October 2013

Kogenate® FS’s New Vial Adapter

Every now and then, I’ll be posting news items from the news wires, to keep our readers updated with new offerings related to their treatment. Today I’m posting about a new reconstitution system for Bayer HealthCare’s Kogenate®FS antihemophilic factor (recombinant). (Please note this is not a paid advertisement but just an educational service to our readers. The new “Vial Adapter” offers Kogenate® FS users an additional choice in reconstitution systems.Vial Adapter is a needleless reconstitution system that contains a sterile chamber with a built-in 15-micrometer filter and a prefilled diluent syringe.

From Bayer’s press release: “Innovating in order to provide two reconstitution systems, Vial Adapter and BIO-SET®, is a great example of our commitment to delivering options that meet the needs of the entire hemophilia A community,” said Pamela Cyrus, MD, Vice President and Head, U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals.

Both Kogenate® FS with BIO-SET® and Kogenate® FS with Vial Adapter are available in Grab and Go packaging, which includes:
·        A prefilled diluent syringe
·        The Terumo® SURFLO® Winged Infusion Set
·        Alcohol swabs, a cotton pad and a latex-free bandage
·        Exclusive anti-counterfeiting and tamper-evident features
·        Alcohol swabs, a cotton pad and a latex-free bandage
·        Exclusive anti-counterfeiting and tamper-evident features

Kogenate® FS, antihemophilic factor (recombinant) is indicated for:
·        Control and prevention of bleeding episodes in adults and children (0-16 years) with hemophilia A
·        Peri-operative management in adults and children with hemophilia A
·        Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in children with hemophilia A and to reduce the risk of joint damage in children without pre-existing joint damage

Kogenate® FS is not indicated for the treatment of von Willebrand disease.

Kogenate® FS is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product or its components, including mouse or hamster proteins.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch, or call 1-800-FDA-1088.

For important risk and use information, please see the full prescribing information at https://www.kogenatefs.com/webapp/prescribing-information.jsp

Great Book I Just Read
Something Wicked This Way Comes by Ray Bradbury

Many of us read Ray Bradbury books, like Fahrenheit 451, in high school. He is so worth reading again. In time for Halloween comes this 1962 October horror/mystery story that reads lyrically, like poetry, like artwork. Two 14-year-old best friends, Jim Nightshade and William Halloway, who live in Green Town, in the midwest, are intrigued by the dark and mysterious carnival that roars into town at 3 am one morning, long after all carnivals have packed up for the season. Headed by a fully illustrated, tattooed Mr. Dark, the carnival has a deep secret to protect, and becomes a threat to the townspeople. Mr. Dark bears a tattoo for each person in town he manages to lure into the carnival–to the carousel that twists time, or the house of mirrors that strands souls… and never lets them go. Only one person can save the townspeople… The writing is absolutely superb, colorful, vivid and memorable. The three page description of the carnival train pulling into the outskirts of town alone will stay with you. Great fall/Halloween reading for young and old… which is, by the way, one of the themes of the book! Five/five stars.

A Memoir of Pain, and Hope

I love discovering new people in our amazing hemophilia community, along with their talents and accomplishments. So I was delighted when Ray Perreault contacted me this year to introduce himself and share a new book he self-published. Ray has severe factor IX deficiency, and his new book, A Life Without IX, is a memoir. To Ray, this is simply a memoir of his life, but his life has been one of unmitigated pain and suffering. He tells this book (mostly) in the third-person, through a character named John. John’s life is Ray’s life. And it is remarkable.

The 173-page book details what it is like to live with chronic, torturing pain, from bleeds treated with plasma, to the “Miracle,” factor IX concentrate. I’ve read people’s stories on pain before, and I have to admit this is the most carefully constructed, detailed recollection of enduring waves of pain that dominate your life that I have read. While reading, you get inside the mind of “John,” experiencing what he experiences. What do people in searing pain think, how do they strategize how to move across the room, what do they dream of at night?

The book covers childhood, high school, adulthood, even a first marriage that brought even more pain to John’s life, then the birth of his child, and at last a second marriage, which brings joy. A book like this will expand your concept of what pain is, and being fresh appreciation for those struggling to survive it minute by minute, day by day.

Ray Perreault is a survivor who deserves the highest respect. His wife summed it perfectly in the book: “You always had the courage to bounce back…it’s the most amazing thing about you.” Resilience in the face of crushing and crippling pain, strength in the face of the negative attitudes and discrimination of “others.” Do not read this book looking for lofty inspiration, though; it reads like the no holds barred memoirs of a war veteran who has seen the worst in battle. But through it all, there is a human spirit that subtly rises above it all, and that embodies itself in this book.

Ray Perreault

I am also an author and editor and so cannot conclude with a few caveats. Self-published books often have their share of problems and glitches, and so it is with this book. Layout: expect many big gaps and white spaces (half page sometimes) in the book where the text flow did not get corrected. Copy: some grammatical errors, many stylistic inconsistencies. Content: the subject matter is vitally important and the story is basically good. But it rambles, and is confusing. It bounces about chronologically.  Why Ray chose to tell it in the third person is a bit of a mystery, and the voice changes sometimes from third-person to first-person with no explanation who the narrator is. But most importantly there is a lack of medical information (HIV is barely explained, leaving too many questions; factor IX is incorrectly explained and referenced; hemophilia is not adequately explained).

My advice is to read this for the story of the man, and it is a story that deserves to be told. It’s a story of perseverance and courage, fortitude and hope. With thorough, professional editing, this book would carry even more impact than it already does. Well done for a first book, Ray!

Keep up with Ray on his blog: http://blog.raymondperreault.com

Order Ray’s book A Life Without IX here

Plasma Awareness Week

This week marks the first ever International Plasma Awareness Week, an opportunity to recognzie the importance of plasma in our lives, and the need to donate blood to provide the life-saving liquid to those in need. It’s especially important to many with hemophilia throughout the world as donated blood donations can be fractionated into factor VIII and IX for many hemophilia patients who use plasma therapy.
You learned this in high school science class but here it is again: Plasma is the clear, straw-colored liquid portion of blood that remains after red blood cells, white blood cells, platelets and other cellular components are removed. It is the single largest component of human blood, comprising about 55 percent, and contains water, salts, enzymes, antibodies and other proteins. It is a clear, straw colored liquid that is 90% water and serves as a transporting medium for cells and a variety of substances vital to the human body.
In the US, we often think only of recombinant factor when we think about treating hemophilia, but plasma therapies remain the mainstay for many with hemophilia, those with inhibitors and undergoing ITI, and those with von Willebrand disease patients.
In addition, plasma protein therapies are used to in emergency and surgical medicine.Plasma protein therapies are not interchangeable and have been defined by regulators as sole-source biologic products because no generics or substitutions exist. In addition, their biological nature demands storage and handling requirements by specialty distributors that ensure their safety.
Source plasma is plasma that is collected exclusively for further manufacturing through a process called plasmapherisis. Recovered plasma is collected through whole blood donation which has been separated into its cellular components.
Safety and quality of plasma protein therapies is the top priority of the plasma protein therapeutics industry. Both collectors and manufacturers adhere to strict regulatory policies and have instituted Good Manufacturing Practices in every step of plasma collection and manufacturing processes.

Plasma-derived therapies depend on the generosity and commitment of healthy donors. Source plasma is collected in over 450 specialized donor centers in the U.S., Canada, Germany, Austria and the Czech Republic. Source plasma collection in the U.S. is regulated by the Food and Drug Administration and by the European Medicines Agency and national regulatory authorities in Europe. Additionally, 436 plasma collection centers are also certified by the International Quality Plasma Program (IQPP), a rigorous, voluntary program that goes beyond regulatory requirements to help ensure donor safety and further improve the quality of plasma used to manufacture therapies.
This is a good week to donate blood then! If you can make the time, please visit your local blood donation center and give the gift of life.
See www.donatingplasma.org and www.pptaglobal.org  for more information.

International Plasma Awareness Week is sponsored by the Plasma Protein Therapeutics Association and its member companies.

Sunny Skies at NHF’s 65th Annual Meeting

Manufacturers set up large booths to view new
programs and products

still in Anaheim, about to enjoy the sunny California weather for two more
days. I’ve already 

enjoyed the past five days with my bleeding disorder
community at the 65th annual National Hemophilia Foundation meeting,
with about 3,000 colleagues, patients, families, corporations and friends.
These just keep betting better and better!
Consumers get to browse many booths
theme was “United in Progress,” aptly named as all the divergent groups in our
community work together to push our agenda through obstacles such as funding
cuts and insurance threats. While I could only attend a couple of sessions,
they were powerful.

Stakeholders’ Meeting
Laurie Kelley with Kevin Shaughnessy 
first was NHF’s Stakeholder Meeting, a comprehensive three-hour session that brought together
key opinion leaders in the US.
Laurie Kelley with Debbi
Adamkin of the Florida Chapter
Rotellini of NHF first discussed the need to establish conflict of interest
boundaries and policies for chapters working with industry (pharmaceutical
companies and specialty pharmacies). There’s even a need to define “industry,”
as HTCs, half of which sell factor, don’t like to be included as industry. 51
chapters have different guidelines so there’s a need to standardized policies
to industry. A lively debate ensued about how to create a template and work with industry’s legal system to approve this.
Laurie with Julie Birkofer and Jan Bult
of the PPTA
we had a federal update from Ellen Riker, of MARC Associates in Washington DC.  As we all know, the federal fiscal year began October 1. And there is no
appropriations bill for health programs or agencies. So now there is an extension
of current funding levels, with a sequester likely to occur again in 2014. I was
amazed at the low level of funding for HTCs. HRSA/MCHB funding remains flat at
$4.9 million for HTCs and coordinating centers. The CDC receives approximately
$7 million (not a lot) for hemophilia for surveillance, research, outreach and
education (grants to HTCs and patient organizations, all impacted negatively by the 2013
sequester). Most alarming is that the head of CDC doesn’t think chronic disorders
should be in CDC to begin with! So new cuts are a threat. Ellen reminded us
that we would not have gotten through our HIV days without CDC; we need each
update on HR 460 Bill, which NHF is supporting: this prevents private insurance
plans from requiring cost-sharing for drugs on specialty tiers. Only 14% of
health plans using specialty tiers; specialty drug spending represents only 2%
of overall health plan spending. NHF wants to prevent insurance plans from
putting factor in the specialty tier, where copays could be astronomical.
The legendary Barry Haarde!
Rice gave an update on the Affordable Care Act “Marketplaces”—those websites
that will allow consumers to compare health care plans to find the right one at
the lowest cost. We learned that the Marketplaces went live just the day
before, and many crashed as huge numbers of consumers logged in!  Her own staff
went on the federal site and it was confusing. Michelle presented a great spreadsheet
that will help breakdown the Marketplace by level (denoted by “metal”—platinum,
bronze, etc), by state, etc. This will be a vital and helpful guide for those with bleeding disorders using the Marketplace. Glitches in the Marketplace include: some had navigators but called them different things, which was confusing. Some organizations
are putting up tools based on what feds said would be there but the tools
aren’t right because the feds haven’t followed through with what they said they
would provide!
Laurie Kelley withAndy Matthews
and Chad Frederickson
primary message was: be patient. We all have until December 15 to pick a plan for
January 1. Wait before accessing the Marketplace so the bugs can be fixed.

Laurie with hemo moms Elizabeth Pulley
and Julie Heinrich
Uniting the World
Laurie with friends from Nigeria
session I attended was about the twinning program of the World Federation of
Hemophilia. Present was Megan Adediran,
my friend and colleague from Nigeria. Megan is the president and cofounder of
the Haemophilia
Foundation of Nigeria, and mother of
two boys with hemophilia. She was present with husband Thomas and another
mother of a child with hemophilia. She gave a fiery speech about the importance
of the twinning program with NHF, to close the gap between two countries, two
worlds, two different approaches to hemophilia. She related how hard the HFN is
working to reach its people in need. Nigeria is a huge country, where only
about 150 people with hemophilia out of approximately 10,000 have been
diagnosed, and where thousands more wait to be identified and helped. It could
take a lifetime to find them, but the twinning assures that we are in this
together for the long haul, United in Progress.
Laurie Kelley with the
lovable Tater, who has hemophilia
was a great meeting, with sessions on social media, von Willebrand Disease,
hepatitis C updates, gene therapy research, obesity, pain management and even
dating when you have hemophilia. There was something for everyone.
We’re really grateful to NHF also for allowing Save One Life, my nonprofit that sponsors children with bleeding disorders in developing countries, a table to help us gather more sponsorships. We did pick up a lot of new sponsors, hopefully helping us to reach 85 new sponsorships before December 31. Go to www.saveonelife.net if you can sponsor a needy child in poverty with a bleeding disorder!
With Debbie de la Riva, former ED of
Lone Star Chapter
is always wonderful to meet up with families I’ve known for so long, going on
23 years for many. One special visitor was 25-year-old Emmanuel, from Ghana,
Africa. I’ve known “Kofi” for about 5 years, when I first went to Ghana, and
now he is attending college in UC Riverside. Living so close, he was able to
take a bus, and was invited in as a guest of NHF. He has factor VIII
deficiency, and is bright, articulate and energetic. It was wonderful to have
him here, especially as he could meet the Nigerians! But I always wish more of
our own US families could attend this remarkable event. I’d like to see a way
to spend less on booths, and more on airfare and hotel for as many families as
possible, to make this truly a community event.
Laurie and Patrick Schmidt,
CEO FFF Enterprises and sponsor of
88 children with Save One Life!
did a splendid job as always of organizing this mammoth event, and we thank
everyone involved, including sponsors and supporters, for mailing this
available. See you in Washington DC next year!

Rich Pezillo, Sonji Wilkes and
Jane Smith
Laurie Kelley with Laurel McDonnell
and Paul and Linda Clement
Janie Davis of Baxter with Emmanuel
of Ghana
Laurie and Chris Bombardier at
Save One Life table
Ellen White, RN, who first “discovered”
Ghana as a country in need of our help

Save One Life sponsors gather
for coffee 
Factor Support Network displays
our books!
Bayer booth
With long time friend
Barbara Chang

Laurie Kelley with
Reid Coleman of NC
Laurie and Zoraida taking
a break
Yeah, I met Iron Man!
No trip to California
without visiting Jim Morrison in
Venice, where it all began for
the Doors!

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