Document Detail


Clinical efficacy and safety of the factor VIII/von Willebrand factor concentrate BIOSTATE in patients with von Willebrand's disease: a prospective multi-centre study.
MedLine Citation:
PMID:  20331755     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
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.
Authors:
S Dunkley; R I Baker; M Pidcock; J Price; M Seldon; M Smith; A Street; D Maher; G Barrese; C Stone; J Lloyd
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Publication Detail:
Type:  Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't     Date:  2010-03-16
Journal Detail:
Title:  Haemophilia : the official journal of the World Federation of Hemophilia     Volume:  16     ISSN:  1365-2516     ISO Abbreviation:  Haemophilia     Publication Date:  2010 Jul 
Date Detail:
Created Date:  2010-07-20     Completed Date:  2011-01-05     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9442916     Medline TA:  Haemophilia     Country:  England    
Other Details:
Languages:  eng     Pagination:  615-24     Citation Subset:  IM    
Affiliation:
Haematology Dept, Royal Prince Alfred Hospital, Sydney, Newcastle, NSW, Australia. scott.dunkley@email.cs.nsw.gov.au
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MeSH Terms
Descriptor/Qualifier:
Adolescent
Adult
Aged
Aged, 80 and over
Child
Child, Preschool
Drug Administration Schedule
Drug Combinations
Factor VIII / administration & dosage,  analysis,  therapeutic use*
Female
Hemorrhage / prevention & control
Hemostasis / drug effects
Humans
Male
Middle Aged
Prospective Studies
Virus Inactivation
Young Adult
von Willebrand Diseases / drug therapy*
von Willebrand Factor / administration & dosage,  analysis,  therapeutic use*
Chemical
Reg. No./Substance:
0/Drug Combinations; 0/von Willebrand Factor; 9001-27-8/Factor VIII

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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