Life With PNH: What to Expect

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that causes red blood cells to break down prematurely, leading to episodes of hemoglobinuria (dark red or brown urine). PNH is a chronic condition, but it can be managed with treatment to reduce symptoms and prevent complications.

What are the symptoms of PNH?

The most common symptoms of PNH include:

- Episodes of dark red or brown urine (hemoglobinuria)

- Fatigue (extreme tiredness)

- Weakness

- Shortness of breath

- Abdominal pain

- Nausea

- Vomiting

Other symptoms of PNH can include:

- Skin rash

- Joint pain

- Headaches

- Back pain

- Blood clots

- Kidney failure

What are the treatments for PNH?

There are several treatments available for PNH, including:

- Eculizumab (Soliris): This medication is approved by the US Food and Drug Administration (FDA) to treat PNH. Eculizumab is an antibody that blocks a certain protein from binding to red blood cells, which helps to prevent them from breaking down prematurely.

- Rapastinel (Ultomiris): This medication is also FDA-approved for treating PNH. Rapastinel is given through an injection every two weeks. It helps to block the complement cascade, which is a part of the immune system that can lead to the destruction of red blood cells.

- Stem cell transplant: This procedure can help to cure PNH in some cases. A stem cell transplant involves removing healthy stem cells from a donor and infusing them into the patient.

- Blood transfusions: Blood transfusions may be needed to replace red blood cells that have been destroyed.

- Other treatments: Other treatments for PNH can include medications to treat symptoms such as pain, fatigue, and nausea.

What is the prognosis for PNH?

The prognosis for PNH has improved significantly with the development of new treatments such as eculizumab and rapastinel. With proper treatment, most people with PNH can live a normal life.

How can I manage PNH?

In addition to following your doctor's treatment plan, there are a few things you can do to manage your PNH:

- Get regular exercise.

- Eat a healthy diet.

- Get enough sleep.

- Avoid stress.

- Quit smoking.

- Avoid exposure to chemicals and toxins.

By following these tips, you can help to reduce your symptoms and improve your overall health.