Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by a mutation in the PIGA gene. This mutation causes defective hematopoietic stem cells, which develop into defective red blood cells. A part of your immune system known as the complement system breaks down the defective red blood cells prematurely.

When your complement system breaks down red blood cells, your body releases hemoglobin in your urine. Hemoglobin is a component of red blood cells. It can cause your urine to appear red or dark colored, especially when you urinate after waking up.

PNH raises your risk of potentially life threatening blood clots and bone marrow failure disorders, including acquired aplastic anemia (AA) and myelodysplastic syndromes (MDS). These disorders cause low blood cell counts.

Multiple treatments are available to manage symptoms and complications of PNH. Read on to learn about your treatment options.

Your doctor may prescribe one or more of the treatments below to manage symptoms and complications of PNH.

Complement inhibitors

The Food and Drug Administration (FDA) has approved multiple medications to treat PNH:

  • eculizumab (Soliris), approved in 2007
  • ravulizumab (Ultomiris), approved in 2018
  • pegcetacoplan (Empaveli), approved in 2021

These medications are complement inhibitors that block your complement system from breaking down red blood cells. They don’t cure PNH, but they may reduce symptoms, lower your risk of complications, and improve your quality of life.

Researchers are also developing new types of complement inhibitors, some of which are being tested in clinical trials. You can learn more about clinical trials for PNH at

Taking a complement inhibitor may raise your risk of infection, including serious meningococcal infection. Before you start taking a complement inhibitor, your doctor will advise you to get the meningococcal vaccine.

Medication to prevent and treat blood clots

To prevent blood clot formation, your doctor might prescribe anticoagulation therapy. This type of medication is also known as a blood thinner.

If you develop signs or symptoms of a blood clot, your doctor may prescribe medication to help break it down. This is known as thrombolytic therapy.

Multiple types of blood thinners and thrombolytic therapies are available.

If your doctor prescribes any of these medications, they will monitor you closely for potential side effects, including excessive bleeding.


If you develop severe bone marrow failure, your doctor might prescribe immunosuppressive medication to reduce the activity of your immune system.

For example, they may prescribe antithymocyte globulin, cyclosporin, or both. These medications don’t cure PNH, but they may improve your bone marrow function.

Among other potential side effects, they may raise your risk of infections. Your doctor can help you learn how to manage such a risk.

Synthetic growth factors

If you have low blood cell levels, your doctor might prescribe synthetic growth factors, such as:

  • erythropoietin (EPO, Epogen, Procrit, and Aranesp)
  • granulocyte-colony stimulating factor (G-CSF, Neupogen, Granix, and Zarxio)

These are human-made versions of hormones that stimulate blood cell production.

Erythropoietin stimulates blood cell production, which may help reduce symptoms of anemia.

Granulocyte-colony stimulating factor stimulates white blood cell production, which may help prevent excessive bleeding.

Androgen therapy

Androgen therapy is another potential treatment for low red blood cell count. Androgen is a sex hormone that stimulates red blood cell production.

Blood transfusions

Many people with PNH require at least one blood transfusion to increase their blood cell count.

In this procedure, you receive donor blood cells to replace blood cells that your body has destroyed or supplement blood cells that it has produced.

You may receive red blood cell transfusions to treat anemia, platelet transfusions to prevent or treat serious bleeding, or both. You may also receive antibiotics to prevent and treat infections.

Receiving multiple red blood cell transfusions raises your risk of iron overload, which happens when you have too much iron in your body. If you develop iron overload, your doctor may prescribe iron chelation therapy to treat it. This type of medication removes excess iron from your body.

Bone marrow transplant

If you have PNH with severe AA or MDS, your doctor might recommend an allogenic bone marrow transplant.

In this procedure, your doctor uses chemotherapy, radiation therapy, or both to destroy your bone marrow. Then they replace it with bone marrow from a healthy donor.

Bone marrow transplant can potentially cure PNH, but it carries a high risk of potentially life threatening side effects. Your doctor can help you decide whether this is a suitable treatment option for you.

It’s not safe to use alternative or complementary treatments alone to manage PNH.

However, your doctor may recommend certain complementary treatments along with medication, blood transfusions, or other conventional treatments.

For example, they may recommend iron supplements, folate supplements, or both to support red blood cell production. Folate is also known as folic acid or vitamin B9.

Your doctor may also encourage you to eat more foods that are rich in these nutrients.

The only known cure for PNH is an allogeneic bone marrow transplant that uses new, healthy stem cells from the bone marrow of a related donor (who is not an identical twin of the patient) or from an unrelated donor who is genetically similar to the patient.

Due to the high risk of side effects, your doctor will only recommend this treatment if you develop serious complications from PNH, particularly severe bone marrow failure.

Other treatments for PNH won’t cure the condition but may reduce symptoms and lower your risk of complications.

In most cases, treatment is needed to manage PNH. Getting treatment can help reduce symptoms and lower your risk of potentially life threatening complications.

In rare cases, PNH goes into spontaneous long-term remission without treatment. When this happens, symptoms resolve and defective red blood cells are no longer detectable in blood tests.

If you have PNH that goes into remission, it’s important to attend regular checkups with your doctor. They will monitor you for changes in your condition.

A small study from 2016 found that in some cases, people who showed signs of spontaneous remission from PNH went on to develop leukemia. The cancer occurs more frequently in people with PNH, AA, or MDS.

PNH can cause fatigue and other symptoms that may affect your quality of life, even if you’re receiving treatment.

If you experience changes in your symptoms or find it difficult to manage your symptoms, let your doctor know. They may recommend changes to your treatment plan to improve symptom management.

Adjusting your daily habits may also help you manage fatigue and promote good overall health.

For example, try to:

  • Get enough high quality sleep.
  • Get regular, moderate-intensity exercise.
  • Limit or avoid alcohol and avoid smoking tobacco.
  • Eat a well-balanced and nutrient-rich diet, including foods rich in iron and folate.
  • Schedule important activities for times of the day when you tend to have more energy.
  • Limit stressors, set realistic expectations for yourself, and take breaks when needed.

If living with PNH is negatively affecting your mood or mental health, consider asking your doctor for a referral to a mental health specialist who has experience supporting people with chronic illness.

You might also find it helpful to connect with other people living with PNH through an online or virtual support program. For example, the Aplastic Anemia & MDS International Foundation operates a peer support network and support groups for people living with AA, MDS, and PNH.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by defective bone marrow stem cells that produce abnormal red blood cells that rupture and release excess hemoglobin into circulation.

To manage symptoms and potential complications of PNH, your doctor may prescribe a combination of medications, blood transfusions, and other treatments. They may also recommend nutritional supplements, changes to your diet, or other lifestyle changes to help manage symptoms and promote good overall health.

If you develop severe bone marrow failure, they may recommend a bone marrow transplant. This is the only known cure for PNH, but it carries a high risk of severe side effects.

Other treatments won’t cure PNH but can help limit symptoms and reduce your risk of complications.

Talk with your doctor to learn more about the benefits and risks of different treatment approaches.