May 2017

Just B with IXINITY®

Laurie Kelley May 28, 2017

It’s refreshing when a company really
connects with the community they’re providing treatment options for, which is
why today, I’m happy to spread the word about the new promotional campaign from
Aptevo, the makers of the factor IX treatment, IXINITY® [coagulation
factor IX (recombinant)].

Read below to get the story on how
people in the hemophilia B community inspired the campaign, plus see it for yourself at JustBIXperiences.com.

This is a paid public announcement from Aptevo and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the IXINITY website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal
identifying information against your will.

Finding inspiration from familiar faces

As IXINITY marks its second anniversary
as a treatment option for those with hemophilia B, the people behind the
product are excited to start the next phase with a fresh new face. They didn’t
have to look very far, because who better to represent the close-knit
hemophilia B community and the company that cares about them than real people actually
using IXINITY?

Introducing…

Just B: Because life is bigger than hemophilia
B, make it your IXperience™ with IXINITY.

Please see Important Safety Information for IXINITY below.

An idea that became an identity, inspired
by everyone who is redefining what it means to have hemophilia B by living life
on your own terms and Just B-ing yourself.

By sharing your experiences, your
expectations, and your aspirations, you’ve shown that, regardless of having
hemophilia or not, everyone deserves the chance to Just B.

Meet the first people to be featured
sharing their IXINITY stories (and see if there are any familiar faces from the
community that you recognize) at JustBIXperiences.com.

Plus, find out more about what it means
to Just B and get connected in the IXINITY Family Room on Facebook.

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.

For more information about IXINITY, please see full Prescribing Information,
including Important Patient Information.

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.

Aptevo
BioTherapeutics LLC, Berwyn, PA 19312

IXINITY [coagulation factor IX
(recombinant)] and
any and all Aptevo
BioTherapeutics LLC brand, product, service and
feature names, logos, and slogans are trademarks or registered trademarks of Aptevo
BioTherapeutics LLC in the United States and/or
other countries.

Hemophilia History Made: Everest Summit!

Laurie Kelley May 21, 2017

Mountaineer Chris Bombardier

He did it! History made! Chris
Bombardier, the young man from Denver, Colorado with hemophilia B, around 10 pm last night became the first person in history with hemophilia to summit Mt.
Everest! Everest alone is an incredible challenge due to the high altitude,
which kicks in on the nine-day trek to base camp at about 10,000 feet. Everest
is 29,029 ft. Chris has hemophilia,
and faces prolonged bleeding from injuries. He uses an extended half-life product,
which will help increase the length of time factor circulates in his blood. So
many have asked how he is protecting himself: factor, prophylaxis, oxygen
tanks, and great sherpas to help guide him. It takes a special kind of person
to dream of this, train for this, and take on this. And he is doing this not just for personal best record or even to make history, but to shine a spotlight on the huge disparity in care between hemophilia treatment in countries like the US and in countries like Nepal.

Read from his blog, Adventures of a Hemophiliac. What does a guy with hemophilia think
and feel before undertaking a history-making adventure?

Infusing on a rock!

TUESDAY May 16

I finished my last infusion at base camp and tomorrow morning
our team will begin our summit push. I’m not exactly sure how to describe the
feelings I am currently having. I’m excited, nervous, scared and hopeful all at
the same time. I know the next week of my life will be incredibly hard, full of
moments of questioning my sanity and of overwhelming joy. I’ll be able to
witness some of the most amazing views on this planet and also be more
exhausted than I have ever been.

Through the hard moments and the amazing moments I know that I have a family
that is supporting me no matter what. My beautiful and strong wife, my
incredible parents, aunts, uncles and friends will be with me ever step of the
way. I also know that I have my bleeding disorder family from all over the
globe cheering me on and that makes me feel strong.

As I pack my gear and put my boots on in the morning and walk into the [Khumbu]
icefall with only the light of my headlamp showing the way, I’ll think about
how fortunate I am to be able to choose this life and this adventure. Choose to
try and push myself farther than I could ever dream and step on the top of the
world. I’ll also think about all my blood brothers and sisters that aren’t able
to have that choice…. yet. I’ll remember that by standing on the summit and
by raising awareness about hemophilia and disparity in care, we can change that.

The hope is that we can summit on May 22. While I am challenging myself on the
mountain I want to challenge all of you. We already met the goal of finding
sponsors for every child on the Save One Life,
Inc. website. 55 kids have now been sponsored since I began
this adventure! Let’s not stop there!

The climbers promoting Save One Life

I would love to see the fundraising page for my Everest climb
reach $8,848, the amount of meters above
sea level the summit reaches. I won’t know if we’ve reached that goal until two
days after the summit and I reach basecamp but that would be icing on the cake.
Those funds will help those with hemophilia in Nepal continue to rebuild after
the earthquake. It will help them climb their own personal “Everest”
and work towards living the lives they choose. I would also urge you to reach
out to Save One Life and put your name on the waiting list to sponsor a child.
There will be more children added soon and they all could use our help.

Thank you all for the support and hopefully in few days no one will be able to
say someone with hemophilia can’t climb Everest.

Navigating the Khumbu icefall: first steps to Everest

And thanks to Octapharma for sponsoring Chris’s historic
climb!

#everest #saveonelife #nepal #hemophilia #summitpush #bombardierblood #bleedingdisorder #climbing #everest2017 #bebrave #playitsmart #sevensummits

Visit our Gallery to see the trek to base camp!
Visit our Gallery to see our visit to Nepal’s hemophilia families!

Happy Mother’s Day !

Laurie Kelley May 14, 2017

A bleeding disorder diagnosis brings out the mama bear in most
mothers who have a child with a bleeding disorder. Maybe even more so in
those who have children with inhibitors. Below, Cazandra MacDonald, a
personal friend and wonderful mom of two boys with hemophilia (and
inhibitors) shares her thoughts on inhibitors in hemophilia.

Happy Mother’s Day to all the incredible moms who have children with bleeding disorders!

What’s in a Number?

Written by Cazandra Campos-MacDonald

Originally published in PEN May 2017

Numbers, numbers, numbers. Our society is flooded with numbers. From Social Security numbers to birthdays, PINs, passcodes and checking our weight, we can hardly get through a day without numbers. When you are living with a bleeding disorder, you monitor the assays of your factor, track the number of bleeds per month, check how many doses of product are on hand, and measure the circumference of a swollen knee. But when you live with an inhibitor, there’s another number that can become the focus of treatment: the Bethesda unit (BU).

The Bethesda inhibitor assay is a test that measures the titer (strength) of the inhibitor, described in Bethesda units. Inhibitor titers may range from less than 1 BU to thousands of BU. Knowing this number will help determine how bleeds are treated. If the inhibitor registers as low titer (less than or equal to 5 BU), bleeds may be treated with high doses of standard factor concentrate. If the inhibitor registers as high titer (greater than 5 BU), standard factor concentrates are ineffective and special factor concentrates called bypassing agents are used instead. Attempting to treat bleeds in the presence of inhibitors is less effective than treating bleeds without inhibitors—so the goal is to eradicate the inhibitor. If the inhibitor registers as less than 10 BU, this is when many providers will have patients begin immune tolerance therapy (ITT), also called immune tolerance induction (ITI), a treatment protocol designed to eliminate the inhibitor.1 Knowing your BU is crucial in order

to take the next step in working toward that goal.

It’s easy to put your faith completely in the numbers. Knowing your current BU is important, but know first that every individual is unique and there are several different ITT protocols. Each person does not react to ITT in the same way. One body may accept ITT easily, and his BU will come down in a short time. Others on the protocol may take years to get the same results. Numbers do not dictate that the treatment for one person will be the same as for another. For example, two brothers, both with severe hemophilia and inhibitors and with the same parents, can live very different lives with an inhibitor. My older son, Julian, was one year old

when he was diagnosed with a low-titer inhibitor; it never rose above 5 BU. He immediately had a port inserted, and he started ITT for two and a half years. He tolerized, meaning his inhibitor dropped to zero, and he has never had an inhibitor resurface.

My younger son, Caeleb, was 11 months old when diagnosed with a high-titer inhibitor that registered over 2,200 BU. His titer dropped to 0 BU at one point after ITT, but now he is living with a low-titer inhibitor, and he receives factor daily

to maintain his tolerance. My sons both reached 0 BU after ITT, but they had different outcomes.

The numbers can be promising and sometimes disappointing. But ultimately, the numbers are a key component to treatment.

Everyone who tracks his BU has an ultimate goal in mind: to lower the titer to zero. If your titer is 323 BU, your goal may first be 299 BU, then 250 BU.2 Another person may be hoping to get to double digits, and another to single digits. Of course, when you’re tracking your BU, you want to get to zero and stay there. When you reach 0 BU, you may think that the inhibitor is now a thing of the past—but not necessarily. Once 0 BU is attained, the next step is to monitor the half-life of the factor. To be successfully considered tolerized (this is also called complete tolerance), the following must be maintained:

• The inhibitor titer can no longer be measured.

• Factor recovery is greater than 66% of normal.

• The half-life of factor VIII is greater than six hours.3

But someone may live with 0 BU for many years without these three characteristics. This is called partial tolerance. For example, if your child has 0 BU and a three-hour half-life of factor in his body, he will probably continue with the same ITT therapy, which may be daily infusions. ITT is not always successful: an ITT attempt in which inhibitor titers fail to decrease at least 20% over three to six months, or remain over 5 BU after three to five years, is considered a failure. This example shows that not only is BU important, but monitoring the number of hours for the half-life is critical to treatment. So how does a family live with the numbers?

“Lab work disappointment” is a phrase Kari Atkinson’s family used when the numbers were not what they had expected for their son. “We had so much hope that the inhibitor would go away.” But now, says Kari, “we are not as concerned about the number because we can tell when [the BU is] up and down by how our son bleeds.” How an individual’s body reacts to treatment is the ultimate measure of success. If you’re living a full life with few bleeds and an active inhibitor, the important thing is that you are healthy, happy, and thriving. Eric Frey’s son, age seven, has lived with an inhibitor for over five years. “After time, we learned two things: First, we already knew what the results [BU] were going to show by the way our son was bleeding, bruising, and behaving. Second, the Bethesda number is far less important than how our son was bleeding, bruising, and behaving.”

Despite living full, healthy lives with an inhibitor, many families still worry about the numbers. “Making peace” with the inhibitor is something that most people don’t want to do. It can feel as if you’re giving in and accepting that the inhibitor will always be present. In order to live a life where hemophilia is not the center of everything, making peace is crucial. “We have had enough experience that we know if the inhibitor is under 7 BU, we are living pretty good,” says Kari. Her family is not focusing on 0 BU, but for now, they know that anything under 7 BU is acceptable. “It’s really hard to not focus on the numbers, especially when you have the active inhibitor and either you need to get below 10 BU to start ITT, or you are doing ITT and trying to get down to zero,” says Eric. “We understand how hard that is. Focus on health. Focus on wellness.”

Numbers are essential for people living with inhibitors. Keep track of bleeding episodes because this is a significant tool to see if your treatment is appropriate. Continue your regular blood draws according to your provider’s recommendations. Even if you’re not a slave to the BU, it’s vital to monitor the progress of your inhibitor. The key is to enjoy life. Savor every moment. When things aren’t going well, try to remember that life will get better. And when life is good, soak it in.

Cazandra Campos-MacDonald is a motivational speaker, educator, and patient advocate for families with bleeding disorders. She writes a blog chronicling the journey of her two sons with severe hemophilia and inhibitors, and has written articles and blog posts for other publications. Cazandra’s older brother, Ronaldo Julian Campos, died of complications from hemophilia as an infant. Cazandra lives with her family, Rev. Joe MacDonald, Julian (20), and Caeleb (11), in Rio Rancho, New Mexico.

Click here to see Caz’s Ted Talk

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Free to families with hemophilia

1. ITT is a proven treatment toward eradicating inhibitors. Larger-than-normal doses of factor are given in the hope of overriding the inhibitor. ITT protocols can differ in frequency of infusing, depending on the physician’s and individual’s needs.

2. Once you achieve 10 BU, it doesn’t matter if the BU gets lower, because all infused factor is inactivated in minutes. Even so, families living with an inhibitor will find emotional relief when the numbers get closer to zero.

3. D. M. DiMichele, W. K. Hoots, S. W. Pipe, G. E. Rivard, and E. Santagostino, “International Workshop on Immune Tolerance Induction: Consensus Recommendations,” Haemophilia 13(2007): 1–22.

AHCA: Crime and Punishment

Laurie Kelley May 8, 2017

Get ready to rumble.
The hemophilia community overall is not just deeply disappointed with what’s
happening in Washington DC regarding healthcare, it’s shocked, angry … and good manners prohibits me from using cruder language. It
won’t take the proposed healthcare bill lying down. Been there before when
government failed our healthcare in the 1980s.

The American Health
Care Act (H.R. 1628)
passed the House of Representatives on Thursday, May 6. Facebook lit up with
angry comments from the community when it knew the bill was headed for a vote.
Some people went so far as to threaten to unfriend anyone who didn’t agree that
this bill was a threat to our coverage and health. Some went so far as to say
that if you didn’t take a stand, you were “privileged,” and that the bill must
not impact you. Strong words and convictions. Advocates urged everyone, as
people with an expensive preexisting condition, to contact their congressperson
and ask them not to vote for this.

The AHCA’s stated overall
goal is to reduce federal spending over time. But it also carries a significant
risk of reducing the number of people covered—the Congressional Budget Office estimates
24 million by 2026— and their benefits. The estimated federal savings of
$300 billion must be officially assessed by the CBO in order to pass in the Senate. And to complicate matters, the Senate may write
its own version of the bill instead of voting on the current one.

The key components of the ACA
(“Obamacare”) that were favorable to those with preexisting conditions like
hemophilia were: eliminating lifetime caps, keeping children on parents’
insurance until age 26, eliminating preexisting condition discrimination,
creation of the Marketplace exchanges, and expanding Medicaid.

Some components of the
AHCA that are of concern include:

• Giving states the
ability to run their own Medicaid program,

• Allowing states to
opt out of any mandate that insurers not raise insurance costs for Americans
with preexisting conditions

• Allowing states to
create a work requirement for people on Medicaid.

• Repealing the
employer mandate

• Repealing the tax
penalty for the ACA’s individual mandate

• Cuts $900 million in
taxes for individuals who earn over $200,000 annually, while also cutting $1
trillion in subsidies for Medicaid.

• Providing $100
billion under the “Patient and State Stability Fund” to help states manage some
of the costs of the most expensive patients

• Permitting
states to allow insurers on the exchanges to charge more (with no upper limit)
for patients with pre-existing conditions, although they still cannot be
technically denied coverage

• Allows insurers on
the exchanges to raise rates more for older people, penalize people who go more
than two months without continuous coverage, and replace Obamacare’s
income-based and cost-based subsidies for exchange coverage with a tax credit
that only adjusted for age.

In today’s TV show This Week, House Speaker Paul Ryan
commented, “Under this bill, no matter what, you cannot be denied coverage
if you have a preexisting condition.”
He tried to reassure viewers by adding, “You
can’t charge people more if they keep continuous coverage. The key of having a
continuous coverage provision is to make sure that people stay covered and they
move from one plan to the next if they want to. It’s kind of like waiting until
your house is on fire to then buy your homeowner’s insurance. You want to make
sure that people stay covered to keep the cost down.”

In response to the
passing of the House bill, Joe Kennedy III said this:

“In
a world view that scapegoats the Struggling and the Suffering, that sees fault
in illness, that rejects the most basic universal truth of the human existence:
that every single one of us, one day, will be brought to our knees by a
diagnosis we didn’t expect, a phone call we can’t imagine, and a loss we cannot
endure. So we take care of each other because but for the
grace of God there go I one day. And we hope that we will be shown that mercy
too. It is the ultimate test of the character of this country confronting our
chamber today. Not the power we give the strong but the strength with which we
embrace the weak.”

Facebook has been not
only a place to vent and share feelings and facts, but also has been a great
way to advocate. Facebook friend Randy wrote:

I just faxed my US
senators using Resistbot. Send a text message to 504-09 and put RESIST as the
message. Resistbot generates a fax to your senators and representative. (A fax
is apparently more effective than a phone call.) You’ll get a few prompts back.
We need them to vote NO on repealing the ACA. Lives are at stake – not to
mention wallets. Please copy, paste and share.

Facebook friend Jeff wrote: Insurers – not the government – define what pre-existing
conditions are, and they are free to be as liberal with their definitions as
they wish…

And
the only good thing to come out of all this so far? More people are becoming activists.
People are contacting their congressperson, some for the first time in their
lives. Young people are reading, learning, talking a stand. Conversations get
heated but also shed light; debates may divide but also deliver.

And
our community, as always, will take a stand against threats to our hard-earned healthcare
gains. Watch the news for continued debate on healthcare reform, and see you on
Facebook!