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Paroxysmal nocturnal hemoglobinuria (PNH) happens when your immune system attacks your red blood cells and platelets. If not treated, it can lead to complications that include blood clots, anemia, and chronic kidney disease. 

What Are the Current Treatments for PNH? 

The only potential cure is a bone marrow transplant, a procedure in which radiation or drug therapy is used to destroy your existing marrow and replace it with marrow from a donor. These new stem cells will grow into healthy red blood cells. 

But this procedure doesn’t work well for everyone. Doctors usually recommend it for very young people whose marrow has failed. 

The first treatments you’ll typically try are meant to ease symptoms and include: 

  • Watchful waiting to see if mild symptoms get worse
  • Supportive care, such as iron supplements or blood transfusions
  • Blood thinners to prevent clots 

Immunosuppressive Therapy for PNH

The trouble in PNH comes from part of your immune system called the complement system. It destroys red blood cells when you have PNH. The most common medicines work by inhibiting, or blocking, a couple of different proteins in your blood that make you lose red blood cells. Eculizumab (Soliris) and ravulizumab-cwvz (Ultomiris) are inhibitors that work to block complement protein C5. Pegcetacoplan (Empaveli) is an inhibitor that works against another protein called C3.

You can get medicine through an IV at regular intervals. With these treatments, you already can expect to live about as long as if you didn’t have PNH. 

How Can PNH Treatment Get Better in the Future?

There’s still room to make treatment even better. Researchers are working to expand your options. They’re working on medicines that would be easier to take. They also hope to come up with medicines that work even better and for more people, so you can live life to the fullest with PNH.

You might be able to get these new medicines with a shot you give yourself. You may be able to treat your PNH in the future with a pill or with a combination of an IV medicine and one you could take by mouth. So treatment for PNH might be easier in the future than it is now.

What About New PNH Treatments or Combinations?

Another way researchers are trying to make PNH treatment better is by looking at new medicines and combinations of medicines. 

There’s another complement protein called C6. Researchers are at work now to develop an inhibitor drug for it. But it’s possible that medicines in the future could hit even more targets. Doctors might put different medicines together to make them work better. For example, it might work to target both C3 and C5 proteins at the same time.

Researchers are also looking at medicines that work against other players in the complement system. Iptacopan is a drug you take by mouth that raises hemoglobin levels by binding to and blocking one called factor B. 

Another, danicopan, is a factor D inhibitor. It’s still being studied, but you would take it by mouth as an add-on to C5 inhibitor therapy with eculizumab or ravulizumab if you still have serious red blood cell damage. 

The hope is that different medicines or combinations of medicines could control PNH in people when existing medicines aren’t enough.

What About Risks and Benefits of Future Treatments?

One thing to keep in mind is that medicines have already made a big difference when you have PNH. New medicines also may come with added risks. Medicines that work better for PNH might make infections more likely. That’s because they target your immune system. Taking two medicines together likely would have more side effects and lead to a greater chance of infections. 

So, researchers and doctors will need to weigh these risks and benefits carefully. They’ll do this through studies called clinical trials. New treatments or combination treatments also might cost more. If you’re worried about paying for your PNH treatment, ask if there’s help available.

Can I Take Part in a Clinical Trial for PNH Treatment?

Clinical trials are now testing new PNH medicines and combinations. They have different goals, depending on what phase they’re in. Some only look to see if a medicine is safe. Others will look to see if a new drug works better than ones doctors already have. There’s no guarantee a new medicine will work for you or that it will work better than what’s already available.

If you’re interested in exploring a trial, ask your doctor what trials in PNH are available near you and whether you might be a good candidate. Each trial will have lots of criteria that define who can be in them. If you’re eligible, you’ll want to ask a lot of questions before you decide if you want to be in a trial. 

How Can I Find a Clinical Trial?

To find trials, you can search at Enter “PNH” into the condition or disease field. To narrow it down to trials that are enrolling people right now, click “recruiting” under status and then hit the “apply” button. 

From there, you can see the trials available and what they’re testing. Check to see where the trial is enrolling people. Some of them will be in other places in the world. Also check the eligibility criteria to get an idea if any of them might be an option for you. In the meantime, talk to your doctor about your treatment options and any questions or concerns you have.

Show Sources

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National Institute on Aging: “What Are Clinical Trials and Studies?” “Find a study.” “Iptacopan.” 

Haematologica: “Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria.”

Blood Advances: “Development of an anti-human complement C6 monoclonal antibody that inhibits the assembly of membrane attack complexes.”

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