Monday, February 10, 2025

A Chance at More Freedom - Living with Hemophilia B

I am your Eyeball Acquisition Specialist. Success in life, in anything, depends upon the number of persons that one can make himself agreeable to. US online shopping has been growing in popularity over the past few years, as evidenced by the increasing number of people choosing to purchase goods and services over the internet. Bigdoggpinc is helping solidify trust from gaining your confidence 1 person at a time if I have to.

A Chance at More Freedom - Living with Hemophilia B

(BPT) - By Brandpoint

Michael was only four years old when he was diagnosed with hemophilia B, a genetic bleeding disorder that affects the body's ability to generate factor IX, a protein crucial for blood clotting. "It was scary in the beginning," Michael said. "Kids that age like to copy stuff on TV, and I wanted to play sports or be like Spider-Man. As a preschooler, I really had no idea how to comprehend my diagnosis. And then to learn that I'd have to stick myself with needles multiple times a month made me anxious."

Hemophilia B causes prolonged bleeding and increases the risk for internal bleeding in the muscles and joints, which can be life threatening if untreated. Missing or insufficient amounts of factor IX can be replaced by medicine to help the body clot properly. The standard treatment for people with hemophilia B involves lifelong, preventative or on-demand factor replacement therapy, which may be infused multiple times a month intravenously (into a person's veins). However, recent advancements in gene therapy offer new hope.

HEMGENIX^® (etranacogene dezaparvovec-drlb) provides a one-time infusion that can elevate factor IX levels for years, offering greater bleed protection and significantly reducing the need for regular infusions. Michael, who received HEMGENIX, shares his journey with this innovative treatment.

This is Michael's personal experience. Every patient experience is different. For more information, visit https://www.hemgenix.com.

Q: Before you tried gene therapy, you were on prophylaxis treatment. What was that like?

A: I had to learn to self-infuse prophylaxis from a very early age. Otherwise, I'd have to go to the hospital for treatment anytime I got injured — even from something small like jamming my finger playing basketball.

Once I was consistent with my infusions, I didn't need to go to the hospital as often, but I still sometimes experienced breakthrough bleeds. I tried not to let hemophilia B hold me back, and I participated in equestrian classes, swimming, basketball and golf, though I had to prepare for activities more than the average kid. I knew if I was going to play basketball, I may get injured, so I would self-infuse before I left the house. I also had to have my medicine with me in case I got hurt or I had to go to the hospital. It was just something I had to deal with.

Q: How did you learn about gene therapy? What motivated you to move forward with treatment?

A: My condition meant I was in and out of the doctor's office a lot, so I became pretty close to my care team. While I was in college a few years ago, they introduced me to a new treatment option called HEMGENIX.

Around the same time, I had suffered a pretty serious injury, tearing one of my pectoral muscles at the gym. I had to undergo surgery, which was scary because my hemophilia puts me at a higher risk of bleeding during the surgery and recovery. I had to self-infuse more frequently during this time and relied more on my family than ever as an adult. I thought, if there's a chance to stop sticking myself and help me become more independent, I'll take it. After discussing my options with my doctor, we decided that HEMGENIX would be a good option for me.

Q: What was receiving gene therapy like?

A: After going through the pre-screening tests and getting approval from my insurance company, I received an infusion date. HEMGENIX was quite new at the time, and there wasn't a treatment center near my home, so my grandmother and I drove from Louisiana to Mississippi for my infusion. I remember being very excited the entire drive. After having to self-infuse prophylaxis multiple times a month for most of my life, I was hopeful to receive a one-time treatment that could potentially reduce or eliminate my regular infusions for years.

The infusion process took a little over an hour, followed by a few hours of monitoring for infusion-related reactions before I could go home. For me, it was a simple process, and I used the time to get homework and studying done while at the center. Even though I knew what to expect, I still couldn't believe how easy it was for me.

Q: What's life like now after your infusion?

A: After receiving HEMGENIX, there were several follow-up appointments I had to do with my doctor, including liver and blood enzyme tests to monitor for any elevations. I also had to watch for any side effects such as headaches, flu-like symptoms, fatigue, nausea, or feeling unwell. Today, my life no longer revolves around my infusion schedule. I haven't experienced a breakthrough bleed since receiving HEMGENIX and I no longer need regular infusions. I've noticed that I feel more confident and less stressed because I know my factor IX levels are steady. I'm not always thinking about if and when I need to infuse. I exercise and play basketball now without having to self-infuse beforehand or afterwards.

I'm currently focused on pursuing my MBA, which I am excited to complete soon. From there, I'm going to pursue a career in business, and I look forward to many of life's new adventures without the stress of constant infusions.

Q: What message would you like to share with the broader community about living with hemophilia B?

A: I've been managing hemophilia B since elementary school and while I tried not to let it stop me from doing the things I wanted, it definitely can be physically and mentally challenging at times. It was always a nagging thought in the back of my head — something I had to constantly consider and be aware of. Having a support system in place is very important.

When my doctor introduced me to HEMGENIX, I was thrilled by the potential benefits but also nervous. Learning all I could about the benefits and risks ultimately helped me make the best decision for myself. However, everyone is different, and needs can change over time, so it's great that we have so many choices available today. I encourage everyone living with hemophilia B to regularly discuss their treatment options with their doctor to find what works best for them.

Important Safety Information

What is HEMGENIX?

HEMGENIX^®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who:

Currently use Factor IX prophylaxis therapy, or
Have current or historical life-threatening bleeding, or
Have repeated, serious spontaneous bleeding episodes.

HEMGENIX is administered as a single intravenous infusion and can be administered only once.

What medical testing can I expect to be given before and after administration of HEMGENIX?

To determine your eligibility to receive HEMGENIX, you will be tested for Factor IX inhibitors. If this test result is positive, a retest will be performed 2 weeks later. If both tests are positive for Factor IX inhibitors, your doctor will not administer HEMGENIX to you. If, after administration of HEMGENIX, increased Factor IX activity is not achieved, or bleeding is not controlled, a post-dose test for Factor IX inhibitors will be performed.

HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.

What were the most common side effects of HEMGENIX in clinical trials?

In clinical trials for HEMGENIX, the most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell. These are not the only side effects possible. Tell your healthcare provider about any side effect you may experience.

What should I watch for during infusion with HEMGENIX?

Your doctor will monitor you for infusion-related reactions during administration of HEMGENIX, as well as for at least 3 hours after the infusion is complete. Symptoms may include chest tightness, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure. If an infusion-related reaction occurs, the doctor may slow or stop the HEMGENIX infusion, resuming at a lower infusion rate once symptoms resolve.

What should I avoid after receiving HEMGENIX?

Small amounts of HEMGENIX may be present in your blood, semen, and other excreted/secreted materials, and it is not known how long this continues. You should not donate blood, organs, tissues, or cells for transplantation after receiving HEMGENIX.

Please see full prescribing information for HEMGENIX.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

You can also report side effects to CSL Behring's Pharmacovigilance Department at 1-866-915-6958.

HEMGENIX is manufactured by uniQure Inc. and distributed by CSL Behring LLC.

HEMGENIX^® is a registered trademark of CSL Behring LLC.

USA-HGX-0907-NOV24

Genius home hacks: Products to make your life a breeze

Bigdoggpinc Publishing presents

From Uncertainty to Strength: Navigating Life with Cervical Cancer

(BPT) - Sponsored by Pfizer Inc. and Genmab A/S

It never occurred to Courtney that the difficulties she was having with sleeping and breathing could be cancer. Then, she was rushed to the emergency room and a CT scan revealed masses. Courtney, a 44-year-old pharmacy technician from Wilmington, North Carolina, was shocked to be diagnosed with Stage 3 cervical cancer.

"It was all just kind of a whirlwind, like, 'Oh my God. This is really happening,'" said Courtney. "Other than being screened for cervical cancer at my gynecologist, I knew hardly anything about it."

The American Cancer Society estimates that in 2024, there will be 13,820 new cases of cervical cancer diagnosed.ⁱ These numbers have improved since the mid-1970s due to advancements in vaccination and screening practices but, despite this progress, it's projected that this year about 4,360 women will die from the disease.ⁱ This underscores the need for more treatment options.

Following her initial diagnosis, Courtney began a treatment regimen, undergoing a hysterectomy and trying numerous therapies. When she progressed after first-line treatment, she was diagnosed with recurrent cervical cancer. "That was really scary. It was a very hard Thanksgiving that year."

When cervical cancer comes back (recurs) or spreads (metastasizes), treatment options are limited, and survival rates are as low as 19.4%.ⁱⁱ The outlook can feel bleak for patients and their loved ones. That's why for patients, particularly those like Courtney who are facing a challenging prognosis, having an open discussion with healthcare providers and ensuring full understanding of treatment options is critical. By staying informed and exploring all options, patients can take an active role in their care and overall treatment journey.

Courtney recalls taking to the internet, researching potential treatment options in between appointments. "TIVDAK^® (tisotumab vedotin-tftv) for injection came up, and I wanted to learn everything about it. I was excited to try something else, that could possibly work for me."

After much consideration, Courtney and her medical team chose TIVDAK as her next treatment option. TIVDAK is a prescription medicine used to treat adults with cervical cancer that has returned or has spread to other parts of the body, and who have received chemotherapy that did not work or is no longer working. It is an antibody-drug conjugate (ADC), which attacks cells that have a special protein, called Tissue Factor. The special protein, Tissue Factor, is also present on some normal cells. So TIVDAK can still harm normal cells, which can cause side effects. In a clinical trial of 502 adult patients with cervical cancer that has returned or spread and who have received chemotherapy that did not or is no longer working, the patients in the TIVDAK group had a median overall survival of 11.5 months, versus 9.5 months for those treated with chemotherapy.

Eye problems are common with TIVDAK and can be severe. TIVDAK can cause changes to the surface of your eye that can lead to dry eyes, eye redness, eye irritation, corneal ulcers, blurred vision, and severe vision loss. Tell your healthcare provider if you develop new or worsening vision changes or eye problems during treatment. Serious side effects of TIVDAK may include eye problems, nerve problems (peripheral neuropathy), bleeding problems (hemorrhage), lung problems, and severe skin reactions. These are not all of the side effects of TIVDAK. Please see below for more Important Safety Information and click here for the full Prescribing Information with Medication Guide.

"[Since starting TIVDAK], I feel positive. I feel hopeful," Courtney says. "It was a really great option for me. I'm still on therapy." While these were Courtney's experiences at the time of publishing, TIVDAK will not work for everyone, and individual results may vary.

Having a support system was crucial for Courtney as she navigated treatment. In addition to feeling bolstered by her healthcare providers, husband, family and work colleagues, Courtney also found support in online patient and caregiver communities.

"It's been amazing the support that comes around you – sometimes from people you didn't even expect. It's been surprising and very encouraging. There are people working every day to help people just like me. I'm so thankful for that."

If you or a loved one has previously treated recurrent or metastatic cervical cancer, speak with your healthcare provider about whether TIVDAK could be part of your treatment plan.

What is TIVDAK?

TIVDAK is a prescription medicine used to treat adults with cervical cancer:

that has returned or has spread to other parts of the body, and
who have received chemotherapy that did not work or is no longer working.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about TIVDAK?

Eye problems are common with TIVDAK and can be severe. TIVDAK can cause changes to the surface of your eye that can lead to dry eyes, eye redness, eye irritation, corneal ulcers, blurred vision, and severe vision loss. Tell your healthcare provider if you develop new or worsening vision changes or eye problems during treatment.

Your healthcare provider will send you to an eye specialist to check your eyes before you start treatment with TIVDAK, before each infusion for your first 9 infusions of TIVDAK, and as needed for any new or worsening signs or symptoms of eye problems. Your healthcare provider will ask if you have any signs or symptoms of eye problems before each infusion. You will be referred to an eye specialist for any new or worsening signs or symptoms of eye problems.

Your healthcare provider will prescribe 3 different types of eye drops before you start treatment with TIVDAK. Bring the eye drops with you to each infusion and use them as directed by your healthcare provider to reduce your risk of eye problems:

Use 1 drop of steroid eye drops in each eye before each infusion and continue to use your eye drops
3 times a day for 3 days after each infusion
Use vasoconstrictor eye drops right before each infusion
Use lubricating eye drops throughout treatment and for 30 days after your last dose of TIVDAK

Do not wear contact lenses throughout your treatment with TIVDAK unless you are told to use them by your eye specialist.

What are the possible side effects of TIVDAK?

Serious side effects of TIVDAK may include:

Eye problems. See "What is the most important information I should know about TIVDAK?"

Nerve problems (peripheral neuropathy) are common with TIVDAK and can be serious. Tell your healthcare provider right away if you have new or worsening numbness or tingling in your hands or feet or muscle weakness.

Bleeding problems (hemorrhage) are common with TIVDAK and can be serious. Tell your healthcare provider or get medical help right away if you have signs or symptoms of bleeding during treatment with TIVDAK, including blood in your stools or black stools (looks like tar), blood in your urine, cough up or vomit blood, unusual vaginal bleeding, or any unusual or heavy bleeding.

Lung problems. TIVDAK may cause severe or life-threatening inflammation of the lungs that can lead to death. Tell your healthcare provider right away if you have new or worsening symptoms, including trouble breathing, shortness of breath, or cough.

Severe skin reactions. TIVDAK may cause severe or life-threatening skin reactions that can lead to death. Tell your healthcare provider or get medical help right away if you have signs or symptoms of a severe skin reaction during treatment with TIVDAK, including:

Skin reactions that look like rings (target lesions)
Rash or itching that continues to get worse
Blistering or peeling of the skin
Painful sores or ulcers in your mouth, nose, throat, or genital area
Fever or flu-like symptoms
Swollen lymph nodes

The most common side effects of TIVDAK include:

Decreased red blood cell counts
Numbness or tingling in your hands or feet
Eye problems (conjunctival disorders)
Nausea
Tiredness
Changes in liver function blood tests
Nosebleed
Hair loss (alopecia)
Bleeding (hemorrhage)

Your healthcare provider may decrease your dose of TIVDAK, temporarily stop, or completely stop treatment if you have side effects.

What should I tell my healthcare provider before receiving TIVDAK?

Tell your healthcare provider about all your medical conditions, including if you:

have a history of vision or eye problems
have numbness or tingling in your hands or feet
have bleeding problems
have liver problems
are pregnant or plan to become pregnant; TIVDAK can harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with TIVDAK
are breastfeeding (nursing) or plan to breastfeed. It is not known if TIVDAK passes into your breast milk. Do not breastfeed during treatment and for at least 3 weeks after the last dose of TIVDAK

Females who can become pregnant:

- Your healthcare provider should do a pregnancy test before you start treatment with TIVDAK
- Use an effective method of birth control during your treatment and for at least 2 months after the last dose of TIVDAK

Males with female partners who can become pregnant:

- Use effective birth control during treatment and for 4 months after the last dose of TIVDAK

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking TIVDAK with certain other medicines may cause side effects.

These are not all of the possible side effects of TIVDAK. Discuss side effects with your healthcare provider. You may report side effects to FDA at 1-800-FDA-1088.

Please see the full Prescribing Information with Medication Guide about TIVDAK including an IMPORTANT WARNING here.

The patient featured in this article is receiving or has received TIVDAK. The patient was compensated by Pfizer/Genmab to share their story. These were their experiences at the time they were interviewed. TIVDAK will not work for everyone. Individual results may vary.

Courtney was compensated for her participation.

Content sponsored and provided by Pfizer and Genmab. Courtney partnered with Pfizer and Genmab to share her experience with recurrent/metastatic cervical cancer.

i American Cancer Society. (n.d.). Key statistics for cervical cancer. Retrieved September 6, 2024, from https://www.cancer.org/cancer/types/cervical-cancer/about/key-statistics.html.

ii National Cancer Institute SEER Program. "Cervical Cancer Statistics." seer.cancer.gov. Retrieved September 6, 2024, from https://seer.cancer.gov/statistics/.

Advertisement Only

Much more than a 'bad cold': Tracy's frightening RSV story

(BPT) - Sponsored by GSK – When Tracy, a GSK patient advocate and 66-year-old retiree, started coughing, she assumed it was just her chronic obstructive pulmonary disease (COPD) acting up again. Little did she know, it was the beginning of a respiratory syncytial virus (RSV) infection.

For most people, RSV infections come with mild symptoms, like a runny nose, sneezing, or coughing, and go away on their own in a week or two. But for older adults like Tracy, who was 64 years old at the time, it can be much more serious, even life-threatening. "Everybody thinks it's a cold, a bad cold," Tracy said. "And it could be a lot worse than a bad cold."

The first thing Tracy noticed was a bad headache, and then the cough came on. Having lived with COPD for a decade, she was used to the coughing. But when the cough became uncontrollable to the point where she couldn't stay standing, her husband insisted they go to the emergency room.

"That coughing was the kind that you cough, and your side hurt, and your stomach hurt, and you couldn't quit coughing no matter what you did," Tracy said.

In the emergency room, she was evaluated and diagnosed with RSV, and her case was deemed so severe that she was admitted to the hospital. As her doctor explained, her COPD had made the RSV symptoms more severe for her than they might have been otherwise.

For older adults like Tracy, RSV can also lead to serious illness like pneumonia. Each year, approximately 177,000 adults 65 years and older are hospitalized in the US due to RSV, and an estimated 14,000 of those cases result in death.

"I was very scared," Tracy said.

There's no specific treatment or cure for RSV, so the doctors provided supportive care for her symptoms. After five days, she was sent home from the hospital, but the ordeal had left her exhausted. It took two weeks for her symptoms to improve, and even then, the cough and fatigue persisted.

Her experience highlights just how severe RSV can be for an older adult. It also reinforces the importance of helping to protect against RSV through vaccination.

Before she got sick, Tracy didn't have the option of getting vaccinated to help protect against RSV, but that changed last year when RSV vaccination became available for older adults. The CDC recommends a single dose of RSV vaccine for adults ages 60-74 who are at increased risk for severe RSV disease and all adults ages 75 and older.

Have you or a loved one been diagnosed with RSV and want to tell your story and help others, like Tracy? Compensation may be available for eligible advocates. Reach out to gsk_rsv@syneoshealth.com and find out if you qualify.

If you're 75 years or older, or you're 60-74 years old with certain underlying health conditions, visit AREXVY.com and talk to your doctor or pharmacist about getting vaccinated against RSV with AREXVY (Respiratory Syncytial Virus Vaccine, Adjuvanted).

Please see full Prescribing Information available at AREXVY.com and the following Important Facts About AREXVY.

Important Facts About AREXVY (Respiratory Syncytial Virus Vaccine, Adjuvanted)

What is AREXVY (Respiratory Syncytial Virus Vaccine, Adjuvanted)?

AREXVY is an FDA-approved vaccine for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in people 60 years of age and older

What is RSV and what causes it?

RSV is a respiratory virus that typically causes mild symptoms but can lead to serious respiratory illness in older adults. It can cause lower respiratory tract disease, which may include infections of the lungs and lower airways

RSV spreads via respiratory droplets. It can lead to more severe symptoms in people with long-term respiratory or heart diseases

How does AREXVY work?

AREXVY is a vaccine that works to boost your immunity to protect against LRTD caused by RSV

How is AREXVY given?

AREXVY is given as a single dose (0.5 mL) into the muscle of the upper arm

Who should not get AREXVY?

You should not receive AREXVY if you are allergic to any of its ingredients

What are the potential side effects?

Fainting can happen after getting injectable vaccines, including AREXVY. Precautions should be taken to avoid injury due to fainting

Some people with weakened immune systems may have reduced immune responses to AREXVY

The most common side effects are injection site pain, fatigue, muscle pain, headache, and joint pain

Additional Important Information:

Vaccination with AREXVY may not result in protection of all vaccine recipients

Ask your healthcare provider about the risks and benefits of AREXVY. Only a healthcare provider can decide if AREXVY is right for you

Need more Information?

This is only a summary of important information

To learn more about AREXVY, talk to your doctor, pharmacist, or other healthcare provider

Visit AREXVY.com or call 888-AREXVY9 (888-273-9899) for the FDA-approved product labeling

You are encouraged to report vaccine adverse events to the US Department of Health and Human Services. Visit www.vaers.hhs.gov to file a report, or call 1-800-822-7967.

Tracy was compensated by GSK for her participation in this program.

Trademarks are owned by or licensed to the GSK group of companies.

PMUS-RSAADVR240002 October 2024

Produced in USA.

Advertisement Only

Rare Diagnosis Reveals Surprising Link Between Mysterious Symptoms

(BPT) - As he approached his 40th birthday, Andrew began experiencing strange symptoms — fatigue, bone pain and frequent trips to the bathroom. Though unsettling, "I didn't think much of them and chalked the symptoms up to things that tend to happen in middle age," recalls the attorney and father of three. "I felt like I was constantly experiencing a low-level allergic reaction: my skin was itchy, and my eyes were watery and itchy. I felt uncomfortable in my own skin."

But when he began to develop unusual spots on his torso, his dermatologist recommended a biopsy and a few other tests. A few days later, his doctor called with a shocking diagnosis: a rare disease called indolent systemic mastocytosis, or ISM. "It was incredible to realize all these different symptoms were related," says Andrew.

ISM is caused by the uncontrolled increase and activity of abnormal mast cells, which play an important role in immune responses. As the cells accumulate and activate in different parts of the body, the disease can cause chronic, potentially debilitating symptoms such as skin lesions, diarrhea, fatigue, bone pain and life-threatening anaphylaxis. Symptoms of ISM are often unpredictable and can be caused by a wide range of triggers including foods and beverages, odors and scents, temperature changes, exercise, stress and more.

In addition to the toll of his physical symptoms, the appearance of his skin spots began to have a negative impact on Andrew mentally. "It made me feel really self-conscious," he remembers. "I didn't like looking at them in the mirror, and I avoided taking my shirt off at the pool or beach — I lost some of my confidence."

Because his dermatologist wasn't familiar with ISM, Andrew and his wife Shannon found a specialist in Boston. Together, they flew across the country, from their home state of Oregon, in search of help.

Seeking symptom relief

With no approved ISM treatments at the time, Andrew's plan before he saw a specialist focused solely on reactive care. "I was treating my symptoms with eye drops when my eyes felt itchy, antihistamines when I felt reactive, and antacids when my stomach was upset. Sometimes the medications worked, sometimes they didn't. I never knew when to expect symptoms. It felt like a game of 'whack-a-mole' using various medications to manage the ever-changing and unpredictable symptoms."

A terrifying experience

A few years after his consultation with an expert on ISM, Andrew was out "in the middle of nowhere" in Idaho on a rafting trip with family and friends, when he was stung by a wasp or bee. "I ended up experiencing anaphylaxis, and because I didn't know I was allergic, I didn't have an epinephrine injector. Everyone around me was panicking. Fortunately, my good friend who is a physician brought a medical kit, which included a vial of epinephrine. He injected me, and I believe he saved my life right there."

"This was an incredibly scary episode for me, and for my family. I would say that was a wake-up call. I became much more interested in researching and exploring different treatment options for ISM that went beyond supportive care. I wanted to take a more proactive approach to my care."

Targeting the underlying cause

Andrew was now seeing a hematologist who recommended a treatment that the U.S. Food and Drug Administration (FDA) approved called AYVAKIT^® (avapritinib) for adults with ISM. Andrew and his hematologist discussed the potential benefits and side effects of AYVAKIT and decided together that Andrew would try it.

AYVAKIT is a once daily pill that works by selectively targeting the KIT D816V mutation, the underlying cause of the disease in ~95% of patients with ISM which results in the overproduction and activation of abnormal mast cells. AYVAKIT is not recommended in people with low platelet counts (less than 50 X 10⁹/L). AYVAKIT for ISM may cause serious side effects such as cognitive effects and skin sensitivity to sunlight (photosensitivity). AYVAKIT can have harmful effects on pregnancy and fertility as well as cause harm to the unborn baby. Talk to your healthcare provider for more information.

"I was encouraged to hear there was another option for me and hopeful that AYVAKIT could improve some of my most bothersome symptoms."

Please see Important Safety Information below and click here for full Prescribing Information, including Patient Information.

'Comfortable in my own skin'

Andrew started taking AYVAKIT in October of 2023. "I'm glad that I took the leap and decided to try AYVAKIT after talking it over with my doctor. Now I'm more than a year into treatment, and so far, I haven't experienced cognitive side effects, which I was concerned would impact my work."

While he still takes precautions on his family adventures rock climbing and skiing, Andrew has noticed changes in his ISM symptoms. "My skin spots are lighter and my tryptase (allergic reactive marker) levels are lower, which is reassuring to me. My spots are still there, and I occasionally feel fatigued, but overall though, I feel my symptoms are better. I feel comfortable in my own skin."

"I would love to encourage other people with ISM to chart their own course and lean in to finding a solution. Everyone's experience is going to be different, but there are solutions out there, you just have to find them. So do your own research, ask your own questions, and do what is right for you."

Please click here to see the full Prescribing Information and Patient Information for AYVAKIT.

WHAT IS AYVAKIT^® (avapritinib)?

AYVAKIT^® (avapritinib) is a prescription medicine used to treat adults with indolent systemic mastocytosis (ISM). AYVAKIT is not recommended in people with low platelet counts (less than 50 X 10⁹ /L). It is not known if AYVAKIT is safe and effective in children.

IMPORTANT SAFETY INFORMATION

AYVAKIT^® (avapritinib) may cause serious side effects, including:

Cognitive effects. Cognitive side effects can happen during treatment with AYVAKIT and can be severe. Tell your healthcare provider if you develop any new or worsening cognitive symptoms including forgetfulness, confusion, getting lost, trouble thinking, drowsiness, trouble staying awake (somnolence), word finding problems, seeing objects or hearing things that are not there (hallucinations), or a change in mood or behavior.

Skin sensitivity to sunlight (photosensitivity). Your skin may be sensitive to the sun or other forms of light (photosensitivity) during treatment with AYVAKIT. Avoid or limit exposure to direct sunlight, sunlamps, and other sources of ultraviolet radiation during treatment and for 1 week after stopping treatment with AYVAKIT. Use sunscreen or wear clothes that cover your skin if you need to be out in the sun.

Before taking AYVAKIT, tell your healthcare provider about all of your medical conditions, including if you:

history of bulging or weakening of a blood vessel wall (aneurysm) or bleeding in your brain
have a history of stroke within the last year
have low platelet counts
have or have had liver problems
are pregnant or plan to become pregnant. AYVAKIT can cause harm to your unborn baby
- Females who are able to become pregnant: Your healthcare provider should do a pregnancy test before you start AYVAKIT. You should use effective birth control (contraception) during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Talk to your healthcare provider about birth control methods that may be right for you. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with AYVAKIT.
- Males with female partners who are able to become pregnant should use effective birth control (contraception) during treatment and for 6 weeks after the final dose of AYVAKIT.
are breastfeeding or plan to breastfeed. It is not known if AYVAKIT passes into your breast milk. Do not breastfeed during treatment with AYVAKIT and for at least 2 weeks after the final dose of AYVAKIT. Talk to your healthcare provider about the best way to feed your baby during this time

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. AYVAKIT may affect the way other medicines work, and certain other medicines may affect how AYVAKIT works. Especially tell your healthcare provider if you take estrogen-containing hormonal birth control or medicines that prevent blood clots.

Do not drive or operate hazardous machinery if you have confusion or trouble thinking during treatment with AYVAKIT.

The most common side effects of AYVAKIT in people with ISM include: swelling around your eyes, dizziness, swelling of your arms and legs, and flushing.

Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with AYVAKIT if you develop certain side effects. AYVAKIT may cause fertility problems in females and males. Talk to your healthcare provider if this is a concern for you.

These are not all of the possible side effects of AYVAKIT. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

AYVAKIT is available in 25 mg tablets.

This promotional content was developed by Blueprint Medicines Corporation, the manufacturer of AYVAKIT. Individual results may vary. Andrew is being compensated by Blueprint Medicines for sharing his story.

AYVAKIT, Blueprint Medicines and associated logos are trademarks of Blueprint Medicines Corporation.

To learn more and connect with others about their experience, including AYVAKIT, visit AYVAKIT.com.

Advertisement Only

Find everything you need at amazing prices on Wish! gadgets to stylish clothing, we have it all. Shop now and save big

From Caregiver to Patient: Michel's Journey with Thyroid Eye Disease Treatment

(BPT) - Michel, a psychiatric nurse from Southern California, has always been someone who is deeply committed to helping others take control of their health. He didn't realize that one day he would be the one needing care.

A few years after being diagnosed with Graves' disease, a common form of hyperthyroidism, Michel noticed his eyes starting to bulge a bit, accompanied by symptoms of dryness, eye pain and difficulty sleeping because his eyes wouldn't fully close.

"I assumed it was part of my Graves' disease and then I went to get a routine eye exam for new glasses and my doctor diagnosed me with Thyroid Eye Disease (TED)," said Michel. "Even though I'm a nurse, I did not know about TED until my diagnosis."

TED is a chronic autoimmune condition that may affect many people with Graves' disease. TED occurs when the body's immune system mistakenly attacks the muscle and fat tissue behind the eyes, causing eye bulging, eye pain, double vision and pressure behind the eyes. These symptoms can be mild or severe and may flare or get worse over time.

Despite receiving this diagnosis, Michel still had no clear treatment path and his symptoms began to severely impact his life. "I began to withdraw from social situations and struggled with changes in my appearance. I was afraid of unwanted attention and comments from others due to my eye bulging," said Michel.

Simple tasks, like using a computer or phone, became difficult because of Michel's double vision and eye pain.

Treatment with TEPEZZA^® (teprotumumab-trbw)

While watching TV, Michel saw a commercial for TEPEZZA, the first and only FDA-approved medication to treat TED, no matter if you'd lived with TED for months or years.

"After four years of struggling with my TED symptoms, I was relieved to learn there was an approved and specific treatment option available for people like me," said Michel.

After discussing the risks and benefits with his doctor, Michel was connected with a home health service and began receiving his infusions at home. TEPEZZA is administered intravenously every three weeks over a course of eight infusions, with the full treatment lasting about five months. By the second infusion, Michel started noticing positive changes, and after completing his eighth infusion, Michel saw a significant improvement in his symptoms of eye pain and eye bulging.

Michel did experience some side effects during his treatment with TEPEZZA. Common side effects include muscle cramps or spasms, nausea, hair loss, diarrhea, feeling tired, high blood sugar, hearing problems, taste changes, headache, dry skin, weight loss, nail problems and changes in menstruation.

After treatment with TEPEZZA, "I no longer fear unwanted attention. My eye bulging has decreased and my eye pain has subsided," said Michel. "I look and feel more like myself again." Infusion reactions, like high blood pressure or difficulty breathing, can happen during or within 24 hours after an infusion of TEPEZZA. Please see Important Safety Information below.

TED Education and Advocacy

With his background in nursing, Michel knows the value of education and self-advocacy. To support others newly diagnosed or living with TED, Michel offers the following advice:

Advocate for yourself and your symptoms. Even if they aren't visible, be vocal about how they affect your daily life. Have you stopped doing a hobby you love or find it difficult to use a computer due to your TED symptoms? Tell your doctor.
Some doctors still think if you have been living with TED for a long time, you are not a candidate for TEPEZZA — but this is not entirely true. Even if you've had bulging eyes for years due to TED, you can still experience improvement through treatment with TEPEZZA.
Not all eye doctors are used to treating TED, which is why you should see a doctor who specializes in it. Make sure you're seeing a TED Specialist, such as an oculoplastic surgeon, neuro-ophthalmologist or endocrinologist, who has experience with the condition.
Visit TEPEZZA.com to learn more and find a TED Specialist.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about TEPEZZA?

Infusion reactions can happen during or within 24 hours after your infusion of TEPEZZA. If you have a reaction while receiving TEPEZZA, your doctor or nurse will slow or stop your infusion and treat your reaction. If you have a severe infusion reaction, your doctor may stop your treatment completely.

Tell your doctor or nurse right away if you have any of these symptoms during or after your treatment with TEPEZZA:

High blood pressure	Difficulty breathing
Fast heartbeat	Headache
Redness of the face/Feeling hot	Muscle pain

If you have inflammatory bowel disease (IBD), such as Crohn's disease or ulcerative colitis, TEPEZZA may make your IBD symptoms worse. Symptoms of worsening IBD may include: an increased number of loose stools with stomach pain or cramps, and blood in your stools. After each TEPEZZA infusion, tell your doctor right away if you have worsening IBD symptoms.

TEPEZZA may cause an increase in your blood sugar. Before starting treatment with TEPEZZA, tell your doctor if you are currently being treated for diabetes, know your blood sugar is high, or have been diagnosed with diabetes. It is important for you to take your treatments and follow an appropriate diet for glucose control as prescribed by your doctor.

TEPEZZA may cause severe hearing problems including hearing loss, which in some cases may be permanent. Tell your doctor if you have any signs or symptoms of hearing problems or changes in hearing.

Before receiving TEPEZZA, tell your doctor if you:

Have inflammatory bowel disease (Crohn's disease or ulcerative colitis).
Are currently being treated for diabetes, have been diagnosed with diabetes, or know your blood sugar is high.
Are pregnant or plan to become pregnant. TEPEZZA may harm your unborn baby. Tell your doctor if you become pregnant or suspect you are pregnant during treatment with TEPEZZA.
- Women who are able to become pregnant should use an effective form of birth control (contraception) prior to starting treatment, during treatment and for at least 6 months after the final dose of TEPEZZA.
Are breastfeeding or plan to breastfeed. It is not known if TEPEZZA passes into your breast milk. Talk to your doctor about the best ways to feed your baby during treatment with TEPEZZA.
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, dietary and herbal supplements. Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

What are the possible side effects of TEPEZZA?

The most common side effects of TEPEZZA include muscle cramps or spasms, nausea, hair loss, diarrhea, feeling tired, high blood sugar, hearing problems, taste changes, headache, dry skin, weight loss, nail problems, and changes in menstruation.

This is not a complete list of all possible side effects. Tell your doctor or treatment team about any side effect you may have.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/safety/medwatch, or call 1-800-FDA-1088.

Please visit TEPEZZA.com for more information.

USE

TEPEZZA is a prescription medicine used to treat Thyroid Eye Disease (TED), no matter if you've had TED for months or years.

Advertisement Only

Valentine's Day Sale! Enjoy 10% off your order. Enter code LOVE24. One day only

Bigdoggpinc Informer

Translate Help

Sharing Agent

Becoming a Govvi Influencer

Diigo with Bigdoggpinc

Monday, February 10, 2025