What does PNH mean?
Living with a rare disease like paroxysmal nocturnal hemoglobinuria (PNH) can be a challenge, but the more you know about PNH, the better you may be able to manage it.
What does PNH mean?
A sudden attack
Even though hemolysis due to PNH is always occurring, symptoms can worsen from time to time. This worsening happens when the immune system is more active, like during an infection or illness.
The part of the immune system that causes hemolysis is more active at night, so symptoms like hemoglobinuria are often most obvious in the morning. However, hemolysis is happening at some level in people with PNH all the time, night and day.
Hemoglobin in the urine
Red blood cells (RBCs) destroyed by hemolysis release a dark red protein called hemoglobin. The body gets rid of the hemoglobin in the urine, which turns reddish or very dark. Not everyone with PNH has hemoglobinuria.
How C5 and intravascular hemolysis cause
PNH red blood cells (RBCs) are vulnerable to attack by complement protein C5
In PNH, a change in the bone marrow causes some RBCs to be made without important protective proteins on their surface.
Without these proteins, the PNH RBCs are vulnerable to a part of your body’s immune system called complement, including a protein within the complement system called C5.
C5 plays a key role in intravascular hemolysis (IVH)
C5 usually works to destroy disease-causing pathogens like bacteria by poking holes in them.
In PNH, C5 also targets RBCs that are missing protective proteins.
Uncontrolled C5 destroys PNH red blood cells inside blood vessels in a process called IVH.
IVH causes PNH symptoms and other effects
IVH is the main cause of PNH symptoms and can lead to blood clots and other PNH-related effects in the body, such as organ damage.
If you have PNH, you are at constant risk of C5-driven IVH.
RBC=red blood cell.
Did you know?
per million people are diagnosed in the
United States each year.
What is clone size?
Clone size, which can be measured by high-sensitivity flow cytometry, is the percentage of blood cells in your body that have been affected by PNH and, therefore, do not have the protective proteins that blood cells usually have on the surface.
Many of your blood cells may be normal, but anyone with PNH will have some clones. A larger clone size means you have more blood cells that are missing protective proteins. But even small clone sizes can lead to PNH-related health problems—a small clone size does not necessarily mean that you have “less PNH.” Your clone size may change over time, and symptoms can get worse over time if PNH is left unmanaged. That is why continued monitoring and management are very important.
The difference between RBC and white blood cell (WBC) clone sizes can indicate the extent of hemolysis
PNH can be tough to identify, since its symptoms are similar to those of other diseases.
You may not see what’s happening below the surface, and even when you feel fine, lab results can reveal potential problems. The signs and symptoms of PNH may not always be obvious, so it’s important to discuss what you notice with your doctor.
Symptoms you may see
Signs you may not see
Common signs and symptoms of PNH
Symptoms you may see or feel
- Dark-colored urine
- Shortness of breath
- Difficulty swallowing
- Yellowing of the skin and eyes
- Erectile dysfunction (ED)
Signs you may not see or feel
- Blood clots
- Kidney disease
- Damage to your organs
- Heart attack
Your doctor will consider all test results, signs, and symptoms
How can I help my doctor monitor my PNH?
Track your signs, symptoms, and lab results. They will show you and your doctor how you are physically affected by PNH.
Be sure to keep track of changes in your symptoms. Monitoring your symptoms is important, since PNH can manifest in serious ways. It can cause blood clots, which block veins and arteries and can lead to heart attack, stroke, and damage to your organs, as well as other problems.
Stay in touch with your physician about your PNH. Make sure you are on the same page and getting the most out of your treatment experience.
You don’t have to accept feeling sick
When you deal with PNH every day, over time, you may learn to cope with your symptoms. But it doesn’t have to be that way. You don’t have to accept feeling sick. Your doctor can seek a treatment that is appropriate for you. That is why it is important to track your signs and symptoms: so you can tell if they’re getting worse over time instead of just accepting them. Talk to your doctor about treatment options—you shouldn’t have to feel like feeling sick is normal.
Speaking with your doctor is key to successful management of PNH
Asking questions will keep you informed.
Here are a few you might want to ask your doctor:
- Can my disease
- How will I know if
my PNH is getting
worse or better?
- I would like a copy of my lab
results. Would you please
help me understand them?
Talk to your doctor about ULTOMIRIS
Talk to your doctor about long-acting ULTOMIRIS for extended control of your PNH between infusions.FIND OUT MORE
PNH may be rare, but you’re not alone
OneSource™ can help you learn about PNH, co-verify your insurance coverage, and identify helpful resources for people living with PNH and those who care for them.LEARN MORE ABOUT ONESOURCE
IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING
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 and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.
- You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you are not vaccinated.
- If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.
- If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
- If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.
- Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms and eyes sensitive to light.
Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.
ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor must: enroll in the ULTOMIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.
ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with ULTOMIRIS. Call your doctor right away if you have any new signs or symptoms of infection.
Who should not receive ULTOMIRIS?
Do not receive ULTOMIRIS if you have a meningococcal infection or have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed.
Before you receive ULTOMIRIS, tell your doctor 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 doctor 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 doctor 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, feeling faint or discomfort in your arms or legs. Tell your doctor 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 doctor 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 doctor or pharmacist. Call your doctor 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.
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.