Self-Advocacy Helped Me Navigate My Rare Disease

Diagnosed at a young age with a rare and potentially life-threatening disease, one man learned the power of taking control.

When South Carolinian Roger was diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) at age 26, he was overwhelmed. “Being diagnosed with PNH was shocking,” he explained. “It was life-altering. In the beginning, I didn’t really know how to prioritize my needs and my health. I was desperate for educational information.”

PNH is a rare disease that causes the body’s own immune system to attack and destroy red blood cells. It is an acquired disease, which means you don’t inherit it. Caused by a mutation in the body’s stem cells, it affects about 12-13 people per million each year. “When I was diagnosed back in 2011, there weren’t a lot of treatment options,” Roger explained. “But you’ve got to take some action. You can’t just sit by and let it take control.”

For Roger, that meant leaning into self-advocacy around managing his PNH. And that has made all the difference.

Self-advocacy

Initially, Roger was treated with blood thinners and steroids — but managing his condition was stressful and destabilizing, especially as a young man with an active life between work, family and friends and hobbies like golf and playing the guitar and piano. “I was getting frustrated,” he said. “I was tired of taking pills twice, three times a day. I said, ‘enough is enough.’”

With the help of his family — especially his mother, who actively researched PNH and available treatment options — Roger identified a less-frequent treatment plan to address his disease. But he felt trapped in a retail job that allowed him the time off he needed to manage his health but left him feeling unfulfilled.

“It was easy to take off time working in retail,” he explained, “but I wanted to advance my career further and have more freedom.”

Roger and his family continued to educate themselves and track the development of new PNH treatments. That paid off for Roger in 2018 when he asked his doctor about ULTOMIRIS^® (ravulizumab-cwvz), an FDA-approved medicine used to treat adults and children 1 month of age and older with PNH.

Sustained disease control

ULTOMIRIS is designed to work by targeting one of the immune system proteins that is attacking red blood cells in the body. Two weeks after an initial loading dose, ULTOMIRIS is infused intravenously every eight weeks for most people. The most common side effects of ULTOMIRIS are upper respiratory tract infection and headache. Roger and his doctor discussed the benefits and risks of treatment including that he would need meningococcal vaccinations at least two weeks before his first dose of ULTOMIRIS due to the risk of serious meningococcal infections. Roger liked that ULTOMIRIS allowed him more time between infusions, which fit his lifestyle and career goals better. “I left retail and pursued a more professional role in staffing and recruiting, which I’m passionate about,” he explained. “With ULTOMIRIS, I’m able to really plan my life, my free time, my vacations. I’m able to schedule things that I previously couldn’t. It gave me a real sense of freedom. This was my own experience and may be different for others.”

Roger credits the doctors and nurses he’s worked with over the years for the success of his current care plan — but also himself and his proactive push to ask questions, initiate conversations, and advocate for himself.

“For me, self-advocacy is really taking back the control that you feel you lost with the diagnosis,” he said. “The first thing I would say to someone facing a diagnosis like this is, life is not over. There is hope. Find the right doctors, but you’re going to have to do some of the work on your own. You can do it.”

To learn more, visit ultomiris.com/pnh

IMPORTANT SAFETY INFORMATION & INDICATION

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

ULTOMIRIS is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.

ULTOMIRIS increases your chance of getting serious meningococcal infections that may quickly become life-threatening or cause death if not recognized and treated early.
1. You must complete or update meningococcal vaccine(s) at least 2 weeks before your first dose of ULTOMIRIS.
2. If you have not completed your meningococcal vaccines and ULTOMIRIS must be started right away, you should receive the required vaccine(s) as soon as possible.
3. If you have not been vaccinated and ULTOMIRIS must be started right away, you should also receive antibiotics for as long as your healthcare provider tells you.
4. If you had a meningococcal vaccine in the past, you might need additional vaccines before starting ULTOMIRIS. Your healthcare provider will decide if you need additional meningococcal vaccines.
5. Meningococcal vaccines do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: fever, fever with high heart rate, headache and fever, confusion, muscle aches with flu-like symptoms, fever and a rash, headache with nausea or vomiting, headache with a stiff neck or stiff back, or eyes sensitive to light.

Your healthcare provider will give you a Patient Safety Card about the risk of serious meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. Your risk of meningococcal infection may continue for several months after your last dose of ULTOMIRIS. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

ULTOMIRIS is only available through a program called the ULTOMIRIS and SOLIRIS Risk Evaluation and Mitigation Strategy (REMS). Before you can receive ULTOMIRIS, your healthcare provider must: enroll in the REMS program; counsel you about the risk of serious meningococcal infections; give you information about the signs and symptoms of serious meningococcal infection; make sure that you are vaccinated against serious infections caused by meningococcal bacteria, and that you receive antibiotics if you need to start ULTOMIRIS right away and are not up to date on your vaccines; give you a Patient Safety Card about your risk of meningococcal infection.

ULTOMIRIS may also increase the risk of other types of serious infections, including Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria gonorrhoeae. Your child should receive vaccines against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with ULTOMIRIS. Certain people may be at risk of serious infections with gonorrhea.

Who should not receive ULTOMIRIS?

Do not receive ULTOMIRIS if you have a serious meningococcal infection when you are starting ULTOMIRIS.

Before you receive ULTOMIRIS, tell your healthcare provider about all of your medical conditions, including if you:

have an infection or fever
are pregnant or plan to become pregnant. It is not known if ULTOMIRIS will harm your unborn baby.
- Pregnancy Registry: There is a registry for pregnant women who take ULTOMIRIS to check the health of the pregnant mother and her baby. If you are pregnant or become pregnant while taking ULTOMIRIS, talk to your healthcare provider about how you can join this registry or you may contact the registry at 1-833-793-0563 or www.UltomirisPregnancyStudy.com to enroll.
are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

If you have PNH and you stop receiving ULTOMIRIS, your healthcare provider will need to monitor you closely for at least 16 weeks after you stop ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in your red blood cell count, tiredness, blood in your urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males.

What are the possible side effects of ULTOMIRIS?

ULTOMIRIS can cause serious side effects including infusion-related reactions. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, stomach (abdominal) pain, muscle spasms, changes in blood pressure, tiredness, feeling faint, shaking chills (rigors), discomfort in your arms or legs, or bad taste. Stop treatment of ULTOMIRIS and tell your healthcare provider right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion-related reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out.

The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory tract infection and headache.

Tell your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your healthcare provider or pharmacist. Call your healthcare provider right away if you miss an ULTOMIRIS infusion or for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

INDICATION

What is ULTOMIRIS?

ULTOMIRIS is a prescription medicine used to treat adults and children 1 month of age and older with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.

Please see full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious meningococcal infections.