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Autoimmune Hemolytic Anemia Treatments: Exploring New and Existing Treatment Options

Updated on November 7, 2024

Researchers are constantly learning more about autoimmune hemolytic anemia (AIHA). This rare immune condition develops when your body attacks and destroys healthy red blood cells (RBCs) in a process called hemolysis. The destruction of the RBCs causes anemia and can lead to a range of health problems. There are multiple treatment options for AHIA. This article will help you understand what treatments are available to help you or a loved one living with AIHA.

What Medications Can Manage AIHA?

If your AIHA symptoms are mild, you may not need treatment, and your health care provider may closely monitor your RBC levels. Medications are often prescribed for people with AIHA when symptoms are more severe or if the RBCs are being destroyed rapidly.

The best treatment for you depends on your type of AIHA. There are two main types of AIHA — cold AIHA and warm AIHA. The warm and cold classification depends on the type of antibodies involved.

Many medications used to treat AIHA are prescribed off-label. This means that the U.S. Food and Drug Administration (FDA) did not specifically approve the drug to treat AIHA. Medications are used off-label for many reasons, including when there aren’t FDA-approved drugs for a certain disease.

Targeted Therapies

Targeted therapies only block or affect specific cells or molecules in the body. Because these drugs only target certain cells, they may cause fewer side effects. Many targeted therapies for AIHA are a type of biologic called monoclonal antibodies — immune proteins made in a lab that attach to a target on specific cells.

Rituximab (Rituxan) is a targeted therapy that’s commonly used off-label to treat warm and cold AIHA. It may be the first treatment that’s tried (called first-line therapy) for people with cold AIHA. In people with warm AIHA, rituximab may be recommended if the first treatment doesn’t work (called second-line treatment). This medication targets B lymphocytes (a type of white blood cells) that create the autoantibodies, which are antibodies that target your own healthy cells responsible for destroying the red blood cells. Rituximab may be prescribed by itself or with other medications.

In 2022, sutimlimab-jome (Enjaymo) was FDA-approved specifically for cold agglutinin disease (CAD), a kind of cold AIHA. This drug works by blocking immune proteins called complements that activate the harmful autoimmune B cells. This type of treatment is called a complement activation inhibitor.

Daratumumab (Darzalex) is a monoclonal antibody that targets B cells. This drug was originally approved to treat people with multiple myeloma. Researchers are currently studying whether it may also be helpful for people with AIHA.

Eculizumab (Soliris) is a complement inhibitor originally approved to treat paroxysmal nocturnal hemoglobinuria (another blood disorder). This medication may be helpful for emergency treatment in people with CAD.

Corticosteroids

Corticosteroids (steroids) are a type of anti-inflammatory drug that helps reduce the activity of your immune system. These medications are used off-label as a first-line therapy for people living with warm AIHA. However, these drugs may not be an effective cold AIHA treatment. Prednisone and methylprednisolone are common corticosteroids used for AIHA.

Corticosteroids are often started at a high dose and then slowly reduced over weeks or months. The medication can be given with other therapies, such as rituximab.

Immunosuppressive Medications

Immunosuppressive medications reduce the activity of the immune cells that produce harmful autoantibodies. These medications may be prescribed off-label for refractory AIHA, meaning they’re used when other treatments, like rituximab or corticosteroids, haven’t worked. Examples of immunosuppressive medications include:

Chemotherapy

Chemotherapy medications may be prescribed to treat cold AIHA. Chemotherapy drugs, such as fludarabine (Fludara) or bendamustine (Treanda) may be combined with rituximab as first-line therapy for CAD.

You may generally think of chemotherapy medications only being used to treat cancer. Medications used to treat blood cell cancer can be effective with AIHA because they may reduce the number of immune cells that produce the autoantibodies that attack the body’s red blood cells.

Erythropoiesis-Stimulating Agents

Erythropoiesis-stimulating agents are used to treat anemia in people with kidney disease or taking anti-cancer medications. These drugs help the body produce more RBCs.

Erythropoiesis-stimulating agents are also sometimes used off-label to treat AIHA in people who haven’t responded to other treatments.

Treatment for Underlying Conditions

When another condition contributes to the development of AIHA, it’s called secondary AIHA. AIHA that’s not associated with another health condition is called primary or idiopathic AIHA. One of the best approaches to treating secondary AIHA is to treat the condition causing the anemia. Secondary AIHA may be triggered by the following diseases or conditions:

  • Infections — Viruses, including Epstein-Barr virus, measles, mumps, rubella, cytomegalovirus, hepatitis, HIV, and varicella (the virus that causes chickenpox and shingles)
  • Cancer — Lymphoma, leukemia, colon cancer, and other solid tumors
  • Autoimmune diseases — Systemic lupus erythematosus, rheumatoid arthritis, Sjögren’s syndrome, thyroid disease, ulcerative colitis, and Hashimoto’s disease
  • Drug-induced hemolysis — Drug-induced immune hemolytic anemia, a rare condition caused by some medications

If you have a condition that’s been linked to AIHA, treating that condition should help reduce the destruction of red blood cells and reduce your AIHA symptoms.

What Medical Procedures Can Help Manage AIHA?

Sometimes, surgery or other medical procedures are needed alongside medications to achieve faster results. If you have severe AIHA symptoms or are not responding to medications, your health care provider may prescribe:

  • Blood transfusion offers temporary relief for people with severe anemia by providing new red blood cells.
  • Plasmapheresis, also called plasma exchange, temporarily reduces the amount of red blood cell-destroying antibodies in people with AIHA.
  • Intravenous immunoglobulin involves administering antibodies (immunoglobulin) in the vein.
  • Splenectomy (removing the spleen) can be an effective warm AIHA treatment for some people when other treatments have failed. It’s not an effective therapy for those with cold AIHA.
  • Stem cell transplantation is only used in people with AIHA if they’re not responding to any of the medications or other procedures.

Ongoing Research in AIHA Treatments

Clinical trials are studies that help researchers learn more about how new or existing drugs can treat AIHA. All drugs must pass through clinical trials to test their safety and efficacy to gain approval from the FDA.

There are several clinical trial options for people with warm or cold AIHA. Talk with your health care provider to learn more about whether you’re eligible to participate in a clinical trial.

What Lifestyle Changes Can Help Manage AIHA?

Certain lifestyle changes can help manage your AIHA. If you’re living with cold AIHA, it’s important to avoid the cold. If you must be in cold temperatures, protecting the head, face, hands, and feet can help decrease RBC hemolysis. In case of a medical procedure or hospitalization, solutions given intravenously (into a vein) should be warmed for people with cold AIHA. Folic acid supplementation is also a supportive measure that helps improve your blood levels in AIHA.

Talk With Others Who Understand

On myAIHAteam, the social network for people with AIHA and their loved ones, more than 1,400 members come together to ask questions, give advice, and share their stories with others who understand life with AIHA.

What AIHA treatments have you taken? Share your experience in the comments below, or start a conversation by posting on your Activities page.

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May 4, 2023
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