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United by Blood

Laurie Kelley October 21, 2018

Madonna Smith, executive director of Oregon Chapter, adopted a child with hemophilia into our “family”

Well I’m ready for a rest! Last week I was in Orlando at NHF’s 70^th annual meeting, and last night, at the New England Hemophilia Association’s Red Tie Soiree in Worcester, Massachusetts. And while there is so much happening with new products, with gene therapy coming closer, new programs for our community, and the spectacular movie Bombardier Blood ready for viewing nationwide, the overriding feeling I have after these major events is… this is family. We are truly a family, united by blood.

As I’ve been in the community for over 30 years now, I’ve come to know so many people, and have watched as babies have grown to young adults, starting families of their own, some also with bleeding disorders. Our family grows, and grows older! My friends and I, all young moms and dads when we came into this family, are now in our 60s, looking back at the incredible path this bleeding disorder family has traveled together.

One path literally is the one that led to the movie Bombardier Blood, which debuted last Saturday in Orlando. To a standing room only crowd of over 500 people at 7:15 am, the Octapharma-sponsored event and movie about Chris Bombardier’s epic Everest summit brought laughter and tears to all. It is a stunning film, directed by Patrick James Lynch—whose name you all should know as well as Chris’s by now! These two young men with hemophilia have skyrocketed to fame with their achievement. It makes us, the older moms and dads, incredibly proud. We all kind of feel like they are “our” sons, or like our sons. Sons of our community, doing daring things for our community.

Go Here to See Photos from NHF’s 70th Annual Meeting!

I was honored and fortunate to travel the actual path to Everest base camp with Chris and his wife Jess, and share in part of that historic journey. All the while, I felt like the mother I am, watching out over both of them (as if Chris needed it!). Viewing the movie that morning brought back so many memories and feelings of that journey in May 2017.

So much of the NHF meeting was connecting, with old friends and new, with families in need and families who can share and give. And at the final night we listened to music and danced the night away. While blood unites us as family, music unites us in joy.

I wish everyone could attend NHF’s or HFA’s annual meeting, though I know it’s improbable for most. The meetings are often held in the same spots: Dallas, Chicago,

Laurie Kelley with Derek Nelson, everyone’s favorite

Anaheim, Orlando, San Diego. You’ll never see one in Boston—too expensive. Not everyone can afford to go away by air and stay at hotels. While there are scholarships for first-time families, it sadly still leaves many in our family on the fringe.

Laurie Kelley with Kevin Shaughnessy, who she met when he was 12!

And that’s why we have local chapters and state bleeding disorder organizations. Does your state or region have one? The New England Hemophilia Association has become one of the best run organizations in the US. It wasn’t always that way. As we heard last night, when Charlie Dougherty, who served as treasurer and who passed away earlier this year, found when he joined, it was poorly run and $22,000 in the red. He straightened it up, but it took Rich Pezzillo, a young man with hemophilia, to rocket it into the highest level, and not just by squaring away financials. Rich has breathed new life into NEHA, infusing it with passion and commitment like never before. I confess I never donated to NEHA, even though I’ve lived in New England all my life. But with Rich’s leadership and his crackerjack team, we are all excited to give and participate once again. Last night proved it. I sponsored three tables and invited the team at Save One Life to come and experience the joy of this family reunion, and they had a blast, amazed by the comradery and caring.

In many ways, I find I can enjoy our bleeding disorder family simply by being local. So there’s hope for you too, to join this family by seeking out your local organization. Is it active? Does it have programs? NEHA has programs every month, sometimes every weekend! Does your chapter do a fundraising walk? Have a women’s or teen program? Is there someone to call to learn how to get involved?

I would really urge you to do everything you can to participate. Bleeding disorders can throw a curve ball at you at any time, as life can too. This family, this “Wicked Strong Family” as we say in Boston, is here to help. These friends have become more than friends—they are truly family.

And when I attended the wake last Friday of my first cousin’s husband, who died unexpectedly at only age 61, you realize how family is always family, no matter what. I visited with my cousins, who I rarely see anymore, except on Facebook. We were playmates as children, but life gets busy, the family gets bigger and reunions consist mostly of funerals now. Still, we have history and we are united by blood. It was good to see everyone again; I still love them all.

See Photos from NHF’s 70th Annual Meeting here!

Rising star: Rich Pezzillo with Laurie Kelley

We’ve lost a lot of people in this community this year and as life goes on will continue to lose more. It’s at this time that family comes together: whether nationally, or locally. Find out more about your local chapter; meet your hemophilia/bleeding disorder family. Get involved; give of your time or support. We are all united by blood, and can create the family we need by connecting, caring, sharing and loving.

And a little celebrating and dancing doesn’t hurt either!

Shire: a hematology company with innovation in their blood

admin October 14, 2018

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 Shire website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

If you had hemophilia in the 1940s, your life would have been very different from the one a person with hemophilia lives today. Then, there were no factor replacement therapies, bypassing agents, or prophylaxis regimens. For a person living with hemophilia, there was joint pain, bleeds treated with whole-blood infusions, and life spans that often didn’t reach adulthood.^1-3 Clearly, a new approach to hemophilia treatment was needed. Fortunately, that’s when Baxter’s hemophilia division stepped in, which is now part of Shire, as of 2016.⁴

A revolution begins

It began with an aggressive and passionate effort to provide people with hemophilia a bleed-free life more than 70 years ago.⁵ Then in 1966, one of our scientists, a hemophilia patient himself, had a significant breakthrough. Dr. Murray Thelin helped create one of the first factor VIII replacement therapies and ushered in the modern age of hemophilia treatment.^3,6

Although the first factor VIII replacement therapies were a life-changing improvement for tens of thousands of people with hemophilia, the innovation didn’t stop there.

In the years that followed, we advanced the treatment of hemophilia A, hemophilia B, hemophilia A or B with inhibitors, von Willebrand disease, and acquired hemophilia A with significant developments. Many of these were firsts: the first recombinant factor VIII treatment, the first needleless transfer device, the first recombinant factor VIII treatment free of blood-based additives, the first recombinant treatment for people with von Willebrand disease, and the first recombinant porcine factor VIII for acquired hemophilia.^6-9

The breakthroughs continue

Thanks to the many contributions that have been made in the past, and which Shire continues to make, to the treatment of bleeding disorders, Shire’s vision for patients with a bleeding disorder is closer to realization than ever before.

Today, the focus is on using individualized prophylactic dosing for preventing bleeds.¹⁰ With a recent Shire development, the first FDA-cleared PK dosing software for use in hemophilia, people with hemophilia can conveniently estimate their PK profile and the factor levels in their body at any given time. This may allow patients to tailor their prophylaxis to their activities while ensuring adequate factor coverage.^11-13

And the innovation continues. Research and development is going strong with 20 ongoing clinical trials in bleeding disorders, including one in gene therapy, as well as advancements in other novel therapies. Shire has engaged hundreds of the world’s leading scientists, researchers, and patient support specialists to help them.⁵

Most fundamentally, Shire is collaborating with the bleeding disorders community, including patient associations that have enabled the diagnosis of more than 30,000 hemophilia patients around the world.⁵ Shire has listened to, learned from, and championed their needs. This bleeding disorders community is our community. It’s why Shire is always pushing ahead, proactively shaping the future of bleeding disorders and continually elevating care for patients.

Philipp C. The aging patient with hemophilia: complications, comorbidities, and management issues. Hematology Am Soc Hematol Educ Program. 2010;2010:191-196.
History of bleeding disorders. National Hemophilia Foundation. https://www.hemophilia.org/Bleeding-Disorders/History-of-Bleeding-Disorders. Accessed August 30, 2018.
Dateline Federation: Honoring Our Past, Building Our Future. 41st ed. Washington, DC: Hemophilia Federation of America; 2014. http://www.hemophiliafed.org/dateline/HFA_Dateline_2014_Q2_Summer/HFA_Dateline_2014_Q2_Summer.pdf. Accessed August 30, 2018.
Shire to combine with Baxalta, creating the global leader in rare diseases [news release]. January 11, 2016. https://www.shire.com/-/media/shire/shireglobal/shirecom/pdffiles/newsroom/2016/shire-to-combine-with-baxalta-pr-1-11-16-final.pdf?la=en&hash=A7A87F964B4026EDB959A608A5D57357795DCC44. Accessed September 14, 2018.
Shire’s 70+ year commitment to the hemophilia community [news release]. June 6, 2018. https://www.shire.com/newsroom/2018/january/7sossj. Accessed August 30, 2018.
Kingdon HS, Lundblad RL. An adventure in biotechnology: the development of haemophilia A therapeutics – from whole-blood transfusion to recombinant DNA to gene therapy. Biotechnol Appl Biochem. 2002;35:141-148.
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;4:186-201.
Janbain M, Leissinger C, Kruse-Jarres R. Acquired hemophilia A: emerging treatment options. J Blood Med. 2015;6:143-150.
Shire, Inc. The first and only recombinant treatment for adults affected by von Willebrand disease, launches in the US. [press release]. https://www.shire.com/newsroom/2016/august/nbtjyp. Accessed September 28, 2018.
Berntorp E, Spotts G, Patrone L, Ewenstein BM. Advancing personalized care in hemophilia A: ten years’ experience with an advanced category antihemophilic factor prepared using a plasma/albumin-free method. Biologics. 104:8;115-127.
Collins PW. Personalized prophylaxis. Haemophilia. 2012;18(suppl 4):131-135.
Hazendonk HCAM, van Moort I, Mathôt RAA, et al. Setting the stage for individualized therapy in hemophilia: what role can pharmacokinetics play? Blood Rev. 2018;32(4):265-271.
US Food & Drug Administration. Substantially Equivalent 510(k) Device Information: BK170028. https://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/SubstantiallyEquivalent510kDeviceInformation/UCM592876.pdf. Accessed September 28, 2018.

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The Importance of Plasma

Laurie Kelley October 7, 2018

“Das Blut ist ein ganz besonderer Saft” (Blood is an absolutely remarkable fluid)–Goethe, in Faust

It’s Plasma Awareness Week! One of my favorite celebrations, because plasma is truly the gift of life. 55% of your blood is made up of plasma, and it is a vital ingredient in many therapeutic products to treat diseases and disorders. Plasma Awareness Week aims to :

Raise global awareness about source plasma collection
Recognize the contributions of plasma donors to saving and improving lives
Increase understanding about lifesaving plasma protein therapies and rare diseases.

It’s so important that we donate blood, or even just plasma, to give companies the needed raw material to make their pharmaceutical therapies. While this week we saw another jump in the evolution of hemophilia treatment with the approval of Hemlibra for non-inhibitor patients, plasma still holds a very important place in our global community. People with hemophilia may need it for IT therapy in combating inhibitors. And the developing world truly needs it as a source of treatment… sometimes it’s the only source.

Read the posters below to learn more, know that people around the world have disorders that absolutely require plasma therapies, and then… donate! Go here to learn more about donating. And visit PPTA to learn more as well!

Wine and hemophilia: what’s the connection?

Laurie Kelley September 30, 2018

“A bottle of wine contains more philosophy than all the books in the world.” Attributed to Louis Pasteur

Was wine ever considered historically as a treatment for hemophilia?

Richard Atwood, our contributing writer from North Carolina, offers this week’s blog about the history of wine as a medicine, by summarizing a book he read.

Salvatore P. Lucia’s 1963 book, A History of Wine as Therapy, purports that wine is perhaps the oldest of all medicines, dating back over 40 centuries of written record. Wine is also used as a menstruum for other therapeutic substances. Wine contains alcohol, aldehydes, ketones, esters, acids, carbon dioxides, nitrogenous compounds, pigments, tannins, sugars, pectins, glycerols, vitamins, and inorganic compounds. As a food, wine provides fluids, calories, minerals, vitamins, and proteins. As a medicine, wine acts an appetite stimulant, stomachic, tonic, tranquilizer, anesthetic, astringent, antiseptic, vasodilator, diaphoretic, diuretic, and antibacterial agent! Whew!

Wine was used in the healing arts in Egypt, India, China, Greece, Rome, Byzantine, Syria, Persia, the Dark Ages, and modern times. The therapeutic use of wine is documented in Egyptian medical papyri, the Bible, the Talmud, the Koran, Hindu vedas, Chinese medical treatises, plus ancient and modern formularies, dispensaries, and pharmacopoeias. Wine is prescribed as a pharmaceutical necessity. Among the prominent physicians promoting wine as therapy are Hippocrates, Socrates, Plato, Aristotle, Celsus, Homer, Pliny, Dioscorides, St. Luke the Evangelist, Galen, Avicenna, Maimonides, Arnald of Villanova, and Ambroise Pare. So maybe there is some philosophy to this!

Research scientists investigated the effects of the polyphenols and tannins found in grapes. These substances are thought to promote the resistance of the capillaries to hemorrhage. This would “confirm the empirical observations of the Spanish court physician who prescribed a diet of grapes and wine for the Prince of Asturias, the first recorded haemophiliac member of the royal family of Spain.” (pp. 197-198). The reference is an undated, unpublished manuscript by A.J. Lorenz.

Richard comments that there is only one reference to hemophilia in the text, but seven references to anemia. The absence of much hematological research on wine is disappointing. The use of wine as a treatment for hemophilia, especially when the patient with hemophilia is Spanish royalty, has seemingly never been replicated.

For the use of wine to treat hemophilia, Lucia omitted the reference to John Cochrane, MD, surgeon in Edinburgh, Scotland, who published in Lancet (ii: 147-150) the 1841 article titled “ On the haemorrhagic diathesis.” Cochrane discounted the effectiveness of the internal use port wine as an effective treatment of hemophilia because the wine increases the general force for circulation, causing the congested vessels to rupture. Ouch.

Maybe wine is best left to the parents or caregivers, to relax them from the aftermath of a hospital visit or surgery in their loved one with hemophilia?

Cheers!

Factor replacement–treatment you can rely on

admin September 23, 2018

I travel to many countries where factor is not available to patients with hemophilia and it’s heartbreaking to see the damage bleeding can do. We are fortunate in this country to have factor replacement therapy. Read below for a quick summary of its benefits, and why it’s so important for patients.

When it comes to living with your hemophilia, having confidence in your treatment is important. For more than 50 years, factor replacement has been the standard of care in the treatment of hemophilia A and B with proven efficacy in preventing bleeds through prophylaxis and low risk of thrombotic events.^1-4

Factor-based therapy replaces what’s missing in the coagulation cascade for people with hemophilia, thus working within the body’s natural hemostatic process.^3,5

Factor replacement represents a comprehensive approach to treating hemophilia. It can be used in multiple scenarios to meet different treatment needs that vary from person to person such as prophylaxis (routine infusion of factor to prevent bleeds), on-demand use (infusion of factor to control bleeding episodes), and during or after surgery.^3,6

A one-size-fits-all treatment approach does not meet the unique needs of each individual.⁷Your healthcare provider can individualize your regimen by adjusting your infusion dose and frequency (how often factor is infused) based on:

How your body uses factor
Your activity level
Your lifestyle needs

Individualizing your prophylactic treatment can help reduce bleed rates in some patients with hemophilia A.⁸

Reducing bleeds is necessary to preserve your joints. Even a single bleed matters, and there is evidence that the number of joint bleeds a patient experiences can lead to the development of joint disease.⁹Remember, if bleeds are not treated immediately, this can result in permanent joint damage.²

Decades of clinical studies and real-world use have shown that early prophylaxis with factor-based treatment can reduce the risk of joint bleeds and preserve joint health in some patients with hemophilia A.^2,10-15

To effectively manage your hemophilia, be sure to ask your healthcare provider about an established treatment that offers options for individualization. With decades of documented success in managing, controlling, and reducing bleeds in more than 170 clinical studies, factor treatment remains a trusted and well-studied treatment option in patients with hemophilia.^{1, 15, 16}

Commitment to the hemophilia community is always a priority to Shire. As a leader in hemophilia research, Shire continues to innovate on your behalf, developing programs and services that support your efforts each step of the way. Shire is focused on providing advanced hematology treatments for today and innovating for the future. ¹⁷

Talk to your healthcare provider to learn more about how factor works and all the ways it can help patients like you with hemophilia. Visit www.bleedingdisorders.com to discover if factor treatment is right for you.

References

Center for Biologics Evaluation and Research. User fee billable biologic products and potencies approved under Section 351 of the PHS Act. http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/ucm122936.htm. Accessed July 17, 2018.
Price VE, Hawes SA, Chan AKC. A practical approach to hemophilia care in children. Paediatri Child Health. 2007;12(5):381-383.
Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388:187-197.
Coppola A, Franchini M, Makris M, Santagostino E, Di Minno G, Mannucci PM. Thrombotic adverse events to coagulation factor concentrates for treatment of patients with haemophilia and von Willebrand disease: a systematic review of prospective studies. 2012;18:e173-e187.
Berg JM, Tymoczko JL, Stryer L. Many enzymes are activated by specific proteolytic cleavage. In: Biochemistry. 5th ed. New York, NY: WH Freeman; 2002. https://www.ncbi.nlm.nih.gov/books/NBK22589/?rendertype. Accessed July 24, 2018.
World Federation of Hemophilia. Guidelines for the management of hemophilia. 2nd ed. Montreal, Quebec: World Federation of Hemophilia; 2012:1-80.
Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia. 2014;20:607-615.
Yamasaki N, Fujii T, Fujii T. Individualized prophylaxis can contribute to decreasing annualized bleeding rate (ABR) in adult persons with hemophilia A. 2018;24(suppl 5):1-194.
Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009;7:413-420.
Bertamino M, Riccardi F, Banov L, et al. Hemophilia care in the pediatric age. J Clin Med. 2017;6(54):1-13.
Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535-544.
Manco-Johnson MJ, Soucie JM, Gill JC for the Joint Outcomes Committee of the Universal Data Collection, US Hemophilia Treatment Center Network. Prophylaxis usage, bleeding rates,and joint outcomes of hemophilia, 1999 to 2010: a surveillance project. 2017;129(17):2368-2374.
Khawaji M, Astermark J, Von Mackensen S, Akesson K, Berntorp E. Bone density and heath-reated quality of life in adut patients with severe hemophilia. Haemophilia. 2011;17:304-311.
Rodriguez-Merchan EC. Aspects of current management: orthopaedic surgery in haemophilia. 2012;18:8-16.
Franchini M, Mannucci PM. The history of hemophilia. Semin Thromb Hemost. 2014;40:571-
NIH Clinical Trials Registry. Ongoing and complete clinical trials using factor in patients with hemophilia. https://clinicaltrials.gov/ct2/results/details?term=Factor+VIII&recr=Closed&fund=2. Accessed July 17, 2018.
Shire website. https://www.shire.com/who-we-are/areas-of-focus/hematology

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