Spring 2004 - Factor Nine News

The Coalition for Hemophilia B

The Coalition For Hemophilia B Breakfast Meeting

SATURDAY, MARCH 27, 2004

7:30 AM - 9:00 AM

RIVIERA HOTEL
LAS VEGAS, CA

CAPRI ROOM 112-113

TOPIC: PROPHYLACTIC TREATMENT IN HEMOPHILIA B

SPEAKER: DAVID CLARK, PH.D.

BABYSITTING - CAPRI ROOM 114
(beginning at 7:15 am - breakfast will be provided, games, movies, toys, etc.)

Sponsored by a Grant from Aventis Behring

Our breakfast meeting will be held at the Hemophilia Federation of America Conference March 26-28, 2004

7:30 AM - Welcome & Introduction
7:40 AM - Dr. David Clark - Prophylactic Treatment in Hemophilia B
8:10 AM - Question & Answer
8:30 AM - Family Discussion

For more information please call Kim at (212) 554-6898 or email: Hemob@ix.netcom.com

We look forward to seeing you there!

RESEARCH SURVEY DEADLINE MARCH 31ST

A research survey was sent out on March 1, 2004 to Physicians, treaters and people with hemophilia B. The purpose of this survey is to gather information regarding the packaging/infusion of clotting factor products. By completing the survey you will help shape future offering of these products.

To complete survey online people with hemophilia B should go to website: www.surveywatch.com/h12

Physicians and Treaters should go to website: www.surveywatch.com/h22

If you need more information please contact Kim at (212) 554-6898 or email: hemob@ix.netcom.com

AVENTIS BEHRING SALE

Aventis and CSL Limited have signed a definitive agreement. CSL will acquire Aventis Behring and will combine this business with their ZLB Bioplasma operations to create ZLB Behring. The business will be managed from its head office in King of Prussia, Pennsylvania. We hope this company has found a home and will become a strong force in hemophilia research.

MEDICARE OUTPATIENT REIMBURSEMENT RATES INCREASED

Josesph Pugliese, Vice President & General Manager at Aventis Behring announced a recent and critical victory regarding Medicare reimbursement in a hospital outpatient setting. Through the determined efforts of the Aventis Health Policy team acting in coordination with patient advocacy organizations and allied trade associations, Medicare has just released a program transmittal dated February 27, that will significantly increase reimbursement for clotting factors and immune globulin therapies.

To read more about the corrected 2004 payment rates visit the website.
Telephone: reimbursement answer line at (800) 676-4266
Email: accesstocare@aventisbehring.com

LIVING WITH HEPATITIS C

a success story by Wayne Cook

In the early 1980’s I was very naive about my hemophilia and everything else that comes with it. By 1983 I was diagnosed with hepatitis B. The doctor’s didn’t know how I contracted it. After long talks with my doctor in Florida, he suggested I move back to New York where the doctors knew how to treat my condition. In 1984 my wife and I moved back to upstate New York.

Growing up, I was taught you did not discuss having hemophilia with anybody, not your friends, your employers and even parts of your own family. One day in 1985 I was having my wisdom teeth removed and ran into complications. I developed an allergic reaction to the plasma and had to go to the hospital. One of the nurses told me that she heard of a new organization in one of the other hospitals that dealt only with hemophilia. I was one of the first patients at the new Hemophilia Treatment Center (HTC)

While visiting the HTC, they discussed various treatments that were available for hemophilia. I was also informed that with the new screening process for HIV and AIDS it would be rare to contract either one but there was still a chance I could contract hepatitis.

In 1992 my HTC called me and asked me to come in and speak with my nurse coordinator and doctor. I was informed that I had a strain of hepatitis called non a and non b. There was a treatment out there but as long as I felt good I would not need it at this time.

By 1996 my hepatitis has reached a new level and I was advised to try te treatment of Interferon.

By this time they had a name for the strain of hepatitis and it was called hepatitis “C”. Interferon was the only product to treat hepatitis C and the side effects were terrible. After six months I stopped treatment because I could not take the side affects.

I went for a second opinion at Mount Sinai Hospital. The doctor told me that that research was being done on a combination of a new drug to go along with the interferon. My wife and I decided to wait and go on with our lives.

One day while visiting a friend who also had hemophilia and hepatitis C, I was told about a good doctor in Florida who was cutting edge when it came to dealing with hemophilia, HIV and Hepatitis C. I went home and made an appointment to see him.

In 2000 my wife and I flew to Florida to see the doctor and discuss my case. I had a heart attack in 1999 and the first thing he did was talk with my cardiologist about the treatment he was going to give me. Next I was told to get myself in better physical shape and healthy before we started the treatment.

In 2001 I lost my best friend to hepatitis C and by 2003 I was not only physically set to begin treatment but I was mind set. I received great support from my family and a few very close friends that I am bound with through hemophilia.

Treatment of pegulated Interferon and Copegasus began in September 2003. The side effects started immediately so I took another drug to help with the depression.

In 23 years of marriage to my wife she has always been the one to push me through and stand by me. She kept telling me I could do it and stood by me with the side effects and being up sick all hours of the night. She was the one who helped me get through this. She was my rock.

After four months of treatment I flew to Florida for a check up with my doctor. He told me that I was looking good and that my blood work was as well. I had lost some weight, my cholesterol was down and my blood pressure was great. But I still needed to stay on the treatment.

A week later I received a phone call from my doctor asking me to call him right away. I was informed my hepatitis C was no longer detectable. I had no signs of any antibodies and my viral load was undetectable.

After sitting at my desk for a few minutes and crying with happiness, I called my wife and told her the good news. We sat down and told the children who were overjoyed. Then I called my best friend’s wife. I told her that my best friend was looking down on me from heaven and helping me get through this.

We cried together with joy and happiness. Later I made four more calls to the people that mean the most to me in my life, they are from the hemophilia community, and I consider these people an extension of my own family.

I still have to go through the treatments and still deal with some of the side effects but I am getting through them a lot easier now. I think because I know that the outcome is going to be great and there is light at the end of the tunnel.

This is my story and I wanted to share it with everyone. I know that we, in the bleeding disorders community all have our own individual issues but we also have a common bond that draws us all close and makes for very good friendships. Know that with all the issues we deal with, there is some kind of hope for all of us.

FACTOR IX PRODUCTS

Dr. David Clark

There are three basic types of factor IX products currently available in the U.S. for treatment of hemophilia B: Factor IX Complex, Coagulation Factor IX (Human) and Coagulation Factor IX (Recombinant).

The major issues involved in the selection of a product are safety and cost. Safety issues include the risk of infectious diseases and the risk of other serious side effects. Products that are made from human plasma can potentially contain viruses or other infectious agents that cause diseases such as hepatitis and AIDS. However, over the two decades since the AIDS crisis in the early 1980’s tremendous advances have been made in improving the viral safety of products made from human plasma. Today’s plasma products are considered extremely safe, but the risk of disease transmission cannot be completely ruled out. For that reason, the National Hemophilia Foundation and many physicians recommend that patients be treated with recombinant products when possible.

Recombinant products are produced in genetically engineered animal cells that are inherently free from human viruses. In addition, the one recombinant factor IX product available is produced without the use of any human or animal proteins either in the broth used to feed the cells or In the additives used to stabilize the product in the vial. The production process also includes a filtration step at the end that can remove any accidental viral contamination.

Factor IX Products currently available in the United States:

Listed below are the Company/Product Name/Purity/Purification Methods Viral Inactivation/ Removal Methods.

Factor IX Complex (Human) Products
Baxter Bebulin(r) VH
Low Purity
Chromatography
Vapor Heating
Baxter Proplex (r) T
Low Purity
Adsorption/PEG precipitation
20% ethanol/Dry Heating
Grifols Profilnine(r)SD
Low Purity
Chromatography
Chromatography/Solvent-Detergent
Coagulation Factor IX (Human) Product
Aventis Behring Mononine(r)
High Purity
Chromatography
Chromatography/Chemical Treatment/
Ultrafiltration
Grifols AlphaNine(r)SD
High Purity
Chromatography
Chromatography/ Solvent-Detergent/
Nanofiltration

Coagulation Factor IX (Recombinant) Product
Wyeth Benefix(r)
High Purity
Chromatography
NanofiltrationSome factor IX products can have another very serious side effect. When used in large amounts for extended periods of time, for instance as support for surgery, some patients have experienced problems with thrombosis.

Thrombotic side effects seem to be associated only with the Factor IX Complex products. So far, there have been no reported cases of thrombosis associated with the use of the higher purity Coagulation Factor IX products.

Why Factor IX Complex caused thrombosis is still not known. It may be that small amounts of clotting factors become activated during processing. Alternatively, the unneeded excess of other clotting factors in the complex may make the blood more prone to clotting.

“Recombinant factor IX is produced by special animal cells that have been engineered to contain the gene for human factor IX.”

Thrombosis is unnecessary clotting within the bloodstream that can plug up blood vessels, cutting off the blood flow. This can have serious, even fatal, consequences. If the blood flow to important organs such as the heart or lungs is affected.

Product cost is a major factor for many patients. As expected, the more highly purified products cost more. Thus, there are often trade-offs that must be made between safety and cost. There are also differences of opinion that enter into the decision. Some physicians believe that all of the products currently in the U.S. have such high levels of viral safety that the decision can be made based on other criteria such as cost.

Factor IX Complex was the first product developed for the treatment of hemophilia. Its introduction, starting in 1959, radically transformed the lives of hemophilia B patients. Prior to its availability, patients could only be treated with plasma.

Plasma is not very effective because the concentration of factor IX in plasma is relatively low, and only a limited amount of plasma can be injected. Compared to plasma, Factor IX Complex is approximately 200 times more purified. However, Factor IX Complex also contains significant amounts of several other clotting factors, which is why it is called a “complex”. These other clotting factors, including factors II and X (and in some products also factor VII), all have similar molecular structures. Therefore they tend to purify together as a group from plasma.

“Coagulation Factor IX (Human) is purified approximately 14,000 times compared to plasma”

With the widespread use of Factor IX Complex, a potentially serious side effect became apparent. As mentioned on page 6, patients that received large amounts of Factor IX Complex for extended periods of time tended to experience problems with thrombosis.

Coagulation Factor IX (Human) is a more highly purified concentrate that was developed to eliminate the thrombosis problems. Coagulation Factor IX (Human) is purified approximately 14,000 times compared to plasma and contains no other clotting factors. Although produced by different methods, both products have similar purities; both are essentially pure factor IX with only insignificant amounts of other plasma proteins.

Coagulation Factor IX (Recombinant) is a highly purified factor IX concentrate produced by genetic engineering, not purified from plasma. Recombinant factor IX is produced by special animal cells that have been engineered to contain the gene for human factor IX. The cells are grown in large tanks suspended in a nutrient broth that contains everything that the cells need to grow and produce factor IX. The factor IX they produce is secreted into the broth, from which it is purified to produce the product.

Recombinant factor IX has the same molecular structure as the factor IX in plasma and functions similarly in restoring the clotting ability of the blood. However, a slightly larger amount of recombinant factor IX than plasma-derived factor IX must be infused to give the same level of factor in the blood. This is probably due to a slight difference between the way that factor IX is produced in the human body and the way it is produced in the animal cells used to manufacture the product. Interestingly, there have also been informal reports that some patients on recombinant products have chosen to use plasma-derived product during surgery because they feel it controls the bleeding faster. However, there have not been any formal studies done on this.

There are many factors to consider in the selection of the appropriate product for an individual patient. In addition to differences among the products, there are also physiological differences among patients. The best recommendation is for the patient and his family to have an informed discussion with his physician to choose the best product for his particular situation.

NEW JERSEY STATE PILOT PROGRAM

Last spring the Hemophilia Association of New Jersey (HANJ) invited their members to participate in a program developed by their own, Joe Caronna (HANJ V.P.). The program focused primarily on men, but also provides benefits to other family members, as well as those in the community where we all must live and work..

As a father of a child with hemophilia, and as an advocate for the bleeding disorder community, Joe has become extremely familiar with the obstacles and challenges of managing a bleeding disorder.

Over the past two years Joe has been devoted to the development of effective and practical support and education tools. The availability of these tools will better equip the bleeding disorder community in resolving some of their daily challenges.

Joe’s first goals was to create a safe forum where men could gather to embrace, learn and share their insight, support, inspiration and practical advice. His next goal was to design and develop a new and improved training tool, which would be efficient, effective and easy to use.

Once the strategy was developed, Joe presented the concept to the HANJ Board of Trustees who immediately recognized the values of this unique program. HANJ was pleased to fund a three-month pilot to determine if their excitement and instincts were correct.

“The program resulted in “life changing” experiences for those who participated”

Over 120 respondents from the community provided written comments to the surveys distributed during the three month pilot program and several major themes emerged: the need for more effective support for men, and the need for effective training tools for the bleeding disorder community. The program resulted in “life changing” experiences for those who participated. 30 Male Participants were in the 3 Month Pilot Program.

Comments from people in the pilot study:

“The session brought tears to my eyse. I felt that the topics were important and were presented in a very heartfelt manner. Thanks!”

“Well needed topic. I have been dealing with anger control for a long time.”

“The interaction with participants was extradordinary and really made me think…it will hep me improve the relationships with my family. I also now realize just how much I have to be thankful for!”

“Beautiful interaction between the men. Very moving and emotional to all and a highly recommended program.”

47% Men with bleeding disorder
42% Men with child/children with bleeding disorder,
10% Men with friend/extended family with bleeding disorder

Age participants between 19 and 74 years old from all socio-economic, religious and racial boundaries.

Each teleconference ran approximately 90 minutes with an average of 16 men per teleconference.

The five topics covered were:

1. For Men Only: The issues we Face when Living with a Bleeding Disorder
2. The role of Men in the Face of a Bleeding Disorder
3. One Hope; One Dream: Strengthening Relationships with your partner
4. Being There: Strengthening Relationships with your Child
5. Positively Energizing Your Anger

For more information please email joe_caronna@inalex.com or see web: www.inalex.com for more information and to review teleconference and workshops

WYETH BOOKLET

PAL - PREVENTION FOR ACTIVE LIVES

“Well written informed booklet to help people living with hemophilia prevent bleeds and lead a healthy, active life.”

The PAL Program was started by Wyeth to raise awareness about a particular kind of prevention - infusions added to regular factor regimens before activities with potential to cause bleeds. By preventing bleeds, you may avoid or minimize the potential for joint damage. A prevention regimen, including preventive infusions can help people play, move and compete in appropriate activities

Prevention includes many good habits that may help enhance health and protection. There are many was to help avoid bleeding episodes and participate more fully in activities. Ask your health care provider about:

• Regular exercise and good nutrition
• Making it a habit to use protective gear, such as seat belts and bicycle helmets
• Utilizing protective padding to help avoid injury at home or office
• Infusing before activities that might cause a bleed.

As you read the booklet, think of the many activities you participate in - or would like to participate in - and contact your health care provided to discuss whether preventive treatment may be right for you.

TO RECEIVE THIS BOOKLET PLEASE CALL:
1-888-999-2349 or visit the web: www.hemophiliavillage.com

PATIENT SERVICES INCORPORATED

DANA KUHN, PHD, PRESIDENT AND CO-FOUNDER

We have found out about a good company that provides families with insurance assistance. Since insurance is a big concern in the hemophilia community, we share this information with you.

Patient Services Incorporated (PSI) was founded in 1989 and today they assit over 9 percent of their entire budget and program on bleeding disorders.

Families are offered assistance based upon the severity of their medical and financial needs. Since PSI’s beginning, over 4,500 people in 50 states have received assistance.

PSI uses donated dollars to help families avoid using social service programs, thus saving the taxpayers. Through PSI’s financial assistance, the Medicaid “roles” are reduced.

At least 50-70% of the people PSI helps experience a better quality of life because of its assistance. PSI also helps families with chronic illnesses return to gainful employment.

Through private, foundation, and corporate donations. 80 cents of each donated dollar goes to direct help.

PSI’s vision is to become the leading national non-profit organization providing services for people with chronic illnesses and diseases. Their well-trained staff and volunteers assist clients with a range of health insurance options by utilizing funds from a diversified donor-base. They are working hard each day to make this vision a reality.

• PSI saves middle-class families from becoming financially devastated when they have a family member with an expensive chronic illness.
• PSI is the only non-profit of its kind providing health insurance premium and pharmacy co-payment assistance.
• PSI is recognized by the Office of the Inspector General, the IRS, Better Business Bureau and Dun and Bradstreet.

For more information please call:
1-800-366-7741 or 1-800-744-3813
Email: uneedpsi@uneedpsi.org
Website:www.uneedpsi.org
Address: PSI, PO Box 1602
Midlothian, VA 23113

WYETH

WYETH HEMOPHILIA PATIENT ASSISTANCE PROGRAM &
REIMBURSEMENT INFORMATION LINE

We wanted to make you aware of some of the patient assistance programs available for insurance issues. This looks like a good one:

The Wyeth hemophilia Patient Assistance Program is designed for ANY eligible patient who is factor VIII or factor IX deficient. Participants are not required to be on a Wyeth brand and patients do not have to earn, accumulate, track or redeem points or coupons.

The following criteria determine patient eligibility for the patient assistance program:

• Patient is not covered by health insurance or other third-party payer coverage-Patient is not eligible for state hemophilia assistance, Medicaid or Medicare reimbursement or billing
• Patient has an annual income below $25,000(single) or $40,000(two ore more family members)
• Patient has no other sufficient resources or assistance to pay for the product
• Use of patient’s own resource assets will cause financial hardship

FOR MORE INFORMATION PLEASE CALL: 1-888-999-2349 (hit option 1)

NACCHO

NORTH AMERICAN CAMPING CONFERENCE OF HEMOPHILIA ORGANIZATIONS

NACCHO conducted by the Hemophilia Association of Arizona and funded by a grant from Wyeth, held their conference in Tempe, Arizona on January 30- February 1. Approximately 165 individuals from 34 states, Canada and Puerto Rico, including volunteers, nurses and social workers attended.

Guest speakers included Michael Brandwein, author of “Learning Leadership: How to Develop Outstanding Teen Leadership Training Programs at Camp, and Tom McDermott, a musician and singer who presented “Music and Games you can take back to Camp”.

During the conference, participants discussed best practices for operating hemophilia camps, and providing an educational and supportive environment for the children in attendance. All in the all the conference was a success and everyone left feeling very positive and motivated for the upcoming camping season.~

NATIONAL HIA FOUNDATION ON THE RESEARCH FRONT…

The Coalition for Hemophilia B has been supporting novel research into the production of human proteins crucial for coagulation. Our goal is to increase the supply and availability of these proteins for treatment purposes. Another goal is to avoid human blood-borne diseases by generating a non-human source for the proteins. Several plasma proteins have already been successfully produced in transgenic animals, including Factor IX and Factor VIII. Human alpha1-antitrypsin produced in goat milk is currently in clinical trials for the treatment of hereditary deficiencies.

The Coalition has supported research on Thrombin, a key regulatory protein in the coagulation cascade. Thrombin has both anticoagulant and pro-fibrinolytic activity. It is currently prepared by extraction from bovine or human plasma. The use of bovine thrombin will decrease in the future, due to immune reactions and a risk of BSE transmission. Emerging applications for thrombin are connected with its use as a component of fibrin sealant, bandages and other fibrinogen-based products. Thus a good source of recombinant prothrombin would fulfill all these needs.

In the future, the Coalition hopes to support research into developing a replacement therapy for von Willebrand’s Disease (vWD). This is the most common coagulation disorder, affecting 1% of the population, and is caused by either the absence of or a defective protein, the von Willebrand Factor (vWF). Patients have increased bleeding times, have low Factor VIII levels and suffer from modest to severe bleeding. VWD affects both men and woman, although many patients, especially women, may have been inaccurately diagnosed and treated for gynecologic problems. In fact, 13% of patients with menorrhagia have vWD. Currently, patients are being treated with DDAVP and plasma-derived cryoprecipitate, or human Factor VIII concentrates that also contain vWF. There is neither a pure plasma-derived nor recombinant vWF product available.