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Treatments for Paroxysmal Nocturnal Hemoglobinuria

Medically Reviewed by Michael W. Smith, MD on March 24, 2021

Most treatments for paroxysmal nocturnal hemoglobinuria (PNH) help to manage symptoms. You can take medicine to prevent blood clots, boost your red blood cell count, and prevent other problems. A stem cell transplant is the only way to cure PNH, but it comes with some risks.

Which treatment you get depends on how severe your symptoms are. If you have mild symptoms that don’t bother you, you may not need any treatment. Your doctor will just watch you to see if you have symptoms later.

Your doctor will start you on treatment if you start to have problems like:

  • Bruising
  • Bleeding
  • Tiredness
  • Shortness of breath

Medicines to Protect Your Blood Cells

The main treatment for PNH is medicine to stop the breakdown of red blood cells, lower your chances of blood clots, and improve your quality of life. 

Eculizumab (Soliris) and ravulizumab (Ultomiris) work in a similar way. Both medicines come as an IV. You get Soliris once every 2 weeks and Ultomiris once every 8 weeks.

Soliris and Ultomiris control symptoms, but they don't cure PNH. You'll need to take medicine for the rest of your life, or until your disease goes into remission. 

Pegcetacoplan (Empaveli) is a targeted therapy that can be prescribed for those who are new to treatment or are switching from Soliris or Ultomiris. It is also taken by IV and is administered twice a week.

Because these drugs block part of your immune system, they can make serious infections more likely. You'll need to get the meningococcal vaccine at least 2 weeks before your first dose of Soliris or Ultomiris. Then your doctor will keep an eye on you for signs of an infection while you take the medicine.

Stem Cell Transplant

Stem cells are the early cells in your bone marrow that grow into new blood cells. The only way to cure PNH is with what's called an allogenic stem cell transplant. Also called a bone marrow transplant, it replaces your damaged stem cells with healthy ones from a donor, usually a close relative because they're often the best match.

A stem cell transplant can have serious risks, including infection and organ damage. It's only for people with very severe PNH who could have life-threatening blood clots or other serious problems.

Before the transplant, you'll get treatments to prepare your body for the new cells. Chemotherapy or radiation destroy the old damaged bone marrow so the new stem cells can grow. You get them through a long, thin tube placed in your chest or neck. The stem cells will travel to your bone marrow, where they'll grow into healthy new blood cells.

Treatments to Ease Symptoms

Your doctor may give you other treatments to ease PNH symptoms by helping your body make new blood cells.

Those can include:

  • Iron and folic acid supplements
  • Blood transfusions
  • Growth factors
  • Hormones called androgens

You might also need to take blood thinners to prevent blood clots from forming. While you're on a blood thinner, you have to be extra careful to prevent injuries because these drugs make bleeding more likely.

Medicines to Suppress Your Immune System

Some people with PNH also have aplastic anemia, which is when your bone marrow fails to produce new red blood cells because your immune system attacks the stem cells in your bone marrow. Medicines such as antithymocyte globulin (Thymoglobulin) and cyclosporine can stop your immune system from attacking your bone marrow.

Clinical Trials

Researchers are studying many new treatments for PNH in clinical trials. Joining one of these studies can give you access to a new therapy before it is available to everyone else. Ask the doctor who treats your PNH if a clinical trial might be right for you.

WebMD Medical Reference

Sources

SOURCES:

Aplastic Anemia and MDS International Foundation: "What are the Treatments for PNH?"

American Cancer Society: "Stem Cell or Bone Marrow Transplant Side Effects."

FDA: "Highlights of Prescribing Information: Soliris," "Highlights of Prescribing Information: Ultomiris."

Johns Hopkins Medicine: "Paroxysmal Nocturnal Hemoglobinuria."

Mayo Clinic: "Aplastic Anemia," "Bone Marrow Transplant."
Medscape: "Paroxysmal Nocturnal Hemoglobinuria Treatment and Management."

National Organization for Rare Disorders: "Paroxysmal Nocturnal Hemoglobinuria."

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