Haemate^®P von Willebrand factor/factor VIII concentrate: 25 years of clinical experience

Abstract

Although von Willebrand disease (VWD) has a very long history, our understanding and treatment of the bleeding disorder has only evolved during the past 50 years or so. It was not until the 1920s that VWD was first recognized as a disease separate from that of classical haemophilia. It then took another 30 years before the first effective treatment was developed. Since then, the medical management of VWD has evolved considerably, but not without its ups and downs. One of the key milestones in the evolution of the treatment of VWD was the development of Haemate P/Humate-P (CSL Behring) - the first virus-inactivated factor VIII plasma product. For 25 years, this concentrate has demonstrated excellent clinical efficacy and safety for patients with VWD and for those with haemophilia. This article provides an historical overview of the early landmark efforts to ensure a safe plasma-derived replacement product and outlines the clinical evolution in the use of Haemate P.

Acquired hemophilia as the cause of life-threatening hemorrhage in a 94-year-old man: a case report

“…Plasma-derived or recombinant human FVIII concentrates can be used in patients with low-titer inhibitors, which should be administered at doses sufficient to overwhelm the inhibitor and thus achieve hemostatic levels of factor VIII [2]. Hemostasis can usually be achieved if plasma levels are raised from 30% to 50% [9,10]. Although Humate-P has been used extensively for treatment of von Willebrand disease, experience with its use in factor VIII inhibitor remains very limited [9,10].…”

“…Hemostasis can usually be achieved if plasma levels are raised from 30% to 50% [9,10]. Although Humate-P has been used extensively for treatment of von Willebrand disease, experience with its use in factor VIII inhibitor remains very limited [9,10]. According to recent recommendations, human plasma-derived or recombinant FVIII concentrates can be used in acquired hemophilia for the treatment of minor bleeding manifestations and acute bleeding episodes when the inhibitor titer is low (≤ 5BU) [2], and no bypassing agent is immediately available, as was the case with our patient.…”

Utility of a high VWF

“…The need for such precautions notwithstanding, it should be born in mind that thromboembolic events in VWD patients receiving VWF/FVIII concentrates have been rare. The long-term safety record of such concentrates [ 35 ] has been further strengthened by a number of recent studies in which no thromboembolic events were encountered [ 30 , 36 – 38 ].…”

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

“…The concentrate (infusion of 4 9 10 3 IU per event, median; 12 9 10 3 IU per patient, median) was given 60 min (median) before the procedure and after surgery (timing of postoperative treatment was dependent on surgery type and patient bleeding tendency) [ Table 4]. These patients received a total of 234 postoperative, surgery-related infusions (median 7.0 per patient, range [1][2][3][4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19]. The median number of postoperative infusions required to treat one event was five (range 1-16).…”

“…Haemate ® P (CSL Behring, Marburg, Germany), a pasteurized VWF/FVIII plasma-derived concentrate in use for almost three decades, has been demonstrated by extensive clinical practice to be an effective and safe treatment for patients with VWD [6,7]. A novel, volume-reduced formulation has been recently developed, maintaining the same characteristics, but with a reduction of 50% of reconstitution volume which could be useful especially when a high dose of VWF/ FVIII is required in a single infusion [7].…”