von Willebrand's disease (VWD) is an inherited bleeding disorder characterized by deficient levels of or dysfunctional von Willebrand factor (VWF). This phase II/III open-label, multicentre study evaluated the efficacy and safety of BIOSTATE, a high purity plasma-derived double-virus inactivated FVIII/VWF concentrate, when used in non-surgical bleeds, surgical procedures and prophylactic therapy in VWD patients for whom desmopressin treatment was deemed ineffective, inadequate or contraindicated. Twenty three patients (7 type 1, 9 type 2 and 7 type 3; 12 male, 11 female), who received FVIII/VWF concentrate as part of their VWD management, were recruited prospectively between December 2004 and May 2007 from eight centres in Australia and New Zealand. BIOSTATE dosing was based on pre-treatment FVIII:C and/or VWF:RCo plasma levels and a predetermined dosing guide. Haemostatic efficacy of BIOSTATE was rated as excellent or good for all major and minor surgery events, long-term prophylaxis, and for four of the six assessable non-surgical bleeding events. Blood transfusions were required by two major surgery patients as well as one patient with a non-surgical bleed. The median overall exposure to BIOSTATE across all groups was 8 days, greater in the prophylactic group (range 53-197) compared with major surgery (3-24), minor surgery (1-8) and non-surgical bleeds (1-10). BIOSTATE was shown to be efficacious and well tolerated when treating patients with VWD. This study also provides important insights into dosing regimens with BIOSTATE and the role of monitoring therapy with FVIII:C and VWF:RCo.
Measurement of health-related quality of life (HR-QoL) is used in patients with haemophilia as a way of assessing the effectiveness of health care, especially as cure is not possible. We report the first such study on patients with chronic coagulation disorders in Australia, using The RAND 36-item Health Survey 1.0 (SF-36), a standardized validated questionnaire combined with a semistructured interview. The mean scores for the eight domains of the SF-36 ranged from 52.5 +/- 42.1 for physical role to 80.0 +/- 20.0 for social functioning. Comparison with normative data obtained from the Australian Bureau of Statistics (ABS) demonstrated a reduction in all domains in this population with statistically significant reductions in general health, physical role limitation and vitality. Comparison with other studies indicates that the HR-QoL of patients with haemophilia and von Willebrand's disorder in Australia is comparable with other Western countries, reflecting the overall similar quality of care available to these patients. This study also provides a good cross-sectional view of the psychosocial factors of life in patients in Australia and recognizes the positive family support available to this population, while raising relevant shortcomings in schools and at the workplace that need to be studied further in a controlled manner.
Flow cytometry-positive cases should be included with those positive on morphology in a summative model to define BM involvement in DLBCL, as it may have a potential impact on predicting outcome.
concentration between those patients co-prescribed simvastatin and those taking warfarin alone. Although dietary vitamin K intake influences warfarin dose requirements, a parameter not assessed in this study, it can be assumed that the two patient groups had similar dietary intakes as there was no significant difference between their mean fasting plasma vitamin K concentrations.In conclusion, we have found that concurrent simvastatin therapy results in alterations in warfarin pharmacokinetics, most likely through inhibition of the P450 enzymes CYP3A4 and CYP2C9 responsible for the metabolism of R-and S-warfarin respectively, rather than by modification of its pharmacodynamics. Clinicians should be aware of the interaction between these commonly co-prescribed drugs and patients receiving warfarin should be monitored carefully when concurrent therapy with simvastatin is initiated to avoid overanticoagulation.
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