Although researchers haven’t found a cure for atypical hemolytic uremic syndrome (aHUS), there are some effective ways to manage it.
The treatment that works best for you will depend on what caused your aHUS and how severe your disease is. Mild forms of aHUS may only need occasional blood transfusions and blood pressure medications to prevent high blood pressure (hypertension).
For other cases, you need treatment that more regularly boosts your red blood cell and platelet counts and protects your kidneys.
Medications for aHUS
The FDA has approved three monoclonal antibodies to treat atypical HUS caused by genetic mutations:
Eculizumab (Soliris). This has been a standard aHUS treatment since its approval in 2011. Kids as young as 2 months can take it. You get it as an infusion at a hospital or clinic. It takes about 35 minutes to get one dose. Doctors use it as a first-line treatment, meaning they usually choose it before other options.
You take it once a week. After 5 weeks, your doctor will move to an every-other-week schedule for your doses.
It helps your red blood cells stay intact longer by blocking an early step in a cascade of steps that lead to their breakdown. It also prevents your red blood cells from clumping together and forming a clot.
You might have some side effects when you take it, such as:
- Back pain
- Cough
- Diarrhea
- Dizziness
- Headache
- Joint pain
- Muscle pain
- Nausea
- Stomach pain
- Swelling in your ankles, hands, or feet
Tell your doctor right away if you’re taking eculizumab and feel lightheaded, have chest pain, or have signs of an allergic reaction like hives, swelling, or trouble breathing.
In May 2024, the FDA approved eculizumab-aeeb (Bkemv), an interchangeable biosimilar to eculizumab for the treatment of aHUS.
Ravulizumab (Ultomiris). The FDA approved this monoclonal antibody in 2019. Kids as young as 1 month can take it. A nurse gives it to you as an infusion. It takes 2 to 4 hours to get a dose. After your first dose, you’ll come back 2 weeks later for your next dose. After that, you get them every 8 weeks.
Another method for getting your dose is an injection system (a prefilled cartridge) you give yourself at home once a week. You stick the cartridge onto the skin of your thigh, upper arm, or stomach. The system delivers the medication into your body over 10 minutes. You may need two separate injections for one dose.
Ravulizumab helps prevent blood clots and preserve red blood cells by blocking certain activity in your immune system.
Side effects of ravulizumab can include:
- Diarrhea
- Nausea and vomiting
- Constipation
- Headache
- Muscle or joint pain
- Pain in the arms or legs
- Runny nose
- Pain or swelling in the nose or throat
- Cough
- Dizziness
- Pain when you pee
- Hair loss
- Dry skin
- Decreased appetite
- Tiredness
- Rash, itching, redness, swelling, bruising, or pain near the injection site (for the skin injection)
Tell your doctor right away if you have stomach pain or signs of an infection when you take ravulizumab.
Plasma Therapy
There are two types of procedures involving plasma that can help manage aHUS. Plasma is one of the four parts of your blood. The other three are red blood cells, white blood cells, and platelets. These options used to be first-line treatments for aHUS for many years before medications for the condition were approved:
Plasma exchange (plasmapheresis). Thisis a procedure where a doctor uses a machine to separate your plasma from your blood and then returns the blood back to your body.
Plasma infusion. This replaces your plasma with fresh plasma from a healthy donor.
Some people may go into remission from their aHUS with plasma therapy, but it means coming back for plasma therapy long-term to maintain the remission. It’s still possible to have kidney failure after plasma therapy.
Kidney Transplant
If other treatment options don’t work to manage your aHUS, you may need a kidney transplant for kidney failure. Although this is a safe surgery, as many as 50% of people who get a new kidney go on to have aHUS in their new organ.
Complications
If your aHUS gets worse, the blood clots in your blood vessels can drastically slow down or cut off blood flow to your organs. If this happens, you may deal with serious issues, including:
- Kidney failure
- Swelling of your legs, ankles, and feet
- Blood in your urine (hematuria)
- Very high blood pressure or pulmonary hypertension
- Heart disease or heart attack
- Stroke
Talk to your doctor to be sure you’re keeping your aHUS under control as best you can to avoid these complications.
Show Sources
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SOURCES:
Cleveland Clinic: "Atypical Hemolytic Uremia Syndrome," "Eculizumab Injection."
Medscape: "Hemolytic-Uremic Syndrome Treatment & Management."
MedlinePlus: "Eculizumab Injection," "Ravulizumab Injection."
NORD: "Atypical Hemolytic Uremia Syndrome."
American Kidney Fund: "aHUS (atypical hemolytic uremic syndrome)."
