October 2017

Help is Coming, Victoria!

Laurie Kelley October 29, 2017

On September 20, Hurricane Maria ripped through the sunny, tropical paradise of Puerto Rico. With sustained winds of 175 mph, Maria has been classified as Category 4 hurricane, and one of the deadliest to hit the island. It’s the tenth most intense storm ever in the Atlantic region. Wind, flooding, downed power lines—the island was battered to its knees. Currently 75% of the island still lacks electricity and many residents—Americans—have lost everything. It’s an ongoing humanitarian crisis that needs our help. For those with hemophilia on the island, access to medical care and factor was cut off. Below is one VWD family’s story of riding out the storm, and the aftermath.

Happier Times: Victoria (R) with sister Larah (L) and mother Maritza

Victoria Pereira Figueroa is only 19 years old and lives in Canovanas San Isidro, Puerto Rico with her family. She has type 1 VWD. When they were warned of the impending twin sister hurricanes, Irma and Maria, she felt her family was well prepared for the arrival of a strong hurricane. With each “tolmentera,” thunderstorm, the family purchased water and necessity items, and prepared packages for each one. They live in a rented concrete house, which seemed very stable.

“During Hurricane Maria, my little sister Larah was very scared, so my mom decided to move us with our beds on the first floor as it seemed safer,” Victoria recounts. “At about six o’clock in the morning, September 21, when Maria lashed in her second round with all her fury, our house completely flooded filling with more than eight feet of water! We had to take refuge in a hurricane in a school that is four houses away from ours—it was very dangerous to go out. At the emergency shelter they received me, my family and even my puppy!” Victoria and her family and puppy spent two weeks in the shelter, with other families. Conditions were difficult. There was a lack of essential necessities such as water, light at night, food and a place to sleep. The
family had to sleep on the floor, with the puppy curled up tightly next to them.

The family also suffered lack of factor, and contracted several viruses that were spreading rapidly in a place lacking sanitary conditions. But the close quarters and survival conditions had one ray of light: “We were able to make good friendship,” said Victoria.

The aftermath was shocking; Victoria’s neighborhood was devastated. Her world was upended.
“Two days after the hurricane my parents went to get the floodwater out of the house and clean up. When I had the opportunity to go to my house it was very shocking for me to see what was destroyed. My bedroom was a mess, my desk was completely smashed together with all my University books and notes; everything was scattered on the floor, under the water and mud. I was very distressed to see my things like that.”

Muddy and unsanitary floodwaters filled the downstairs

Victoria was heartbroken, but her family was safe, her puppy still by her side, and her spirit unbroken. Remarkably, she adds, “Rather than just worry about ourselves, I am trying to continue helping others in need, and I’m returning to the shelter to help the children, with games and activities like painting.” Victoria, in the midst of terror, then devastation, has still managed to find a way to give back. Her community has rallied together. Can we do the same for our hemophilia families there, from the comfort of our homes on the mainland?

Hemophilia Federation of America has a fund dedicated to raising money solely for the families of Hurricane Maria, to be given directly to them to aid in rebuilding their homes and lives. We will ensure the money will be destined in their hands and bank accounts, and we will track its usage.

Having helped impoverished families in developing countries for over 20 years, I’ve seen the promise and pitfalls of donating money. I can ensure this money will be used properly and with accountability to benefit families directly. It’s our mission and method of operating. HFA Executive Director Kimberly Haugstad and I are working together on this. Please consider donating today! We will be making trips to PR soon to deliver funds and oversee their usage.

Help is coming,Victoria!

Friends and colleagues, please go to
http://www.hemophiliafed.org/ and make a donation today!

Learn about factor treatment, an established option in hemophilia

Laurie Kelley October 22, 2017

Since 1990, we’ve been educating the hemophilia community about choice of therapies. Please read about coagulation, half-life, and a type of hemophilia treatment.

For people with hemophilia, factor treatment temporarily replaces missing factors (proteins) in your body.1 Factor treatment helps to control a bleed by producing a proper blood clot when you need it, and where you need it.1-6 It does so by following the natural balance of on-and-off steps in the blood clotting process.2,7-9 You can compare the on-and-off steps to the way a light switch turns on and off.

Before a bleed occurs, the factor activity remains “off,” ready but waiting until it is needed.10 Once there is a bleed, the blood clotting process is turned “on,” like a light switch, and factor is activated.2,8 After factor is activated, it can help stop a bleed in 3 tailored ways: when the bleed occurs, at the site of the bleed (where), and (how much) in an amount needed to control the bleed.2-4,9,11 After the bleed is controlled, the blood clotting process is turned off, factor is inactivated again, and the unused factor is eliminated from the blood.2,12

Factor treatment can be used in multiple scenarios to meet different treatment needs. Because factor is infused intravenously (IV), the proteins for clotting are immediately available for use, allowing for bleed protection when you need it.13

Prophylaxis treatment is the routine infusion of factor with the goal of preventing bleeding episodes before they happen.1
On-demand treatment is infused to control bleeds once they begin.1
Factor can also be used to help prevent and manage bleeds during or after surgery.14

Keep in mind that every person with hemophilia is different. You and another person with hemophilia who are the same age and weight may require different amounts of factor—depending on how your bodies use factor. Your healthcare provider can individualize your treatment regimen by adjusting your infusion dose and frequency, based on how your body uses factor, your activity level, and lifestyle needs.15,16

Healthcare providers can reliably measure factor levels in your blood. Doing so can help individualize your treatment and help minimize bleeding risk.15,16 Talk with your healthcare provider to learn more.

Your lifestyle and activities can have an impact on your bleeding risk.16 Use of factor over the years has shown benefits in managing, controlling, and reducing bleeds.17,18 It is proposed that all bleeding, even bleeding you may not readily feel or see (subclinical or micro-bleeding), can result in significant damage to joint and bone health.19,20

For over 50 years, healthcare providers and patients like you have continued to rely on factor treatment.18 With substantial bleed-reduction benefits and experience in over 170 clinical trials, factor treatment is considered a well-studied and established treatment option.16,17,21

Shire has been dedicated to pursuing advancements in hemophilia for more than 60 years. As a leader and innovator in hemophilia research, we offer a vast array of patient programs and support—all of which are proudly brought to you by our dedicated people.

Explore the Possibilities. Talk to your healthcare provider to learn more about factor treatment.

To learn more about treating hemophilia, you can visit: http://www.bleedingdisorders.com/bleeding-disorders-info/ http://www.hemophilia.org
http://www.wfh.org
http://www.hemophiliafed.org

References
1. Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388:187-197.
2. Johari V, Loke C. Brief overview of the coagulation cascade. Dis Mon. 2012;58:421-423.
3. Gale AJ. Current understanding of hemostasis. Toxicol Pathol. 2011;39(1):273-280.
4. Stavenuiter F, Bouwens EAM, Mosnier LO. Down-regulation of the clotting cascade by the protein C pathway. Hematol Educ. 2013;7(1):365-374.
5. Antovic A, Mikovic D, Elezovic I, Zabczyk M, Hutenby K, Antovic JP. Improvement of fibrin clot structure after factor VIII injection in haemophilia A patients treated on demand. Thromb Haemost. 2014;111(4):656-661.
6. Hvas AM, Sørensen HT, Norengaard L, Christiansen K, Ingerslev J, Sørensen B. Tranexamic acid combined with recombinant factor VIII increases clot resistance to accelerated fibrinolysis in severe hemophilia A. J Thromb Haemost. 2007;5:2408-2414.
7. Lenting PJ, van Mourik JA, Mertens K. The life cycle of coagulation factor VIII in view of its structure and function. Blood. 1998;92(11):3983-3996.
8. Berg JM, Tymoczko JL, Stryer L. Many enzymes are activated by specific proteolytic cleavage. In: Biochemistry. 5th ed. New York, NY: WH Freeman; 2002.
9. Hoffman M, Monroe DM. A cell-based model of hemostasis. Thromb Haemost. 2001;85:958-965.
10. World Federation of Hemophilia. The clotting process. https://www.wfh.org/en/page.aspx?pid=635. Accessed September 5, 2017.
11. Colvin BT. Physiology of haemostasis. Vox Sanguinis. 2004;87(suppl 1):S43-S46.
12. Orlova NA, Kovnir SV, Vorobiev II, Gabibov AG, Vorobiev AI. Blood clotting factor VIII: from evolution to therapy. Acta Naturae. 2013;5(12):19-39.
13. Canadian Hemophilia Society. Factor replacement therapy. http://www.hemophilia.ca/en/bleeding-disorders/hemophilia-a-and-b/ the-treatment-of-hemophilia/factor-replacement-therapy/. Accessed September 20, 2017.
14. World Federation of Hemophilia. Guidelines for the management of hemophilia, 2nd ed. World Federation of Hemophilia, Montreal, Quebec, 2012.
15. Castellone DD, Adcock DM. Factor VIII activity and inhibitor assays in the diagnosis and treatment of hemophilia A. Semin Thromb Hemost. 2017;43(3):320-330.
16. Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia. 2014;20:607-615.
17. Franchini M, Mannucci PM. The history of hemophilia. Semin Thromb Hemost. 2014;40:571-576.
18. Center for Biologics Evaluation and Research. User fee billable biologic products and potencies approved under Section 351 of the PHS Act. http://www.fda.gov/AboutFDA/CentersOffices/ OfficeofMedicalProductsandTobacco/CBER/ucm122936.htm. Accessed September 20, 2017.
19. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535-544.
20. DiMinno MND, et al. Magnetic resonance imaging and ultrasound evaluation of “healthy” joints in young subjects with severe haemophilia A. Haemophilia. 2013;19:e167-e173.
21. NIH Clinical Trials Registry. Ongoing and complete clinical trials using factor in patients with hemophilia. https://clinicaltrials.gov/ct2/results/details?term=Factor+VIII&recr=Closed&fund=2. Accessed September 20, 2017.

©2017 Shire US Inc., Lexington, MA 02421.
All rights reserved. 1-800-828-2088.
SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates. S35567 10/17

What’s Happening with Helixate® FS?

Laurie Kelley October 16, 2017

We’ve had a tremendous number of factor products enter the US hemophilia market the past few years. in fact, we have exploded from under 10 to about 26 now, not including inhibitor products!

Rarely do we speak of a product exiting the market. But as of December 31, one will no longer be manufactured—Helixate® FS (Antihemophilic Factor (Recombinant)).

I’ve read on Facebook some conspiracy-theory explanations! But here are the facts:

1. Helixate® FS is the exact same product as Kogenate® FS. Exactly.

2. Both products are made by Bayer Corporation. The therapy that makes Kogenate FS has been sold to CLS Behring annually as part of a lawsuit settlement many years ago. CSL then markets the product as Helixate® FS. This was good for CSL, which at the time did not have a recombinant factor VIII product.

3. The resultant contract is coming to an end; CSL Behring and Bayer will no longer partner to product and market Helixate FS.

4. And CSL now has recombinant FVII products that it has created, namely Afstyla (Antihemophilic Factor [Recombinant], Single Chain, a next-generation factor VIII with twice-weekly dosing available).As with Helixate FS, Afstyla uses the Mix2Vial® system, which will keep patients’ reconstitution process consistent.

5. CSL wants to assure the community that it will update patients, prescribers and specialty pharmacies with updated supply information. Community members can sign up here for direct updates.

6. The products that are already manufactured will still be in the distribution pipeline, probably through to early 2019. This gives patients and their families plenty of time to consider treatment options.

If you currently use Helixate FS, you should speak with your HTC hematologist soon about what treatment options you have, given your insurance policy and specialty pharmacy. You can also contact a CSL Behring My SourceSM Care Coordinator at 1-800-676-4266 Monday–Friday, 8 AM to 8 PM ET–but please speak with your hematologist, who can help you find the right therapy for you.

Helixate FS is a great product, and there are many more also great to choose from. Use our factor chart here to start looking. Good luck and always Factor Up!

This is an unpaid public service announcement.

“The Only Real Magic …”

Laurie Kelley October 8, 2017

Music, as far as I have seen in the world so far, is the only real magic that I know. There is something really honest and clean and pure and it touches you in your heart. Tom Petty

Tom Petty, incredible musician and performer, sadly passed away on October 2, and joined other rock stars in the sky this year: Greg Allman ( Allman Brothers), Sib Hashian ( Boston), Chris Cornell (Soundgarden, Audioslave), and more. Last year we lost heavy hitters like Prince, and David Bowie. Musicians provide the lyrical and emotive background of our lives. When we hear a certain song, our minds can recall towns, people, news events, lost loves, and hopes that happened at the same time we first heard that song.

I love music, from Bach to Metallica, and feel it unites people in ways that are almost spiritual. You can meet someone completely different from you, in age, race, ethnicity, religion, gender… but when you learn they love the same band you adore ( the Doors!), you suddenly find you have everything in common with them!
So, I have to shout out two amazing artists in our bleeding disorders community: Billy Conde and Mathew Tache, who are more than wonderful musicians. They’ve put together a way for music to unite those with hemophilia.

They’ve created Blood Vibrations ( a tip of the hat to Beach Boys’ Good Vibrations). It’s a website devoted to bringing musicians with bleeding disorders together to publish their songs. From the website: “Blood Vibrations is an ongoing grassroots project that collects sound and music created by people with bleeding disorders. The goal of the project is to provide a forum for creativity, expression, sharing and learning- all contributing to whole being wellness. Anyone with a bleeding disorder is welcome to participate. No musical experience is required. Record in hi-fi, lo-fi, no-fi. We encourage collaborations and group works.”

Billy and Matt facilitate all the ongoing work themselves– soliciting music/art, website, and promotion. The project is free ( no cost to share/submit and no cost to listen). They do not accept finding from outside sources– all costs are paid by these two wonderful men with hemophilia directly.

Check out Blood Vibrations, and email Billy and Matt at bloodvibrations@yahoo.com if you have an idea for a song, or one to submit to create an album.

You can also download music from the latest Blood Vibrations album, Blood Vibrations 6. You might see some talented community members you know on some of the albums, like Shelby Smoak, Kevin Finkle, Josef Zwier, and Jenny Massey.

Coincidentally, when Billy and I communicated, he was just listening to No Rest for the Wicked by Ozzy Osbourne, Zakk Wylde on lead guitar. I had just met Zakk Wylde in Utah; unbelievably great and intense guitarist! “Counterfeit God” is my favorite song of his so far.

Billy adds, “I’ve also been immersing myself since Monday in the music of Tom Petty, which was important to me as I was discovering rock ‘n’ roll as a kid.”

Listen to music, all types. It stirs memory, enriches the mind, feeds the soul. And if you can… make music. Now you can, free and easy, and with our community, at Blood Vibrations.

Our Vietnam Wall: The Hemophilia Memorial

Laurie Kelley October 1, 2017

I’m stunned into silence while watching Facebook, as I see the names on
banners…Papo Gonzalez, Patty Kuhn, Charles Carman, John W. Cavanaugh, Tom Fahey, Christopher Pitkin, Loras Goedken, Terry Stogdell, Greg and Tim Haas, Michael Sutton, Bill McAdam, George McCoy, Brent Runyon, Larkey DeNeffe, Brian Craft…

So many of these people with hemophilia were known to me personally; some I shared a drink with, or travel, or swapped stories. Brian Craft and I were once in a video together, back in 1993! Tom Fahey and I met several times as he was nearby in Boston. I had removed their names from my mailing list, over and over, as each one passed. I still keep a framed photo of Tom in my home office. All are gone, victims of one of the greatest tragedies in the history of medicine. The infection of our nation’s blood supply by HIV.

They are like our veterans. Each November we remember those who sacrificed their lives so that we could live more free ones, so that we can escape the threat of tyranny, or the threat of invasion. Their deaths meant better quality of life for future generations. The massive deaths, up to 10,000, of those with hemophilia from AIDS spurred research, better factor products and ultimately better medicine to combat HIV, sparing thousands, perhaps millions around the
world from infection through blood products.

On September 16, these “veterans” were finally given a federal memorial, The Hemophilia Memorial, residing at the National AIDS Memorial Grove. The Memorial is similar to the Vietnam Wall, where names will be remembered forever. I wish I could have been there, but our community was well represented by the surviving family members, loved ones, physicians who treated patients and our national advocates. What an incredible and moving memorial to the
unsuspecting patients caught up in a maelstrom of disease and even deceit. Nothing like this virus had happened in history and it was the stuff of science fiction.

While we have needed this memorial to help heal, even 30 years later, it took a long time to make it happen. AIDS has been a painful reminder of our failures, and young lives lost. Now, as more and more hemophilia patients survive AIDS and live normal lives, even having children, it seemed right to have a memorial.

My son was born in 1987 as the AIDS firestorm had fully ignited; the very week he was born the Ray brothers were bombed out of their home in Florida. The next month was National AIDS Awareness Month. We lived in fear, of the disease but also of public sentiment, which was against us. This community has prevailed, in spirit, in attitude and in results. The slogan at the Dachau Prison Camp I visited at age 16 was “Never again.” I hope this can become our
slogan too. Success can sometimes breed complacency, and one thing this community has prided itself on is to never be complacent. Our future and our children’s lives depend on that. Hemophilia changed history twice: the overthrow of Tsar Nicholas II of Russia, and the way we with blood and disease worldwide.

Let’s change it once more with a cure. And this Memorial will be our everlasting remembrance of an ancient disorder and a modern age disease, whose time came, changed history, and went.

“Our story will be remembered. The Hemophilia Memorial will ensure
we never forget those we loved but lost.” —Jeanne White-Ginder, mother of Ryan White

LA Kelley Communications donated $1,000 to the Memorial during the NHF Annual Meeting opening night. Please consider making a donation
to the Hemophilia Memorial here.