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Long-term Prophylaxis in von Willebrand Disease – How Should It Be Given and for Whom?
Table 2: VWD International Prophylaxis Study Clinical Laboratory Assessments
Type of Assessment Entry Interval Visit If Product Is Switched Termination
(Every 3 months) During Study
Complete blood count * * *
VWF:RCo
a
***
VWF:Ag
b
FVIII:C
a
*
Platelet count (most recent) *
ABO blood group
c
*
Inhibitory antibodies to VWF
d
**
a: Trough and peak levels measured at entry and if a product switch occurs during follow-up; trough levels measured every three months at interval visits and at termination; b: Trough and
peak levels; c: From diagnostic laboratory evaluation; d: Batched for processing in central laboratory. VWF:RCo = von Willebrand factor ristocetin cofactor; VWF:Ag = von Willebrand factor
antigen; FVIII:C = coagulation factor VIII:C.
document the number of bleeding episodes, management and outcome. Of The steering committee includes the following members: Erik
interest is the interval from the onset of the first documented episode of GI Berntorp, Principal Investigator, Malmö University Hospital, Malmö,
bleeding to the point of discontinuation of follow-up or death. The analysis Sweden; Thomas Abshire, Co-Principal Investigator, Aflac Cancer
will be descriptive, characterising the population in terms of diagnosis Centre and Blood Disorders Service, Atlanta, Georgia, US; Mayte
(definitive versus presumptive diagnosis of angiodysplasia), bleeding Alvárez, Unidad de Haemofilia Hospital, Universitario La Paz, Madrid,
frequency and severity, treatment and outcomes. Spain; Manuel Carcao, Haemophilia Program, Hospital for Sick
Children, Toronto, Ontario, Canada; Jorge DiPaola, Mountain States
Study Status Regional Haemophilia and Thrombosis Centre, Aurora, Colorado, US;
The VIP study currently contains 27 centres in Europe and Joan Cox Gill, Comprehensive Centre for Bleeding Disorders, The Blood
North America. Many of these are in the process of completing the ethical Research Institute, Milwaukee, Wisconsin, US; Sharyne Donfield, Rho
review process. We believe that the efforts to identify and implement Inc., Chapel Hill, North Carolina, US; Augusto Federici, Angelo Bianchi
optimal prophylactic treatment regimens for VWD will greatly benefit a Bonomi Haemophilia and Thrombosis Centre, Milan, Italy; Peter
substantial proportion of people, especially those with type 3 disease, who Kouides; Mary M Gooley Haemophilia Centre, Rochester, New York,
are most severely affected. We are actively enrolling treatment centres to US; Karin Kurnik, Dr von Haunersches-Children’s Hospital, Munich,
participate with us in this effort and encourage investigators who might be Germany; Frank Leebeek, Erasmus Medical Centre, Rotterdam, The
interested to contact us or members of the steering committee. For more Netherlands; Stefan Lethagen, Copenhagen Haemophilia Centre,
information email
Erik.Berntorp@med.lu.se,
Thomas.Abshire@emory.edu or Copenhagen, Denmark; Michael Makris, Royal Hallamshire Hospital,
SDonfield@rhoworld.com ■ Sheffield, England; Pier Mannucci, Angelo Bianchi Bonomi
Haemophilia and Thrombosis Centre, Milan, Italy; Prasad Mathew, Ted
Acknowledgements R Montoya Hemophilia Center, Albuquerque, New Mexico, US;
The VIP study is an initiative of the VWD PN, an investigator-initiated Rochelle Winikoff, Haemophilia Treatment Centre, Hôpital Ste-Justine,
study funded through an unrestricted grant provided by CSL Behring, Montreal, Québec, Canada.
Marburg, Germany. The VIP study has been approved by the Haemophilia
and Thrombosis Research Society (HTRS) and the American Safety oversight is provided by Christine Lee, Emeritus Professor of
Thrombosis and Haemostasis Network (ATHN), and is officially recognised by Haemophilia at the University of London and an honorary consultant
the Von Willebrand Factor Standardisation Committee (SSC) of the haematologist at the Oxford Haemophilia Centre, UK; and John
International Society of Thrombosis and Haemostasis (ISTH). It is registered Hutter, Professor Emeritus of Pediatrics at the University of Arizona,
at
www.ClinicalTrials.gov Tucson, Arizona, US.
1. Rodeghiero F, Castaman G, Dini E, Epidemiological (DDAVP) responsiveness in children with von Willebrand 12. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al., Prophylaxis
investigation of the prevalence of von Willebrand’s disease, disease, J Pediatr Hematol Oncol, 2003;25(11):874–9. versus episodic treatment to prevent joint disease in boys with
Blood, 1987;69:454–9. 7. Federici AB, Mazurier C, Berntorp E, et al., Biological response severe hemophilia, N Engl J Med, 2007;357(6):535–44.
2. Castaman G, Federici AB, Rodeghiero F, Mannucci PM, Von to desmopressin in patients with severe type 1 and type 2 von 13. Berntorp E, Petrini P, Long-term prophylaxis in von Willebrand
Willebrand’s disease in the year 2003: towards the complete Willebrand disease: results of a multicenter prospective disease, Blood Coagul Fibrin, 2005;16(Suppl. 1):S23–6.
identification of gene defects for correct diagnosis and European study, Blood, 2004;103:2032–8. 14. Blanchette VS, Manco-Johnson M, Santagostino E, Ljung R,
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Textbook of Hemophilia, Oxford: Blackwell, 2005:265–71. 9. National Institutes of Health/National Heart, Lung, and Blood blood loss using a pictorial chart, Brit J Obstet Gynaec, 1990;
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Italian Association of Hemophilia Centers (AICE), Haemophilia, 10. Berntorp E, Abshire T, for the von Willebrand Disease Hematol, 1993;30:3–6.
2002;8:607–21. Prophylaxis Network, The von Willebrand Disease Prophylaxis 17. Pettersson H, Gilbert M, Diagnostic imaging in hemophilia:
5. Rodeghiero F, Castaman G, Di Bona E, Ruggeri M, Consistency Network: exploring a treatment concept, J Thromb Haemost, musculoskeletal and other hemorrhagic complications, New York:
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Willebrand’s disease and hemophilia A, Blood, 1989;74(6): 11. Nilsson IM, Berntorp E, Löfquist T, Pettersson H, Twenty-five 18. Centers for Disease Control and Prevention, Measuring Healthy
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EUROPEAN HAEMATOLOGY 37
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