Realizing I Was a Runner, Despite My Hemophilia

From a young age, Thomas Carine was told he would always need to manage the life-threatening risks, chronic pain, and bleeding associated with hemophilia A, a rare disorder that prevents his blood from properly clotting due to a missing or defective protein called factor VIII. “The first doctor we met told my parents, ‘Your son has hemophilia A,’” he recalls. “‘He’s probably not going to live past 12 years old. Be ready for that.’” Now, at age 32, Carine runs two to three miles each day, plays intramural softball, and enjoys a stable career in media—an expansive, active life he has cultivated despite his chronic condition.

An Early Diagnosis

Carine was diagnosed with hemophilia A soon after birth, when his parents noticed substantial bruising from crawling and delayed healing time after his circumcision. Like many medical conditions, hemophilia is not experienced the same way by everybody. Carine’s condition is severe and can cause life-threatening internal bleeding even in the absence of an inciting injury. At the time of his diagnosis, there weren’t many hematology departments around Carine’s hometown of San Jose, California, and finding adequate care (and clinical understanding) was difficult.

“The early years were very hard,” he says. “Luckily my dad was a doctor, so he was there to handle my factor VIII replacement infusions at home.” Bleeding episodes in joints—such as the ankles, knees, and elbows—sometimes occurred with little warning. These prevented him from participating in many childhood activities.

Outside Looking In

Carine recalls gearing up for a sleepover with a laundry list of to-dos. “My parents and I prepared so many questions for the doctors, had calls with the parents and the other kids, talked through everything I’d need to bring with me,” he recalls. “Finally, we get to the sleepover. That night there was blood in my urine. The next morning, we had to go to the hospital where I found out I was bleeding into my kidneys. That was a linchpin moment: I realized that I couldn’t wish this away.” At age 10, Carine’s doctor recommended treating with factor VIII prophylactically, aiming to prevent bleeding episodes rather than treating them after they happen in order to help improve his symptoms.

Even then, sports were still daunting. Carine remembers trying out for the baseball team: “By the second week of tryouts, my joints were painful and swollen.” Doubting his ability to lead a physically active life, he began to explore the theater program in high school, building sets and doing audio-visual work—a serendipitous pivot that would ultimately lead Carine to a job in the A/V department for a professional hockey team.

Embracing His Reality

As Carine transitioned into adulthood, his experience managing the physical and mental effects of hemophilia A evolved. “It just becomes your life. It becomes another day,” he says. “The next day becomes a little easier, and then you get through the one after that. I remember the first factor VIII infusion I did for myself was a huge milestone: How do I give myself my own infusion so I don’t have to wait for my parents, so that I don’t have to go to a hospital?” With more confidence in learning to manage his condition, Thomas started participating in more physical activities too.

Still, independence was not without challenges. While he had a career he loved, managing his condition and treatment schedule still posed a burden. His factor VIII infusions required careful preparation and administration several times a week. Carine has always looked for new strategies to streamline his routine and at one point he optimistically pursued a gene-therapy trial, but was denied eligibility due to certain existing antibodies to the virus used in that therapy.

Still, he continued searching for new treatment options.

Finding the Right Treatment

Four years ago, upon his doctor’s advice, he began using Hemlibra® (emicizumab-kxwh)*, a prescription medicine used to prevent or reduce the frequency of bleeding episodes in adults and children living with hemophilia A. It is the first medicine for hemophilia A that can be subcutaneously injected, like a shot under the skin, similar to the way someone with diabetes injects insulin. Because Hemlibra has a unique four-week half-life (how long it takes for half the amount of a medicine to leave the body), the medicine sustains longer in the body between doses, enabling patients to self-administer once a week, every two weeks, or every four weeks after loading doses.

*What is the most important information I should know about HEMLIBRA? HEMLIBRA increases the potential for your blood to clot. Discontinue prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. Carefully follow your healthcare provider’s instructions regarding when to use an on-demand bypassing agent, and the dose and schedule you should use. HEMLIBRA may cause serious side effects when used with aPCC (FEIBA®), including thrombotic microangiopathy (TMA) and blood clots (thrombotic events). If aPCC (FEIBA®) is needed, talk to your healthcare provider in case you feel you need more than 100 U/kg of aPCC (FEIBA®) total. See more safety information below.

Running Forward

The administration of Hemlibra, taking less than a minute to inject after preparation, allowed Carine to integrate Hemlibra into his busy schedule, and he turned his main focus back to his life. “I made an effort to figure out my workout schedule and run with other people to encourage me,” he says. Carine talked to his doctor to make sure it was okay for him to participate in these activities and to learn how to take the necessary precautions.

While he still struggles with joint damage and issues from childhood, Carine continues to regularly participate in physical activities and recreational sports. With more flexibility in his schedule, he runs every day, plays on an intramural softball team with friends, and practices calisthenics with weights regularly throughout the week. His experience makes him hopeful for the future of people with hemophilia. “I just want people with this condition to lead more normal, active lives,” he says. “It’s all about the next generation.”

Every person’s hemophilia A journey is different, and Thomas’ physical practices may not be appropriate for everyone.

*In the Phase III HAVEN 3 study in people aged 12 years and older without factor VIII inhibitors, the average number of treated bleeds per year (ABR, annualized bleed rate) for people receiving HEMLIBRA prophylaxis every week (N=36) or every two weeks (N=35) was 1.5 (95% CI: 0.9, 2.5) and 1.3 (95% CI: 0.8, 2.3), respectively, compared to 38.2 (95% CI: 22.9, 63.8) for people receiving no prophylaxis (N=18). The median time on HEMLIBRA for people in HAVEN 3 study was 30 weeks (once every week), 31 weeks (once every 2 weeks), and 24 weeks (no prophylaxis). In the Phase III HAVEN 4 study, the ABR for people aged 12 years or older with hemophilia A with or without factor VIII inhibitors receiving HEMLIBRA prophylaxis every four weeks (N=41) was 2.4 (95% CI: 1.4, 4.3). The median time on Hemlibra for people in HAVEN 4 study was 26 weeks.

Important Safety Information

What is the most important information to know about Hemlibra?

Hemlibra increases the potential for blood to clot. People who use activated prothrombin complex concentrate (aPCC; Feiba®) to treat breakthrough bleeds while taking Hemlibra may be at risk of serious side effects related to blood clots.

These serious side effects include:

Thrombotic microangiopathy (TMA), a condition involving blood clots and injury to small blood vessels that may cause harm to one’s kidneys, brain, and other organs

Blood clots (thrombotic events), which may form in blood vessels in the arm, leg, lung, or head

Patients should talk to their doctor about the signs and symptoms of these serious side effects, which can include:

• Confusion

• Stomach, chest, or back pain

• Weakness

• Nausea or vomiting

• Swelling, pain, or redness

• Feeling sick or faint

• Decreased urination

• Swelling of arms and legs

• Yellowing of skin and eyes

• Eye pain, swelling, or trouble seeing

• Fast heart rate

• Numbness in your face

• Headache

• Shortness of breath

• Coughing up blood

If patients experience any of these symptoms during or after treatment with Hemlibra, they should get medical help right away.

Patients should carefully follow their healthcare provider’s instructions regarding when to use an on demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. If aPCC (Feiba®) is needed, patients should talk to their healthcare provider in case they feel they need more than 100 U/kg of aPCC (Feiba®) total.

Patients’ bodies may make antibodies against Hemlibra, which may stop Hemlibra from working properly. Patients should contact their healthcare provider immediately if they notice that Hemlibra has stopped working for them (e.g., an increase in bleeds).

The most common side effects of Hemlibra include: injection site reactions (redness, tenderness, warmth, or itching at the site of injection), headache, and joint pain. These are not all of the possible side effects of Hemlibra. Patients can speak with their healthcare provider for more information.

What else should patients know about Hemlibra?

Patients should see the detailed “Instructions for Use” that comes with Hemlibra for information on how to prepare and inject a dose of Hemlibra, and how to properly throw away (dispose of) used needles and syringes.

• Patients should stop taking their prophylactic bypassing therapy the day before they start Hemlibra.

• Patients may continue taking their prophylactic factor VIII for the first week of Hemlibra.

Hemlibra may interfere with laboratory tests that measure how well blood is clotting and create an inaccurate result. Patients should speak with their healthcare provider about how this may affect their care.

Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide.

Patients should only use Hemlibra for the condition it was prescribed. Patients should not give Hemlibra to other people, even if they have the same symptoms that they have. It may harm them.

Patients should tell their healthcare provider about all the medicines they take, including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements. Patients should keep a list of them to show their healthcare provider and pharmacist.

Before using Hemlibra, patients should tell their healthcare provider about all of their medical conditions, including if they are pregnant, plan to become pregnant, are breastfeeding, or plan to breastfeed.

Since Hemlibra was tested in males, there is no information on whether Hemlibra may impact an unborn baby or breast milk. Females who are able to become pregnant should use birth control during treatment.

Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. Side effects may also be reported to Genentech at (888) 835-2555.

Please see Important Safety Information, including Serious Side Effects, as well as the Hemlibra full Prescribing Information and Medication Guide.

M-US-00022169(v1.0)

© 2024 Genentech USA, Inc. All rights reserved.

This is a paid partnership between Genentech and Men’s Health.

Read More

Leave a Reply

Your email address will not be published. Required fields are marked *