Graft-versus-host disease (GVHD) treatments focus on preventing or controlling attacks by transplanted donor immune cells against your body’s healthy cells and tissues. Many of these approaches use medications that calm your immune system and reduce inflammation. Doctors choose treatments depending on the type of GVHD, your specific symptoms, and their severity.
In this article, we’ll discuss eight types of treatments for GVHD, including those for acute and chronic forms. Some treatments may also help individuals with overlap GVHD, where both acute and chronic symptoms occur together.
GVHD develops in people who receive an allogeneic stem cell transplant — also known as a bone marrow transplant. This procedure replaces damaged or diseased blood-forming cells in your bones with healthy cells from donors. Bone marrow transplants help treat certain blood cancers, such as types of leukemia and lymphoma.
Acute GVHD occurs when the donor’s immune cells mistakenly attack the recipient’s healthy cells and tissues, thinking they are harmful. This immune response can damage organs and cause a wide range of symptoms. Acute GVHD typically occurs within the first 100 days after a transplant but can sometimes appear up to six months later. It can range from mild to severe, depending on the extent of organ involvement.
Doctors treat acute GVHD based on where symptoms occur and how severe they are. They try to prevent it from happening in the first place by prescribing preventive treatment. If this doesn’t work, other options include corticosteroids and Janus kinase (JAK) inhibitors to help control the immune system’s response and reduce inflammation.
Chronic GVHD develops gradually over time and can affect nearly any organ in the body. Doctors usually divide chronic GVHD treatments based on how severe a person’s symptoms are and where they’re located.
Mild cases of chronic GVHD are typically managed with local treatments for the affected areas. Moderate and severe cases may need systemic treatments taken by mouth or IV to control inflammation and relieve symptoms.
Read on for a list of treatment options for GVHD.
Doctors start preventive treatment right after a transplant to lower the risk of GVHD. This often includes a combination of immunosuppressive medications, such as calcineurin inhibitors (CNIs). CNIs work by blocking specific proteins to stop certain immune cells (called T cells or T lymphocytes) from attacking your tissues.
Examples of CNIs used to prevent acute GVHD include cyclosporine (Neoral) and tacrolimus (Prograf). You may take cyclosporine and tacrolimus treatments by mouth or through a vein via an IV infusion.
Corticosteroids (commonly called steroids) help suppress the immune system and reduce inflammation. They mimic cortisol — a hormone naturally made by the adrenal glands, which sit atop the kidneys. Corticosteroids fight inflammation and stop the donor cells from damaging your body. Steroids work by calming the immune response and preventing donor immune cells from attacking your body.
For mild acute GVHD, doctors usually recommend topical corticosteroids, such as creams, ointments, or eye drops, to reduce symptoms in affected areas. Moderate to severe acute GVHD treatments may require systemic (bodywide) steroids such as methylprednisolone. Systemic medications are taken by mouth or by IV.
Many health experts recommend treating moderate to severe chronic GVHD with steroids. Studies show that almost half of those with chronic GVHD respond well to prednisone. Unfortunately, using steroids for too long can cause unwanted side effects. It’s important to let your doctor know if:
Your doctor may combine topical therapies with steroids to treat your chronic GVHD symptoms. This works best for skin symptoms limited to one area of your body.
JAK inhibitors work by blocking proteins involved in inflammation. The U.S. Food and Drug Administration (FDA) has approved many JAK inhibitors for inflammatory skin diseases like eczema and psoriasis.
Ruxolitinib (Jakafi) was approved in 2019 to treat steroid-refractory acute GVHD — that is, GVHD that doesn’t respond to steroids. Doctors typically prescribe ruxolitinib pills to take by mouth.
Topical therapies are applied directly to the skin, eyes, and/or mouth to treat mild GVHD symptoms. If you’re dealing with skin rashes, creams and ointments can provide relief. Some topicals are made with anti-inflammatory medications to treat dry, flaky skin. They help calm the immune system and stop the donor cells from attacking your cells and tissues.
Your doctor may prescribe topical medications like steroids or immunosuppressants. Examples include calcineurin inhibitors like tacrolimus.
As their name suggests, immunosuppressants work by “suppressing” or dampening the immune system. This stops the donor cells from attacking your healthy tissues. These medications are often used when chronic GVHD doesn’t respond to steroid treatments or when a doctor wants to reduce steroid side effects.
Examples of immunosuppressants for chronic GVHD include:
Additionally, the FDA has approved an immunosuppressant called remestemcel-L-rknd (Ryoncil) to specifically treat steroid-refractory acute GVHD in infants and children ages 2 months and up. Unlike traditional immunosuppressants, this medication is a mesenchymal stromal cell (MSC) therapy, which is thought to modulate immune system activity and reduce T-cell-driven inflammation, though its exact mechanism is still being studied.
If you have GVHD skin symptoms that affect more than half your skin, your doctor may suggest a procedure called extracorporeal photophoresis (ECP). ECP is an immune-modulating therapy that helps regulate the immune response rather than directly “stopping” donor cells from attacking. It is often used for chronic GVHD, particularly when symptoms do not respond well to steroids or topical treatments.
During ECP, a machine draws your blood through a catheter (thin tube) and separates white blood cells, including T cells. These cells are then treated with a medication called 8-methoxypsoralen (8-MOP), which makes them sensitive to ultraviolet (UVA) light. The treated cells are exposed to UVA light, which alters their function before they are returned to your bloodstream. This process helps balance the immune system and reduce inflammation associated with GVHD.
According to Cleveland Clinic, the ECP process takes around three to four hours. Most people need multiple treatments to improve their chronic GVHD symptoms.
The FDA has approved several targeted treatments for chronic GVHD that does not respond to standard treatments, such as steroids. These treatments work by selectively blocking immune pathways involved in inflammation and tissue damage.
Bruton’s tyrosine kinase (BTK) inhibitors are drugs that block B cells — specialized immune cells that make proteins called antibodies. Ibrutinib (Imbruvica) is approved for treating chronic GVHD in adults and children who have not responded to at least one prior systemic treatment. Side effects of ibrutinib include fatigue, diarrhea, and abnormal heart rhythms.
For severe chronic GVHD that does not respond to steroids, a doctor may prescribe either ruxolitinib or belumosudil (Rezurock). These are both approved for steroid-refractory chronic GVHD. The FDA approved belumosudil in 2021. Doctors also use this oral medication in people who have tried at least two other GVHD treatments that weren’t effective.
Axatilimab (Niktimvo) is another treatment option for people who haven’t had success with at least two other chronic GVHD treatments. The FDA approved axatilimab in 2024. This is a protein drug called a monoclonal antibody that blocks certain immune cells. Studies show that axatilimab controls inflammation and stops tissue damage in GVHD. Axatilimab is given by IV.
Health experts also recommend clinical trials for anyone with GVHD. These are large studies on potential treatments for health conditions. If you’re interested in participating in clinical research, ask your hematology/oncology doctor if they know of any studies for which you may be eligible.
Overlap GVHD is diagnosed in people who have both acute and chronic GVHD symptoms at the same time. Doctors treat overlap GVHD with the same treatments as for chronic GVHD.
Mild symptoms affecting small parts of your body can be managed with topical creams and ointments. If you haven’t had steroid treatment before, your doctor will likely prescribe prednisone. They can adjust your dose if your symptoms start worsening.
Moderate to severe cases of overlap GVHD usually need additional treatments. Your doctor can add on a second or third medication — like an immunosuppressant or targeted therapy if your symptoms don’t improve with steroids alone.
On myGVHDteam, the site for people with graft-versus-host disease and their loved ones, people come together to ask questions, give advice, and share their stories with others who understand life with the condition.
What treatments have you tried for GVDH? Share your experience in the comments below.
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