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Resources for PNH

Understanding more about PNH and your management plan will help you gain confidence in living with a rare condition. Here are some resources to help you continue an open communication with your healthcare team.

Learn more about PNH

When it comes to living with PNH, a good first step is education. Learn more about PNH and how to manage it with these helpful materials.

Ask your healthcare provider or contact Alexion OneSource™ about getting the ULTOMIRIS Patient Starter Kit.

Downloadable resources (PDF)

Resources for people living with PNH

It is natural to think you are alone when you are diagnosed with PNH because it is a rare disease. Communicating with others who have had similar experiences and who understand can make a difference.

The Aplastic Anemia and MDS International Foundation (AAMDSIF)

Supports, connects, and educates patients, caregivers, and health professionals on bone marrow failure diseases worldwide. It promotes and invests in collaborative clinical research to accelerate the discovery of better treatments and cures for aplastic anemia, myelodysplastic syndrome (MDS), PNH, and related bone marrow failure diseases.

Learn more
National Organization for
Rare Disorders (NORD)

A not-for-profit organization dedicated to helping people with rare disorders such as PNH.

NORD and the NORD logo are registered trademarks of the National Organization for Rare Disorders.
NORD is a registered 501(c)(3) charity organization.

Learn more

These organizations are independent nonprofit organizations. Alexion is not responsible for information they may provide.

Glossary

anemia
The condition of having a lower-than-normal number of red blood cells or amount of hemoglobin. Anemia reduces the ability of the blood to carry oxygen and is sometimes found in PNH.
blood clots
Blood clots form when parts of your body’s blood clump together. In a healthy body, this clumping can stop bleeding when you’re cut or injured, but in certain conditions, these clumps can block blood flow in the veins and arteries, which can be dangerous. In PNH, a clot can happen at any time and can cause serious health problems.
bone marrow
Soft tissue inside your large bones. Stem cells, contained in your bone marrow, work to create parts of your blood: red blood cells, white blood cells, and platelets.
breakthrough hemolysis
Breakthrough hemolysis is defined as at least 1 new or worsening symptom or sign of hemolysis that occurs along with elevated LDH levels (after LDH levels were previously reduced through treatment). Breakthrough hemolysis may lead to complications.
complement
Part of the immune system. In healthy individuals, complement is a sequence of reactions in the blood that is part of the body’s natural defense system. It helps fight against bacteria and other foreign matter in the body.
complement protein 5 (C5)
Part of the complement system. C5 usually works to destroy disease-causing pathogens like bacteria by poking holes in them. In PNH, C5 also targets red blood cells that are missing protective proteins.
FACIT-Fatigue
FACIT stands for Functional Assessment of Chronic Illness Therapy. The FACIT-Fatigue scale can be used to measure fatigue. FACIT-Fatigue scores can range from 0 to 52, with higher scores indicating less fatigue.
hemolysis
Breakdown of red blood cells. Hemolysis is the main cause of major health problems in PNH.
intravascular hemolysis (IVH)
Hemolysis that occurs inside the blood vessels. In PNH, IVH is caused by complement, including the protein C5, and is the major cause of PNH signs and symptoms.
kidney damage
Healthy kidneys clean your blood by removing excess fluid, minerals, and wastes. They also make hormones that keep your bones strong and your blood healthy. In PNH, the blood cells that burst release iron and hemoglobin into your system. As a result, blood vessels and tissues in the kidneys can get injured. This injury reduces the level at which your kidneys work.
lactate dehydrogenase (LDH)
LDH is an enzyme found in red blood cells that is released during hemolysis. Lab tests to measure LDH levels can help to assess PNH disease activity.
paroxysmal nocturnal hemoglobinuria (PNH)
A disease where red blood cells are created without certain protective proteins. This causes them to break down (a process called hemolysis) and can result in serious health problems. Signs and symptoms include stomach pain, difficulty swallowing, anemia, shortness of breath, and fatigue. Life-threatening complications from PNH include blood clots, which may lead to kidney failure and damage to your other organs.
proteins
Proteins are the building blocks of life. The body needs proteins to repair and maintain itself. In PNH, some or all red blood cells lack an important protective protein. Without this protein, PNH red blood cells are attacked by complement, part of the body’s natural defense system, resulting in hemolysis.
red blood cells (RBCs)
A type of cell found in your blood that delivers oxygen and removes waste (carbon dioxide) in your body. Red blood cells affected by PNH are attacked and destroyed because they are missing a protective protein.
white blood cells (WBCs)
A type of cell found in your blood that helps your immune system fight disease and infection.

Talk to your healthcare provider about ULTOMIRIS

Talk to your healthcare provider about long-acting ULTOMIRIS for extended control of your PNH between infusions.

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IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING

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

ULTOMIRIS is a medicine that affects your immune system and can 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, body 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 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, and are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS will harm your unborn baby or if it 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, tiredness, feeling faint, discomfort in your arms or legs, bad taste, or drowsiness. Stop treatment of ULTOMIRIS and tell your healthcare provider or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion 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 the full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious meningococcal infections.