Living Fully With PNH: One Patient’s Journey
Sponsored by Alexion, AstraZeneca Rare Disease
For Mike, life with paroxysmal nocturnal hemoglobinuria (PNH) hasn’t been defined by limitations—it’s been shaped by resilience, family, and a deep appreciation for the everyday joys that make life meaningful.
Now aged 68 and retired, Mike lives with his wife Sara in Delaware. “The beach is in our backyard, we can walk out the back door and be on our boat,” he says with a smile. Boating is more than a hobby—it’s a symbol of the freedom Mike has reclaimed since he began treatment as part of the clinical trial in 2017 with ULTOMIRIS® (ravulizumab-cwvz), an FDA-approved medicine used to treat adults and children 1 month of age and older with PNH.
Navigating the Unknown
Diagnosed with PNH in 1987 at age 30, Mike’s journey began as a mystery: red-colored urine after a day of yard work. “I thought I’d overdone it,” he recalls. But when it happened again, he sought answers. A series of referrals led him to a hematologist who recognized the signs and diagnosed him with PNH—a rare, chronic blood disorder characterized by red blood cell destruction within blood vessels and white blood cell and platelet activation, which may lead to blood clots. Symptoms may include fatigue, trouble swallowing, shortness of breath, chest and/or stomach pain, and other complications.
At the time, Mike had never heard of PNH. “I’m someone who likes to research, but there wasn’t much out there,” he says. “It was described as progressive—but I didn’t fully understand what that meant.” Over the years, he experienced increasing fatigue and other symptoms that made daily life unpredictable, though others can experience more serious symptoms such as blood clots.
Reclaiming Freedom Through Treatment
Everything changed when his doctor introduced him to a clinical trial for ULTOMIRIS, a complement inhibitor designed to target one of the immune system proteins that attack red blood cells in the body. No less than two weeks prior to starting treatment, Mike received meningococcal vaccinations due to the risk of severe meningococcal infections. Two weeks after an initial loading dose, ULTOMIRIS is infused intravenously every eight weeks for most adults. The most common side effects of ULTOMIRIS are upper respiratory tract infection and headache. “It was a turning point,” Mike says. “The treatment fit into my life instead of taking it over. I get an infusion every 8 weeks, and then I don’t think about it for the next 55 days. I call it my oil change.”
That freedom has allowed Mike to embrace the things he loves such as hiking, biking, and playing racket sports. He and his wife recently picked up golf— “we’re terrible,” he laughs—and in the winter, he volunteers as a ski patroller at a local resort. “It’s a great way to stay active and give back,” he says.
A Strong Support System
The most important part of Mike’s journey has been the unwavering support of his wife, Sara. “There were times I couldn’t stand in the shower. She helped me up the stairs. We put our heads together and got through it,” he says. Their partnership, now nearly 40 years strong, has been a constant source of strength. “You can’t shield your spouse from something like this, but at the same time, you don’t want to scare your kids.”
Mike and Sara raised two daughters, now grown, who are just beginning to understand the full scope of what their father endured and continues to manage. “They knew something was going on, but we didn’t make a big deal of it,” Mike says. “Now, it’s interesting to talk about it with them as adults.”
Helping Others Navigate Their Journey
Today, Mike shares his story with others through patient ambassador programs, speaking with newly diagnosed patients and caregivers. “I didn’t get lucky having this disease,” he says, “but I got lucky in how I’ve been able to live with it.”
His message to others is clear: “This is something you can live with. Don’t self-diagnose. Don’t isolate. Advocate for yourself. Ask questions. This was my experience, but know that everyone’s experience is different.”
Mike doesn’t sugarcoat the hard parts. “There were times I felt depressed. I didn’t know what the end was going to look like,” he says. “But I’ve learned that if you avoid dealing with the hard parts, it only makes life more complicated. There are treatments out there that can help.”
For Mike, ULTOMIRIS played a key role in improving his health—and once he began feeling better, he was able to embrace more of life’s meaningful moments with his family. “Other than around the time of my infusions every 8 weeks, I don’t see myself as a patient,” he says. “I don’t know what the future brings, but so far, it’s been pretty good.”
To find resources and learn more, visit ultomiris.com/pnh or reach out to OneSourceTM, Alexion’s patient support program at https://alexiononesource.com/ultomiris.
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.
- You must complete or update meningococcal vaccine(s) at least 2 weeks before your first dose of ULTOMIRIS.
- 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.
- 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.
- 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.
- 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.
ALEXION, ULTOMIRIS, and SOLIRIS are registered trademarks and OneSource is a trademark of Alexion Pharmaceuticals, Inc.
© 2025, Alexion Pharmaceuticals, Inc. All rights reserved.
US/ULT-P/0648 V1 08/2025
