During the 24-month observation, 55 patients
underwent surgery, dental extractions and other invasive procedures (total number of interventions, 126) and therefore received Haemate® P VR as short-term prophylaxis. The procedures were mostly dentistry interventions (52.1%) and invasive procedures/endoscopy (25.6%). The concentrate (infusion of 4 × 103 IU per event, median; 12 × 103 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 PD0325901 in vitro of 234 postoperative, surgery-related infusions (median 7.0 per patient, range 1–19). The median number of postoperative infusions required to treat one event was five (range 1–16). In this subgroup of patients, most events were treated successfully with a response rated as excellent in 56.7% of the events treated (only one patient, with VWD type 2A, had a moderate response to treatment) [Table 4]. The treatment with Haemate® P was generally well-tolerated during the 24-month observation after the switch to the volume-reduced formulation of Haemate® P. There were no reports of adverse reactions related to the
study drug or development of inhibitors against VWF in the entire population, including patients on secondary prophylaxis and thus receiving more infusions. No thrombotic events were reported. During the 24-month follow-up period, almost half of the patients missed days at school/work because of VWD, 36.4% were hospitalized selleck and 35.5% underwent surgery because of the disease (Table 5). The efficacy and safety of Haemate® P for the treatment of VWD in a setting of real-life clinical practice have so far been addressed in a number of retrospective and prospective studies [9, 13-17]. To our knowledge,
Cobimetinib solubility dmso however, this is the first prospective study on a large (121 patients) population of patients with VWD receiving Haemate® P to treat a bleeding episode, as long-term prophylaxis or as short-term prophylaxis for surgery. Treatment history and dosing information was very detailed, and data on patients undergoing surgery/invasive procedures, a treatment setting still under intense investigation, were also collected. Also, the population considered was quite unique in terms of VWD-type distribution, as a relatively high proportion of patients had disease type 3 (31/121), a low prevalence subgroup in the general population of patients with VWD and most severely predisposed to bleeding events due to the virtual absence of VWF [2, 3]. The study population was also assessed in terms of BS, a parameter useful for the objective assessment of disease severity and for guiding therapeutic choices. The high average BS observed clearly indicates that a large proportion of the recruited patients had a significant bleeding tendency, thus strengthening the relevance of the observed results.