A Source of Support and Strength

Trying
to “go it alone” is a noble and brave course in the face of adversity, but not
always an ideal one, especially if you have a bleeding disorder. There is a
huge community filled with caring people, knowledgeable professionals and
valuable resources to help you. This week’s blog shares the experience of
Casey, who realizes how much he needs the support of the community, especially
when it comes to selecting a product that he and his physician believe is right
for him and his body’s individual needs.

Indications

ALPROLIX is an injectable
medicine that is used to help control and prevent bleeding in people with
hemophilia B. Hemophilia B is also called congenital Factor IX
deficiency.

Your healthcare provider may
give you ALPROLIX when you have surgery.

Selected Important Safety Information

Do not use ALPROLIX if you
are allergic to ALPROLIX or any of the other ingredients in ALPROLIX.


Please see ALPROLIX.com for the Indications and Important SafetyInformation , as well as the full Prescribing Information.

Growing
up, I usually didn’t talk about my hemophilia B. I was the first person in my
family to have hemophilia; diagnosed in early childhood. At school, when I would
have bleeds, I remember thinking that none of the other kids did. Without a
mentor, I kept my emotions to myself, no matter how vulnerable I felt.

My
dad gave me my treatments 99% of the time. Always getting the vein on the first
stick. However, my mom was a nervous wreck. One time she had to give me a
treatment, she tried again and again with no luck. She was in tears. In that
moment, she became my motivation to learn how to self-treat. So at the age of
10, I began managing my own hemophilia infusions. It was a liberating feeling.
I wasn’t so vulnerable after all.

It
was one of my nurses who told me about ALPROLIX. After discussing the risks and
benefits with my doctor, he explained that the recommended starting prophy
regimens are either 50 IU/kg once weekly, or 100 IU/kg once every 10 days. And
that the dosing regimen can be adjusted based on individual response. No two
people with hemophilia are alike, so it made sense to me that medication could
work differently in everyone. I started on a 10-day schedule, but when I had my
labs taken, my doctor told me that I could extend my infusions to once every
two weeks. That really fits my busy schedule. Thinking back on my childhood, I
never believed infusing every 14 days would be possible.

I
didn’t start taking ALPROLIX just for myself. I was also thinking of my two
nephews who also have hemophilia. After seeing my experience and talking with
their doctors, they started on ALPROLIX, too. I try to help with my nephews
when I can. I’ve even occasionally been their caregiver and given them a couple
treatments. I make sure they know they have me to look up to if they need
advice.

Watching
my nephews grow up brings back so many memories of my own childhood. We have a
special connection because I understand some of the challenges they face. They
have an advantage I didn’t have growing up: a close family member who knows
what they’re going through. I can help my nephews in a way no one could help
me. My goal is to help them find confidence in themselves and encourage them to
have fun being kids.

I
hope that by sharing my experiences, I might encourage others to figure out
what they have learned from their own experiences and take control over their
choices.

Thank
you.
To hear from other people
living with hemophilia B, check out thesevideos.
Talk to your healthcare team
about whether ALPROLIX may be right for you.
This blog was sponsored by Biogen, for educational
purposes.
FIX-US-0807   
06/2016

Together we can FACTOR IT FORWARD™

The following is a paid public service announcement.
LA Kelley Communications always advises you
to be a savvy consumer when contacting any company; do not reveal personal
information (PI) or protected health information (PHI) carelessly or against
your will.
Every day, I’m reminded of how much I have to be thankful for. Like
the life-changing support I’ve had the privilege to both give and receive within
the hemophilia community.
As a parent personally touched by a
bleeding disorder, I’ve witnessed first-hand the power a strong support system
can provide.
This truly is a community that cares, and I’m proud to be a part
of it.
That’s why today, I’m excited to spread the word about a program that
embraces the spirit of this community—FACTOR IT FORWARD.
With FACTOR IT FORWARD, the people behind IXINITY® [coagulation
factor IX (recombinant)] have partnered with AmeriCares, a non-profit global
health organization, to donate IXINITY to people with hemophilia B who can’t
afford to be without it right here at home.
And it doesn’t end there. Visit www.factoritforward.com or read on to find out how you can help.
FACTOR IT FORWARD and help lead
the way
As part of their commitment to the hemophilia B community, the
makers of IXINITY will donate 10% of IXINITY used to nines who need it most
here in the U.S. That means:
For
every 10 units of IXINITY used, 1 unit is donated to those in need in the United
States.
So when you use IXINITY, you help
give back to the hemophilia B community.
You can try IXINITY for free to see how it works for you. With
your IXINITY FREE Trial, you’ll receive 1 month of treatment up to 20,000 IU. Learn more about the FREE Trial, download the FREE Trial Form, and talk to your doctor. 
Plus, there are even more ways to join in and help FACTOR IT
FORWARD in addition to using IXINITY.  
Get involved today, right now, when you take the giving quiz at www.FactorItForward.com. You’ll earn points that will contribute to
factor donations just by answering a few quick questions.
Or chat with an IXINITY rep
at a local chapter event and snap a
selfie with your giving pledge. Share the pic on your favorite social media
channel to get points just for posting.  
More points equal more factor donated, making it even easier for
everyone to do their part!
And since there’s no limit to the number of IUs donated, every IU
of IXINITY used and every point earned count toward that end goal: to leave no
nine behind.
Visit www.FactorItForward.com
 today
to learn more about the program and how you can participate, and talk to your
doctor to find out how you can try IXINITY for free.   
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 accompanying 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.
 
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)], FACTOR IT FORWARD, 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. CM-FIX-0048

The Most Powerful Man in America–with Hemophilia No Less

The man who built Fox News into a political media giant has been called the most powerful man in America by President Obama… and also happens to have hemophilia! Roger Ailes. 
During this raucous political season, it’s worth looking at the man who helped create the news outlets for conservative voters in America.

The following is a book review by Richard Atwood, our columnist for PEN: Gabriel Sherman’s 2014 biography The Loudest Voice in the
Room. How the Brilliant, Bombastic Roger Ailes Built Fox News — and Divided the
Country
is fascinating for its look at this interesting man, who once sat on the National Hemophilia Foundation board of directors.
Roger Eugene Ailes was born on May 15, 1940 in
Warren, Ohio. When two, he bit his tongue and bled, leading to a diagnosis of
hemophilia. Ailes attended Ohio University where he enrolled in Air Force ROTC
for two years, but his eyesight and other physical problems were an issue. He
even tried to enlist. Not knowing how long he would live, Ailes did not openly
discuss his hemophilia with most of his classmates. His older brother, a
physician, linked Ailes’ temper to his medical condition. His parents divorced
while he was in college. Ailes married Marjorie White in 1960. Beginning in
1961, he worked as a producer for The Mike Douglas Show at a television
station in Cleveland. Ailes kept his hemophilia a secret. When the show moved
to Philadelphia in 1966, Ailes was its executive producer. He treated politics
as entertainment. After an interview with Richard Nixon in 1968, Ailes worked
as a media advisor, or image consultant, for Nixon’s presidential campaign
before being fired by the RNC in 1970. While separated from his wife in 1972,
Ailes was agent for, and romantically involved with, the actress Kelly Garrett.
As a Broadway producer in the 1970s, Ailes had both successes, such as Hot l
Baltimore
, and several flops. Ailes took risks with sky diving, tearing his
ankle ligaments, while also becoming paranoid about his physical safety. He had
brooding moods, increased weight and pent up anger. He demonstrated his
conservative political beliefs when he was a consultant and news director of
TVN (Television News Inc) for two years before it shut down in 1975. Ailes
broke up with Garrett in 1977, then became romantically involved with Norma
Ferrer who had two children. He divorced White that year and married Ferrer in
1981. 
Ailes was most successful as a political consultant from 1980 to 1986.
After a fist fight, he had swollen hands. He was known to make sexual
approaches to female employees when he was executive producer for the Tomorrow
Show
with Tom Snyder. His father died of Alzheimer’s in 1983, and his
weight ballooned again. Ailes published his book of self-help wisdom, You
Are the Message
, in 1988. He became an executive producer for Rush Limbaugh
in both radio and television. He worked for 28 months at CNBC, beginning in
1993, and learned how to turn news into entertainment. He moved to the America’s
Talking
cable channel in 1994. He divorced Ferrer in 1995, this time a more
expensive change. Ailes now walked with a limp. He moved to MSNBC in 1995, and
left a year later. Roger Murdoch hired Ailes in 1996 to be chairman and CEO of
Fox News. Ailes’ book became the cable channel’s sacred text. His fear for his
personal safety increased. He married Beth Tilson, a divorced producer whom he
met at CNBC. Ailes’ son, Zachary Joseph Jackson Ailes, was born on January 1,
2000. Ailes hired Bill O’Reilly, his brightest star. Fox News passed CNN for
good in ratings in 2002 after the Monica Lewinsky affair in 1998 and the 2000
elections.  Ailes hired Glenn Beck
from CNN in 2008 and supported the Tea Party in 2009. Many conservative
politicians became employees of Fox News. 
After building a mansion in Garrison,
New York, 46 miles north of NYC, Ailes bought the Putnam County News &
Recorder
in 1988, installed his wife as editor, and changed the local paper
to reflect the conservative views of Fox News. Ailes continued to demonstrate
his fear for personal safety and his paranoia, and he shared his conspiratorial
worldview with his wife. Ailes also was in charge of Fox Business Network that
was launched in 2007. 
Ailes remembered his father’s lessons and had a ‘Rosebud’ story.
“The cruelest lesson Roger would speak of occurred in the bedroom Roger shared
with his brother. Roger was standing on the top bunk. His father opened his
arms wide and smiled.
   “Jump Roger, jump,”
he said.
   Roger leapt
off the bed into the air toward his arms. But Robert took a step back. His son
fell flat on the floor. As he looked up, Robert leaned down and picked him up.
“Don’t ever trust anybody,” he said.” (p. 7).
This biography covers Ailes’ childhood rather
quickly, but his hemophilia is stressed more often during his younger years.
Yet a specific diagnosis and family history of hemophilia is never provided.
The book is more an analysis of Fox News than a life story of Ailes for his
older adult years. There are 6 pages for A Note on Sources, 98 pages of Notes,
13 pages for Selected Bibliography, and 20 pages for the Index. Hemophilia is
cited 10 times in the text, and is listed twice in the Index, under ‘Ailes,
Roger’ and by itself. Oddly, there are no photographs. The author interviewed
614 individuals in addition to referencing the written sources. Ailes was not
subjected to a psychological analysis, yet he was frequently described as
impulsive, paranoid, aggressive, and a ruthless competitor, all influenced by
his ego and temper. He was also quite successful. The author, who never
received permission to interview Ailes, is a journalist and editor living in
New York City with his wife.

It’s worth Googling Ailes to read about his work, his attitudes and thoughts about the political candidates; a fascinating man with hemophilia!

Another book read by Richard, but not as good, is Zev Chafets’s 2013 book Roger Ailes: Off Camera.
New York, NY: Sentinel. 258 pages.

Standing on the Shoulders of Giants

Dr. Olaf Walter reviews Octapharma’s history
I
was privileged this week to be invited to a special scientific symposium in
Heidelberg, Germany, sponsored by Octapharma. Octapharma is the largest
privately owned plasma manufacturer in the world. It recently launched  the first and only fourth generation recombinant factor VIII (FVIII)  produced in a human cell line without any chemical modification or protein fusion, NUWIQ®, now available in the
US. This unique product is important as it is hoped that a recombinant product
that originates from a human cell line might have low immunogenicity—in other
words, it might not trigger an inhibitor, which is the greatest complication of
hemophilia.
Octapharma
was founded in 1983 by Wolfgang Marguerre; the name comes from the Greek
for the word “eight.” Octapharma manufactured the world’s first plasma-derived FVIII that employed a viral inactivation step using solvent-detergent. I cannot overestimate how important this process is. My own son used product
initially that had no SD or pasteurization and miraculously escaped HIV and
hepatitis C infection.
 
Octapharma
now serves patients in 105 countries with 6,200 employees in 32
countries, of which half are in the US, the largest market for hemophilia
products.
Laurie Kelley with Marcia Boyle,
executive director of the IDF
The
Saturday symposium featured speakers from the US, Denmark, Canada, and Germany,
including our own Dr. Craig Kessler from Georgetown University. While all the speakers
were excellent, I learned a tremendous amount from Dr. Roger Kobayashi, of UCLA
School of Medicine, who is an immunologist. Patients with immune deficiency
share so many of the same concerns as those with hemophilia. Our need for
constant products, safe products, easy to administer and affordable. Dr.
Kobayashi reminded us that physicians like him, and companies like Octapharma, “stand on the shoulders of giants”–those researchers who have come before us. He told us that the very first Nobel Prize was awarded to a doctor who
discovered gamma globulins, proteins that can be used commercially to treat
infections in those with malfunctioning immune systems. He described the pain patients
felt years ago when intramuscular injection were given; the poor children! They
could not walk for days. Finally, intravenous infusions were manufactured and
successfully treated patients with immune deficiency. While he spoke, I had the honor
to be sitting next to Marcia Boyle, the executive director of the Immune Deficiency
Foundation, an exceptional leader who for 32 years has provided education,
advocacy and help to those with immune deficiency in the US. Indeed, Dr.
Kobayashi mentioned her three times in his speech!
We
learned about Alzheimer’s (discovered here in Germany), and Octapharma’s research into treating this
insidious disease, the fifth leading cause of the death in the US. There is
currently no treatment and after hearing Dr. Shawn Kile’s excellent presentation,
I pray that this company discovers a treatment soon. I recently witnessed the devastating
effects of this disease in a dear elderly friend. It strips a person of their
very identify and almost of their humanity. How long will we wait for a
treatment to slow it or stop its progression? Companies like Octapharma work
daily to answer this.
But
I was most interested in Dr. Craig Kessler’s speech on personalized
prophylaxis. Dr. Kessler described how half-lives differ dramatically among
patients and only a pharmacokinetic (PK) test can determine each person’s half-life. That’s the number one place to start with a proper treatment regimen. In
the “old” days, like when we were raising a child with hemophilia, we dosed
by weight and bleed severity only. Now we realize this is not enough. Each person
will have a different half-life. He displayed one slide that showed 66 patients
with half-lives varying up almost 5 hours. Once you know your half-life, you
can find the proper prophylaxis treatment. This might include looking through the vast
array of factor products. Dr. Kessler reminded us that patients want 1) reduced
number of infusions, 2) reduced inhibitor development and 3) to adopt prophy to
their own lifestyle.
Dr.
Kessler reported that NUWIQ® is Octapharma’s new fourth generation recombinant FVIII from a human cell line—the
first such product. Because it is entirely from humans, and not animal cell
lines, this could potentially reduce the rate of inhibitor development and avoid possible
allergic reactions, and allow personalized prophylaxis with fewer
infusions.  He described several
clinical studies with NUWIQ®, including one with 135 previously treated patients,
none of whom developed an inhibitor. We also know that inhibitors can develop
in about 30% of previously untreated patients with factor VIII deficiency, usually
within 20 exposure days. Octapharma designed NUWIQ® to be less immunogenic by using a human cell line. I will want to follow clinical studies closely on the
product to see what else they uncover regarding this.
Laurie Kelley with Octapharma friends

I
was also interested to hear from Dr. Olaf Walter, senior vice president, and MC for the day, that Octapharma is conducting research for a  subcutaneous (under the skin) delivery of factor. I know of two other companies
that are also working on this, and it will again be interesting to watch
developments. As we write in our May issue of PEN, this is the most exciting
time in hemophilia!

The symposium
closed with a delicious lunch and then a black tie gala that evening at the
Heidelberg Castle. It was not only the 33rd anniversary of
Octapharma, but also the 75th birthday of founder and chairman Wolfgang
Marguerre. I have a special indebtedness to Wolfgang, as he is Save One Life’s
largest supporter and also sponsor the largest number of children, 120.

Laurie Kelley with Octapharma Founder and Chairman
and Save One Life’s leading donor Wolfgang Marguerre



The evening was
dazzling, with over 40,000 flowers, 500 guests including Octapharma employees from around the world, 150 wait staff, at least two high quality bands, delicious food and drink. This was a wonderful way to celebrate
the accomplishments of this company and its indefatigable and brilliant
founder. I met up with my hemophilia colleagues, including three of our
top sponsors for Save One Life: Wolfgang, and also Patrick Schmidt, CEO of FFF
Enterprises, Neil Herson, president of ASD Healthcare, and Paolo Marcucci of Kedrion S.p.A.

Together, all three sponsor
about 280 children with hemophilia in poverty annually. Many of the other
attendees also sponsor children, including hemo-mom and Octapharma employee
Sherri Rojhani, who just sponsored a child in honor of Wolfgang Marguerre’s
birthday.

The evening
ended with spectacular fireworks, accompanied by songs representing many
countries, but the final song was a favorite of mine, the Irish pub-song The
Wild Rover.

When I thanked
Wolfgang for such a lovely evening, I told him he needed to manufacture a new
product—his energy! I want an infusion of whatever he has to keep me going strong
at 75 to do the humanitarian work I love to do. If only!
Thanks to Octapharma for a fascinating
symposium, such amazing festivities, and for all its support for Save One Life.

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