You’re taking your paroxysmal nocturnal hemoglobinuria (PNH) medication as prescribed, but you still feel exhausted, have symptoms, or need blood transfusions. Is this the best you can expect?

There are more PNH treatment options than ever before. If your treatment plan isn’t working as well as you’d hoped, you may have other choices.

Here are signs it may be time to discuss switching treatments with your PNH specialist, along with other treatment options you might consider.

🗳️ Have you ever switched treatments for paroxysmal nocturnal hemoglobinuria (PNH)?

No, my initial treatment plan is still working well for me

No, but I’d like to talk to my doctor about other treatment options

Yes, I’ve switched treatments for PNH once

Yes, I’ve switched treatments for PNH twice or more

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You’re Still Having Anemia Symptoms

PNH treatments help protect red blood cells from being destroyed, but some people continue to have anemia despite treatment. Symptoms of anemia include:

• Unusual tiredness or weakness
• Shortness of breath
• Dizziness or headaches
• Racing heartbeat
• Pale skin

If these symptoms persist or interfere with daily activities, tell your PNH specialist.

Sometimes, anemia continues because your immune system destroys red blood cells outside your blood vessels. This is called extravascular hemolysis.

Extravascular hemolysis can keep happening even when medications such as eculizumab (Soliris and its biosimilars) or ravulizumab-cwvz (Ultomiris) control red blood cell destruction inside blood vessels. Your specialist can run tests to determine whether a change to your treatment plan might help.

You Still Need Blood Transfusions

Blood transfusions can treat severe anemia. However, if you continue to need transfusions despite following your treatment schedule, your medication may not be controlling PNH well enough.

Some people taking eculizumab or ravulizumab-cwvz continue to have anemia because red blood cells are being removed by the liver and spleen.

Newer medications work differently and may reduce anemia and the need for transfusions. If you’re still receiving regular transfusions, ask your specialist about other treatment options.

You’re Experiencing Breakthrough Hemolysis

PNH treatment aims to prevent hemolysis, or the destruction of red blood cells. Sometimes, however, hemolysis suddenly returns or worsens despite treatment. This is called breakthrough hemolysis.

Breakthrough hemolysis can happen when medications called complement inhibitors don’t provide enough protection or when an infection activates your immune system. Symptoms may include:

• Sudden or worsening fatigue
• Dark or reddish-brown urine
• Shortness of breath
• Abdominal or back pain
• Difficulty swallowing

Blood tests may show a sudden or sharp decrease in hemoglobin levels, along with significantly increased levels of lactate dehydrogenase (LDH). Breakthrough hemolysis can cause severe anemia requiring transfusions. Rarely, it can cause blood clots.

A 2025 review found that breakthrough hemolysis can occur with different PNH treatments, although how often it happens and what causes it vary.

Contact your care team promptly if you develop symptoms of breakthrough hemolysis. Severe breathing difficulty, chest pain, and fainting require emergency care because they may be signs of a serious complication, such as a blood clot. Your specialist may adjust your medication schedule or recommend another treatment.

Finding the right treatment may take some trial and error, as one myPNHteam member reported: “Next month will mark a year since I started my new treatment, and I feel much better. I had previously tried other treatments, but none were effective enough to stop the severe hemolysis I was experiencing.”

“Next month will mark a year since I started my new treatment, and I feel much better. I had previously tried other treatments, but none were effective enough to stop the severe hemolysis I was experiencing.”

— A myPNHteam member

You’ve Developed a Blood Clot

Blood clots are among the most serious complications of PNH. Although treatment reduces the risk, clots can still occur. Warning signs include swelling or pain in one leg, sudden chest pain, abdominal pain, breathing difficulties, or dizziness.

Seek immediate medical attention if you suspect a blood clot. Chest pain, sudden breathing difficulty, or strokelike symptoms require emergency care.

If you develop a clot during treatment, your specialist will investigate whether your PNH is adequately controlled. A blood clot doesn’t automatically mean you need different medication, but it’s a reason to reassess treatment.

Blood Test Results Suggest Your PNH Isn’t Well Controlled

Even if you’re feeling relatively well, regular blood tests may reveal that your treatment isn’t controlling PNH well enough. Important tests include the following:

Hemoglobin

A hemoglobin test measures the level of the oxygen-carrying protein in red blood cells. Levels below about 11.6 grams per deciliter in adult women or 13.2 grams per deciliter in adult men may indicate anemia, according to Mayo Clinic. Persistent low levels warrant investigation.

Lactate Dehydrogenase

LDH is an enzyme that increases in the blood when red blood cells break apart. Levels above the laboratory’s upper limit of normal may suggest inadequate control of hemolysis.

Reticulocyte Count

Reticulocytes are young red blood cells. High levels may indicate your body is replacing destroyed red blood cells. Unexpectedly low levels in someone with anemia may signal reduced blood cell production.

Bilirubin

This substance forms when red blood cells break down. Elevated levels may indicate ongoing hemolysis, although other conditions can also cause them.

Flow Cytometry

Flow cytometry is currently the gold standard for diagnosing PNH. This test identifies blood cells missing protective proteins called CD55 and CD59 and measures the percentage of cells affected by PNH. Your specialist may repeat it to monitor changes in these cell populations over time.

Your specialist may also check iron levels and other markers. No single result automatically means you need to switch treatments. Your doctor will consider trends in your test results, symptoms, and other possible causes.

Your Treatment Is Difficult To Keep Up With

Some PNH treatments require infusion appointments, whereas others involve injections or pills taken at home.

Even the most effective medication doesn’t work well if you can’t take it consistently. People may find it challenging to take their PNH medication as directed for many reasons, such as the following:

• Medication or copays are too expensive.
• Side effects are difficult to tolerate.
• Traveling to an infusion center is inconvenient.
• Appointments interfere with work or family responsibilities.
• Medication schedules are difficult to remember.

If you face any of these challenges, tell your PNH specialist. They may be able to help manage side effects, find financial assistance, or simplify your treatment routine.

If these solutions aren’t enough, another medication may fit your lifestyle better and make it easier to stay on treatment.

What Other PNH Treatments Could You Consider?

If your current treatment plan isn’t working well enough, your specialist may recommend a medication that targets a different part of the immune system.

Eculizumab, ravulizumab-cwvz, and crovalimab-akkz (PiaSky) block a protein called C5 to prevent red blood cells from breaking apart inside blood vessels. However, they don’t always prevent red blood cell destruction outside blood vessels.

Newer medications target other proteins in the complement system, a part of the immune system involved in PNH.

Pegcetacoplan

Pegcetacoplan (Empaveli) targets a protein called C3, helping prevent red blood cell destruction inside and outside blood vessels.

In the PEGASUS trial, people who switched from eculizumab to pegcetacoplan had greater hemoglobin improvements than those who continued eculizumab.

Pegcetacoplan may be an option if you’re taking ravulizumab-cwvz or eculizumab but still experiencing anemia or needing transfusions.

Although ravulizumab-cwvz is typically given through an IV every eight weeks in adults, pegcetacoplan is administered through an infusion under the skin, generally twice weekly.

Iptacopan

Iptacopan (Fabhalta) blocks a protein called factor B, helping prevent red blood cell destruction through a different part of the complement system.

In the APPLY-PNH trial, people with persistent anemia who switched from a C5 inhibitor to iptacopan had greater hemoglobin improvements and were more likely to avoid transfusions than those who continued their C5 inhibitor.

Iptacopan is a pill taken twice daily, making it worth discussing if you’d prefer oral medication over infusions or injections. However, you’ll need to remember to take it every morning and evening.

Danicopan

Switching treatments isn’t always necessary. Sometimes, adding a medication can help.

Danicopan (Voydeya) blocks factor D, another protein involved in red blood cell destruction. This medication is approved for use alongside eculizumab or ravulizumab-cwvz in adults with PNH who have extravascular hemolysis.

You continue your existing medication while taking danicopan three times daily. This combination may improve anemia when your treatment isn’t adequately preventing red blood cell destruction outside blood vessels.

What About the Risks of Switching?

Like all medications, each PNH treatment has potential benefits and potential risks.

Complement inhibitors can increase the risk of serious infections, particularly those caused by certain bacteria. Depending on the medication, you may need vaccinations against meningococcal, pneumococcal, or Haemophilus influenzae type b infections. Vaccination reduces but doesn’t eliminate the risk of infection.

If you decide to switch treatments, your PNH specialist will consider side effects, medication interactions, and ways to make the transition safely.

Never stop or change your PNH medication without medical guidance. Interrupting treatment can cause severe hemolysis and other complications.

Finding a Treatment That Fits Your Life

Your PNH treatment should do more than improve blood test results. Ideally, it should help you feel better and make daily life easier. Tell your specialist what matters most, whether that’s having more energy, avoiding transfusions, managing side effects, or spending less time at appointments.

Ask what improvements you can expect and how long they might take, and discuss the risks of switching. Your care team may also suggest ways to manage side effects or simplify your treatment routine. Together, you can decide whether it’s time for a change and choose a plan that supports your health and goals.