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In the past couple of decades, researchers have developed several new ways to treat paroxysmal nocturnal hemoglobinuria (PNH). Some treatments block the processes that cause the disorder. Others help manage PNH symptoms and boost quality of life.
The wide range of PNH symptoms varies from person to person. Your symptoms help determine which treatments you’ll receive. Treatment may also be based on whether you have bone marrow failure — whether the hematopoietic stem cells (HSCs) that live in your bone marrow produce enough healthy blood cells. How well you tolerate certain side effects can also help guide your doctor’s treatment recommendations.
The following therapies may be part of your PNH treatment plan. Together, you and your doctor can determine which treatments will best help relieve your symptoms and improve your well-being.
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’ll need to attend regular follow-up visits. Your doctor may run tests to look for signs of excessive hemolysis — the breakdown of red blood cells. They’ll also check to make sure your heart and kidneys are working properly. If you develop PNH symptoms, you have blood clots, or your body no longer makes enough blood cells, you may need to begin treatment.
In PNH, hemolysis occurs when the immune system mistakenly attacks red blood cells that lack protective surface proteins. These vulnerable cells are made by abnormal hematopoietic stem cells in the bone marrow. The complement system — a part of the immune system that normally helps fight infections — then targets these red blood cells and causes them to break apart.
Several drugs are designed to block your complement system, stopping intravascular hemolysis — the process in which the immune system attacks and destroys red blood cells within blood vessels. These medications are known as complement inhibitors.
Eculizumab (Soliris) was the first PNH medication approved by the U.S. Food and Drug Administration (FDA). This monoclonal antibody (laboratory-made protein) is usually given as an intravenous (IV) infusion every two weeks at a doctor’s office or clinic. In some cases, you may be able to receive the treatment at home.
Because eculizumab weakens part of your immune system, it can increase your risk of infections, including urinary tract infections. Eculizumab also raises the risk of a life-threatening illness called meningococcal infection. To help protect you, your doctor will recommend a meningococcal vaccine before starting treatment.
The FDA has also approved biosimilar versions of eculizumab. One of them, eculizumab-aeeb (Bkemv), is considered interchangeable with eculizumab, which means a pharmacist may be able to swap it for eculizumab in some states. Eculizumab-aagh (Epysqli) is another FDA-approved biosimilar. Ask your doctor or pharmacist if a biosimilar might be right for you.
Ravulizumab (Ultomiris) was approved by the FDA as a PNH treatment in 2018. This medication works the same way as eculizumab and is similarly effective. Like eculizumab, ravulizumab is given through an IV infusion, but you don’t need it as often. Most people receive ravulizumab once every eight weeks.
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 to a doctor’s office.
Pegcetacoplan blocks a different complement protein than eculizumab and ravulizumab. While these latter two drugs prevent hemolysis only within blood vessels, pegcetacoplan also blocks extravascular hemolysis. This means it can help stop the destruction of red blood cells in locations outside the blood vessels, such as the spleen.
Pegcetacoplan may be right for you if you still have anemia (low levels of red blood cells) after trying eculizumab or ravulizumab. However, pegcetacoplan also carries a significant risk of serious infections, including meningococcal infections.
The FDA approved iptacopan (Fabhalta) in 2023. Iptacopan is the first oral drug for PNH. As a factor B inhibitor, iptacopan prevents hemolysis within blood vessels and extravascular hemolysis.
In a 2022 study, participants with PNH who received iptacopan had better results than those given eculizumab and ravulizumab. The iptacopan group had higher levels of hemoglobin (a protein found in red blood cells), needed fewer blood transfusions, and had less fatigue.
In 2024, the FDA approved crovalimab-akkz (PiaSky) to treat PNH in people 13 and older who weigh at least 88 pounds. This monoclonal antibody works in a way that’s similar to eculizumab. After an initial IV loading dose, crovalimab-akkz is given as a subcutaneous (under the skin) injection every four weeks.
In a clinical trial, crovalimab-akkz was shown to work as well as eculizumab in controlling hemolysis and reducing the need for blood transfusions.
The FDA approved danicopan (Voydeya) in 2024 as an add-on treatment to ravulizumab or eculizumab in adults with PNH who still experience extravascular hemolysis. Danicopan is a complement factor D inhibitor in tablet form. It’s taken by mouth three times a day.
Danicopan isn’t effective on its own and must be used with ravulizumab or eculizumab. In people who continue to have symptoms despite treatment with standard C5 inhibitors, danicopan may help raise red blood cell levels and reduce the need for blood transfusions.
Because danicopan affects the immune system, it raises the risk of serious infections caused by encapsulated bacteria (bacteria lined with a protective barrier), such as Neisseria meningitidis. Vaccination is required before starting the drug.
Eculizumab, ravulizumab, and pegcetacoplan all work by preventing hemolysis. However, they don’t help produce more blood cells, which is also a problem for many people with PNH.
Medications that weaken the immune system can protect HSCs from being attacked. These immunosuppressive therapies include anti-thymocyte globulin and cyclosporine. Your doctor may prescribe these if you have a bone marrow failure condition such as aplastic anemia in addition to PNH.
PNH develops due to abnormalities in hematopoietic stem cells. Replacing HSCs through bone marrow transplantation — often called stem cell transplantation — can sometimes lead to a cure for PNH.
The first step of a bone marrow transplant involves finding a donor with compatible HSCs. In cases in which an unrelated donor is needed, this search can take a long time. Next, defective HSCs must be destroyed. This is typically done using chemotherapy, radiation therapy, or immunotherapy. All these methods destroy cells throughout your bone marrow. Your doctor will then 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, the procedure sometimes fails and always comes with serious risks. For example, you may develop graft-versus-host disease, in which 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, so doctors recommend bone marrow transplants only for people with very severe PNH.
Blood transfusions don’t block the biological processes that cause PNH, but they can help manage symptoms. The procedure involves infusing blood cells from a donor into veins. This may help you feel better quickly if you have low levels of red blood cells. Also, platelet transfusions may help prevent or treat bleeding problems caused by low levels of platelets (blood cells that help form blood clots).
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, so you may need extra iron and folate. Your doctor can test your blood levels and tell you whether supplementation may help.
Your body normally makes growth factors such as erythropoietin to help spur the formation of new blood cells. If you have low red blood cell counts, your doctor may prescribe an erythropoiesis-stimulating agent (ESA) to help your body make more red blood cells. Epoetin alfa is a form of erythropoietin, and darbepoetin alfa is a related, longer-acting ESA.
If you have low levels of white blood cells, your doctor may recommend a growth factor such as granulocyte colony-stimulating factor (G-CSF), like filgrastim.
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 may help increase levels of red blood cells if you have anemia.
Anticoagulants — also called blood thinners — are medications that keep blood from clotting. PNH leads to an increased risk of thromboses (blood clots), which can cause serious health problems. However, blood thinners can help reduce this risk.
Steroids like prednisone are not a long-term treatment for PNH. They usually don’t control hemolysis or prevent clots and can cause side effects. Doctors may use them briefly in certain situations, but for most people, long-term prednisone isn’t recommended and other treatments are preferred.
Researchers regularly study potential new treatments for blood disorders like PNH. Clinical trials look into how safe and effective these therapies are. By participating in a clinical trial, you may be able to try a treatment that wouldn’t otherwise be available to you.
Right now, a bone marrow transplant is the only treatment that may cure PNH. However, it doesn’t work for everyone — and it comes with serious health risks, so it may not be the right option for you.
In general, PNH is treated as a long-term condition. Your healthcare team will recommend treatments based on your symptoms and health problems. Current therapies usually keep the condition under control, and most people with PNH can expect to live as long as someone without the condition.
On myPNHteam, people share their experiences with PNH, get advice, and find support from others who understand.
Have you tried any treatments for your PNH? Let others know in the comments below.
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