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Puerto Rico: Soy Radames

Laurie Kelley November 13, 2017

Lily Gomez delivering supplies to the family

Meet Radames Castillo-Toa, and parents Auraliz Martinez and Francisco Castillo. Real Americans with hemophilia who suffered catastrophic loss when Hurricane Maria struck Puerto Rico on September 20. Lily Gomez, a mother of a child with hemophilia who lives in Miami, visited the island to assess damage, meet families and return with a report and recommendations for aid.

Lily shares that the oldest son Radames described the hurricane landfall as terrible. “Everything was flying around outside, and then inside our home we lost the roof. It was a long day.”

The father, Francisco, indicated that the family lost everything when part of the roof flew away, because everything got wet and the walls cracked. They had to evacuate and go to his mother-in law’s home and try to wait for FEMA there. In the aftermath, Francisco tried to cover the roof but the panels and the tarp he got are not enough.

“When it rains outside, it rains inside,” he commented. He added that an electrical pole flew away with the wind, leaving the family in the dark. Fortunately, he was able to borrow a
generator to keep the factor refrigerated. They are back at the house now, but are in desperate need of a new roof. Each time it rains, everything gets wet again.

Lily said, “We found them retrieving water from a spring coming out through a PVC pipe on the side of the mountain. He said he doesn’t know where the water is coming from but that’s the only water they have available for bathing, washing and toilets. They boil the water when they need drinking water. He said he knows about the problems with contaminated water. He said his wife leaves early in the morning to find drinking water.”

Communication is almost impossible, as there is barely any signal. The family submitted a request to FEMA for a roof, but FEMA is overwhelmed too. The family is still waiting. The father told Lily, “Thank God we are alive and working together to meet our needs.”

What to do? I am on the phone weekly with Kimberly Haugstad, executive director of HFA, wonder woman and also mother of a child with hemophilia. Hemophilia Federation of America has a disaster relief program, the only one of its kind for Americans with hemophilia, and probably this week we will confirm a plan to get funds to Radames and his family for a new roof. Imagine if your home looked like the photos below. Americans need to help Americans now, and our hemophilia community needs to start giving. During this month of Thanksgiving, it’s the right thing to do. www.hemophiliafed.org

Remembering Renée

Laurie Kelley November 6, 2017

This past week I’ve been working diligently on updating my book, A Guide to Living with von Willebrand Disease. I feel guilty saying it’s my book—it was actually “our” book, my and Renée Paper’s. It’s been out of print a while, and we truly need this resource. While thinking of Renée as I edited it, I realized that tomorrow marks the 10th anniversary of her passing, and working on this book made me realize again what a valuable leader, friend and advocate she was.

She was only 49 when she died, after an eight-week hospital stay following a fall. She had dealt with multiple health challenges: von Willebrand disease, diabetes, hepatitis C. Compounding this was obesity. After she lost her sister Michelle, who suffered from similar health concerns, she decided to have gastric bypass surgery, in an attempt to improve her health. She lost a remarkable amount of weight, but the years of illness had taken their toll on her body. What’s truly amazing is that nothing, nothing, seemed to slow Renée down!

She traveled and lectured frequently. She was a powerhouse when lecturing. I saw her absolutely command a room full of nurses and doctors with her photographic memory, brilliant knowledge of VWD and her deep-seated passion. She spoke with authority, compassion and a call to arms, for everyone to find unidentified VWD patients, get them the treatment they deserve and need, to stop the silent suffering of women. She herself had had a hysterectomy in her early 20s, rendering her unable to have children, when doctors did not correctly diagnose her with VWD and sought to end her uncontrollable bleeding. I think in part her burning dedication and fiery style of lecturing was fueled by the embers of what was left of her ability to control her life, to have children. She didn’t want this to happen to any other woman.

Fiery style of lecturing? If you never heard a Renée Paper speech, you missed some great and shocking speeches. One of my favorite lines by her was when she blamed the medical community for misdiagnosing women who had VWD as being “hysterical,” or “imagining” their illnesses. “You know why this happens?” she would bark out. “Because men dominate the medical scene! And you know why they don’t take us seriously? Because men don’t have uteri!” Leave it to Renée to always use the correct Latin plural of uterus.

Renée traveled with me to Puerto Rico and the Dominican Republic on business together, and she and I presented in places as close as Connecticut and far away as Australia. She was brilliant; she was fun-loving. She was could be irreverent yet compassionate. She loved animals, and one of her favorite gifts to her friends was to send a photo of herself each New Year’s with a different animal from different parts of the world: kissing a dolphin in the Caribbean; draping a boa constrictor around her neck in Mexico; nuzzling an alligator in New Orleans; atop a camel in the Canary Islands. Renée knew how to grab life by the horns and tame it, and she wanted others to do that, too, regardless if they had a disorder or disability.

Renée walked the talk: her message was always to get educated about VWD. Don’t let it keep you from enjoying life. Play the hand you were dealt. (Yes, she was from the Las Vegas area!) Play it and win.
Renée was a visionary leader. As an emergency room nurse in Nevada, she saw the need for a patient-based hemophilia organization and an HTC. Nevada had neither when she first lived there. Typical of Renée, she saw the need and figured out how to meet it. She founded Nevada’s first patient organization and HTC, both of which continue to this day. In fact, November 1 is Renée Paper Day in Nevada! How many people can claim such an honor?

So we remember an inspiring and action-oriented leader, who was warm and loving, and also at times, a pain! And she knew it, and didn’t care. She had places to go and things to do, and a higher calling. As if she knew the clock was ticking, she made incredible accomplishments that continue to benefit patients to this day—including the world’s first book on VWD, which will be re-released in 2018. A legacy like that is the mark of a true leader.

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.