In the past couple of decades, researchers have developed multiple new treatments for paroxysmal nocturnal hemoglobinuria (PNH). Some PNH treatments block the processes that cause the disorder. Others help manage PNH symptoms and boost your quality of life.

PNH causes a wide range of symptoms that vary from person to person. Your symptoms help determine which treatments you will receive. Your treatment may also be based on whether you have bone marrow failure — in other words, whether the hematopoietic stem cells (HSCs) that live in your bone marrow can produce enough healthy blood cells.

The following therapies may be part of a PNH treatment plan that helps relieve your symptoms and improve your well-being. You can discuss the possible benefits and side effects with your doctor.

1. Watchful Waiting

Some people with PNH don’t need any treatment when they’re first diagnosed. Your doctor may recommend waiting to start treatment if you have very mild symptoms and your body is producing enough healthy blood cells.

When using a watchful waiting approach, you will need to attend regular follow-up visits. During these visits, your doctors may run tests to look for signs of hemolysis (the destruction of red blood cells). They will also check to make sure your heart and kidneys are working properly. If you develop PNH symptoms or your body can no longer make enough blood cells, you may need to begin treatment.

2. Complement Inhibitor Medications

PNH causes your immune system to destroy your red blood cells in a process known as hemolysis. In PNH, abnormal hematopoietic stem cells in the bone marrow make defective red blood cells. These red blood cells are targeted and broken apart by the complement system, a part of your immune system that helps protect you against infection.

Several drugs are designed to block your complement system, stopping intravascular hemolysis — the process in which your immune system attacks and destroys red blood cells within your blood vessels. These medications are known as complement inhibitors.

Eculizumab

Eculizumab (Soliris) was the first medication approved for the treatment of PNH by the U.S. Food and Drug Administration (FDA). This monoclonal antibody (laboratory-made protein) is given as an IV infusion every two weeks. You usually need to go to a doctor’s office or clinic to receive an eculizumab infusion, but in some cases, you may be able to receive treatment at home.

Eculizumab also blocks aspects of your immune system you need to fight certain infections. If you use this medication, you will have an increased risk of urinary tract infections, gastrointestinal infections, and respiratory infections. Eculizumab also raises your risk of a certain type of life-threatening bacterial infection called a meningococcal infection. For this reason, it’s important to get a vaccine against this illness before you start using eculizumab.

The FDA has also approved biosimilars for eculizumab. This means they’re considered interchangeable with Soliris. Approved biosimilars for eculizumab include:

• Eculizumab-aeeb (Bkemv)
• Eculizumab-aagh (Epysqli)

Ravulizumab

Ravulizumab (Ultomiris) was approved by the FDA as a PNH treatment in 2018. This medication works in the same way as eculizumab and has a similar rate of effectiveness. Ravulizumab is also given as an IV infusion. However, it can be taken less often. You will need to receive a dose of ravulizumab once every eight weeks.

Pegcetacoplan

Pegcetacoplan (Empaveli) was approved by the FDA in 2021. This drug is also given as an infusion under the skin. However, you give yourself the infusion rather than going into a doctor’s office.

Pegcetacoplan blocks a different complement system protein compared to eculizumab and ravulizumab. While these latter two drugs only prevent hemolysis within your blood vessels, pegcetacoplan can also block extravascular hemolysis (the destruction of red blood cells in locations outside of your blood vessels, such as your spleen).

Pegcetacoplan may work well if you still have anemia (low levels of red blood cells) after trying eculizumab or ravulizumab.

Iptacopan

The FDA approved iptacopan (Fabhalta) in 2023. Iptacopan is the first oral drug for PNH. It is a factor B inhibitor. Iptacopan prevents hemolysis within your blood vessels and extravascular hemolysis.

In a 2022 study, participants with PNH had better results when they used iptacopan compared to eculizumab and ravulizumab. They had higher levels of hemoglobin (a protein found in red blood cells), didn’t need as many blood transfusions, and had less fatigue.

Crovalimab-akkz

In 2024, the FDA approved crovalimab-akkz (PiaSky) to treat PNH in people aged 13 and older who weigh at least 88 pounds (40 kilograms). Crovalimab-akkz is a monoclonal antibody that works in a similar way as eculizumab. Crovalimab-akkz is administered as a subcutaneous (under the skin) injection every four weeks, after loading doses.

In a clinical trial, crovalimab-akkz or eculizumab was given to study participants, and results were compared. Crovalimab-akkz was found to work as well as eculizumab to control hemolysis and prevent the need for blood transfusions.

3. Immunosuppressive Therapy

Eculizumab, ravulizumab, and pegcetacoplan all work by preventing hemolysis. However, they don’t help your HSCs produce more blood cells, which is also a problem for many who have PNH.

Medications that weaken your immune system can protect your HSCs from being attacked. These immunosuppressive therapies include antithymocyte globulin (Thymoglobulin) and cyclosporine (Gengraf). Your doctor may prescribe these if you have a bone marrow failure condition such as aplastic anemia in addition to PNH.

4. Bone Marrow Transplant

PNH occurs due to abnormalities in your HSCs. Therefore, replacing these cells through bone marrow transplantation can sometimes lead to a cure for PNH.

The first step of a bone marrow transplant is finding a donor whose HSCs are compatible. This can take a long time in some cases. Next, it’s necessary to destroy your defective HSCs. This is usually accomplished with chemotherapy, radiation therapy, or immunotherapy. All of these methods destroy cells throughout your bone marrow. Next, your doctor will give you an infusion of healthy HSCs from the donor. The new HSCs will travel to your bone marrow and start making healthy blood cells.

A transplant works well for some and can even be a cure for PNH. However, it sometimes fails, and it always comes with large risks. You may develop other conditions such as graft-versus-host disease, in which your newly transplanted HSCs attack other tissues in your body. Other potential side effects include infection, infertility, organ damage, and cancer. Some of these side effects can be life-threatening. For this reason, doctors only recommend bone marrow transplantation for people with very severe PNH.

5. Blood Transfusions

Blood transfusions don’t block the biological processes that cause PNH, but they can help manage your symptoms. During a blood transfusion, blood cells from a donor are infused into your veins. This may help you feel better quickly if you have low levels of red blood cells. Additionally, blood transfusions may be able to prevent or treat bleeding problems caused by low levels of platelets, the cells that help form blood clots.

6. Dietary Supplements

If your blood levels of iron or folate (also called folic acid or vitamin B9) fall too low, taking supplements of these nutrients may help improve your red blood cell levels.

Your body needs iron and folate to make new red blood cells. Most people get enough of these nutrients from their diet. When you have PNH, however, your body needs to make many red blood cells to make up for the ones that were destroyed. In this case, your body may need extra iron and folate. Your doctor can test your blood levels and tell you whether supplementation may help.

7. Blood Cell Growth Factors

Your body normally makes growth factors such as erythropoietin (EPO) to help spur the formation of new blood cells. If you have low red blood cell counts, your doctor may prescribe laboratory-made EPO, sold under brand names like Epogen and Aranesp, to encourage your HSCs to make more red blood cells.

If you have low levels of white blood cells, your doctor may recommend taking other growth factors, including granulocyte colony-stimulating factor or G-CSF (Neupogen).

8. Androgens

Like growth factors, androgens are natural substances found in your body. Your body uses androgen hormones while producing red blood cells. Androgen medications such as danazol (Danocrine) may help increase levels of red blood cells if you have anemia.

9. Blood Thinners

Anticoagulants, also called blood thinners, are medications that keep your blood from clotting. PNH leads to an increased chance of developing blood clots, which can cause serious health risks. However, blood thinners can help reduce this risk.

10. Steroids

Steroids like prednisone may help eliminate hemolysis and restore red blood cell levels. While many people take steroids for PNH, doctors are starting to avoid prescribing them. Steroids don’t always help, and they can lead to side effects when they are used for long periods of time.

11. Clinical Trials

Researchers regularly study potential new treatments for blood disorders like PNH. Clinical trials measure the safety and effectiveness of these therapies. By participating in a clinical trial, you may be able to try a new treatment option that you wouldn’t otherwise be able to receive from your doctor.

Can PNH Be Cured?

There is only one kind of treatment that might cure PNH: a bone marrow transplant. However, this treatment won’t cure PNH for everyone, and it has serious health risks.

In general, PNH is treated as a chronic condition. Your health care team will recommend treatments based on your symptoms and health problems. Current therapies are usually good at keeping your condition under control, and most people with PNH can expect to live as long as someone without the condition.

Talk With Others Who Understand

On myPNHteam, the site for people with paroxysmal nocturnal hemoglobinuria and their loved ones, members come together to ask questions, offer advice, and share their stories with others who understand life with PNH.

Have you tried any treatments for your PNH? What has been most helpful for you? Share your experience in the comments below, or start a conversation on your Activities page.