November 2015

Blood: Time to Donate!


National Hemophilia Foundation recommends recombinant factor as the standard of choice for treatment of hemophilia, but did you know that many factor products used to treat
hemophilia are developed from human blood, specifically human plasma? In fact, one person with hemophilia can require up to 1,200 plasma donations for a one year’s supply of factor
products. Plasma products are especially important for those undergoing immune tolerance therapy to treat inhibitors, for those with von Willebrand disease and for those in developing countries.
And plasma can come from you! Think of making a blood donation, during this time of Thanksgiving and holidays.
First, learn a bit more about plasma, Plasma is the straw-colored liquid that makes up
approximately 55 percent of total blood volume. A single liter of plasma yields
coagulation factors essential for blood clotting, immunoglobulins used to
combat viruses and bacterial infections, and albumin, a major plasma protein
that regulates blood volume and other essential functions.
In addition to treating hemophilia, plasma-derived therapies
are used in everyday medicines, emergency and critical care situations, as well
as preventive medicine. Albumin, for example, is used to treat burns, shock,
trauma, liver conditions and cardiopulmonary illnesses; immunoglobulins are
indicated for Rh incompatibility, pediatric HIV, hepatitis, and animal bites.
Plasma is an expensive raw material and represents between 40 to 60 percent of the cost
of plasma-derived product production. This is due primarily to its biologic
nature: plasma protein therapies are not interchangeable, have no generic
variations or substitutions, and are defined as sole-source biologic products
by global regulators.
Since plasma is biological in nature, complex regulation and
oversight measures are in place—including collection, processing, and storage
and handling requirements—to ensure plasma donor health, as well as product
purity and efficacy for patients.
The majority of the world’s plasma comes from plasma donors
in the U.S. Collectively, the Food and Drug Administration (FDA) and its Center
for Biologics and Research (CEBR) are responsible for regulatory oversight of
the U.S. blood supply. Blood collection centers are either registered or
licensed by the FDA, and are held to quality standards comparable to those of pharmaceutical
manufacturers. Blood establishments located outside of the U.S. that import or
offer for import blood products are also required to register with the FDA.  
CEBR regulates the collection of blood and blood components
used for transfusions, as well as for the manufacture of pharmaceuticals
derived from blood and blood components. CEBR develops and enforces quality
standards, inspects blood establishments and monitors reports of errors,
accidents and adverse clinical events.
Manufacturing processes begin with fractionation,
followed by purification and virus inactivation. Fractionation is a time
consuming and complex process that extracts, or “fractions off,” specific
plasma proteins that have a proven health benefit. Fractionation requires
multiple processing steps, which involve manipulating solution pH, temperature,
ionic strength and alcohol concentration.
Once fractionated, plasma proteins are further subjected to
virus inactivation, a complex purification processes that includes prion
removal, nanofiltration, solvent/detergent
treatments and incubation, to ensure sterility and purity of the final product.
The complete manufacturing process, from plasma collection at a donor
center to the FDA’s lot release, takes seven to 12 months.


So donate now! Next year at this time, your donation could be used to save a life!
Sponsored by ASD Healthcare.
Great Book I Just Read
The Climb to Hell  [Kindle]
Jack Olsen
Olsen sure can write a page-turner. In 1957, one of the most audacious and stunning rescues to ever take place occurred on the north face of the Eiger, one of the most notoriously difficult challenges to scale, located in the heart of the Swiss Alps. The Eiger is feared for its avalanches, fast-changing weather, and sheer facade. When two poorly-prepared Italians set off to make history by being the first Italians to scale it, they become stuck for eight days, in zero-degree weather, unable to get off the mountain. This launches an international rescue effort, that leads to surprising outcomes, including national rivalry, clashing personalities, extreme risk taking and uncertain success. Fantastic story, well told and must-reading by all mountaineers. Four/five stars.

Dealing the Hand Your Dealt: Pulse on the Road in Las Vegas!

I’m in Henderson, Nevada, a suburb of Las Vegas, the day after we gave another Pulse on the Road symposium about health care insurance. The air is dry and cool, the sky a perfect robin’s egg blue, and I’m surrounded by red and purple mountains. What an enchanting place to visit!

Whenever I am here, I think of my dear friend and colleague, Renee Paper. Renee was a champion of those with von Willebrand disease. She became the face of those with VWD, and advanced care for it, and for Nevada, like no one else. Nevada has an HTC thanks to her; Nevada has an NHF Chapter, thanks to her. Trailblazer, activist, she was also an emergency room nurse who had VWD and her personal story, how she was given a hysterectomy in her early 20s to stop her excessive bleeding and thus became childless, led her to become a VWD Warrior when she was later properly diagnosed as having VWD. She vowed no one else would suffer as she had.

Rewarding a partipant for a correct answer!

It seems we might have another Renee in our midst! We invited Nevada resident Kelly Lynn Gonzalez to speak at Pulse on the Road, upon recommendation of Michelle Rice, vice president of public policy and stakeholder relations at National Hemophilia Foundation. Good gamble. Kelly has VWD, as does her daughter Jacey. Kelly is the mother of 5 (yes, 5!!) and also worked in education, and now works for a specialty pharmacy. She participated here as a mother, to share the story of the birth of her activism.

In a nutshell, it was the struggle to get a diagnosis for Jacey, who suffered terrible bleeding, and who missed 47 days of school in one year, after doctor upon doctor told her it was one thing or another. Jacey had already fought off cancer, and now faced more health problems. Dealing with the multiple needs of her many children, Kelly still was able to fight the system, honor her maternal instinct which told her the doctors were wrong, and push and push not only to get a proper diagnosis, but also then health care coverage! Her insurer would not allow her to use the HTC!

Kelly Lynn Gonzalez implores
the audience to advocate and never give up

Well, that changed eventually.

Kelly is a powerful, emotive storyteller, and her presentation had many in tears. The energy level in the room, even following two hours of insurance lectures, was palpable. Kelly inspired many, I am sure, to rise up and become better advocates for their children. The audience was 60% families with VWD; an anomaly for us!

I thank everyone from Nevada who came to this symposium. Executive director Kelli Walters and her team did an amazing job handling this event. Thanks to our own Zoraida Rosado for organizing, shipping and set up. Thanks to Michelle Rice of NHF, and also thanks to Baxalta for sponsoring this!

Michelle Rice, NHF, speaks about
preserving healthcare options

Above all, thanks to every single audience member. Michelle and I agreed, from the podium, that in 7 years we have never seen a more attentive, engaged audience. (Well, Alabama was a close second!!) In appreciation, LA Kelley Communications is going to make a donation to the chapter for the wonderful audience. When you are a speaker, an attentive, receptive and engaged audience is everything.

Laurie Kelley with Renee Paper
in Puerto Rico, 2006

Renee used to tell me, “It’s not the hand you are dealt, it’s how you play that hand,” using an analogy fitting for a Las Vegas lady. In other words, you have VWD (or hemophilia). So what? The important thing is what are you going to DO about it? Renee was a no-nonsense, fix-it-now kind of lady. She never engaged in self-pity, and didn’t exactly coddle others. But she was compassionate. Not intimidated by anyone, she took the hand she was dealt (VWD and losing her ability to have children) and turned it into a national and then international crusade for better health care for those with VWD, especially women. Sadly, Renee passed away in November 2007 due to health complications. She is terribly missed. I feared for a long time there would be no one to fill her shoes; who could ever speak with that fiery passion? Who would have that dedication?

People like Kelly Gonzales give me hope.

Click here to learn more about Renee Paper, RN.

Amazing Book I Just Read

The Witch of Lime Street: Seance, Seduction and Houdini in the Spirit World  [Kindle]
David Jaher

It’s fitting to have read a book about Houdini on a plane ride to Las Vegas. We all know he was the master illusionist (much like David Copperfield, who I will see tonight at MGM, is today). But who knew the rest of the story? It’s fascinating! Following World War I, families were so grieved by the tremendous loss of life, they turned to seances and “spiritualists” to help them contact their deceased loved ones. One outspoken believer in contacting those in the afterlife was Sir Arthur Conan Doyle, the author of Sherlock Holmes! He traveled and spoke widely about spiritualism. It became such a hot topic, with so many engaged in it, that Scientific American decided to hold a national contest to see if anyone could prove that spiritualists could really communicate with the dead. On the committee was Sir Arthur’s good friend, Harry Houdini, who did NOT believe in spiritualism. As a master illusionist, he believed everything could be explained as a deception. And he successfully debunked many who claimed to have contact with the dead. But the hardest case was little sweet Mina Crandon, who lived on Lime Street in Boston, the wife of a very successful surgeon. She wasn’t a publicity seeker and didn’t want the prize money. And no one could seemingly detect how she was able to levitate a table, conjure spirits that spoke, command items that flew around the room. Without giving away this fascinating and sometimes lurid tale, this case destroyed the friendship between Sir Arthur and Houdini, made Houdini more famous, and yet reviled by the spiritual community, embarrassed the esteemed Scientific American publication, and brought to light even more bizarre facts than anyone expected! It is a riveting page-turner, extremely well written and informative, full of wild characters and celebrities, and the ending is a shocker. Read it! Five/five stars!

B the Inspiration!

I’m a believer in the power of stories. By sharing our personal stories, we can connect, inspire and support each other. This week I’d like to share an exciting new opportunity for the hemophilia B community. 
What’s your story? A new RIXUBIS [Coagulation Factor IX (Recombinant)] program gives you the chance to share it. 
Whether you’re a patient or caregiver, you’ve got stories about life with hemophilia B: stories of resilience, triumphs, and what keeps you going each day. 
RIXUBIS has launched a vibrant new program called B the Inspiration, created just for the hemophilia B community. See real people’s stories. Show your support and share your story, too. 
For now, check out the RIXUBIS Facebook page. And be sure to check soon for a special new section of the RIXUBIS website,, where these stories truly come to life. Get inspired—and be inspiring to others. 
And here’s the technica/medical info I need to include in this blog…
Indications and Important Risk Information for RIXUBIS [Coagulation Factor IX (Recombinant)] 

RIXUBIS is an injectable medicine used to replace clotting factor IX that is missing in adults and children with hemophilia B (also called congenital factor IX deficiency or Christmas disease). 
RIXUBIS is used to control and prevent bleeding in people with hemophilia B. Your healthcare provider may give you RIXUBIS when you have surgery. RIXUBIS can reduce the number of bleeding episodes when used regularly (prophylaxis). 
Detailed Important Risk Information 
You should not use RIXUBIS if you are allergic to hamsters or any ingredients in RIXUBIS. 
You should tell your healthcare provider if you have or have had any 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. 
Allergic reactions have been reported with RIXUBIS. Call your healthcare provider or get emergency treatment right away if you get 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 factor IX. An inhibitor is part of the body’s defense system. If you form inhibitors, it may stop RIXUBIS from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for development of inhibitors to factor IX. 
If you have risk factors for developing blood clots, the use of factor IX products may increase the risk of abnormal blood clots. 
Common side effects that have been reported with RIXUBIS include: unusual taste in the mouth, limb pain, and atypical blood test results. 
Call your healthcare provider right away about any side effects that bother you or if your bleeding does not stop after taking RIXUBIS. 
Please see RIXUBIS full Prescribing Information

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit, or call 1-800-FDA-1088. 

Baxalta and Rixubis are trademarks of Baxalta Incorporated. This is a paid annoucement by Baxalta.

To Market, To Market

At last! I am not traveling, and I have time to attend the
hemophilia events in my own back yard. This one is Fall Fest, New England Hemophilia
Association’s biannual event. Held in charming Providence, Rhode Island,
we had about 100 people attending to hear speakers like Barry Haarde, who rode
a bike across the US four times; Dawn Rotellini of NHF; and Perry Parker, golf
pro. I consider all these speakers not only interesting, but also friends!
In fact, and I know this isn’t original, but it really was like a
family reunion, to be surrounded by so many friends and families, some of whom
known for 15 or 20 years.
The general session opened the day, with guest speaker Dr.
Kapil Saxena, Director, Global Clinical Leader, Global Clinical Development,
Hematology, of Bayer Corporation, formerly a pediatric hematologist with
the Boston Hemophilia Treatment Center. He spoke about how a drug like factor
is brought to market. It was excellent and informative, and stunning. Some
Every drug on the market starts as one of almost 10,000 compounds.
Which one has a chance to be brought to market one day?  The Drug discovery journey sorts through
those 10,000 compounds, to narrow the field to 259 compounds (this can take up
to 6 years). These are furthered narrowed to 5 compounds (another 6 years!),
which then are brought to the FDA for review. When approved, they go into
clinical studies, which can last up to 1.5 years. Altogether, a drug like
factor can take almost 14 years to get to the market.
Laurie Kelley with Lee Hall, CoRe Manager
and person with hemophilia
Dr. Saxena explained the stages of clinical studies. There are
four stages, and some get killed off in the first stage, Phase I, for safety
reasons. Phase II examines efficacy and safety. Phase III, if it makes it that
far, involves a large number of patients, from 1,000-3,000 (although that can’t
possibly be true for hemophilia). More data is gathered over many clinical
centers. For hemophilia, these involve sites now in India, Egypt, China and
more. After the drug is brought to market, Phase IV looks for any more
problems, now that the drug is in widespread use.
His talk was relevant to our hemophilia marketplace today. With
the patents expired on factor VIII, IX and FVII molecules, our R&D and
production pipelines are filled with new products. Pharmaceutical companies are
banking on selling these, and yet the marketplace is getting crowded. Dr.
talk really made us think about the extraordinary effort—and cost–that goes
into making any one hemophilia product. On the one hand, these are among the
most scrutinized products on earth, and that’s great. On the other, how will the
market sustain all these great products?
Barry Haarde and Christian Mund
It will be interesting to monitor and to check in a year from
The afternoon sessions were a bit lighter! I attended one
mean featuring Barry Haarde! Of course, Barry is pretty much a celebrity by now
in this community for his heroic rides across America. Six weeks after completing
a 108 mile ride in one day, I still am having severe shoulder pain. Barry does
that in a day, and then goes out and does another 100, then another, then
Superman! He and Christian Mund, a young man with hemophilia, spoke on setting
goals, making dreams come true. Christian lives right near me and I’ve
known him since he was 12. And he went his first 12 years without being
involved in the community at all. Why? Life was good; there was factor, prophy,
and no need! But as he got involved, he enjoyed the rich relationships and
friends he made. He went through Bayer’s Leadership program, and landed an
excellent job at a marketing firm in Boston! Way to go!
Barry shared his story about how he got involved too. Barry went
most of his adult life without being involved. Shielding his HIV/hep C positive
status from everyone kept him from making connections. But he eventually joined
our community when his brother passed away. And boy did Barry join it! No one
in the US can compete with Barry on the cycling front. He’s
made history at this point, and raised about $170,000 for my nonprofit Save One
Life. But his message was simple: find what you can do and love to do. You’ll
never know where it will lead.
The day finished off with a dynamic presentation by CoRe managers
from Biogen, and also community members: Lee Hall, person with hemophilia, and
Lisa Schmidt, former program manager for NEHA. It was inspiring and fun. I’m
not going to give away their presentation, because… you should go
yourself! I give presentations, and know all the tricks and topics, been doing this
for 25 years. But I learned a few really wonderful things at this presentation.
Jane Smith with her
Volunteer Award 
We had to hustle out the Omni as the Redskins were approaching for their game with the Patriots! Like Rhode Island, the hotel was kind of small, so we headed home, really feeling happy after spending a day with “family.”

Heather Case of NEHA, John Bruno and Maryann May
The lovely ladies of NEHA!
Laurie Kelley dwarfed by golfer Perry Parker and
baseball player Jesse Schrader, who both
have hemophilia!
Patrick Mancini, NEHA president, presents
Dennis Mackey with an award
Laurie with Leslie Oygar, RN, who she wrote to 26 years
ago and now just met!

Good Book I Just Read

The Paths We Choose
Sully Erna [Kindle]
This autobiography by the founder and lead singer of the band Godsmack is not technically a great read, but it’s raw and real. The first full 60% of the book is a detailed catalog of Sully’s scrappy and difficult childhood, growing up poor in Lawrence, Massachusetts, with street fighting, crime and later on, drugs and alcohol. His breezy writing style is actually sweet, and bittersweet. You wonder how he ever escaped the beatings (received and doled out), the heartbreaks. Music was his driving force, his salvation. When he gets to the bands, and the formation of Godsmack, it all tied in. His survival skills gave him what he needed to take the huge risk to break out on his own, and become a frontman and founder of a great band, and a great sound. The book ends with his advice to all those with dreams and broken hearts to never give up, no matter what you have to go through. A quick, two-hour read, and I really enjoyed it and love the band’s music. Two/five stars.

Don’t Fear Heights— or Hemophilia

This is an impressive family with hemophilia, who did an impressive thing. I’m so inspired I want to go climb this mountain myself! We published this in the August issue PEN, but in case you missed it… here it is! Read and be inspired.

by Elizaveta Temidis
It is not the
mountain we conquer, but ourselves. 
—Sir Edmund Hillary
My son John and I are avid hikers, and we love a good dose
of adventure. John is 15, a sophomore at Wallkill High School, New York, and
has severe hemophilia A. He keeps busy on the school’s Nordic ski team, playing
piano and French horn, and reading.
last summer, John literally rose to new heights: he summited Mt. Whitney, the
highest mountain in the contiguous US. Mt. Whitney is 14,509 feet above sea
level, in the Sierra Nevada Range in California. A one-day permit means
completing the 11-mile ascent and return hike—with an elevation gain and loss
of 6,145 feet—in 24 hours.
to the West Coast from New York in the family car was an adventure in itself!
We departed July 30, a beautiful summer morning, with Ramen noodles, factor,
and audiobooks.
American West is beautiful and fascinating. Dust devils wander aimlessly on the
Utah and Nevada plains. In Nebraska, a gigantic gate on Rt. 80 welcomes
everybody to the Wild West! Carbon County in Wyoming proudly holds a Cow Plop
annual event. Mustangs still roam free in Utah. Warnings about rattlesnakes are
mundanely posted on garbage cans at rest stops. American pronghorn antelopes
are the second fastest land animals after cheetahs. Our car can outrun a Nevada
a four-day drive through 11 states, we arrived in California on August 3 and
pitched our tent at a campground 8,000 feet above sea level, to get acclimated
to the altitude and explore before the big hike. We protected ourselves against
black bears that roam the campground at night, gawked at the amazing beauty of
magnificent mountain ranges, and chopped enough firewood for evening campfires.
August 7, we packed up and moved to Whitney Portal campground. We set up our
tent, gathered our supplies, replenished our drinking water, and went to bed
around 6:00 pm. We awoke four hours later, John infused his factor, and we set
off on our grand new adventure at 11:45 pm.
flashlights moving on the side of the dark mountain assured us we were not
alone. We stopped every hour for a snack and every 15 minutes for a gulp of
water, watching for signs of altitude sickness. The last two hours before
sunrise were the darkest and made us yearn for the sun like never before!
greeted the sunrise at six miles, or halfway up the mountain, before going on
to the infamous section of 99 switchbacks. The switchbacks were helpful and
refreshing, and we named them after family members as we hiked. Freezing
temperatures faded, and the views were astonishing! Reaching Trail Crest, we
gasped at the amazing view of the Sequoia Kings Canyon Wilderness all the way
to the Pacific Ocean. We were happy to see patches of fresh snow in mid-August,
and even threw a few snowballs.
reached the summit at 10:30 am—tired, but relieved, grateful, and at peace. The
absolute quietness of this incredible place was interrupted only by unobtrusive
shouts of joy from arriving hikers. We took a photo of us holding a hand-drawn
birthday card for my mom, whose birthday is August 8; and of a toy squirrel
monkey that we’d promised Alex, John’s younger brother, to take to the top with
us. Before heading back down, we celebrated with two little bottles of Coke,
and spent the next hour in a relaxed, contemplative mood, taking our time,
looking at the endless mountain ranges all around, while shivering in the cold,
unrelenting wind.
hike down to Whitney Portal seemed harder than our hike up. We were extremely
tired and had one desire: to lie down in our tent and sleep. Yet the wondrous
scenery of mountains and cliffs, which we hadn’t seen during our ascent in the
dark, made us stop in awe, taking photos and marveling at the beauty and
ruggedness of this corner of the world.
last two miles were the hardest. We needed to finish before dark. We sang every
Russian and American song we knew, and recited every Russian poem we could
remember. We got some curious looks, yet our method worked so well that we
barely noticed the miles pass.
the hike, John had no traumas or joint problems. He’s been on prophylaxis since
age one, and since he began training on the Nordic ski team two years ago, he’s
had fewer joint bleeds.
a 22-mile round trip, a total of 19 hours and 15 minutes on the trail, and a
6,145-foot elevation gain and drop, we returned to our tent happy and
exhausted. We texted our family that we were victorious, overjoyed, nauseous,
and tired. Then we went to sleep.
arrived home five days later, hungry and happy, full of news and impressions,
eager to hug our family and grateful for everybody’s support. It’s possible
that John, at age 14, might be the youngest person with severe hemophilia ever
to climb Mt. Whitney! This climb proved to us that whatever challenges might
stand in the way—hemophilia, fear of heights, or pain—our children with
hemophilia need to pursue their dreams and live life to the fullest.
Elizaveta Temidis, 40, was born in St. Petersburg, Russia,
and came to the US to study business in college at age 19. She is a high school
mathematics and Russian language teacher with New Paltz High School and online
Virtual High School. She helps run the Nyack Russian School and a Russian
summer camp in the Catskill Mountains. She lives in Wallkill, New York, with
her husband George and sons John, 15, who has severe hemophilia A, and Alex,
 Great Book I Just Read

Denali’s Howl: The Deadliest Climbing Disaster on America’s  Wildest Peak
Andy Hall [Kindle]

Andy Hall was just a child when his father, employed with the National Park Service as park superintendent, was present when Joe Wilcox and his 12-member team made a daring ascent in 1967 of Mt. McKinley, which President Obama has now called Denali, its native name. Only 5 of these ambitious and brave young men survived, and Hall, an accomplished journalist, recreates their journey and respectfully and accurately dissects the personality, the team problems, leadership conflicts, and the unforgiving Perfect Storm that hit Denali that fateful climb. Fantastic writing and a gripping story make this a page turner. Four/five stars.

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