Efficacy and safety during formulation switch of a pasteurized VWF/FVIII concentrate: results from an Italian prospective observational study in patients with von Willebrand disease

Abstract

Summary. Von Willebrand disease (VWD) is an inherited bleeding disorder caused by the quantitative or qualitative deficiency of von Willebrand factor (VWF). Replacement therapy with plasma-derived VWF/factor VIII (FVIII) concentrates is required in patients unresponsive to desmopressin. To assess the efficacy, safety and ease of use of a new, volume-reduced (VR) formulation of VWF/FVIII concentrate Haemate ® P in patients requiring treatment for bleeding or prophylaxis for recurrent bleeding or for invasive procedures. Pharmacoeconomic variables were also recorded. Data were analysed using descriptive statistics. This was a multicentre, prospective, observational study. Consecutively enrolled patients received Haemate ® P VR according to their needs, and were followed for 24 months. Of the 121 patients enrolled, 25.6% had type 3 VWD and more than 40% had severe disease. All patients were followed for 2 years, for a total of 521 visits. Ondemand treatment was given to 61.9% of patients, secondary long-term prophylaxis to 25.6% and prophylaxis for surgery, dental or invasive procedures to 45.5%. The response to treatment was rated as good to excellent in >93-99% of interventions. The new formulation was well tolerated by all patients with no report of drug-related adverse events. The switch to volume-reduced Haemate ® P was easy to perform and infusion duration was decreased twofold compared with the previous formulation. Volume-reduced Haemate ® P was at least as effective and well-tolerated as the previous formulation.

Practical aspects of factor concentrate use in patients with von Willebrand disease undergoing invasive procedures: a European survey

“…Eighteen studies used concentrate to control bleeding , but one was excluded due to lack of detail. The 17 remaining studies covered 614 patients undergoing 914 surgical procedures (where reported 178 interventions were classified as major and 257 as minor surgery) (see Table S1).…”

“…Two guidelines give recommended dose of concentrate to cover major surgery in terms of dose of FVIII:C. However, a crucial consideration is that different preparations of factor concentrate have different ratios of FVIII:C to VWF:RCo. The manufacturers quote values from 2.4:1 (Haemate) to 0.75:1 (Immunate) in their SMPCs (Table ), but not all countries label VWF/FVIII concentrates with their VWF:RCo content, so in these cases, FVIII content must be used to guide replacement therapy and this may lead to less than optimal dosing of VWF:RCo.…”

Laboratory monitoring of replacement therapy for major surgery in von Willebrand disease

“…All the products except the recombinant VWF lack to a variable degree high molecular weight VWF multimers and also differ regarding the ratio of VWF:RCo to FVIII:C and the virucidal methods employed (Table ). Their use in the setting of major surgery has been evaluated by a number of prospective studies .…”

Prise en charge des patients atteints d’une pathologie de l’hémostase et d’avulsions dentaires : efficacité, tolérance et compliance

“…Cela entraîne un faible nombre de références concernant ce sujet précis [4][5][6][7][8][9]. En revanche, au cours de ces dernières années, de nombreux articles ont été publiés en utilisant un protocole semblable mais en utilisant un concentré VWF/FVIII comme traitement substitutif pour les patients atteints de maladie de Willebrand [10][11][12][13][14]. L'utilisation de ce traitement permet une meilleure prévention hémorragique des patients, notamment ceux résistants à la desmopressine.…”