“Exploring the Next Frontier” was the theme for the 69th annual National Hemophilia Foundation meeting in the dazzling city of Chicago. A record-breaking 2,987 community members flocked to the Windy City (do you know why it’s called that?)—patients, treatment center staff, industry representatives and hemophilia organization advocates—to share stories, to educate, to network, and to learn.
Inside a blood vessel!
For me, it was my 25th annual meeting, and more like a huge family reunion. With so many friends from past meetings and local chapter meetings and correspondence, on top of all my new friends on Facebook, it was almost impossible to go from point A to point B without bumping into someone I knew!
Brian Andrews, chair of NHF, opened the weekend-long event Thursday evening by welcoming everyone; Val Bias, CEO, stressed inclusiveness and our diversity: individually he welcomed the VWD community, the FX, FI, FXIII, women with bleeding disorders (not VWD but hemophilia!) communities, who all stood up. Val then placed the focus on the National AIDS Memorial Grove, San Francisco, on which names of those with hemophilia lost to AIDS are carved. A touching video showed the memorial, with comments from community members, in particular Jeanne White-Ginder, whose son Ryan White, our own national hero, in 1982 put a tender young face to the scourge of hemophilia/AIDS by refusing to accept being ousted from his school. His stand led to a national movement to better understand the suffering of AIDS patients, the discrimination they faced and the erroneous fear that electrified Americans, most of whom believed you
could contract HIV just from a handshake.
It was a beautiful video, and Jeanne concluded it with a heartfelt, tearful speech about her love for our community. Val asked for donations, to raise $50,000 for the Memorial, and by the close of the conference, $41,000 had been raised!
The next few days were jam-packed with activities, educational sessions, and walks through the industry and nonprofit booths
downstairs, where consumers could play games, speak with reps, and pick up literature on products and services.
Our own Save One Life had a booth that actively received inquiries on how to sponsor a child with a bleeding disorder in a developing country.
The highlight of my visit was the Octapharma symposium Friday morning, showcasing the documentary trailer for “Bombardier Blood,” directed by Patrick James Lynch, who has hemophilia A. Patrick shared the incredible story of the making of the documentary—a project of which I was a part! I traveled to Nepal (visit #4) to introduce Patrick and his team to the Nepalese Hemophilia Society, and to watch as the team filmed Chris Bombardier (factor IX, from Denver) visit the treatment center, travel to patients’ homes, and attend a fun cultural evening before heading out to attempt to summit Mt. Everest. I also accompanied Chris, his wife Jess and photographer Rob Bradford, all the way to Everest Base Camp.
Although I was with them the week in Kathmandu, and then endured the rugged 9-day trek to base camp at 17,500 feet, and then shivered three days at base camp, with 1° temps at night, nothing, nothing stirred me as much as watching the documentary. The full impact of Chris’s sacrifices, the months of training, overcoming fears, and the pressure on this young man to succeed, hit me full force as we saw in six minutes scenes from Denver, from Nepal, patients, base camp… and Chris on the summit, talking through his oxygen mask, holding a banner on which was written the names of Nepalese patients with hemophilia. He did it for them; he did it for us.
Patrick J.Lynch, Laurie Kelley, Chris Bombardier
Over 360 people had permits to climbing Everest that season; 60 summited, including Chris; 10 died, including a world class alpinist, Ueli Steck. Chris risked his life to achieve something no one in history had done: being the first with hemophilia to summit Mt. Everest. Listening to Patrick, and seeing the beautiful trailer, we were all wiping away tears. The human heart has so much potential for courage, for sacrifice for our fellow humans, for overcoming fear and pain. Chris embodied all this.
I worried for the next speaker: how do you top that? But you know, Seth Rojhani, a young man from Denver, nailed it. His story was incredibly motivating and uplifting: being born with hemophilia, then losing your ability to walk after having a spinal bleed, and the surgeons severing your spine accidentally. But nothing has stopped Seth. He loved sports, and with the full support from his wonderful parents (who I am proud to say I know) he participated in many sports!
With his favorite basketball team the Denver Nuggets, Seth Rojhani went on to form “Rolling Denver Nuggets,” a basketball team for wheelchair participants. I loved when he shared his formula for success:
Seth Rojhani and Laurie Kelley
He stayed on a consistent prophy schedule; He rested until all injuries healed; He visited his HTC often. Seth received numerous athletic awards, including the Bronze medal for his team in the Maccabiah Games in Israel this past July. And he promptly pulled out the gleaming medal for the audience to see! Seth said, “Hemophilia is a speed bump, not an obstacle.” He also shared that his father, Ira, told him, “Think positive and good things will happen.” His belief in this way of thinking has never let him down.
When asked of the three participants—Patrick, Chris and Seth—what was the biggest challenge they faced in life, Patrick mentioned losing his brother Adam. His brother never identified with the hemophilia community, and felt isolated, alone. He might as well lived on the outskirts of Nepal, exclaimed Patrick, without factor or comprehensive care. Patrick’s greatest
challenge is overcoming the loss of his brother. Chris’s? Not Mt. Everest but needles! Chris has a needle phobia! And Seth? Being told no so much in his life.
The speakers deservedly received a standing ovation for their incredible stories and work. The three days were filled with symposia and sessions. For first time, the LGBT community had their own session, led by our own (New England-based) Justin Levesque. And I am proud to say that PEN was the first publication in our community to publish an article about the community needs, also written by Justin.
Women with bleeding disorder and those with VWD were also given lots of meeting and air time. You can see a big shift in mindsets this year about inclusion in our community. Those on the fringe are now being heard.
There were also sessions for siblings and one for men only; sessions about pain management, addiction, and gene therapy. The only bad thing about NHF’s Annual meeting is that there is so much to see, hear and do! I couldn’t take in everything unfortunately.
The event ended with a stunning visit to the world famous Field Museum, sponsored by Bioverativ, where families could see the wonders of nature and natural history. My favorite display are “The Ghost and the Darkness,” two man-eating lions from Tsavo, Kenya, which were killed in 1898 after they had killed many workers on the railroad. A Hollywood movie starring Val Kilmer and Michael Douglas tells a somewhat fictional version of the story. It’s a good story but better to see them for real at the museum.
This is a paid public announcement from Grifols 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 Grifols website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.
Treating Your Hemophilia A and von Willebrand Disease With ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) A diagnosis of hemophilia A can be an overwhelming experience, not only for patients, but for their families as well. The best way to combat the feeling of helplessness that often follows a diagnosis is by becoming empowered with information. Grifols, the manufacturer of ALPHANATE—the #1 prescribed plasma-derived factor VIII/von Willebrand factor treatment—has just launched a new patient website. ALPHANATE.com provides experienced and newly diagnosed patients and their caregivers with an understanding of hemophilia A and von Willebrand disease (VWD) along with tools for living with these bleeding disorders.
Living With Hemophilia A
ALPHANATE.com answers some of the most common hemophilia A questions in one convenient location.On the new site, patients and caregivers can learn exactly what hemophilia A is; why a plasma-derived FVIII could be a treatment option; and details about inhibitors, the greatest challenge in hemophilia A treatment.1,2 ALPHANATE.com also outlines how patients can identify, manage, and prevent bleeds. In other words, ALPHANATE.com is not just about providing patients and caregivers with information, it’s about helping people with hemophilia A or VWD.
Patient
Support & Resources
All patients should have access to proper medications, resources, and strong support. With this commitment to care in mind, Grifols offers Factors for Health, a comprehensive patient support and assistance program. The Factors for Health program offers:
$0 Copay Assistance Program, which may cover out-of-pocket expenses not
covered or partially covered by insurance
Free Trial Program, for eligible patients new to ALPHANATE
Benefits investigation and support services
Patient Assistance Program
Care Coordination, helping patients gain access to and remain on
ALPHANATE
Other resources on thenew ALPHANATE.com include a Doctor Discussion Guide to prepare you to get the most out of your conversations with your doctor and a blog with tips for living with and managing a bleeding disorder.
ALPHANATE
ALPHANATE is an FDA-approved human plasma-derived treatment for adult and pediatric patients with hemophilia A or VWD. The manufacturing process for ALPHANATE is specifically designed to preserve FVIII and VWF together, the way they are naturally found in the body.3
Since product launch in 1997, there has never been a confirmed case of virus or prion transmission with ALPHANATE. Grifols is committed to safety before, during, and after ALPHANATE manufacturing. In fact, ALPHANATE is manufactured using a 10-steps-to-safety process, with full traceability from donor to patient.
Because ALPHANATE is made from human plasma, it may carry a risk of transmitting infectious agents, eg, viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, despite
steps designed to reduce this risk.
To meet the needs of a variety of patients, ALPHANATE is offered in a range of convenient vial sizes with low diluent volume.3
Visit Plasma City on the new ALPHANATE.com to see how others with hemophilia A and VWD have been building foundations for success with ALPHANATE for 20 years. And don’t forget to sign up to stay up to date on what’s happening in Plasma City.
Indications
ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) is indicated for:
Control and prevention of bleeding episodes and perioperative management in adult and
pediatric patients with factor VIII (FVIII) deficiency due to hemophilia A
Surgical and/or invasive procedures in adult and pediatric patients with von Willebrand
disease (VWD) in whom desmopressin (DDAVP) is either ineffective or
contraindicated. It is not indicated for patients with severe VWD (type 3)
undergoing major surgery
Important Safety Information
ALPHANATE is contraindicated in patients who have manifested life-threatening immediate hypersensitivity
reactions, including anaphylaxis, to the product or its components. Anaphylaxis and severe hypersensitivity reactions are possible with ALPHANATE. Discontinue use of ALPHANATE if hypersensitivity symptoms occur, and initiate appropriate
treatment. Development of procoagulant activity-neutralizing antibodies (inhibitors) has been detected in patients
receiving FVIII-containing products. Carefully monitor patients treated with AHF products for the development of FVIII inhibitors by appropriate clinical observations and laboratory tests.
Thromboembolic events have been reported with AHF/VWF complex (human) in VWD patients, especially in the
setting of known risk factors. Intravascular hemolysis may occur with infusion of large doses of AHF/VWF complex (human).
Rapid administration of a FVIII concentrate may result in vasomotor reactions.
Because ALPHANATE is made from human plasma, it may carry a risk of transmitting infectious agents, eg, viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, despite steps
designed to reduce this risk.
Monitor for development of FVIII and VWF inhibitors. Perform appropriate assays to determine if FVIII and/or VWF inhibitor(s) are present if bleeding is not controlled with expected dose of ALPHANATE.
The most frequent adverse drug reactions reported with ALPHANATE in >1% of infusions were pruritus, headache, back
pain, paresthesia, respiratory distress, facial edema, pain, rash, and chills.
In 2007 I was honored to be invited to Hiroshima, Japan, by Novo Nordisk to speak at a meeting of Japanese hematologists, and later a group of hemophilia patients and families. While in Japan, I was able to visit the A-Bomb Dome, ground zero where the atomic bomb was dropped. I’m a huge history buff, and read voraciously about history of all types, and try to visit as many historical places in the world as I can. Of special interest is World War II. I wondered how many
Americans even remembered the dates when “Fat Man” and “Little Boy” were dropped?
We should. I was thinking of Japan and my visit last week, mainly because it is the anniversary of the bombing of Hiroshima (August 6, 1945) and three days later, Nagasaki (August 9, 2017). Also because for some reason, my visit to Hiroshima is one of the highest hit pages on my blog, consistently every year. I reflected on my memorable trip, and what I saw at the museum in Hiroshima. I planned on running a return visit by reposting my blog, when national events suddenly made it more urgent that I do.
Last week, in between those two infamous dates, President Trump chose to use threatening (yet characteristically vague) rhetoric toward North Korea. Everyone since has been whispering and worrying about nuclear war. Guam is now preparing its inhabitants for a pre-emptive strike. In 2007 I wrote: “Yesterday in Hiroshima, before I gave my presentation, I had time to slip out to the Peace Museum. This expansive, eerily quiet edifice preserves the memory of those killed on August 6, 1945, when the first atomic bomb was used. Inside, Yuko and I read in hushed silence what life was like in Hiroshima the morning the bomb fell, and the devastation that followed. The museum has recovered the actual clothing of some of the children who died in the attack, and most notably several watches, with hands frozen at 8:15 am, when the bomb struck. It was hard to sit through the 30 minute opening film without tears filling our eyes, viewing how the victims suffered. Hiroshima has since become the epicenter of a world peace movement, and every mayor since 1968 has written to world leaders whenever a nuclear weapon is tested, urging them to stop the spread of nuclear weapons. The photo above shows the A-Bomb Dome, now a World Heritage site, which is all that remains of a building near the hypocenter of the blast. It was also about the only thing left standing in the city.”
The atomic bomb killed 140,000 people in Hiroshima. The bombing of Nagasaki three days later killed 70,000 more. Japan surrendered days later, effectively ending World War II.
“Nuclear weapons are incompatible with mankind,” said Yoshitoshi Fukahori, an 88-year-old survivor of the Nagasaki bombing who lost his sister in the blast. He said that as he rushed home the morning after the bombing, the shocking view
from the hilltop — his hometown flattened and the landmark Catholic church on fire — made him cry. (LA Times, August 9, 2017)
Since the nuclear destruction of their cities, Japan has become one of the most peaceful nations on earth. The entire Japanese culture is based on respect, desire for peace, and abhorrence of nuclear weapons. Careless throwing around of vague threats sets the entire world on edge. I went to Japan to speak about hemophilia, but came home with renewed respect of how Japan suffered, how it changed their national identity, and with amazement at the peaceful culture.
Let’s hope and pray we do not have to suffer such extremes to learn what peace is and means.
If you know little about the events of World War II that led to the bombings, or know little about the bombings themselves, please read about them. Learn. Study. We learn from the past, to create a better future. And to guide our world leaders, who I fear have not studied about Hiroshima… or learned from it.
* Never again. Plus jamais. Words inscribed at Dachau, a German concentration camp.
Recommended: the movie Fat Man and Little Boy, starring Paul Newman
Great Book You Must Read
Hiroshima by John Hersey. This book, which I read just in time to honor the 49th anniversary of the August 6 bombing of Hiroshima, is one of those absolutely rare gems: short, full of prose and
thoughtfulness, and leaving a lasting impression. You can read this book in one evening, and probably will because it’s hard to put down. Simply told, powerfully effective and unforgettable. A classic. Five stars.
This is a paid public announcement from Shire 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 ADVATE® [Antihemophilic Factor (Recombinant)] website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company, do not reveal identifying information against your will.
Proven Clinical Experience
Prescription drug candidates today go through clinical trials and rigorous efficacy and safety testing but what does that mean for its use in the real world? What does that mean for you?
Clinical studies are very important for bringing products to market, and for expanding the indications for current products. At any given time there are a number of clinical studies happening in hemophilia with the goal of bringing you safe and efficacious drugs. Please read below about one drug and its clinical study results.
Indications
ADVATE is a medicine used to replace clotting factor (factor VIII or antihemophilic factor) that is missing in people with hemophilia A (also called “classic” hemophilia).
ADVATE is used to prevent and control bleeding in adults and children (0-16 years) with hemophilia A.
Your healthcare provider may give you ADVATE when you have surgery.
ADVATE can reduce the number of bleeding episodes in adults and children (0-16 years) when used regularly (prophylaxis).
ADVATE is not used to treat von Willebrand disease.
SELECTED IMPORTANT RISK INFORMATION
You should not use ADVATE if you:
• Are allergic to mice or hamsters.
• Are allergic to any ingredients in ADVATE.
Tell your healthcare provider if you are pregnant or breastfeeding because ADVATE may not be right for you.
Extensively Studied
ADVATE has been extensively studied, with results spanning over a decade.1-6
In one study it was shown that:
• 42% of patients experienced zero bleeds during one year on prophylaxis7
• prophylaxis with ADVATE improved physical health-related quality of life compared with on-demand treatment7*
*Clinically meaningful changes were not seen in the physical health-related sub-categories of General Health and Physical Functioning, and the mental health-related component score and sub-categories of Mental Health, Role Emotional, Social Functioning, and Vitality.
We’re proud that only ADVATE has 13 years of real-world experience as a third-generation plasma/albumin-free recombinant factor VIII and it has physical health-related quality-of-life results in the prescribing information for people with hemophilia A.7,8
SELECTED IMPORTANT RISK INFORMATION
Your body may form inhibitors to factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop ADVATE from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to factor VIII.
Side effects that have been reported with ADVATE include: cough, headache, joint swelling/aching, sore throat, fever, itching, unusual taste, dizziness, hematoma, abdominal pain, hot flashes, swelling of legs, diarrhea, chills, runny nose/congestion, nausea/vomiting, sweating, and rash.
Tell your healthcare provider about any side effects that bother you or do not go away or if your bleeding does not stop after taking ADVATE.
ADVATE [Antihemophilic Factor (Recombinant)] Important Information
Indications
ADVATE is a medicine used to replace clotting factor (factor VIII or antihemophilic factor) that is missing in people with hemophilia A (also called “classic” hemophilia).
ADVATE is used to prevent and control bleeding in adults and children (0-16 years) with hemophilia A.
Your healthcare provider may give you ADVATE when you have surgery.
ADVATE can reduce the number of bleeding episodes in adults and children (0-16 years) when used regularly (prophylaxis).
ADVATE is not used to treat von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
You should not use ADVATE if you:
• Are allergic to mice or hamsters.
• Are allergic to any ingredients in ADVATE.
Tell your healthcare provider if you are pregnant or breastfeeding because ADVATE may not be right for you.
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 mice or hamsters.
• Have been told that you have inhibitors to factor VIII (because ADVATE may not
work for you).
Your body may form inhibitors to factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop ADVATE from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to factor VIII.
You can have an allergic reaction to ADVATE.
Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting.
Side effects that have been reported with ADVATE include: cough, headache, joint swelling/aching, sore throat, fever, itching, dizziness, hematoma, abdominal pain, hot flashes, swelling of legs, diarrhea, chills, runny nose/congestion, nausea/vomiting, sweating, and rash. Tell your healthcare provider about any side effects that bother you or do not go away or if your bleeding does not stop after taking ADVATE.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
1. Valentino LA, Mamonov V, Hellmann A, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on–demand and prophylaxis treatments in hemophilia A management [published correction appears in J Thromb Haemost. 2012;10(6):1204]. J Thromb Haemost. 2012;10(3):359–367.
2. Shapiro AD, Schoenig-Diesing C, Silvati-Fidell L, Wong WY, Romanov V. Integrated analysis of safety data from 12 clinical interventional studies of plasmaand albumin-free recombinant factor VIII (rAHF-PFM) in haemophilia A. Haemophilia. 2015;21(6):791–798.
3. Tarantino MD, Collins PW, Hay CR, et al, and the RAHF–PFM Clinical Study Group. Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin–free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A. Haemophilia. 2004;10(5):428–437.
4. Négrier C, Shapiro A, Berntorp E, et al. Surgical evaluation of a recombinant factor VIII prepared using a plasma/albumin–free method: efficacy and safety of ADVATE in previously treated patients. Thromb Haemost. 2008;100(2):217–223.
5. Blanchette VS, Shapiro AD, Liesner RJ, et al, for the rAHF–PFM Clinical Study Group. Plasma
and albumin–free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost. 2008;6(8):1319–1326.
6. Auerswald G, Thompson AA, Recht M, et al. Experience of Advate rAHF–PFM in previously untreated patients and minimally treated patients with haemophilia A. Thromb Haemost. 2012;107(6):1072–1082.
7. ADVATE Prescribing Information.
8. Grillberger L, Kreil TR, Nasr S, Reiter M. Emerging trends in plasma-free manufacturing of recombinant protein therapeutics expressed in mammalian cells. Biotechnol J. 2009;4(2):186–201.
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Our own Save One Life had a booth that actively received inquiries on how to sponsor a child with a bleeding disorder in a developing country.
The event ended with a stunning visit to the world famous Field Museum, sponsored by Bioverativ, where families could see the wonders of nature and natural history. My favorite display are “The Ghost and the Darkness,” two man-eating lions from Tsavo, Kenya, which were killed in 1898 after they had killed many workers on the railroad. A Hollywood movie starring Val Kilmer and Michael Douglas tells a somewhat fictional version of the story. It’s a good story but better to see them for real at the museum.
