How Is Paroxysmal Nocturnal Hemoglobinuria Treated?

Even though paroxysmal nocturnal hemoglobinuria (PNH) is rare, there are effective treatments—some of which can potentially cure the condition. However, the treatments have risks that people with the condition need to know about.

This article will review the current treatments for PNH, as well as possible options for future therapies.

Home Remedies and Lifestyle

There are no home remedies or lifestyle changes that are recommended to treat PNH. That said, there are some steps that you can take to manage the condition and the symptoms you have, such as:

• Avoiding hormonal birth control pills
• Taking precautions in high altitudes or during air travel to prevent blood clots
• Reducing stress
• Staying up to date on vaccinations

If you have PNH, you may have fewer red blood cells, white blood cells, and platelets than normal, which can put you at risk for health consequences. You will need to take steps to protect yourself from infections and blood clots.

If you are taking blood thinners as a part of your treatment for PNH, you will need to take steps to prevent excessive bleeding.

Over-the-Counter (OTC) Therapies

There are no over-the-counter (OTC) remedies or medications that are approved to treat PNH. If you have PNH, you'll need to discuss your treatment options with your doctor, as well as the steps that you can take in your daily life to manage the condition and any symptoms that you experience.

Prescriptions

The two prescription medications that have been approved by the Food and Drug Administration (FDA) to treat PNH are:

• Soliris (eculizumab) is a medication that is created using monoclonal antibodies that can block your immune system's efforts to attack and destroy your red blood cells. Soliris is given as an intravenous (IV) medication, usually over the course of 35 minutes. You might need to be observed for another hour after you receive the medication to watch for any adverse reactions. People who are treated with Soliris usually receive one infusion each week for five weeks, and then one infusion every other week. The dose might need to be increased over time.
• Ultomiris (ravulizumab) is also a monoclonal antibody medication. Like Soliris, Ultomiris blocks your immune system from destroying or damaging your red blood cells. Ultomiris is given over the course of two to four hours as an intravenous (IV) medication by a doctor or nurse in a medical facility. People treated with Ultomiris usually have a second dose two weeks after the first, and then subsequent infusions every eight weeks. The dose that you receive is based on how much you weigh.

While both medications are effective in interrupting the process that leads to PNH, there are risks involved with the treatments. The medications can increase your risk of certain infections, including meningococcal disease and infections with the Neisseria genus of bacteria that target your respiratory system.

Your doctor will determine the best treatment option for you and make sure that you understand how to reduce your risk of infection, including taking prophylactic antibiotics and adhering to a strict vaccination schedule.

Based on your symptoms or any complications of PNH that you develop, your doctor might also want you to take other medications, including:

• Steroids to suppress your immune system
• Iron supplements to combat anemia
• Anticoagulants or other blood-thinning medications to prevent blood clots
  

Surgeries and Specialist-Driven Procedures

The only curative treatment for PNH is bone marrow transplantation. Some people with PNH can be treated with an allogeneic bone marrow transplant.

For this type of transplant, your own marrow is destroyed using chemotherapy, radiation, or both. Healthy bone marrow is obtained from a donor and transplanted into you through an IV. As the new stem cells migrate to bone marrow, they will start to produce new blood cells that do not have the genetic mutation that causes PNH.

The best-case scenario is to identify an identical twin or a sibling with the same bone marrow type as a person with PNH. However, for many people with the condition, an unrelated matching donor must be found.

People who are not eligible for bone marrow transplants and still require therapy beyond the available medications may receive blood transfusions. While the transfusions are not curative and will not fix the underlying cause of PNH, they can help address problems that have been caused by the destruction of blood cells.

Complementary and Alternative Medicine (CAM)

Complementary and alternative medicine (CAM) therapies are not used to treat PNH. Even though CAM therapies are not therapeutic or curative if you have PNH, taking steps to improve your overall health can still be beneficial. One aspect of managing the condition is avoiding triggers.

Potential triggers for PNH symptoms that you should avoid include:

• Stress
• Exercise
• Alcohol
• Infection
• Certain medications

Summary

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic condition that affects the blood cells in your body. It requires treatment to prevent complications, including death.

There are medications that can help treat the symptoms of the condition, but they come with risks and do not cure PNH. A special type of bone marrow transplantation can be curative, but it is very risky and may not be possible or advisable for every patient with PNH.

A Word From Verywell

If you are diagnosed with PNH, your doctor will talk to you about your options for treatment. There are no OTC or alternative treatments that are approved for PNH, but there are two prescription medications. However, each comes with serious risks.

The only potential cure for PNH is a special type of bone marrow transplant, but it carries significant risks as well. Therefore, it's only used for people with very severe cases of PNH.