National Hemophilia Foundation Applauds First von Willebrand Disease Clinical Practice
Guidelines Issued by NHLBI
Common But Widely Undiagnosed Bleeding Disorder
Can Lead to Serious Health Complications
NEW YORK, NY—May 6, 2008
The National Hemophilia Foundation (NHF) welcomes and applauds the issuance of the
first clinical guidelines in the United States for the diagnosis and management
of von Willebrand disease (VWD), the most common hereditary bleeding disorder, by
the National Heart, Lung and Blood Institute (NHLBI), part of the National Institutes
of Health (NIH) and the U.S. Department of Health and Human Services. The guidelines,
developed in consultation with the American Society of Hematology by a panel of
multi-disciplinary medical experts and released on February 29, include recommendations
on screening, diagnosis, disease management and opportunities for future research.
“Because it is under-recognized and difficult to diagnose, many people with VWD
may not realize they have it, which is why the National Hemophilia Foundation has
long focused efforts on educating the public and health professionals about this
serious and potentially life-threatening disease,” said Howard A. Balsam, interim
chief executive officer of the NHF. “We are pleased that the NHLBI guidelines will
help to elevate public awareness of this important health issue and provide the
medical community with evidence-based direction on how to detect and manage VWD.”
NHF provides resources on bleeding and clotting disorders to the general public
through HANDI, their resource center and library of over 13,000 materials. Information
specialists are available through telephone, e-mail and fax to answer questions,
refer individuals to a hemophilia treatment center in their local area and provide
articles, brochures and news releases regarding bleeding and clotting disorders.
The library contains an extensive amount of resources particularly on von Willebrand
disease and the effects of this disease and other bleeding disorders on women. Contact
HANDI at 800-42-HANDI, handi@hemophilia.org or through fax at 212-328-3799.
About von Willebrand Disease
Von Willebrand disease is caused by a deficiency or abnormality of von Willebrand
factor (VWF), a protein in the blood that is necessary for normal blood clotting.
VWD is classified by types, ranging from Type 1 (the most common and usually mild)
to Type 3 (the most severe). Patients with Type 2 VWD have VWF that does not work
properly, while patients with Type 1 or Type 3 do not have enough VWF to produce
a normal clot. 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.
NHF, in partnership with the Centers for Disease Control and Prevention (CDC) and
with an educational grant from CSL Behring, has developed Project Red Flag, a national
public awareness campaign to educate women and their healthcare providers about
the symptoms of bleeding disorders, especially VWD, and encourage proper diagnosis
and treatment. Not only was CSL Behring part of the efforts to develop the guidelines,
but has been working with state bleeding disorder organizations to advocate for
legislation encouraging greater screening of VWD in cases of menorrhagia. Already
introduced in the Pennsylvania and Minnesota state legislatures, efforts for similar
legislation are currently underway in California.
According to available statistics from the Centers for Disease Control and Prevention
(CDC), VWD affects 1 to 2 percent of Americans—as many as 2.8 million people, more
than half of whom are women. Women may suffer severe health consequences if their
condition is not correctly diagnosed and are at greater risk for miscarriage, for
life-threatening bleeding following surgery or childbirth and for undergoing unnecessary
hysterectomies. Studies conducted by the CDC show that every year 30,000 women undergo
hysterectomy for the diagnosis of abnormal uterine bleeding. Many of these women
have an undiagnosed bleeding disorder that could be managed medically rather than
surgically. According to the CDC it takes a woman, on average, 16 years to get a
diagnosis for VWD.
“The NHF has long collaborated with other organizations and industry to help local
chapters, partner associations, hemophilia treatment centers and consumers raise
awareness of VWD in their local communities,” said Barbara A. Konkle, MD, Professor
of Medicine, University of Pennsylvania, Director, Penn Comprehensive Hemophilia
and Thrombosis Program, and Chair, NHF Women with Bleeding Disorders Task Force.
“We’re pleased to see the Institute affirm the importance of VWD, and the need for
professional guidance and consumer education around this disorder.”
Screening and Treatment
The NHLBI guidelines recommend that physicians take a complete medical history and
physical examination, and order a sequence of blood tests to evaluate individuals
whose history suggests VWD or any bleeding disorder.
According to the guidelines, treatment for VWD depends on its type and severity.
Medicines may be prescribed to increase the release of von Willebrand factor into
the bloodstream (desmopressin, administered either intravenously or through a nasal
spray like Stimate®), replace von Willebrand factor (Humate-P®
or Alphanate SD-HT®), prevent the breakdown of clots (antifibrinolytics)
or to control heavy menstrual bleeding in women (oral contraceptives).
For more information on the NHLBI guidelines go to http://www.nhlbi.nih.gov/guidelines/vwd/index.htm.
For more information on VWD, you can visit the Project Red Flag web site at www.projectredflag.org or
call the National Hemophilia Foundation’s Information Resource Center at 1-800-42-HANDI
(e-mail to handi@hemophilia.org).
Trained staff members are available Monday through Friday, 9 am to 5:00 pm EST to
answer your requests.
About the National Hemophilia Foundation
The National Hemophilia Foundation is dedicated to finding better treatments and
cures for bleeding and clotting disorders and to preventing the complications of
these disorders through education, advocacy and research.
Established in 1948, the National Hemophilia Foundation is a non profit 501(c)3
organization with chapters throughout the country. Its programs and initiatives
are made possible through the generosity of individuals, corporations and foundations
as well as through a cooperative agreement with the Centers for Disease Control
and Prevention (CDC).
For more information about the National Hemophilia Foundation visit www.hemophilia.org.
Important Safety Information for Stimate®
Stimate Nasal Spray is indicated for patients with hemophilia A with Factor VIII
coagulant activity levels greater than 5%. Desmopressin acetate is not indicated
for the treatment of hemophilia A with Factor VIII coagulant activity levels equal
to or less than 5%, or for the treatment of hemophilia B, or in patients who have
Factor VIII antibodies.
Stimate® Nasal Spray is indicated for patients with mild to moderate classic type
1 von Willebrand disease (VWD) with Factor VIII levels greater than 5%. Stimate®
Nasal Spray is not indicated for the treatment of severe classic VWD (Type I) and
when there is evidence of an abnormal molecular form of Factor VIII antigen.
Stimate® Nasal Spray is contraindicated if you have a known hypersensitivity to
desmopressin or any of the components of Stimate. Stimate should not be used by
patients with type 2B VWD, since platelet aggregation may be induced. Stimate Nasal
Spray is for intranasal use only.
To reduce the potential for water intoxication or hyponatremia, especially in the
young and the elderly, patients using desmopressin may be advised to reduce fluid
intake, ingesting only enough to satisfy thirst. Your doctor will monitor for a
rare occurrence of an extreme decrease in plasma osmolality that could result in
seizures, possibly leading to coma.
Other adverse reactions reported with use of injectable and/or intranasal desmopressin
acetate include headache, nausea, somnolence (sleepiness), dizziness, chest pain,
palpitations and tachycardia (rapid heartbeat), and severe allergic reactions, including
anaphylaxis. See full prescribing information for a complete list of adverse reactions.
Please see full prescribing information.