More than three-fourths of patients with Type 3 von Willebrand disease
(VWD), the most severe form of the most common hereditary bleeding
disorder in the world, had bleeding events in the past year that required
treatment, according to results of a study announced at the 2006 World
Federation of Hemophilia (WFH) Annual Congress. The research also found
that 22 percent of patients with Type 3 VWD had bleeding patterns severe
enough to warrant preventive therapy. These findings were released from
the first international census to examine the use of prophylaxis in
treating VWD by the von Willebrand Disease Prophylaxis Network, a
research group of 14 top bleeding disorder physicians and researchers from
Europe and North America.
"The VWD Prophylaxis Network was
established to help determine the optimal treatment of VWD patients, with
a goal of minimizing bleeding-related morbidities," said Erik Berntorp,
MD, PhD, principal investigator and Professor, Department of Hematology
and Coagulation Disorders, Malmö University Hospital, Malmö, Sweden.
"Using the data provided by our census registry and future studies, we
hope to develop more effective regimens for the management of VWD."
The data collected by the Prophylaxis Network from 74 centers in
Europe and North America include a total of 6,208 people with VWD: 4,687
with Type 1; 1,181 with Type 2; and 340 with Type 3, the most severe form
of VWD. The census data show a significant difference in the distribution
of types of VWD diagnosed in patients living in North America and those
living in Europe – with the more severe Type 2 and 3 VWD patients twice as
likely to reside in Europe as in North America (32.1 vs. 16.0 percent).
Researchers also found regional differences in the clinical management of
people with Type 3 VWD.
The data registry also assessed measures
to prevent and control VWD symptoms, a particular interest of the VWD
Prophylaxis Network researchers. Of the 101 patients in the census
registry under treatment with prophylaxis, 75 were Type 3 patients. The
most common reasons for initiating prophylaxis were joint (40 percent),
epistaxis/oral (23 percent) and gastrointestinal bleeding (14 percent).
"The findings of the VWD Prophylaxis Network highlight the risk of
spontaneous bleeding faced by patients with Type 3 VWD and the need to
better understand steps that can be taken to reduce bleeding episodes,"
said Miklos Fulop, chief executive officer and executive director of the
WFH. "Raising awareness of VWD is a major goal of the Federation, and we
welcome future findings of the Prophylaxis Network that may shed light on
the best treatment options to help all those afflicted with this
potentially serious bleeding disorder worldwide."
About von Willebrand Disease
Von Willebrand disease (VWD) is caused by a
deficiency or abnormality of the von Willebrand factor, a protein in the
blood that is necessary for normal blood clotting. Men and women are
equally likely to be affected by VWD. VWD is classified by types, ranging
from Type 1 (the most common and mild) to Type 3 (the most severe). VWD
affects about 60 million people worldwide.
Women with VWD are more
likely to experience heavy, prolonged menstruation. Other common symptoms
of VWD include frequent nosebleeds and easy bruising. Bleeding can be mild
or serious and can occur as a result of injury, or without any obvious
cause. More serious symptoms include bleeding into joints and internal
organs. The VWD patient may require special care during dental procedures,
surgery and childbirth.
There is no cure for VWD, but it can be
treated. Specific treatments may include desmopressin acetate to release
stored von Willebrand factor, or von Willebrand factor replacement
therapy, where required, with Humate-P®, Antihemophilic
Factor/von Willebrand Factor Complex (Human), the only product licensed by
the FDA for the treatment of von Willebrand disease.
von Willebrand Factor (VWF)/factor VIII concentrate Humate-P®
(marketed in Europe as Haemate® P) is a factor replacement
therapy with more than 20 years of demonstrated safety and efficacy around
the world. It is an injectable drug that works to achieve proper
hemostasis by replacing the most active forms of VWF (high molecular
weight VWF multimers) and factor VIII that are missing in patients with
von Willebrand Disease (VWD).
Possible adverse events with the use
of Humate-P include allergic reaction, urticaria (hives), chest tightness,
rash, pruritus (itching) and edema (swelling). Anaphylactic reaction can
occur in rare instances. Thromboembolic events have been reported in VWD
patients receiving coagulation factor replacement therapy, especially in
patients with known risk factors for thrombosis. In these patients,
caution should be exercised and antithrombotic measures should be
Humate-P is derived from human plasma. As with all
plasma-derived products, the risk of transmission of infectious agents,
including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD)
agent, cannot be completely eliminated. Please visit www.ZLBBehring.com to read the
prescribing information for Humate-P.
About the von Willebrand
Disease Prophylaxis Network
Founded in 2001, the Von Willebrand
Disease Prophylaxis Network is an international study group of VWD
physicians and researchers formed with the goals of investigating the role
of prophylaxis measures in protecting against or preventing bleeding
episodes in clinically severe VWD, and educating healthcare professionals
and consumers about VWD. The VWD Prophylaxis Network steering committee is
made up of 14 top bleeding disorder physicians and researchers (six
European, eight North American), including:
Chair: Erik Berntorp, MD, PhD,
Malmö University Hospital,
Co-Chair: Thomas Abshire, MD,
AFLAC Cancer Center and Blood Disorders Service,
Data Coordination and Statistics: Sharyne Donfield, PhD, Rho, Inc.,
- Manuel Carcao, MD, Hemophilia
Program, Hospital for Sick Children,
- Joan Cox Gill, MD, The
Blood Research Institute, US
DiPaola, MD, Great Plains Regional Hemophilia Treatment Center,
- Peter Kouides, MD, Mary M.
Gooley Hemophilia Center, US
Kurnik, MD, Dr. von Haunersches-Children's Hospital,
- Frank Leebeek, MD, Erasmus
Medical Center, The
- Stefan Lethagen, MD,
Copenhagen Hemophilia Center,
- Michael Makris, MD, Royal
Hallamshire Hospital, UK
Mannucci, MD, Angelo Bianchi Bonomi Hemophilia / Thrombosis Center,
- Prasad Mathew, MD, Ted R.
Montoya Hemophilia Center,
- Rochelle Winikoff, MD,
Ste-Justine Hospital, Canada
The VWD Prophylaxis Network is
supported by an unrestricted grant from ZLB Behring.
ZLB Behring is a global leader in the plasma protein
biotherapeutics industry. Dedicated to improving the quality of life for
patients throughout the world, ZLB Behring provides safe and effective
plasma-derived and recombinant products and offers patients a wide range
of related services. The company’s broad portfolio of life-saving
therapeutics is used in the treatment of individuals with hemophilia and
other bleeding disorders, immune deficiency disorders and inherited
emphysema; the prevention of hemolytic diseases for the newborn; cardiac
surgery patients; and shock and burn victims. Additionally, ZLB Behring
operates one of the world’s largest, fully owned plasma collection
networks. ZLB Behring is a subsidiary of CSL Limited, a biopharmaceutical
company that operates worldwide from its headquarters in Melbourne,
Australia. For more information, please visit www.ZLBBehring.com. The website www.AllAboutBleeding.com is a
resource dedicated to increasing awareness about VWD.