Clinical efficacy of highly purified, doubly virus‐inactivated factor VIII/von Willebrand factor concentrate (Fanhdi^®) in the treatment of von Willebrand disease: a retrospective clinical study

Abstract

The goal of therapy in patients with von Willebrand disease (vWD) is to correct the dual defect of primary haemostasis and intrinsic coagulation reflected by low levels of von Willebrand factor (vWF) and factor VIII coagulant activity (FVIII:C). Factor VIII/von Willebrand factor (FVIII/vWF) concentrates are currently the treatment of choice in vWD patients unresponsive to desmopressin (DDAVP). However, only few studies on their clinical use are available so far. The main objective of this study was to retrospectively evaluate the clinical efficacy of a highly purified, doubly virus-inactivated FVIII/vWF concentrate with a high content of FVIII/vWF (Fanhdi). Twenty-two patients with congenital vWD have been treated from 1999 to 2001 at eight specialized centres belonging to the Italian Association of Hemophilia Centers (AICE). Ten males and 12 females, median age 28.5 years, range 5-70 years) had type 3 vWD (six cases), DDAVP-unresponsive type 1 (nine cases) and type 2B (seven cases). The study drug was given to stop or prevent 12 bleeding episodes or to prevent excessive bleeding during 14 surgical or invasive procedures. Overall, replacement therapy with the concentrate showed an excellent to good clinical efficacy in 92% of bleeding episodes and in 93% of surgical procedures. No adverse events occurred during 1,601 infusions, accounting for a total of 304,500 IU of FVIII:C administered. These results confirm the efficacy and safety of this concentrate in the management of bleeding episodes and in the prevention of excessive bleeding during major and minor surgery.

The bleeding score predicts clinical outcomes and replacement therapy in adults with von Willebrand disease

“…27 The final classification according to VWD types was confirmed by the coordinating center in Milan by also using multimeric analysis and mutations of the VWF gene. 28 DNA was available in all affected patients with VWD2A, VWD2B, VWD2M, and in most VWD3 (82%) and VWD1 (63% 16,29 Three plasma-derived concentrates containing both VWF and FVIII were licensed in Italy for VWD at the time of the study [30][31][32] : Alphanate (Grifols), Fahndi (Grifols), and Haemate-P (CSL-Behring). Patients were treated on demand at each participating center with varying doses of VWF (25-80 IU VWF:RCo/kg) chosen according to sites and severity of bleeds.…”

Pharmacokinetics, Efficacy and Safety of IMMUNATE® Solvent/Detergent (IMMUNATE® S/D) in Previously Treated Patients with Severe Hemophilia A: Results of a Prospective, Multicenter, Open-Label Phase III Study

“…No lack of efficacy was reported. These data demonstrate that IMMUNATE S/D has an efficacy and safety profile favorably comparable to other FVIII products [27,28,29,30]. …”

Endovenous laser ablation of the great saphenous vein in patients with von Willebrand disease

“…The hereditary haemorrhagic diathesis in these patients increases the risk of bleeding during surgical intervention, and follow-up care is more complicated. Through adequate preoperative and postoperative replacement, the coagulation factors were corrected, and the risk of bleeding was minimised [12][13][14][15][16][17][18][19][20] . If venous reflux is not treated appropriately, it can lead to disease progression, the development of superficial thrombophlebitis, vein rupture and bleeding, and the occurrence of venous ulcers and skin lesions.…”

“…In patients with vWD who require surgery or other invasive procedures, it is necessary to adequately maintain haemostasis preoperatively and postoperatively to prevent bleeding 12,13 . Three main therapeutic modalities are used for the treatment of vWD: desmopressin (1-deamino-8-Darginine vasopressin, DDAVP) acetate, plasma derivatives (complex concentrate FVIII/vWF; cryoprecipitate) and antifibrinolytics 2,5,[12][13][14][15][16][17][18][19][20] . If bleeding occurs, it is important to determine whether it resulted from surgery or inadequate haemostasis.…”