With Other Hereditary Clottinmentioning

confidence: 99%

The use of recombinant factor VIIa in the treatment of bleeding disorders

Roberts

Monroe

White

2004

Blood

252

207

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Recombinant factor VIIa was initially developed for the treatment of hemorrhagic episodes in hemophilic patients with inhibitors to factors VIII and IX. After its introduction, it has also been used "off-label" to enhance hemostasis in nonhemophilic patients who experience bleeding episodes not responsive to conventional therapy. Evidence so far indicates that the use of factor VIIa in hemophilic patients with inhibitors is both safe and effective. Anecdotal reports also suggest that the product is safe and effective in controlling bleeding in nonhemophilic patients. However, its use in these conditions has not been approved by the FDA, and conclusive evidence of its effectiveness from controlled clinical trials is not yet available. Several questions pertaining to the use of factor VIIa require further investigation, including the mechanism of action; the optimal dose; definitive indications; ultimate safety; and laboratory tests for monitoring therapy. (Blood.

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Treatment of Factor VII Deficiency with Recombinant Factor VIla

Cited by 37 publications

References 9 publications

Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation – the surgical STER

Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation – the surgical STER

Prophylactic effect of recombinant factor VIIa in factor VII deficient patients

The use of recombinant factor VIIa in the treatment of bleeding disorders

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