Exploring New Worlds When You Have Hemophilia: John Oliver

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By Richard Atwood

Have you ever thought of traveling overseas?

A benefit of current treatment of hemophilia is the freedom to safely travel. This was not always possible before the use of factor concentrates. Now with just a few precautions, such as carrying a medical ID, a letter from your hematologist, and a travel case of factor concentrate with necessary supplies, along with your passport and a credit card, you can easily travel to many destinations in the world.

Suppose you wanted to travel across the Atlantic Ocean from England to America. Today you could take a five-hour flight from London to Boston. Now compare that to the voyage of John Oliver (1613-1641), the first recognized individual with hemophilia to travel across the ocean almost 400 years ago. Newly married in 1639, Oliver left England to settle in the Colonies.

John Oliver born in Bristol. The eldest child of James Oliver (1586-1629) and Frances Cary (1590-1635), Oliver had two sisters and two brothers who lived to be adults, and possibly three sisters who died as children. It’s unknown whether a family history of hemophilia existed. At 16, Oliver was apprenticed for eight years to his uncle, Walter Stephens of Bristol, a dealer in textile fabrics. Oliver belonged to a multigenerational extended family of seemingly prosperous merchants.

By 1639, Oliver finished his apprenticeship and received his inheritance after both parents died. His 22-year-old brother James died that year, allowing the speculation that he had hemophilia. Oliver received his brother’s inheritance of £50. After marrying Joanna Lowle (later spelled Lowell), Oliver, at age 26, packed their belongings. Then accompanied by his in-laws, the couple probably traveled by wagon to London, where everyone boarded the Jonathan.

The Jonathan was about 100 feet in length with a capacity of 200 to 300 tons. There were two or three small cabins for important passengers plus a hold for 100 passengers (with maybe room for cattle on the deck). There were over 50 individuals who were aboard. A regular fare was £5 (around $4 in today’s market) and a cabin cost £17, plus £8 for one group’s luggage, and even more for food.

Sailing under Master John Whetstone, the Jonathan departed London on April 12, stopped briefly in Southampton, and arrived in Boston Harbor on June 23. A voyage across the ocean at that time was expected to take six weeks. This trip lasted over nine weeks, indicating bad weather or other problems. Some of the passengers died on board from childbirth complications, infant illnesses, and possibly infectious diseases. Sanitary conditions were most likely impossible to maintain, and food supplies were limited.

John Oliver traveled in a party of 17 led by his father-in-law Percival Lowle (1571-1665), an importer and merchant in Bristol. Lowle was accompanied by his wife, his two married sons and their families, his two married daughters and their families, plus business associates and apprentices. This was the beginning of the prominent Lowell pedigree in New England. (The city of Lowell is named after them, and is home to University of Massachusetts Lowell.)

Soon after arriving in Massachusetts Bay Colony, Oliver and his wife settled in Newbury, about 35 miles north of Boston. The couple bought house lots and arable lands, meadows, marsh, orchard, fences, privileges, and commons from Mr. Stephen Bachelor and Christopher Hussey for six score pounds. Oliver was a merchant of imported goods from England, probably working from his home. In 1640, he was admitted as a Freeman and his daughter, Mary, was born. The next year he was chosen to serve on the General Court appointed commissioners for small causes in Newbury.

Oliver must have died in January 1642, probably due to complications of his hemophilia, leaving an estate worth £420 in lands and goods. In 1645, his widow married Captain William Gerrish, another merchant who traveled in the Lowle party. Later in 1656, 16-year-old Mary Oliver, a hemophilia carrier, married Major Samuel Appleton Jr. from that prominent New England pedigree in Ipswich. Their marriage started the extensive Appleton-Swain pedigree of hemophilia A in Massachusetts—the first known family with hemophilia in the Americas.

Today we are often critical of air travel. High cost, lack of leg and elbow room, inadequate bin size, rude passengers, extra charges, security checks, long delays. Consider what John Oliver had to endure during those nine weeks he was aboard a ship to appreciate how we travel today in airplanes! Yet like Oliver, we also have to pay extra for luggage and food, so some complaints about traveling seem to persist forever. But don’t let that hinder your travels abroad!

A Personal Approach to Healthcare

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Ever hear of personalized healthcare (PHC)? PHC means tailoring a treatment regimen specific to an individual patient. It acknowledges that every patient is different, with different physiology, biology, reactions to medicine, and lifestyle. A treatment regimen that works for one patient may not work for another with the exact same ailment.

The goal of PHC is to improve your quality of life as a patient with a specific disease or disorder, by taking into
account your individual needs and lifestyle, and then tailoring a treatment plan. How does that happen? It’s a collaborative process involving you and your medical team. Your HTC medical team knows all the treatment options, but you know yourself, your body, and your lifestyle. To develop your own PHC, you’ll need to share with the team every aspect of your past health, current health, needs, and desires. Only then can you all explore treatment options that best suit you.

Your Personalized Healthcare Team

The best place to get help with hemophilia and all bleeding disorders is at a hemophilia treatment center (HTC). These are centers of excellence that specialize in diagnosing, treating, and monitoring bleeding disorders. There are over 140 HTCs throughout the US. They follow the model of comprehensive healthcare, which means that they’re not just treating bleeding episodes, but all aspects of living with a bleeding disorder. From orthopedics to psychosocial needs to genetics, the HTC team knows how a bleeding disorder can impact your life.

By using comprehensive healthcare, isn’t your HTC team already implementing PHC?

Maybe not. First, not all HTCs are created equal. Some may lack certain experts you need, such as a pediatric hematology department or a geneticist. And some may have strong beliefs, different from yours, about when—or whether—to start prophylaxis (prophy), or about whether you should try new products.

Parents and patients need to become partners in PHC. Are they ready for this collaboration? Parents of newly
diagnosed children may be too shocked at first, and not even know what questions to ask. Older patients may be overlooked for PHC because they’ve been on the same treatment plan for a long time, their blood work is good, and they don’t complain or ask questions.

It’s important for you to be ready to partner with your HTC and let the team know your needs. But even if you’re comfortable standing up and being heard, what will you say? What will you ask?

1. CHOOSE THE RIGHT TREATMENT

One of the first decisions you need to make about your PHC is which treatment to use. All products licensed in the US are considered safe and effective, but they’re not all the same. How do you find one that’s best for you?

You know that new products are entering the market. You can choose between plasma-derived (made from human blood) and recombinant (made from animal cells containing human genes). Within the recombinant products, there are categories: first, second, or third generation. And there are novel therapies, that are not even factor! So begin by asking your hematologist for opinions on all products.

Your decision about treatment may come down to lifestyle, when selecting a particular brand for prophy. Perhaps the brand you’re using now, three times a week, works well for you or your child. Does a product with an extended half-life offer fewer infusions, saving veins from wear and tear? Or would you benefit from a new, subcutaneous product? Some products might not be available through your insurance. Talk through these choices with your HTC.

2. BLEEDING PATTERNS

Perhaps nothing is more personal than your individual bleeding pattern. People with hemophilia bleed differently, in different places, from different causes. Once your child begins getting bleeds, notice his bleeding pattern, if any. This is where you can really help your HTC team personalize your treatment. The information you share can help prevent a target joint from starting, or can compel the HTC team to put your child on prophy.

You may hear severity levels described like this: Children with severe hemophilia will bleed from trauma, or spontaneously, with no known trauma. Children with moderate hemophilia may bleed on average once a month, with known trauma. Children with mild hemophilia may bleed only after dental extractions and surgery. But what’s the reality? Some children with severe hemophilia bleed only monthly; some with moderate bleed every week, with no known trauma. Your child’s bleeding is unique!

And know his personal symptoms of a bleed: Tingling? Hot? Swollen? Your HTC staff can help you identify symptoms, so you can infuse more quickly at the first sign of a bleed.

3. ESTABLISH A TREATMENT REGIMEN

Personalized healthcare really shines when it’s time to devise a treatment plan. You can use the new, subcutaneous novel therapy. Or you have two options for infusing factor: on-demand (also called episodic) or prophylaxis. On-demand means infusing at the first sign of a bleed. Prophy is the scheduled infusing of factor. It’s designed to keep factor levels in the bloodstream high enough—greater than 1%—to prevent most spontaneous bleeds.

Prophy is the recommended therapy for children with hemophilia in countries like the US with ready access to clotting factor.

And what about your schedule for prophy? This is about as personal as PHC gets! Your HTC team will offer a schedule based on your child’s needs: your family lifestyle, activity level, perhaps pharmacokinetics (PK) data to determine how quickly factor is cleared from the blood after an infusion, and any breakthrough bleeding that might happen.

4. DISCUSS PERSONALIZED MEDICINE

Personalized healthcare is not the same as personalized medicine, a common term. Personalized medicine often refers to using a person’s genetic profile—genomes and specific genetic markers—to guide therapy for cancer and other diseases and disorders, including hemophilia. But PHC includes personalized medicine, and it’s worth discussing with your HTC team because more and more, the hemophilia community is focusing on personalized medicine.

National Hemophilia Foundation (NHF) recognized the importance of this genetic research and launched My Life, Our Future (MLOF). Through this program, you can get a blood test that enables you to learn more about the specific genetic mutation that caused your child’s hemophilia.

So PHC uses personalized medicine to examine your genetic makeup, help predict which medical disorders or diseases your child is most at risk for, and suggest which treatments will be safe and effective (or not) for him. This is particularly important for complications like the risk of getting inhibitors.

We’ve shown you examples of treatment and lifestyle areas to focus on, potential needs to address, and questions to ask your HTC team to design the best life possible with your bleeding disorder. Your HTC team will become one of your most important partners.

How Takeda’s Hematology Support Center can help

This week’s blog provides a website with helpful insurance resources for you to consider. Please read below and click the links to learn more.
This is a paid public announcement from Takeda 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 a Takeda website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.
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If you live with or care for someone with a bleeding disorder, you know how important it is to have helpful resources. It takes teamwork to manage this type of condition.

At Takeda, we understand this. That’s why we created the Hematology Support Center (HSC). Takeda’s HSC offers dedicated help to patients prescribed Takeda hematology products and provides them with information, guidance, and treatment resources. HSC is here for patients and caregivers. We can help with insurance hurdles, copay assistance, and getting access to treatment. HSC can also assist healthcare providers with reimbursement and access issues.

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HSC provides patients and caregivers with dedicated support.

Case Managers are the first point of contact. They can:

  • Give information about financial assistance options
  • Help eligible patients enroll in our CoPay Assistance Program
  • Connect patients to other HSC team members

Reimbursement and Access Managers (RAMs) are insurance experts. They can:

  • Assist with health insurance challenges
  • Provide insurance education tools

Healthcare Educators (HCEs) offer useful information. They can:

  • Provide English or Spanish one-on-one health education support about hemophilia and von Willebrand disease
  • Give information about a patient’s prescribed Takeda treatment
  • Connect patients with informational and community resources, including Takeda’s educational programs
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HSC is committed to providing support for those who need it.

HSC support comes in more ways than one. For example, Case Managers can help patients know if they qualify for our CoPay Assistance Program. This program may cover up to 100% of eligible out-of-pocket expenses, up to the program maximum. Only patients who have been prescribed a Takeda hematology product* and have commercial insurance are eligible for the program.

HSC support continues online.

HSC’s website for patients, hematologysupport.com, can provide additional help. For example, the patient resource library has information about:

  • Insurance
  • Medicare and Medicaid
  • Staying organized while on treatment
  • Other support resources

Living with a bleeding disorder can feel overwhelming at times. But with a Takeda hematology treatment, HSC is here to help. And that can make all the difference.

HSC is here for healthcare providers too.

For healthcare providers prescribing Takeda hematology treatments, HSC can make the process go more smoothly. Need help with an access challenge? We may know how to resolve it. Our website for healthcare providers, hematologysupportpro.com, has useful tools, such as:

  • Prescription referral forms
  • Prior authorization and appeals checklists
  • Billing and coding information

HSC team members are trained to handle all types of support situations. Make HSC the go-to resource for any Takeda hematology access–related questions or concerns.

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Support from HSC is only a call away.

With HSC, patients, caregivers, and healthcare providers have a partner on their side. HSC is here and ready to help. Call us Monday through Friday, 8:30 am to 8:00 pm ET, at 1-888-229-8379.

To learn more about HSC, please visit our website:
For patients: hematologysupport.com
For healthcare providers: hematologysupportpro.com

*Must meet specific program eligibility requirements and terms and conditions. Certain restrictions apply. Not all Takeda hematology products are available for each program.
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Copyright © 2019 Takeda Pharmaceutical Company Limited. All rights reserved. 1-800-828-2088.
The Hematology Support Center logo is a trademark of Shire, a Takeda company.
TAKEDA and the TAKEDA logo are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited.

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Factor your way: empowering you to achieve your goals

Sponsored Content

This is a paid public announcement from Takeda 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 bleedingdisorders.com website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

Submitted by Takeda

What is hemophilia?

Hemophilia is a rare genetic bleeding disorder that prevents blood from clotting normally.1

At the time of a bleed, proteins in your blood called clotting factors form a clot and stop the bleeding.2,3 However, if you have hemophilia, your blood lacks clotting factors (such as factors VIII and IX), and as a result, you may bleed for a longer time after an injury than you would otherwise.4,5

Replacing what’s missing

The current standard of care for hemophilia is factor replacement therapy.6 Factor therapy is a proven treatment with decades of real-world use and an established safety record.7,8

Factor therapy replaces the missing blood-clotting proteins that are naturally found in your blood.9 It is administered via an intravenous injection (directly into your bloodstream), making these proteins available immediately for use.

Factor therapy can be used in different situations such as on-demand (to treat an ongoing bleed), prophylaxis (to prevent bleeds before they occur), and before or after surgery.6,10

Other treatments for hemophilia

The first non-factor therapy option more recently became available.11 To reduce bleeding, it mimics part of the function of missing clotting proteins by bridging other factors. It is administered subcutaneously and is intended for prophylactic use.

Individualizing your treatment with factor

With factor treatment, your healthcare provider (HCP) can individualize your regimen to align with your unique needs and help you achieve treatment goals.

When determining an appropriate treatment plan, your HCP looks at how factor is processed in your body; this is called pharmacokinetics (PK).13 Many factors such as body weight, age, joint status, activity levels, and bleeding frequency are used to better understand how much and how often you require factor.12

A treatment tailored to your lifestyle and activities may help make it easier for you to stick to your prophylaxis. This may help preserve your joint health by significantly reducing bleeding.12

CASE STUDY

Individualizing factor therapy can make a world of difference

Josh
Age 15 years

Josh is an active teen who enjoys playing sports. He is currently taking factor every other day (25 IU/kg) but has recently been experiencing more bleeds, particularly in his ankles. At his HCP visit, Josh mentions that he has increased his tennis practice from once weekly to 3 nights/week.

Due to an increase in Josh’s activity level, and a considerable growth spurt, he may not always be receiving optimal coverage from his current dosing regimen. His HCP recommended adjusting Josh’s dose of factor VIII and changing his infusion schedule.

After taking a few blood samples, they were able to determine what Josh’s factor levels would be at various time points following an infusion. They agreed to an individualized dosing schedule that was appropriate for Josh’s current activity level. Josh’s new dose is 35 IU/kg three times a week.

Since switching to the new dosing schedule, Josh’s bleeding has been reduced even with his increased activity.

 

 

 

 

 

 

 

 

 

Visit bleedingdisorders.com for more information about hemophilia and individualized factor therapy.

Supporting literature:

  1. Livnat T, Barg AA, Levy-Mendelovich S, Kenet G. Rare bleeding disorders—old diseases in the era of novel options for therapy. Blood Cells Mol Dis. 2017;67:63-68.
  2. Smith SA, Travers RJ, Morrissey JH. How it all starts: initiation of the clotting cascade. Crit Rev Biochem Mol Biol. 2015;50(4):326-336.
  3. Berg JM, Tymoczko JL, Stryer L. Many enzymes are activated by specific proteolytic cleavage. Biochemistry. 5th ed. New York, NY: WH Freeman; 2002. https://www.ncbi.nlm.nih.gov/books/NBK22589/. Accessed April 18, 2019.
  4. Hemophilia. National Heart, Lung, and Blood Institute (NHLBI). https://www.nhlbi.nih.gov/health-topics/hemophilia. Accessed April 17, 2019.
  5. Hemophilia. Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/hemophilia/symptoms-causes/syc-20373327. Accessed April 17, 2019.
  6. Rodriguez-Merchan EC. What’s new in orthopedic surgery for people with hemophilia. Arch Bone Jt Surg. 2018;6(3):157-160.
  7. Pipe SW. New therapies for hemophilia. Hematology Am Soc Hematol Educ Program. 2016;(1):650-656.
  8. Franchini M. Current management of hemophilia B: recommendations, complications and emerging issues. Expert Rev Hematol. 2014;7(5):573-581.
  9. Rolstad EB. Perceptions of men with moderate to severe hemophilia regarding the management of their chronic disorder and utilization of community-based support. Am J Mens Health. 2015;9(6):486-495.
  10. Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388(10040):187-197.
  11. National Hemophilia Foundation. MASAC document #255. Available at: www.hemophilia.org. Accessed May 2, 2019.
  12. Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia. 2014;20(5):607-615.
  13. Pharmacokinetics. The Free Dictionary by Farlex. http://medicaldictionary.thefreedictionary.com/pharmacokinetics. Accessed April 17, 2019.
Commitment to our community is always a priority for Takeda Hematology (previously part of Shire and Baxalta). As a leader in hemophilia research, Takeda continues to innovate on your behalf, developing programs and services that support your efforts each step of the way. Takeda is focused on providing advanced hematology treatments for today and innovating for the future.

Copyright © 2019 Takeda Pharmaceutical Company Limited. All rights reserved. All trademarks are the property of their respective owners.

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Patient Newsletter Article 20190226 0.6

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

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