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Celebrating World Hemophilia Day

Laurie Kelley April 18, 2016
Today is World Hemophilia Day, celebrated by thousands of people with hemophilia around the world. We’re a small community: only an estimated 400,000 worldwide. Of this, only 25% are estimated to have adequate treatment. The majority with hemophilia have little to no access to medical care or factor, the blood clotting medicine that stops bleeding.

Today is also the birthday of World Federation of Hemophilia founder Frank Schnabel, an American who moved to Canada to seek better access to treatment and financial coverage for that expensive treatment. It was there he founded the WFH over 50 years ago. Today the WFH represents a staggering 120 countries as members. With membership, these countries receive NGO training, medical help, donations of factor and educational materials.

This week I am traveling to one of WFH’s member countries, Uganda. This is my first time there, and I go to invite the country to join our child sponsorship program Save One Life. Save One Life has over 1,200 children and young adults with bleeding disorders enrolled and has over 400 sponsors who provide financial support. Nurse Kate Khair, from the Great Ormond Street Hospital in London, has been to Uganda several times to help diagnose children with hemophilia. She wrote to me about the challenges of working in a country where so many are undiagnosed and live in rural areas:

Pictures from Barbados’s first World
Hemophilia Day!

“I met a family of 4 boys who have a small holding about a 5 hour drive from Kampala (the capital). When we were there, mum was 8 months pregnant, the boys all have terrible joints and told us about having to go to the river to get water – imagine that with a big knee bleed. There was one sister; she and the dad were essentially holding the family together. The boys’ wish was to be able to go to school, but it was too far to walk. I’m sure this is a familiar story for you, but my 2 team members and I have been profoundly affected by them. Save One Life makes, I know, massive impacts on families like these.” 

Indeed it does! I am especially encouraged today by the efforts of a mother of a child with hemophilia in Barbados, Erica Worrell, who has created the first hemophilia society there. Two years ago I traveled to Barbados to try to motivate the patients to start a national patient organization. The interest was there, the needs are great; we met quite a few families and planted some seeds of advocacy. All it took at last was one brave mother to take the first steps. Today, Barbados celebrated World Hemophilia Day for the first time, and has joined our global community. This is what the day is all about, celebrating the efforts of the many who work so hard to bring support and medical help to those in need, while bringing attention to the public about this rare disorder. Together, we can do it, and fulfill a dream Frank Schnabel had so long ago.

http://www.barbadostoday.bb/2016/04/17/barbados-observes-world-haemophilia-day/#comment-96261

Remembering Ryan

Laurie Kelley April 11, 2016

On April 8, 26 years ago, 18-year-old Ryan White died.
He is remembered in the bleeding disorders community for his extraordinary courage. Born with hemophilia, he was diagnosed with AIDS when he was only 13, in 1984. AIDS was not well understood at this time, and fear was rampant in the public. People thought you could catch HIV by shaking hands with someone with it, or just being near them. Ryan contracted HIV from his clotting factor, which at the time was not treated to destroy viruses. He posed no threat to anyone.
When his school tried to keep him from attending, Ryan and his mother Jeanne White Ginder launched a legal battle, and Ryan became a celebrity. And celebrities stood by him, including Michael Jackson and Elton John. He became the face of discrimination against those with HIV, and became an advocate for AIDS research and public education. He died in April 1990, one month before his high school graduation.
In August 1990, four months after White’s death, Congress enacted The Ryan White Comprehensive AIDS Resources Emergency (CARE) Act (often known simply as the Ryan White Care Act), in his honor. The act is the United States’ largest federally funded program for people living with HIV/AIDS. The Ryan White Care Act funds programs to improve availability of care for low-income, uninsured and under-insured victims of AIDS and their families. The legislation was reenacted in 1996, 2000, 2006, and 2009, and is now called the Ryan White HIV/AIDS Program.
Today, his mother Jeanne continues to advocate to spread Ryan’s message and story. 
For an excellent multimedia look at Ryan’s impact on the hemophilia community, visit HFA’s website. 

Considering Long-Term Health into Your 20s

Laurie Kelley April 4, 2016
By the time teens with hemophilia transition into their 20s, ideally they’ve built a firm foundation on which to manage their disorder. Among many skills, young adults should be able to self-infuse and negotiate health insurance. But when you’re healthy and your hemophilia is under control, it’s easy to overlook general health maintenance beyond hemophilia.
            It’s uncommon for 20-somethings to consider the long-term impact of their lifestyle choices. So what if I’ve gained a few pounds? I only smoke when I’m out with friends. I look so much better with a tan. But young adulthood is the time to develop the habits that will help maintain a healthy body in the decades ahead.
I’m Independent. Now What?
Perhaps more than any other time in our lives, the 20s are a decade of major transformation. Many young adults move out of their parents’ home. Others graduate from college. Even those who’ve diligently managed their hemophilia may be thrown for a loop by all the changes in their lives.
            Your hemophilia treatment center (HTC) may no longer be in the same town or even the same time zone. And finding an HTC is just one of the myriad issues you’ll need to address: Where will I buy groceries? How long will my commute be? When will I find time to renew my driver’s license?
            These changes may take priority, and if you’re feeling healthy, finding a local dentist or doctor doesn’t seem all that pressing. Plus, who has the time?
Not Enough Hours in the Day
Entering the 9-to-5 world for the first time is a big deal that can have a major impact on your health—and not just for those in Deadliest Catch-type occupations. For many, this is their first experience sitting behind a desk for several hours a day. A tangible and common consequence is weight gain. The Centers for Disease Control and Prevention (CDC) reports that an astounding 20% of Americans in their 20s are obese. Not only is extra weight bad for your joints, but obesity is a contributing factor to several leading causes of death, including heart disease, stroke, and certain types of cancer.
            The effects of “a few extra pounds” may not be immediately apparent, but that’s the point. In your 20s, you need to establish good habits to protect your body for the long run. This means adjusting what you’re eating while maintaining some form of routine exercise. Neither is easy to accomplish when you’re just starting life on your own.
            Ian Muir, a 25-year-old with hemophilia, took a new job recently and is slowly figuring out how to get all the pieces of his life to mesh. In college, Ian competed in triathlons, training 25 hours a week. “In school, you had the motivation of working out with your teammates,” says Ian. “And you had a relatively flexible schedule.” Now he’s struggling to find the hours to train for just a fraction of that time. And like many of his peers, Ian has let his diet suffer: “I know I need to get back to eating food that’s good for me, and not just what’s convenient.”
            Living healthy in your 20s doesn’t necessarily require big time commitments. In some cases, you just have to make better choices: When you can, take the stairs instead of the elevator. Cut back on the amount of alcohol and caffeine you consume.
And if you’re among the 1 in every 5 Americans who smoke, quit now. According to the CDC, smoking causes more deaths in the US than alcohol, illegal drugs, and motor vehicle accidents combined.
            Sure, you may not have all the time in the world, but your general health should sit atop your list of priorities.
Molehills Can Become Mountains
While you lived at home with your family, you probably had a stable network of medical resources. But once you’re on your own, you may need to rebuild that network by establishing a relationship with your new HTC, plus maintaining all facets of your health.
            Dental health is one of the most neglected aspects of overall well-being. By the time you’re in your 20s, Mom or Dad probably don’t schedule your dentist appointments, which means that nobody does…until a minor toothache becomes something worse.
            Ian tells a common tale. “I’ve been a bit derelict when it comes to the dentist. What probably wouldn’t have been a big deal had I stayed on top of it, ended with a root canal.”
            Your primary care physician (PCP) can be your best ally. You’ll turn to your PCP when you can’t kick that cough you’ve had for three weeks or are worried about anything from a wart on your foot to your sexual health.
            As Ian assembles the medical resources he needs near his new home, he sees the value in finding the right PCP. “You want someone you can go talk to about health concerns that aren’t hemophilia related,” he says. “Someone who knows you and your medical history and if necessary, who can point you to the right specialists.”
            In addition to addressing your current health concerns, your PCP will review your family history with you, assessing your risk for ailments like heart disease, diabetes, and certain cancers. Armed with this information, together you can develop a plan to reduce some of the risks.
Why Do Today What I Can Put Off ’til Tomorrow?
Throughout your 20s, medical concerns may surface that have nothing to do with hemophilia, and everything to do with the natural aging process. You’ll begin to shed the cloak of invincibility you donned as a teen, and realize that you need to act with an eye to your future.
            So manage your total health as attentively as you manage your hemophilia:
            • Eat a healthy, balanced diet.
            • Maintain your optimal body weight.
            • Make exercise a priority.
            • If you drink or smoke, reduce your alcohol consumption, and quit smoking!
            • Schedule all the exams you’ve neglected for so long.
            • If you don’t have a local dentist, eye doctor, or PCP, contact your HTC or insurance company. They can help you find one.
True, for now, you can ignore the incessant commercials for “old people” drugs like Lipitor. But let them serve as a reminder for the not-too-distant future. What’s Lipitor anyhow? It’s a drug that controls cholesterol. What’s cholesterol?

It’s one of many topics you and your doctor should start discussing.

by Kevin Correa
PEN 2.11     © 2011 LA Kelley Communications

Hear About Nathan’s “IXperience” With His Factor IX

Laurie Kelley April 4, 2016
Last year I told you about the FDA approval of IXINITY® [coagulation factor IX (recombinant)] for people with hemophilia B (FDA Approves a New Factor IX Treatment for Hemophilia B!).  Now people are sharing their firsthand experiences with this factor IX. Watch Nathan’s full video to see more of his story on IXINITY.com.
Be sure to speak with your doctor about any new treatment that you’re considering. This is Nathan’s experience and it may not be typical. 
(The following is a paid placement of product information by Emergent BioSolutions.)
Nathan
Here’s what Nathan says about his IXINITY IXperience™. 
I’m 19 now and have severe hemophilia B. Over the years I’ve had a lot of bleeds. What interested me in IXINITY was that there were no inhibitors found in the clinical trials, the recovery rate was 98%,* and it’s not a plasma-derived product.1 

My hematologist and I collaborated and thought that with a 98%* recovery rate, we should be able to at least try to lower the dosage. Now I’m infusing 4,000 IU per infusion—less than in the past. 
Since I started IXINITY in July of 2015, I’ve experienced no bleeds. I also like the IXINITY reconstitution device. It’s so simple, and there’s really nothing to it. It’s just right there in front of you. 

Nathan’s experience with IXINITY may not be typical. Speak with your doctor to see if IXINITY may be a good option for you. 

*IXINITY recovery is an average based on lab tests of patients in the clinical study.1 Your actual recovery and dose may be different. Speak with your healthcare professional about the right dose for you. 
Visit IXINITY.com to learn more about IXINITY and the FACTOR IT FORWARD program. You can also call an IXINITY IXperience Concierge™, toll-free, at 1-855-IXINITY (1-855-494-6489). 
IXINITY INDICATIONS AND IMPORTANT SAFETY INFORMATION 
What is IXINITY®? 

IXINITY [coagulation factor IX (recombinant)] is a medicine used to replace clotting factor (factor IX) that is missing in adults and children at least 12 years of age with hemophilia B. Hemophilia B is also called congenital factor IX deficiency or Christmas disease. Hemophilia B is an inherited bleeding disorder that prevents clotting. Your healthcare provider may give you IXINITY to control and prevent bleeding episodes or when you have surgery.
IXINITY is not indicated for induction of immune tolerance in patients with hemophilia B. 
IMPORTANT SAFETY INFORMATION for IXINITY® 

• You should not use IXINITY if you are allergic to hamsters or any ingredients in IXINITY. 
• You should tell your healthcare provider if you have or have had medical problems, take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements, or herbal remedies, have any allergies, including allergies to hamsters, are nursing, are pregnant or planning to become pregnant, or have been told that you have inhibitors to factor IX. 
• You can experience an allergic reaction to IXINITY. Contact your healthcare provider or get emergency treatment right away if you develop a rash or hives, itching, tightness of the throat, chest pain, or tightness, difficulty breathing, lightheadedness, dizziness, nausea, or fainting. 
• Your body may form inhibitors to IXINITY. An inhibitor is part of the body’s defense system. If you develop inhibitors, it may prevent IXINITY from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for development of inhibitors to IXINITY. 
• If you have risk factors for developing blood clots, the use of IXINITY may increase the risk of abnormal blood clots. 
• Call your healthcare provider right away about any side effects that bother you or do not go away, or if your bleeding does not stop after taking IXINITY. 
• The most common side effect that was reported with IXINITY during clinical trials was headache. 
• These are not all the side effects possible with IXINITY. You can ask your healthcare provider for information that is written for healthcare professionals. 
Please see full Prescribing Information for IXINITY here.
You are encouraged to report side effects of prescription drugs to the Food and Drug Administration. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. 
Reference: 1. IXINITY [coagulation factor IX (recombinant)] prescribing information. Winnipeg, MB, Canada; Emergent BioSolutions Inc.; April 2015. 
Manufactured by Cangene Corporation, a subsidiary of Emergent BioSolutions Inc. and distributed by Cangene bioPharma, Inc., a subsidiary of Emergent BioSolutions Inc. 
IXINITY® [coagulation factor IX (recombinant)], IXperience, and any and all Emergent BioSolutions Inc. brand, product, service and feature names, logos, slogans are trademarks or registered trademarks of Emergent BioSolutions Inc. or its subsidiaries in the United States or other countries. All rights reserved. 
©2016 Emergent BioSolutions Inc. All rights reserved. CM-FIX-0024 

An Inspiring ELOCTATE Story

Laurie Kelley March 14, 2016

The famous Nigerian poet and novelist Ben Okri wrote,
“Stories can conquer fear, you know. They can make the heart bigger.” In this
week’s blog, a young mother shares her personal story about hemophilia, about
overcoming fear, handling her emotions and learning about a therapy that could work
for her family. She shares her story, to help others facing the same fears. We
hope to bring you more such stories throughout the year.

Thanks! Laurie

Indications ELOCTATE is an injectable medicine that is used to help
control and prevent bleeding in people with Hemophilia A (congenital Factor
VIII deficiency). Your healthcare provider may give you ELOCTATE when you have
surgery. Important Safety Information

Do not use ELOCTATE if you have had an allergic
reaction to it in the past.

My name is Sara, and my
8-year-old son Evan has severe hemophilia A.
The circumstances surrounding
Evan’s diagnosis are likely familiar to many other families in the community—a standard
heel prick that wouldn’t stop bleeding, eventually soaking his newborn clothes.
I remember the nurse quickly whisking Evan down to the nursery, and I watched
as three, four, then five nurses dropped what they were doing and frantically
tended to our baby over the course of the next hour. The doctor finally told me
he suspected Evan had a bleeding disorder and gave a very brief description of
hemophilia.
This was not the life I had
envisioned for my family.
Fast forward sixteen months
later—I had studied every pamphlet and book and memorized signs and symptoms of
every possible bleed like it was my job. I kept on top of appointments and
diligently brought Evan to the ER each time he knocked his head. And then it
happened, my greatest hemophilia fear: a spontaneous head bleed. Hearing things
like, “subdural hematoma, evaluate the need to drill, lucky to be alive,”—all
of it was so surreal. I felt helpless watching my innocent 16-month-old fight
for his life.
I somehow found it in myself
to channel that fear and anger into fuel for managing Evan’s hemophilia. I
forced myself to attend social events and conferences, and ended up making a
great handful of friends who have become a part of my hemophilia lifeline. At
doctor appointments, I began to ask questions without censoring myself in order
to get answers I could understand.
I also began researching
treatment options. By learning about ELOCTATE myself, I was able to work with
my doctor to find the answers to my lingering questions, evaluate the risks and
benefits, and make an informed, educated decision with our physician to see how
Evan would respond to it.
Evan has been taking ELOCTATE
for his severe hemophilia A since August 2014. He does typical 8-year-old
things like riding his bike, running, or playing sports with his cousins.
They’re boys so they want to tackle and hit. Now he knows he has to play
two-hand touch and with the Nerf ball. His cousins have grown up with him so
they’re used to it. They’re attitude is just, “Okay, cool. Let’s go play.”
Since starting on ELOCTATE,
he hasn’t had any spontaneous bleeds. And he’s becoming more receptive to being
involved in his own care.
I’ll always be a protective
mom, but it means so much knowing I can count on ELOCTATE to help care for
Evan’s hemophilia. I am not only grateful for the opportunity to share my
experiences as a caregiver of a child with severe hemophilia A, but also to
provide some wisdom, and, hopefully, some inspiration to a unique, capable, and
fantastic community of people.
You can see more about Evan
and me in our video here  on the
ELOCTATE website.
__________________________________________________________________________________
Talk to your healthcare
provider about whether ELOCTATE may be right for you. Please visit www.ELOCTATE.com to read the Indications and Important Safety Information , as well as
the full Prescribing Information .
ELO-US-0880
02/16
This blog was sponsored by Biogen, for educational
purposes.

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