Explore the possibilities of individualization

When I was raising a child with hemophilia over 20 years ago, all factor dosing was based on our child’s weight and severity of an injury. No one ever talked about PK. What is PK, you ask? Read below and find out! It’s essential to know if you want to individualize the dosing schedule for you or your loved one with hemophilia!

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.

We know that regardless of any similarities, no two individuals with hemophilia are the same. For example, you and another person with hemophilia who are the same age and weight may require different amounts of factor, depending on how your body uses factor.1

One of the key elements in individualizing factor treatment is understanding and utilizing pharmacokinetics (PK). This is the study of how your body uses the medicine you take,2 which in turn helps predict the factor coverage that is available within your body. PK helps predict how the treatment is working with your body. Remember, no two individuals are the same, so factor is used or removed from the blood at different rates depending on your individual PK profile.3

Your PK profile is developed during PK analysis, where your healthcare provider (HCP) draws your blood at very specific times to determine your factor levels.4,5 PK analysis helps your HCP identify and understand4,6,7:

  • Highest level of factor in your body after infusion (peak)
  • Lowest factor level after time has passed (trough)
  • Amount of time it takes for half of the infused factor to be removed from the bloodstream, known as the half-life

This PK information can help your HCP determine an optimal treatment plan for you by adjusting your infusion dose and frequency (how often factor is infused), based on how your body uses factor.1 Your HCP will also take a close look at your lifestyle and other activities, as these can have an impact on your bleeding risk. As you can see, there are many different characteristics to consider when determining the right treatment plan for you.

Individualizing prophylaxis (routine infusion of factor to prevent bleeds) may help improve the likelihood of zero bleeds while also helping to preserve joint health.8 Your joint health is very important; every joint bleed matters. It is recommended to start prophylaxis early, especially in childhood or adolescence, to help preserve your joints.9

We at Shire understand that individualized factor treatment is key. It is necessary to tailor your factor treatment to meet your unique needs. When it comes to treating hemophilia, there is no one-size-fits-all approach.1

Talk to your healthcare provider about factor treatment— an option that lets you individualize your prophylaxis regimen to meet your unique needs. Visit www.bleedingdisorders.com to learn more about factor treatment.

References

  1. Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia. 2014;20:607-615.
  2. Le J. Overview of pharmacokinetics. Merck Manual. https://www.merckmanuals.com/professional/clinical-pharmacology/pharmacokinetics/overview-of-pharmacokinetics. Accessed July 17, 2018.
  3. Collins PW, Björkman S, Fischer K, et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost. 2010;8(2):269-275.
  4. Poon MC, Jackson S, Brown M, McClure W. Clotting factor therapy. All About Hemophilia: A Guide for Families. Montreal, Quebec, Canada: Canadian Hemophilia Society; 2010:1-33.
  5. Lee M, Morfini M, Schulman S, Ingerslev J; and Factor VIII/Factor IX Scientific and Standardization Committee on the International Society on Thrombosis and Haemostasis. The design and analysis of pharmacokinetic studies of coagulation factors. International Society on Thrombosis and Haemostasis. https://c.ymcdn.com/sites/www.isth.org/resource/group/d4a6f49a-f4ec-450f-9e0f-7be9f0c2ab2e/official_communications/fviiipharmaco.pdf. Published March 21, 2001. Accessed June 1, 2018.
  6. University of Virginia Health System. Obtaining trough blood levels. https://med.virginia.edu/neurology/wp-content/uploads/sites/235/2015/11/obtaining-trough-blood-levels.pdf. Accessed July 27, 2018.
  7. Medical Dictionary. Definition of Cmax. Farlex Inc: Huntingdon Valley, PA; 2018. http://medical-dictionary.thefreedictionary.com/Cmax. Accessed July 30, 2018.
  8. Poon M, Lee A. Individualized prophylaxis for optimizing hemophilia care: can we apply this to both developed and developing nations? Thromb J. 2016;14(suppl 1):65-71.
  9. Bertamino M, Riccardi F, Banov L, et al. Hemophilia care in the pediatric age. J Clin Med. 2017;6(54):1-13.

 

©2018 Shire US Inc., Lexington, MA 02421.
All rights reserved. 1‐800‐828‐2088.
SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates. S41614 08/18

Hemophilia Fiction for Young Readers

written by Richard J. Atwood

Originally published in August PEN

Learning to read is a significant milestone in our personal development. As adults, we nudge young readers along in this quest. First we read to them, and later we encourage and supervise for age- and skill-appropriate reading materials.

Our bleeding disorder community is fortunate to include notable authors who write about hemophilia. Their books for young readers are educational and entertaining. Look for short reviews of these books in PEN’s Biennial Bleeding Disorder Resource Guide.

Other authors who write for younger readers sometimes include hemophilia. I suggest the following books.

Main Characters with Hemophilia

Starring Peter and Leigh (Delacorte, 1979); The Friendship Pact (Scholastic, 1986)

Susan Beth Pfeffer

What could be better than a fictional leading character with hemophilia? Peter Sanders, a 17-year-old with hemophilia on Long Island, becomes stepbrother to 16-year-old Leigh Thorpe. Leigh, a former child actor, wants to be a normal teen, while Peter, often bedridden, wants to attend school. Pfeffer also included hemophilia in her pre-teen novel The Friendship Pact. Tracy Newfield, age 12, has a crush on a young actor, Ross Perlman. Ross is a Dartmouth graduate who gives a benefit concert for National Hemophilia Foundation and dedicates his unreleased song to Tracy. Hemophilia in these novels seems dated, yet the descriptions are accurate for the time period.

 

Panda Bear Is Critical (Macmillan, 1981); Picture Perfect (Severn House, 2000)

Fern Michaels

Michaels wrote Panda Bear, a suspense novel, and later retitled it Picture Perfect after making some changes. Five-year-old Davey Taylor stays with his aunt and uncle while his scientist parents travel to Florida to testify in a mob-related drug trial. Davey has hemophilia, for which he receives daily “antigen shots.” During a camping trip, Davey is kidnapped without his hemophilia treatment. He must cunningly escape, aided by his Yorkshire terrier and his CB radio, so he can be treated before it’s too late.

 

She Died Too Young (Bantam, 1994); All the Days of Her Life (Bantam, 1994); A Season for Goodbye (Bantam, 1995)

Lurlene McDaniel

This series of three inspirational novels deals with life-altering situations for children and young adults, ages 10 and up. In the series, teens with various medical conditions attend summer camp at Jenny House in Asheville, North Carolina. One camper is Jeff McKensie, who has hemophilia. Jeff moves from Colorado to Miami to study to be an architect, yet his romantic opportunities at camp are stymied due to his medical condition.

 

Diving for the Moon (Macmillan, 1995)

Lee F. Bantle

Bantle, a lawyer, tackles issues of puppy love, adolescence, hemophilia, and HIV in this novel for ages 9–12. Josh Charkey, age 12, has hemophilia and AIDS. Spending the summer at a Minnesota lake, Josh shocks his close friend Carolina Birdsong with his medical revelations, while still fostering their budding romance.

 

Uphill and Into the Wind (Royal Fireworks, 1996)

Willard Helmuth

In this young adult novel, 12-year-old Eric Kenton, who has hemophilia, wants to be normal. While his parents worry about hemophilia and HIV, Eric dreams of, then successfully accomplishes, riding his fat-tired Huffy bicycle almost 400 miles to a summer camp in Ohio.

 

Kinetic (DC Comics, 2005)

Kelley Puckett and Warren Pleece

Puckett and Pleece wrote and illustrated this storybook comic fantasy. Tom Morrell is a high school senior who has hemophilia, plus about a dozen other syndromes. Tom gains superpowers when he is hit by a truck, and the absorbed energy amazingly “cures” his medical conditions.

 

The Healing of Ryne O’Casey: A Novel (FaithWalk, 2004)

Scott Philip Stewart

Ryne O’Casey, a 10-year-old from Tynbee, Tennessee, is first diagnosed with “hemophilia type A” when just over a year old, and then diagnosed with HIV at age 8. Some of the medical details are questionable for 1995, but the social reactions are truly believable.

The Lorin Solo (self-published, 2006)

Chuck Edwards

This young adult romance novel follows Lorin Lenki, a music student at San Francisco State who has mild hemophilia. Lorin meets Tracy Martin, a nursing student at Saint Rita’s Hospital. Tracy has severe hemophilia, as did her identical twin sister who died. The budding romance tragically ends when Tracy dies from a brain bleed in 1986.

 

Jovenes vampiros. El codice secreto (versos y trazos, 2007)

Jose Aguilar

This illustrated novel, written in Spanish, tells the tragic love story of young Marco Tulio and Isabel, who has hemophilia. The setting is ancient Italy, and includes vampires and Leonardo da Vinci.

 

Supporting Characters with Hemophilia

 

Something Different in the Bank (E. J. Arnold and Son, 1989)

Grace Moulton

Something Different in the Bank is one of a series of books about children with medical conditions. The narrator describes his cousin Michael, who has hemophilia. The treatment of hemophilia seems out-of-date even for 1989, though some of the behavioral concerns are timeless.

 

Monkey Island (Bantam, 1991)

Paula Fox

This teen novel’s awards include an American Library Association Best Book for Young Adults, and a Horn Book Fanfare Selection. Clay Garrity, age 11, is homeless in New York City. When Clay is hospitalized for pneumonia, his roommate, an experienced hospital patient with hemophilia, provides sage advice.

 

Play to the Angel (Scholastic, 2000)

Maureen F. Dahlberg

Greta, age 12, has an older brother named Kurt who dies due to his hemophilia. Set in 1938 in Vienna, this motivational teen novel with historical detail involves children from a musically gifted family who fulfill their dreams, even using the death of a sibling with hemophilia for inspiration.

 

Angel of the Square (HarperTrophy, 2001)

This novel for ages 10 and up tells the story of Ekaterina Ivanova, called Katya, the 12-year-old daughter of a lady-in-waiting to Empress Alexandra in Russia before 1918. Katya is a playmate of Anastasia and learns what is wrong with Alexei, who has hemophilia. Although Katya is fictional, her perspective on the Russian Revolution is believable.

 

A Home for Garth (self-published, 2010)

  1. Reed

Jake Stevens, age 11, finds a lost dog that belongs to Robert Higgins, a 10-year-old with hemophilia. Jake’s moral dilemma is deciding whether to lie to keep the lost dog, or to be truthful and return the dog to its owner. Jake learns about hemophilia and how it limits what Robert can do.

 

The Hardy Boys Undercover Brothers, #32 Private Killer (Aladdin, 2010)

Franklin W. Dixon

This novel for ages 8–12 features teens acting as detectives to solve mysteries, and includes a female with hemophilia. Destiny Darity is the troubled daughter of the headmaster at an exclusive private school near Boston. Destiny has a rare blood type that she inherits from her mother and a mild case of hemophilia that she inherits from her father. The Hardy brothers, Frank and Joe, may not completely understand the genetics of bleeding disorders, but they determine who plays pranks on Destiny.

 

Blood Ties: A Blood Coven Vampire Novel (Berkley, 2011)

Mari Mancusi

This young adult novel includes a main character named Sunshine McDonald, a 17-year-old fairy princess living in Las Vegas. She loves Jayden, a mortal with hemophilia. When Jayden is bitten by a vampire, he needs blood transfusions—not for his hemophilia, but for his turning. Sunshine and Jayden travel to London and Tokyo to find a cure for vampirism by drinking from the Holy Grail, and by doing so, cause Jayden’s hemophilia to disappear.

 

I Hunt Killers (Little, Brown, 2012); Game (Little, Brown, 2013); Blood of My Blood (Little, Brown, 2013)

Barry Lyga

This trilogy of young adult mystery/thrillers delves into the world of serial killers, and the vivid carnage may be inappropriate for young readers, or for any readers. Seventeen-year-old Jasper Dent, called Jazz, is the son of an imprisoned serial killer. With the help of his best friend Howie, Jazz helps police identify serial killers. Howie is a “type-A hemophiliac” with an overprotective mother, so his assistance is sometimes limited when extracting Jazz from dangerous situations. Thankfully, Howie provides some comic relief to an otherwise serious topic.

***

Reading should be a life-long endeavor and source of pleasure. Take advantage of these books to help young readers learn something about bleeding disorders. And keep reading good books to your child!

America’s First Family with Hemophilia

New England, 1639. Imagine that you are standing on the deck of the sailing ship Jonathan. You have just glimpsed the shore of your new home, the Massachusetts Bay Colony. Imagine the brilliant New England foliage, the bright chilly wind. Imagine your dream of farming your newly acquired land. Imagine the adventure. Now, imagine that you are the first European with hemophilia to step on the North American shore.appleton-farms.jpg (1024×682)John Oliver (1613–1642) traveled from Bristol, England with his family to settle under the leadership of the Massachusetts Bay Colony. He lived for only three years after he reached North America, fathering one child, Mary, and dying young as a consequence of his hemophilia. Not until after 1800 did the medical community begin using the term hemophilia to describe his disorder. John’s daughter, Mary Oliver (1640–1698), was likely the first hemophilia carrier of European descent born in the colonies. With her husband, Major Samuel Appleton, Jr. (1625–1696), Mary had three daughters and five sons. One of these sons, Oliver Appleton (1677–1759), was the first American colonist born with hemophilia.

Early Ipswich Roots
Mary and Major Appleton lived in a settlement known to native Americans as Agawam, but re-christened by the English in 1633 as the town of Ipswich. What would life in Ipswich have offered their son, Oliver Appleton? Thirty miles north of Boston on the Atlantic shore, Ipswich was owned by the Massachusetts Bay Colony; it was purchased earlier in the century from Native Americans for 20 British pounds. By the mid-1600s, Ipswich ranked second only to Boston in population and wealth. The Appletons were a wealthy colonial family. Major Samuel Appleton, Jr., Oliver’s father, was the son of Samuel Appleton Sr., one of the “landed gentry,” and a good friend of John Winthrop, the first governor of the Massachusetts Bay Colony. Appleton’s fertile 460 acres of farmland had been granted to him by the Colony in 1638, and left to his son, Major Appleton, around 1670.
Major Appleton, who served as a judge at the infamous Salem Witch Trials in 1692, died in 1696. He left his now nearly 600 acres, split into four parcels, to his four sons: Oliver, Isaac, Samuel and John. Oliver’s 100-plus acre inheritance included his father’s sawmill, ox pasture, and farmland bordering his brothers’ parcels.

In 1701, Oliver married Sarah Perkins. Well-to-do millers, farmers and traders, Oliver and Sarah possessed numerous household and farm goods. They were involved in local politics, church affairs and business. Together they raised fourteen children; several sons and their descendants would become fine cabinetmakers. At the turn of the eighteenth century, Oliver and his three brothers were working their adjoining farms in a loosely communal style. Each brother might grow a crop that the other brothers could use. Yet each brother farmed separately, produced his own goods for trade (like basket hoops), and kept his own business ledger. The brothers owned cattle, sheep, turkeys and hogs, and traded goods with family and friends in Ipswich.

A Dangerous Occupation?
On their “new” land (already cleared and cultivated by Native Americans), the Appletons cut and milled timber, raised livestock and worked the farm. Today, farming is still one of the most dangerous occupations. In the seventeenth and eighteenth centuries, its hazards were surely compounded by Oliver’s hemophilia, and the harsh New England winters. Yet Oliver lived to be 82—a considerable age in any century.

Late in life, Oliver was confined to his bed and developed bedsores on his hips. At age 82, his cause of death is recorded as bleeding from his bedsores and his urethra. Oliver appears to have been a generous and fair man, dividing his estate equitably among his children and his wife Sarah.

Making Medical History
Oliver and Sarah had six daughters and eight sons. Two of the daughters, Sarah and Hannah, had sons with hemophilia.

Interestingly, Hannah’s sons, Oliver and Thomas Swaim, were doctors. What would they have thought of their family’s disorder?

Without letters or other documents, we can only guess. Yet it was the Swaim branch of the Appleton family that attracted the attention of the medical community. Based on his personal connection with the Swaim family, Dr. John Hay, a Massachusetts physician, published an article on the Appletons in a New England medical journal in 1813. Following this publication, the Appleton family history appeared in numerous medical journals, at least as late as 1962. By then, the family had been traced through 350 years and 11 generations: 25 males with hemophilia, and 27 carrier females. In 1961 a blood sample, drawn from the last known living carrier in the family tree, revealed factor VIII deficiency, or hemophilia A.

Are the Appletons America’s “First Family” with hemophilia? Perhaps, in the sense that our knowledge of hemophilia has been enriched by the study of this large and long-lived colonial family. Thanks to our American Revolution, we have no “royal family” with hemophilia. Yet we can still honor and remember the Appleton family. This Thanksgiving, we can recall the challenges faced by earlier generations with hemophilia—people who contributed to our heritage as Americans, and as a hemophilia community. To understand ourselves, and create our vision for the future, we must always remember the past.

You can visit Appleton Farms in Ipswich, Massachusetts. 

From the November 2002 Issue Parent Empowerment Newsletter
“THE APPLETONS: America’s ‘First Family’ With Hemophilia”
by Richard J. Atwood and Sara P. Evangelos
© 2002 LA Kelley Communications, Inc.

United by Blood

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 70th 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.

With the California gang!

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

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.

  1. Philipp C. The aging patient with hemophilia: complications, comorbidities, and management issues. Hematology Am Soc Hematol Educ Program. 2010;2010:191-196. 
  2. History of bleeding disorders. National Hemophilia Foundation. https://www.hemophilia.org/Bleeding-Disorders/History-of-Bleeding-Disorders. Accessed August 30, 2018.
  3. 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.
  4. 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.
  5. 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.
  6. 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.
  7. 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.
  8. Janbain M, Leissinger C, Kruse-Jarres R. Acquired hemophilia A: emerging treatment options. J Blood Med. 2015;6:143-150.
  9. 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.
  10. 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.
  11. Collins PW. Personalized prophylaxis. Haemophilia. 2012;18(suppl 4):131-135.
  12. 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.
  13. 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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