I’m back from a fantastic time at the Florida Hemophilia Association’s annual family meeting. About 330 people attended, and we presented Pulse on the Road, which I’ll write about in a few days, once I have the photos back! There was a motivational speaker, our insurance symposium, a men’s rap session, a woman’s session, and a thorough presentation by Dr. Glenn Pierce of Biogen Idec on long acting factor and gene therapy, and an
Dr. Joanna Davis: excellent presentation on prophy
energetic kids program.
This morning I attended a very detailed and comprehensive review of prophylaxis. You know, many families do prophy, and I have read about it, and researched it, and written about it for years. But today reminded me, you just can’t get enough education on the subject, and we all need to be reminded of the basics. Dr. Joanna Davis, Medical
Director, Div. of Pediatric Hematology/Oncology, University of Miami Hemophilia Treatment Center, and local “celebrity” as she was introduced, gave a five star review of hemophilia basics and how to do prophy the right way.
I liked how she described prophy as an attempt to mimic “nature” by boosting factor levels up in the normal range by planned and frequent infusions, whether a child needs them or not. She reviewed the difference between different prophy regimens:
Primary prophylaxis: begins with the first bleed, and is often an indefinite course of therapy. It prevents joint damage.
Secondary prophylaxis: aimed at treating a target joint or providing coverage during a high-risk situation such as team sports, or after a surgical procedure. It stabilizes or improves joint function.
Then she covered some basic definitions that we all need to know:
Peak level: the level of factor activity reached immediately after an infusion.
Trough: the lowest level of factor activity left over before infusing again.
Half-life: the time it takes for the factor levels to drop to half of what was infused. For FVIII: about 8-12 hours, for FIX, about 24-48 hours.
She explained in practical terms what this meant for prophy: let’s say you infuse on a Monday and you peak at 100%, for example. By Monday evening, a factor VIII patient may be then at 50% factor activity (which means still in the normal range). By Tuesday, the factor activity drops to 25%, and by Tuesday evening, maybe down to 12%. Just before infusing on Wednesday morning, the patient’s levels may be down to 2%–the trough. So prophy is scheduled according to a series of the peaks and troughs of factor activity.
Dr. Davis reminded the audience that on Friday, you may do your third prophy shot of the week. By Monday you’ll be depleted. She reported that
some families do a double dose of factor on Friday, to boost levels high, so the half-life does not bring the factor activity down to 2% by Monday morning.
Some of the barriers to prophy? Venous access—many children’s veins can’t take all the infusions and needle sticks, so a central venous access
device might be in order. This presents its own risks, such as blood clots. And sometimes, children may balk at such frequent infusions, and teens can be in denial and refuse to do their infusions (oh yeah, I can vouch for that one!). And the big issue is cost. Prophy ain’t cheap.
The best time for prophy is the morning, even though our ridiculously over-scheduled lives often make us want to wait till evening. Think about it: infuse at night, when things are quiet, and by morning you’ll only have half the factor working! Best to infuse in the morning, when your child is most active and when the factor is working strong, at 100%.
This was a great overview of prophy and the audience responded
with excellent questions.
After Dr. Davis’s talk, Alan Shier, of ATHN, spoke about the importance
of keeping logs about bleeds and presented the ATHNAdvoy, and wow! What a great
presentation too and what a great device. This is a web-based tool that will
help you track your bleeds and product usage, is accessible by your treatment
center, and can produce reports in a heartbeat. This is also available as a
mobile phone app, so you can use your Smartphone to quickly scan in your product,
report your bleed, and then you will always have a constant record, which you
can even print out in colorful reports. THIS COULD COME IN HANDY IN THE NEW
INSURANCE REFORM CLIMATE! Have I said that clearly enough? We should all be
recording every vial of factor we use. I would highly recommend using the
ATHNAdvoy.
About 11,032 hemophilia patients have already registered for this, which is astounding. All the data is kept in aggregate and no personal information,
including your name is kept. A bit hard to explain all this in a blog; I recommend
visiting www.ATHN.org to lean more!
Thanks to Debbi Adamkin and team for a fabulous time and meeting! (Debbi, aka Wonder Woman, I owe you a dance off)
Great Book I am Reading
In Cold Blood by Truman Capote
After watching the movie “Capote,” starring Philip Seymour Hoffman (fantastic), I felt compelled to read this, especially as the movie ended with the fateful words that Capote never wrote another book, and died of alcoholism. If the story of the writing of the book is compelling, the book is sheer brilliance. Capote does more in this book than document the shocking 1959 murders of Herbert Clutter, a wealthy farmer from Holcomb, Kansas, his wife, and two teens. Capote interviewed everyone and anyone connected to the case and living in Holcomb, including the murderers themselves, taking six years, and created a whole new genre in literature—the non-fiction novel. It certainly became the first true crime novel and was a raging success. Skillfully, almost lyrically written, well paced although it seems to weave its way through the minds of the killers at leisure, this is a masterpiece and not to be missed.
On Monday, August 6, 46-year-old Barry Haarde will be the first person living with HIV and hemophilia to have ridden across the United States by bicycle!
He’ll dip his bicycle wheel into the Atlantic Ocean in Portsmouth, New Hampshire after 50 days and 3,667 miles.
I rode 12 miles on Saturday. I cannot even imagine. Barry is the hemophilia community’s new national hero!
Barry’s “Wheels for the World” ride is about more than setting a record: he is riding to help raise money for Save One Life, the nonprofit I founded in 2001. Many of you are aware that this program provides personal sponsorships of individual children with hemophilia who live in poverty in developing countries. We’re hoping to raise $50,000!
Barry is on Day 35 today, in Fond du Lac to Manitowoc, Wisconsin; 57 miles in one day, elevation gain: 1,350 feet. The Wisconsin hemophilia group took Barry out to dinner to welcome him! Nice going, Wisconsin!
Barry has heart, physique and courage. Each day he dedicates his ride to someone with hemophilia who lost their life to AIDS. For example, today, day 35 of the ride, Barry devotes today to Brandon Hendrickson. I know Brandon’s mom, Angie. How wonderful that someone still remembers Brandon, and urges us all to remember him and the hundreds, thousands more who died!
But let’s not forget those still living, who suffer with no treatment and extreme poverty. Barry is also riding for them.
So to honor Barry’s most certain achievement, I am doing something I’ve never done before. I’m hosting a party for the hemophilia community locally! On August 6, Save One Life will celebrate Barry’s ride. It’s at my house… so contact me if you want to come! laurie@kelleycom.com (An attendance of 75-100 guests is expected. Tickets to the celebration are $25 per guest; $50 per family.)
And please, readers, can you also help? Make a donation today in any amount to our Wheels for the World fundraiser. Go to http://www.saveonelife.net and click on Wheels for the World. You’ll go directly to a page where you can use PayPal or other means. And if you do, I will double your donation by matching it! Please help today and show Barry you are cheering for him!
See Barry’s Interview with the KDLT Evening News, a South Dakota news agency!
“Are you a madman or a guinea pig?” asked a man from France at the final symposium of the World Federation of Hemophilia Congress in Paris, France last Thursday. He addressed his question before the 500 or more attendees in the huge amphitheater at the Palais de Congres to our own George McCoy, from North Carolina, one of the fascinating speakers on the panel debating and comparing long-acting factor to gene therapy.
Laurie Kelley with George McCoy, from North Carolina
Great question, particularly as George had just revealed that he was the very first human ever to be injected with recombinant factor VIII, back in March 1987. I was riveted to George and his reply. The whole audience was. But more on that in a bit!
The session opened with Dr. Paul Giangrande, director of Oxford Haemophilia Centre in England and world-renowned hematologist, purposely supporting continued recombinant therapy, and listing the many pros of our current treatment. Paul reminded us of the Hippocratic oath Primum non nocere: First, do no harm, and cited the long and many years of improved quality of life and quantity of life on these therapies.
(Dr. Giangrande also prefaced his presentation with an outrageously humorous skit on how he gets roped into presenting so many talks at symposia, all seemingly taking contrary positions! He feels it is his duty to provide food for thought on the pros and cons of all treatment; here, he was asked directly by WFH to make a case for recombinant therapy)
Long acting factor, Dr. Giangrande said, which many manufacturers are working on, is based on recombinant technology, a stable and known technology, which is considered safe. It provides breadth of product for factor VIII, IX, and inhibitor patients. WFH president Mark Skinner even said earlier at the Congress that treatment should aim for a trough level of 15%—which recombinants can provide. Giangrande added that we know the manufacturers already so we know in essence from where the product comes and what we are getting.
Giangrande then cited some of the downside of what we know and can expect from current gene therapy, still in clinical trials: more hospitalvisits and blood tests. He asked if patients truly were giving informed consent. Most parents don’t understand what the studies involve. And he questioned the
ethics of using patients in developing countries in these clinical trials—there are medical risks, and the patients could be desperate to have product no
matter what.
Will gene therapy really lower costs? Currently Dr. Kathy High from Children’s Hospital of Philadelphia is investigating a factor IX gene therapy treatment. If successful, Giangrande reminded us, it will be commercialized, just as all the past research in hemophilia has been.
Next came Dr. Kathy High herself, who reminded the audience right away that the ultimate goal is to cure hemophilia. We aren’t used to thinking of cures for genetic diseases. Some have been radical, such as bone marrow transplants or liver transplants. But we have alternatives now. In a recent published gene therapy article, research in the UK showed that a single infusion led to more than 18 months expression of factor IX at
about 5% levels. This was repeated in two other patients. Unfortunately, eight weeks later one patient required steroids. While it looks like a virus vector may trigger an immune response, Dr. High felt that the risk of a short course of steroids outweighed the risk of bleeds. Other risks noted included the viral vector appearing in semen; and one mouse model showed an increase in carcinoma (but he was an old mouse with lysosomal storage disorder! The things you learn at these symposia).
How long will this gene therapy last in the body? We don’t know. In dogs, over ten years. And it only requires a simple infusion, with no organ transplant.
Richard Minja (Tanzania), Neil Herson (President, ASD Healthcare), and Simba (Zimbabwe)
Dr. High posed the question on everyone’s mind: when will gene therapy happen for hemophilia A patients? High believes it will start in 3-5 years. Some studies are already underway, such as one with AV canine factor VIII, with Dr. David Lillicrap of Canada. High gave a positive and detailed summary of currently gene therapy—very exciting presentation!
So back to George McCoy, who then spoke, eloquently, sincerely and humbly. He described his childhood, and how he was diagnosed at age two. One of four boys, his eldest brother Richard had hemophilia for years and no one knew. Sadly, he died. George’s speech was moving and thoughtful, especially when he cited WFH founder Frank Schnabel, who described patients with hemophilia as “tortured pieces of human geography
confined to a wheelchair.”
Dr. Gil White contacted him about being the first rFVIII patient ever in March 1987, and hence the question: is he a madman or a guinea pig? George simply replied that he would do it all again if he had to—he would volunteer for gene therapy if—or when—the time comes. And he stressed that we need volunteers for gene therapy now.
He closed with the beautiful thought: “What brings us here is the will to live, the
will to prosper, the will to learn. We all do need each other.” And while all
the speakers weighed in on whether they would elect gene therapy over
long-acting factor for their hypothetical grandchildren with hemophilia, were
they to have one, it was Dr. High who made a memorable statement: we need access to all therapies, both gene therapy and long-acting, for individual patients with
individual biological make-ups, for those in developing countries— access to all products and treatments for us all. George was right: we all do need each other, and this past week at WFH reminded us all that 300,000 have little or no access to hemophilia treatment. The burden—to me the joy— is on us to help them all.
CEO of Biogen, George Scangos, Laurie,
Sr. VP Medical Affairs, Biogen, Glenn Pierce
I’m in a soggy Paris (not complaining though!), attending the wonderful 50th anniversary of the World Federation of Hemophilia, taking place during its biannual Congress. The WFH, founded by American Frank Schnabel, is the premiere hemophilia nonprofit in the world, dedicated to improving the lives of those with hemophilia around the world, uniting those with hemophilia globally, and advancing access to care in developing countries. Today was the opening day, and a staggering, record-breaking 5,300 are in attendance from 130 countries! It’s a great opportunity for me to meet with our many country partners, who work with us to get factor to patients in developing countries.
I’ve been attending WFH meetings since 1996, and attend every two years. It’s a great chance to learn more about WFH efforts, to meet up with those we serve, and to make new acquaintances. Right away I bumped into Dr. Tatiana Andreeva, a pediatric hematologist from St. Petersburg whom I’ve known since 1998. She even visited my home once!
The incomparable Jill Smith, RN from Australia—who traveled with me once to Zimbabwe for a week to do an assessment of hemophilia care there (and shared a tent with me on safari!)—and incredible Anne Gillham, RN from South Africa, who has been a vital source of training and support for so many countries in Africa besides her own.
(L to R) Jill Smith, RN (Australia), Anne Gillham, RN (South Africa), Laurie Kelley
The opening ceremony was excellent. Mark Skinner, past president of NHF and who has been president of WFH for 8 years now, gave an insightful talk about the accomplishments of WFH in helping advance care. But never have I seen the WFH so intensely focused on developing countries. This made my heart sing, for it’s been my mission and desire since 1996, that we do not forget those at the bottom of the wait list, the ones whose government does not provide funding for treatment. A very touching and powerful movie was shown about how WFH has worked in Senegal to improve care, and I was pleasantly surprised to see that producer Marilyn Ness had created it! It was beautiful, so well done. I’ve been to Africa many times and have traveled those same dusty roads, been in the same hospitals with paint-peeled iron beds, no AC, suffering patients. Marilyn captured the feel of hemophilia care in Africa well.
The WFH is now making a serious push to “Close the Gap” with a new campaign, designed to raise $5 million to help create programs to reach the impoverished countries where care is lacking. We still have four more days of the Congress, and already the tone has been set: we must act now to help save the lives of those without treatment. It’s what we’ve been focusing on at Save One life, my nonprofit, and I applaud WFH for putting the spotlight on developing countries as it moves forward, and asking everyone to join in.
After the ceremony, we all moved into the exhibit area and visited the various booths by the pharmaceutical companies. There we met even more colleagues and friends. I was thrilled to meet up with my friends from Africa, especially Megan Adediran, president of the Nigerian Haemophilia Foundation, who just published a new book about her experiences with hemophilia, called A Light at the End of the Tunnel.
More to come!
Laurie Kelley With Simba (Zimbabwe) and Megan (Nigeria)
It’s fair to say we’ve all been holding our breath to see what the Supreme Court would rule this past week on the Affordable Care Act, often referred to as “Obamacare.” The ruling will have a long-term effect on the bleeding disorder community. Indeed, I’ve been to Hawaii, North Carolina and Tennessee as part of our insurance symposia Pulse on the Road to ask families and patients to pay careful attention to their healthcare policies, and to what’s happening in Washington DC. Would lifetime caps be reinstalled? Would pre-existing conditions be allowed again? Our children with hemophilia would face terrible battles for healthcare access if these provisions were reinstalled.
But in a 5-4 vote, the US Supreme Court ruled to uphold most of the health care law. One central controversy was over mandatory insurance; many Americans saw this as unconstitutional and a threat to our freedom to choose our own protection and access to care. Chief Justice John Roberts gave an opinion, which upheld the constitutionality of the individual mandate because it is a tax. The Medicaid expansion has been limited.
We’re lucky to have such a vigilant community that jumped at the news and shared right away; our vigilance was earned the hard way, through massive loss of life. From the Hemophilia Federation of America: “HFA is reviewing the ruling and will closely monitor its impact and are committed to ensuring adequate access to care for all people with bleeding disorders,” said Kimberly Haugstad, Executive Director of HFA. “We will continue our advocacy to protect beneficial provisions of the ACA, so that the overall health care system is improved. We encourage everyone in the community to join us.”
ACA was made into law March 2010, in the hopes of reforming our convoluted and expensive healthcare system, to try to lower costs and to keep all Americans insured. About 45 million Americans (out of an estimated 300 million) do not have insurance. In fact one young man, age 27, called me two weeks ago with a severe bleed, telling me he didn’t have insurance! He wanted me to send factor that I normally give to patients in developing countries! His only resort would be to go to the ER, get factor, incur massive bills and pay the rest of his life, even just $10 a month, for this enormous cost, easily a $20-30,000 visit. The hospital would eventually write off his treatment as bad debt.
With mandatory insurance, this young man will be forced to find insurance, and his healthcare should be covered. There are so many provisions in the ACA that benefit patients with bleeding disorders, indeed anyone with chronic health disorders. But the stage is set for November: how will this ruling impact the presidential elections?
Maybe it will inspire more Americans to get out and vote. It’s only by persistent advocacy that we have even come this far. I don’t know what the future will bring, but I am breathing a little easier tonight knowing my son is covered for one more year, even while wondering what financial impact this will have on us all long term.
For more information, visit www.hemophiliafed.org or call 1-800-230-9797.
Thanks to Tom Bennett, Executive Director, Hemophilia of North Carolina for sharing the HFA alert with us.
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This was a great overview of prophy and the audience responded
Great Book I am Reading
