Wolfgang Miesbach1, Stefanie Krekeler2, Zsuzsanna Wolf2, Erhard Seifried3. 1. University Hospital Frankfurt Haemophilia Centre, Medical Clinic III, Institute of Immunohaematology and Transfusion Medicine, Theodor-Stern-Kai 7, 60596 Frankfurt, Germany. Electronic address: Wolfgang.Miesbach@kgu.de. 2. University Hospital Frankfurt Haemophilia Centre, Medical Clinic III, Institute of Immunohaematology and Transfusion Medicine, Theodor-Stern-Kai 7, 60596 Frankfurt, Germany. 3. Institute of Immunohaematology and Transfusion Medicine, University Hospital Frankfurt, Theodor-Stern-Kai 7, 60596 Frankfurt, Germany.
Abstract
INTRODUCTION: von Willebrand disease (VWD) is caused by dysfunction or diminished levels of von Willebrand factor (VWF). VWF-containing plasma concentrates are used for treatment of patients with VWD for whom desmopressin treatment is insufficient or contraindicated. A single-centre, retrospective observational study over a period of up to 25 years was conducted to evaluate the effectiveness and safety profile of Haemate(®) P (CSL Behring, Marburg, Germany), a plasma-derived, purified, pasteurised and lyophilised VWF-containing factor VIII (FVIII) concentrate. MATERIALS AND METHODS: The study included 71 patients who had been treated with Haemate(®) P over a period of > 5 years. RESULTS: A total of 663 treatments with individual laboratory evaluations were recorded. Two patients had both mild type 1 VWD and haemophilia A (HA), three had HA only, 39 had type 1 VWD, 16 had type 2 VWD, nine had type 3 VWD, one had acquired VWD and one had uncategorised VWD. Haemate(®) P treatment indications included bleeding events (37 patients) and surgical interventions (70 patients). Thirteen patients received Haemate(®) P as long-term prophylaxis (n = 3), as part of rehabilitation treatment (n = 5), or in context of recovery measurements (n = 5). Treatment with Haemate(®) P was generally well tolerated; only one thromboembolic event occurred. In cases of bleeding events, Haemate(®) P was haemostatically effective in all patients. Furthermore, during operations where Haemate(®) P was administered, >90% incurred no complications. CONCLUSION: In this retrospective observational study, Haemate(®) P was shown to be effective and safe for the treatment of VWD in adult patients.
INTRODUCTION:von Willebrand disease (VWD) is caused by dysfunction or diminished levels of von Willebrand factor (VWF). VWF-containing plasma concentrates are used for treatment of patients with VWD for whom desmopressin treatment is insufficient or contraindicated. A single-centre, retrospective observational study over a period of up to 25 years was conducted to evaluate the effectiveness and safety profile of Haemate(®) P (CSL Behring, Marburg, Germany), a plasma-derived, purified, pasteurised and lyophilised VWF-containing factor VIII (FVIII) concentrate. MATERIALS AND METHODS: The study included 71 patients who had been treated with Haemate(®) P over a period of > 5 years. RESULTS: A total of 663 treatments with individual laboratory evaluations were recorded. Two patients had both mild type 1 VWD and haemophilia A (HA), three had HA only, 39 had type 1 VWD, 16 had type 2 VWD, nine had type 3 VWD, one had acquired VWD and one had uncategorised VWD. Haemate(®) P treatment indications included bleeding events (37 patients) and surgical interventions (70 patients). Thirteen patients received Haemate(®) P as long-term prophylaxis (n = 3), as part of rehabilitation treatment (n = 5), or in context of recovery measurements (n = 5). Treatment with Haemate(®) P was generally well tolerated; only one thromboembolic event occurred. In cases of bleeding events, Haemate(®) P was haemostatically effective in all patients. Furthermore, during operations where Haemate(®) P was administered, >90% incurred no complications. CONCLUSION: In this retrospective observational study, Haemate(®) P was shown to be effective and safe for the treatment of VWD in adult patients.
Authors: M V Ragni; N Machin; L M Malec; A H James; C M Kessler; B A Konkle; P A Kouides; A T Neff; C S Philipp; D J Brambilla Journal: Haemophilia Date: 2016-02-04 Impact factor: 4.287