Winter 2004 – Factor Nine News

The Coalition for Hemophilia B

From Hospital to Home: (Continuous Infusion Factor Replacement (CIFR)

Sunday, October 17, 2004, Marykay Thrower, RN presented at the World Federation of Hemophilia in Bangkok to the Advanced Nursing Group.  The presentation was a discussion of her Poster “From Hospital to Home: Continuous Infusion in 8 day-old male with severe factor IX deficiency”. 

The case study depicts the seamless delivery of care provided with good collaboration with the physician and nurse at the HTC.  Savings were determined by hospital bed days eliminated by reducing the length of stay.  The positive response with therapy allowed the infant to acclimate to his home with  the support of his family.  Early education helped the family realize that although this is a chronic disorder, it is manageable and life can be normalized by providing care at home, in lieu of the traditional institutional management.

The case study was written in collaboration with the Nurse Coordinator, Rhonda Fritz, at the HTC in Boston and reviewed by another Nurse Coordinator, Jim Munn, at the HTC in Michigan. It was accepted first as a poster and later as an oral presentation to the Advanced Nursing Group attending the meeting.

Haemophilia magazine published the abstract in the October 2004 issue.  When we asked Marykay how she felt about presenting she said “This experience was one of the greatest in my professional career!”  The Coalition for Hemophilia B was proud to sponsor Marykay’s attendance at the meeting.

Jim, Rhonda, and Marykay

The Eshton Hewitt Quality of Life Project

Eshton has severe Hemophilia B and EDS.

Tekoa and Eshton

Eshton Hewitt has severe hemophilia B with inhibitors and anaphylaxis (a severe, sometimes fatal allergy) to factor IX.  He also has a connective tissue (ligaments, tendons, skin) disorder that causes dislocations.  There is no family history of the hemophilia so he was not diagnosed until he was six months old.  Joyce and Jay Hewitt also learned of the Connective Tissue Disorder known as Ehlers-Danlos Syndrome  (EDS) three and a half years ago when both the boys (Eshton’s twin brother Tekoa) and their mother, Joyce were diagnosed with EDS. The combination of disorders has wreaked havoc on Eshton’s body causing severe joint damage and making it difficult at best to control the bleeding episodes that inevitably follow a subluxation, or dislocation.

After a great deal of research and struggle to find adequate long-term care for Eshton, his parents decided that his best opportunity to slow the progression of disease in his joints was to strengthen the muscles supporting them through exercise.  Eshton has four target joints and has already undergone a radionucletide Synovectomy which was considered a failure after he hemorrhaged six months post procedure.

The problem with using exercise to strengthen is the inability of his joints to handle the pressure of even the lowest impact exercise or therapy.  Therefore aquatic exercise, which has basically no impact on the joints, is the best option.  Eshton is not able to swim in a public swimming pool due to the combination of his fragile skin and having a port (which makes him susceptible to infection).  There is no way to regularly test a public pool for bacteria and the Health Department does not require that logs be kept showing their testing.  This puts him at a very high risk.

The Hewitts started out with an outdoor above-ground pool.  When Eshton was swimming his bleeding episodes decreased and they had to infuse him less.  (When Eshton has a bleed he has to be infused every 3-6 hours (depending on the dosage) for 1-4 days until he has full range of motion due to the EDS).

As published in the Michigan Flint Journal (Title: Pool to Help Boy Be Like Any Other Kid; Businesses come through for mom; July 26, 2003) “When Eshton is in water, everything is normal for him.” Joyce Hewitt explained.  “One time he was swimming and said he was so happy we had the pool and that when I’m in here, it’s like I’m like any other kid!” The difference was enough that Joyce and Jay wanted to give him a better quality of life year-round and that meant building an in-door swimming pool.

To date the exterior shell is complete, the pool is now enclosed and the room is heated. However, additional funding is needed to bring the project to completion.  Information on specific areas of funding can be found on our website: http://www.coalitionforhemophiliab.org/ or call Kim Phelan at (212) 554-6898

The Coalition for Hemophilia B formed an umbrella project called “The Eshton Hewitt Quality of Life Project.”  Joyce and Jay Hewitt mailed out many letters and made numerous phone calls to prospective donors for this project.  Soon after, donations for building supplies, pools, roofing material, windows and doors, a boiler and plumbing supplies came in.  Individuals, companies and The Coalition for Hemophilia B donated monetarily as well.  The Hewitts were completely overwhelmed and never expected an out-pouring of such magnitude.  Joyce Hewitt said “without the support we would never have been able to make this dream a reality for our son.”

The good news of all this is that Eshton has been able to be more active than ever before.  He is actually able to play outside with friends and the range of motion in his ankles (two of his target joints) has begun to increase.  The fact that he has any range of motion is an improvement and only the beginning of what aquatic therapy can and will do for him.

As the Eshton family is still knee deep in construction, they said if they had to do it all again they would.  Joyce Hewitt stated “when NovoSeven was licensed it was a blessing and a life-altering occasion (Eshton was four then) but to not bleed even half as much as he has is a big, if not bigger, moment for us. We are talking about the difference between trying to stop the damage that occurs every time he bleeds into his joints versus a dramatic decrease in bleeding which slows down the progression of joint disease and gives him a more normal life. We contribute this change directly to his ability to strengthen his muscles while in the pool without causing joint damage.”

“It is very difficult for me to put into words what it has meant for Eshton to be able to go outside and play.  As a mother it is almost impossible to measure what that means to my heart. As he has found some freedom and hope, my heart has as well.  I can see a much different future for my little boy than I have been able to in many years and all I can say is thank you.  Thank you to all who gave and to all who have supported us with their help and donations. We are richly blessed!”
- Joyce Hewitt

National Hemophilia Foundation Conference

November 4-6 2004
Dallas, Texas

Conference was held at the Hyatt Regency at Reunion Center, Dallas, Texas

We held a Factor Nine Meeting on November 6 at the NHF Conference in Dallas.  The meeting was sponsored by ZLB Behring.  John R Taylor, Jr., Founder and President of The Coalition for Hemophilia B made the opening remarks, Dr. Jorge DiPaula, Assistant Professor of Pediatrics and Director of the Hemophilia Treatment Center, University of Iowa gave an overview of various treatment options available for hemophilia B.  The history manufacturing process and pros and cons of each FIX product on the market were discussed.

Linda Price, Ellis Sulser, Nina Duggan and Axel Freese

Afterwards, we held our Factor Nine Family support meeting.  We had old-timers, first timers and the in-betweeners.  It was great to see all come together, share ideas and support each other.  Wayne Cook made the closing remarks.  Our next breakfast meeting will be held at the HFA in Nashville, TN., Friday, April 1, 2005- 9:30am.~

John McNeill, Spencer Duggan, Matthew DeRatto, Paul and Frances Brayshaw

John Taylor Newman, Dallas Cheerleader, Shenythia Frazier, Matthew DeRatto, and Spencer Duggan at Novo Nordisk sponsored Stadium Event

Birth Announcements!

Daddy Felix and Felix Jr.

Yolanda Garcia gave birth to baby Felix Louis Garcia Jr. at 9:16 pm on December 12, 2004. Felix Jr. weighed 8 pounds 4 oz. Some people are calling him “Lupito” since he was born on the day of our lady of Guadalupe.  Yolanda, Felix and Felix Jr. are all doing well!

Congratulations!

Baby Felix and Colby were born one day apart!

Daddy Chris and Colby

Amy Templin gave birth to Colby Mitchell Templin at 1:55 pm on December 13, 2004. Colby Mitchell weighted 9 pounds 6½ oz.  Daddy’s already put his boots on!

Wyeth Hemophilia B Brochure

“Living with Hemophilia B” – a brochure that specifically discusses the diagnosis and treatment of hemophilia is now available from Wyeth.  One copy is enclosed with our newsletter.  If you need additional copies please contact The Coalition for Hemophilia B at (212) 554-6898.

Living with Hemophilia – A Community Education Series

Success from the Start
Overview of hemophilia and treatment options.

Choosing a Factor Replacement Therapy
History of Hemophilia, development of recombinant clotting factors, treatment options.

Making Treatment Choices
Progression of joint disease, the preventive role of effective treatment.

Day-to-Day Life
Community Resources.

If you think one of these programs would be helpful, you can contact us, your local chapter or HTC, who can contact their Wyeth representative to schedule a seminar in your area.

For information on Wyeth’s programs and activities directly from the company – as well as information on living with hemophilia – please visit Wyeth at  www.hemophiliavillage.com to sign up for the newsletter.~

NACCHO

North American Camping Conferenceof Hemophilia Organizations

NACCHO’s 3^rd Annual North American Conference of Hemophilia Organizations (NACCHO) will be conducted by The Hemophilia Association in Tempe, Arizona on February 11-13, 2005.

Representatives of hemophilia camps are invited to attend to network and share best practices for conducting summer camp.  If you are interested in attending, contact the Hemophilia Association at (602) 955-3947 or (888) 754-7017 (toll free).

NACCHO is sponsored by a grant from Wyeth.

The Coalition for Hemophilia B Breakfast Meeting

Friday, April 1, 2005 9:30 am – 11:30 am
Sheraton Music City Hotel
Nashville, Tennessee
Topic: Making Treatment Choices
Dr. Christopher Walsh will speak about the effect of bleeding into joints over time and options for preventing joint damage.  This program will also discuss the advantages and disadvantages for prevention and then accessing veins.

Our Factor Nine family support meeting will follow the speech.  It is a place where people with hemophilia B and their families can come together and share their concerns, gain knowledge, support, hear interesting stories and form lasting friendships.

Hemophilia B Reception
Please join us for a hemophilia B reception on Friday April 1, 1:00 pm – 2:00 pm at the Sheraton Music City Hotel, Nashville, TN - Lounge Area – Signs will be posted in Lounge/Lobby.

Please see the pre-registration forms (enclosed) for Meetings.  If you have any questions please Call Kim at (212) 554-6898.   Meetings above are held in conjunction with the Hemophilia Federation of America Conference.

Both meetings sponsored by The Hemophilia Federation of American in conjunction with Wyeth Phrmaceuticals.

Building Strong Relationships for Men

(with Joe Caronna & Robert Berkley)
Thursday, March 31, 2005
Sheraton Music City Hotel
12:00 pm – 5:00 pm
(lunch and snacks served)
Building Strong Relationships for Men is a coaching workshop on relationships specifically for men with a bleeding disorder, or for those with a child who has one.  It is effective for singles who anticipate being in a great relationship; men in a new relationship who want tools to start off on the right foot; and men in long-term, committed relationships who want a deeper, more fulfilling connection.

Please see pre-registration form (enclosed) for more information. Seating is limited please return pre-registration form ASAP.

Research Update by John Taylor, Jr.

Things are happening in Hemophilia research, mostly good.  Several new Factor VIII products that are focused on inhibitor patients are wending their way through trials, which is a very good sign for those involved.  It is also a good sign for all of us with Factor IX, as the scientists, companies, and the FDA are getting more practice at specialized recombinant products.  Products are being tailored for a smaller population and that means us.

The bad news is that Avigen is dropping the gene therapy product for Factor IX, but Dr. Kathy High is still working hard on what I believe will eventually be a product that will make her famous.  We have heard that she is making progress and that this effort will reach a positive conclusion, but there are hurdles ahead.

A project that many of us have watched for years is the transgenic production of recombinant Factor IX in the milk of pigs.  The science has advanced a long way, but there are still no transgenic products that have made it through the FDA.  As a result, many of the big pharmaceutical companies have shied away from this project even though it looks great.

The issue that holds the key to the future now is not the science but the money.  There is a third possibility, and that is the production of a “follow on” recombinant Factor IX, incorporating the knowledge that science has gathered since BeneFIX was created and making a less expensive and equally as good product. Of these three, gene therapy, transgenic IX or a follow-on IX, which one should come first?  If we were to throw our weight behind one, which one would it be? We wonder what the right answer is.  What is your opinion?  Please call or write and tell us.~

We would like to offer a special thanks to the following contributors of our newsletter:
Novo Nordisk
Wyeth Pharmaceuticals
Zlb Behring LLC