Prophylactic Use of Recombinant Factor VIII Prevents Joint Disease in Young Boys with Hemophilia A



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    Landmark Study Published in New England Journal of Medicine Demonstrates
    Benefits of Early Prophylaxis and Suggests New Explanation for
    Development of Joint Damage in Hemophilia A
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    TORONTO, Aug. 9 /CNW/ - Prophylactic (preventive) infusions of
recombinant factor VIII (rFVIII) significantly reduce the risk of developing
joint damage associated with joint bleeding in young children with hemophilia
A, according to a new landmark study published today in the New England
Journal of Medicine (NEJM). The findings from the first and only randomized,
prospective trial comparing prophylactic(1) with episodic on-demand(2)
treatment in children with hemophilia A, showed that 93 percent of children
who received prophylactic treatment with recombinant factor VIII had normal
joints at the age of six years compared to only 55 percent in the episodic
treatment group.
    Participants in the five-year clinical trial, known as the 'Joint Outcome
Study', used the Kogenate(R) product line, a recombinant factor VIII therapy
for the treatment of hemophilia A. In Canada, Kogenate(R) FS is indicated for
the treatment of hemophilia A in which there is a demonstrated deficiency of
activity of the plasma clotting factor VIII. Kogenate(R) FS provides a means
of temporarily replacing the missing clotting factor in order to correct or
prevent bleeding episodes. The multicenter study involved 15 academic and
major treatment institutions throughout the United States, and included
collaboration with the Centers for Disease Control and Prevention and the
National Institutes of Health.
    "Our results show for the first time that prophylaxis, initiated between
six and 30 months of age, is effective at preventing joint bleeds and
preserving joint function in young boys with hemophilia A. These results
provide healthcare professionals - as well as parents of children with
hemophilia A - with solid information to guide optimum treatment," said
Marilyn Manco-Johnson, M.D., principal investigator of the study, and
director, Mountain States Regional Hemophilia and Thrombosis Center,
University of Colorado at Denver and Health Sciences Center.
    Joint damage caused by repeated bleeding into the joints - often referred
to as hemophilic arthropathy - is one of the most debilitating and costly
consequences of hemophilia A. It can result in long-term inflammation and
deterioration of the joint, and ultimately lead to loss of mobility.(3)
However, previous retrospective patient studies have suggested that regular,
preventive infusions of factor VIII, given to young patients before they
develop permanent joint damage, may reduce the risk of hemophilic
arthropathy.(4, 5) The randomized, controlled clinical trial published this
week in NEJM provides the strongest medical evidence to date comparing joint
outcomes associated with prophylactic and episodic treatment approaches.
    "Results from the study indicate the importance of initiating prophylaxis
before recurrent bleeding has occurred in individual joints in young boys with
severe hemophilia," said Keith Hoots, M.D., study investigator, and professor
of pediatrics and division head/pediatric hematology, University of Texas
Medical School at Houston.
    A subset of patients in the study were identified with joint damage
despite an absence or low occurrence of overt bleeds into these joints. The
investigators suggest that subclinical bleeds - bleeds that are asymptomatic
and often go unnoticed - play a role in the development of joint damage. It is
speculated that subclinical bleeds may be prevented through prophylactic
infusion.
    Early signs of joint damage were detected in the study using
state-of-the-art magnetic resonance imaging (MRI) techniques. By the patients'
last year in the study (at around five or six years of age), joint and
cartilage damage was significantly greater in the episodic group compared to
the prophylaxis group.
    "Through the donation of 17 million units of our recombinant Factor VIII
used to treat the children involved in the trial, Bayer is proud to have been
a part of this significant study. We are fully committed to research-driven
initiatives that provide data for improving patient care and creating a better
quality of life for the thousands of people around the world who live with
this often-debilitating disease," said Georg Lemm, MD, PhD, Director of Global
Clinical Development, Bayer Schering Pharma.
    Further research in the prophylactic treatment of Hemophilia A in young
children is also currently on-going at The Hospital for Sick Children in
Toronto, Canada and these results will reinforce the need for prophylaxis in
young children.
    "In Canada, preventive treatment of Hemophilia A with recombinant Factor
VIII is widely practiced," said Dr. Victor Blanchette, Chief, Division of
Hematolgy/Oncology, Professor of Pediatrics, University of Toronto, The
Hospital for Sick Children. "The results of the USA randomized joint outcome
study provide solid evidence to guide hemophilia care specialists toward use
of prophylactic treatment for young boys with severe hemophilia A."

    Joint Outcome Study Methodology

    The five-year prospective, multi-center, randomized study was conducted
at 15 hemophilia treatment centers in the United States, and enrolled 65 boys
with hemophilia A between the ages of 6 months and 30 months. The study was
supported by a grant from the Centers for Disease Control and Prevention and
the National Institutes of Health. The Hemophilia and Thrombosis Research
Society contributed through the recruitment of participating sites. Bayer
HealthCare donated a total of 17 million units of recombinant Factor VIII to
treat children for the duration of the trial. The children were randomized to
receive either a prophylaxis regimen consisting of 25 IU/kg rFVIII every other
day (n=32) or enhanced episodic treatment consisting of at least three doses
of rFVIII totaling a minimum of 80 IU/kg at the time of a joint bleed (n=33).
The children were followed until the age of 6 years, when they were assessed
for bone or cartilage damage using X-ray and magnetic resonance imaging (MRI)
of damage-prone joints (elbows, knees, and ankles). The study also assessed
joint function, number of joint hemorrhages, and amount of product consumed.

    Detailed Study Findings

    Prophylaxis patients had significantly fewer joint hemorrhages per year
and overall number of bleeds per year compared to patients on episodic
treatment (mean joint bleeds 0.63 vs. 4.89, respectively; mean total bleeds
3.27 vs. 17.69, respectively; P less than 0.001 for both comparisons). When
evaluated by MRI at the age of 6 years, 93 percent of children in the
prophylaxis group had joints that appeared normal, compared to 55 percent of
patients who received episodic treatment (P=0.002). This translated to an 84
percent reduction in the risk for joint damage in patients who received
prophylaxis from an early age. The correlation of hemarthroses with joint
scores as measured with MRI was weak (r=0.26 with P less than 0.001). Based on
MRI, bone or cartilage damage was confirmed in 7 percent of children in the
prophylaxis group compared to 45 percent of those in the episodic group
(P=0.002).

    About Kogenate(R) FS/KOGENATE(R) Bayer

    Kogenate(R) FS (Antihemophilic Factor (Recombinant)) is a recombinant
factor VIII treatment. In Canada, Kogenate(R) FS is indicated for the
treatment of hemophilia A in which there is a demonstrated deficiency of
activity of the plasma clotting factor VIII. Kogenate(R) FS provides a means
of temporarily replacing the missing clotting factor in order to correct or
prevent bleeding episodes. The most frequently reported adverse events were
local injection site reactions, dizziness and rash. Known intolerance or
allergic reactions to constituents of the preparation is a contraindication to
the use of Kogenate(R) FS. Known hypersensitivity to mouse or hamster protein
may be a contraindication to the use of Kogenate(R) FS/KOGENATE(R) Bayer.
Please see the full prescribing information for important risk and use please
visit www.bayer.ca

    About Hemophilia A

    Hemophilia A, also known as factor VIII deficiency or classic hemophilia,
is largely an inherited bleeding disorder in which one of the proteins needed
to form blood clots in the body is missing or reduced. Hemophilia A, the most
common type of hemophilia, is caused by deficient or defective blood
coagulation proteins, known as factor VIII. Hemophilia A is characterized by
prolonged or spontaneous bleeding, especially into the muscles, joints, or
internal organs. Approximately 400,000 people around the world have hemophilia
A.
    About Bayer Inc.

    Bayer Inc. (Bayer) is a Canadian subsidiary of Bayer AG, an international
research-based group with core businesses in health care, crop science, and
innovative materials.
    Headquartered in Toronto, Ontario, Bayer Inc. operates the Bayer Group's
HealthCare and MaterialScience businesses in Canada. Bayer CropScience Inc.,
headquartered in Calgary, Alberta operates as a separate legal entity in
Canada. Together, the companies play a vital role in improving the quality of
life for Canadians - producing products that fight diseases, protecting crops
and animals, and developing high-performance materials for applications in
numerous areas of daily life.
    Canadian Bayer facilities include the Toronto headquarters and offices in
Ottawa and Calgary. Bayer Inc. has approximately 1,000 employees across Canada
and had sales of over $910 million CDN in 2006. Globally, the Bayer Group had
sales of over 28 billion Euro in 2006.
    Bayer Inc. invested approximately $47 million CDN in research and
development in 2006. Worldwide, the Bayer Group spends the equivalent of over
2 billion Euro in 2006 in R&D.

    Forward-looking statements

    This news release contains forward-looking statements based on current
assumptions and forecasts made by Bayer Group management. Various known and
unknown risks, uncertainties, and other factors could lead to material
differences between the actual future results, financial situation,
development, or performance of the company and the estimates given here. These
factors include those discussed in our public reports filed with the Frankfurt
Stock Exchange and the U.S. Securities and Exchange Commission (including our
Form 20-F). The company assumes no liability whatsoever to update these
forward-looking statements or to conform them to future events or
developments.

    
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    (1)  Prophylaxis - Regular, continuous infusions to prevent bleeding
         episodes and the progression of joint disease.
    (2)  Episodic on-demand -Treated at the time of and in response to a
         bleed.
    (3)  Manco-Johnson M, et al. Children with hemophilia. Seminars in
         Thrombosis and Hemostasis. 2003; vol. 29, number 6: 585-594.
    (4)  Nilsson IM, et al. Twenty-five years' experience of prophylactic
         treatment in severe haemophilia A and B. J Intern Med 1992;
         232:25-32.
    (5)  Manco-Johnson M, et al. Results of secondary prophylaxis in children
         with severe hemophilia. Am J Hematol 1994; 47: 113-117.
    





For further information:

For further information: Dara Willis, Fleishman-Hillard Canada, (416)
645-8179, dara.willis@fleishman.ca; Alison Bing, Bayer Inc., (416) 240-5298,
alison.bing.b@bayer.com


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