Did you know that about 31 percent to 36 percent of people with chronic or acute graft-versus-host disease (GVHD) don’t respond to steroid treatment? Fortunately, doctors and researchers are working hard to find new and better ways to manage GVHD. The U.S. Food and Drug Administration (FDA) recently approved several treatments for this condition.

In this article, we’ll explore five new treatment options for GVHD, the way they work, and their possible side effects. We’ll also discuss what to expect during treatment. To learn more about these therapies and if any might be right for you, talk to your healthcare team.

Doctors Treat GVHD Based on the Type and Symptoms

Before digging into the new GVHD treatments, it helps to understand how doctors typically treat the condition. Graft-versus-host disease develops in people who receive stem cell transplants (also called bone marrow transplants). These procedures usually help treat blood cancers such as leukemia and lymphoma. In GVHD, the donor’s immune cells (the graft) attack the transplant recipient’s tissues (the host).

There are two main types of GVHD — acute and chronic graft-versus-host disease. The type you have depends on your symptoms and how severe they are. Standard treatments vary based on whether GVHD is acute or chronic, but most work by calming the immune system to reduce inflammation. Typical options include:

• Immunosuppressive medications, like mycophenolate mofetil (CellCept, Myhibbin) and cyclosporine (Gengraf, Neoral, Sandimmune)
• Corticosteroids (steroids) like prednisone (Rayos)
• Calcineurin inhibitors like tacrolimus (Astagraf XL, Envarsus XR, Prograf)
• Extracorporeal photopheresis (ECP), a therapy that removes white blood cells, treats them with UV light, and returns them to the body to help regulate immune activity

Although these treatments help many people, some individuals develop resistance, especially to steroids. This can lead to steroid-refractory GVHD, which is harder to control, and taking steroids long term can also cause serious side effects. Many doctors try to limit steroid use.

New Treatment Options for GVHD

In recent years, the FDA has approved several new treatments for graft-versus-host disease. The FDA also expanded the approval of existing treatments to treat additional types of GVHD. Doctors and researchers hope these advances will improve the quality of life for people living with this disease.

1. Remestemcel-L-Rknd

In December 2024, the FDA approved remestemcel-L-rknd (Ryoncil) for children as young as 2 months old with steroid-resistant acute GVHD. Remestemcel-L is an exciting development because it uses mesenchymal stromal cells (MSCs), which come from the bone marrow — the spongy tissue inside bone that makes blood cells. This therapy contains donor cells from healthy adults, which are collected and grown in a lab.

A key advantage of MSCs is that they lack major histocompatibility complex (MHC) markers — proteins on cell surfaces that help the immune system recognize what belongs in the body. Because MSCs don’t have MHC markers, the immune system is less likely to attack them, reducing the risk of rejection.

In clinical trials, adverse reactions of remestemcel-L included:

• Heart problems — Irregular heartbeat, swelling, and high or low blood pressure
• Skin issues — Rash
• Stomach problems — Stomach pain, diarrhea, and vomiting
• Blood issues — Bleeding and low platelets (cells that help blood clot)
• Liver problems — High liver enzymes and high bilirubin (signs of liver issues)
• Infections — Bacterial (like staph), fungal, and viral infections
• Muscle and bone pain — Joint pain
• Lung problems — Cough and trouble breathing

Remestemcel-L is given by IV infusion (directly into a vein). The number of cells used is based on the child’s body weight. The entire course takes eight infusions, given twice a week for four weeks. Sessions must be spaced at least three days apart.

2. Axatilimab-Csfr

Another relatively new GVHD treatment is axatilimab-csfr (Niktimvo). The FDA approved this therapy for adults and children weighing more than about 88 pounds who have chronic GVHD and didn’t respond to at least two other medications.

Axatilimab-csfr is a monoclonal antibody — a lab-made protein that binds to a specific target in the immune system, such as a receptor or signaling molecule, to regulate immune response. This antibody works by blocking colony-stimulating factor 1 (CSF-1R), a protein that certain immune cells use to trigger inflammation and damage in GVHD.

In a clinical trial (a study of a treatment’s safety and effectiveness), results showed that blocking CSF-1R with axatilimab helps reduce inflammation and may improve fibrosis (buildup of scar tissue). Overall, 75 percent of participants responded to treatment, and 60 percent maintained their response for at least 12 months.

Reported side effects of axatilimab include:

• Infections
• Muscle and joint pain
• Diarrhea
• Headache
• Fever
• Nausea

Axatilimab is given as an IV infusion administered over 30 minutes. Treatments occur every two weeks and continue until side effects become too severe or GVHD symptoms return.

3. Belumosudil

The FDA approved belumosudil (Rezurock) for adults and children 12 and older with chronic GVHD who didn’t respond to at least two other treatments. Belumosudil is taken once daily by mouth with food.

Belumosudil belongs to a class of drugs known as kinase inhibitors. This drug blocks a protein called Rho-associated coiled-coil containing protein kinase 2 (ROCK2) that turns on immune cells and increases inflammation. By inhibiting ROCK2, belumosudil helps calm the immune system and prevents fibrosis in GVHD.

In a clinical trial, most of the 65 participants responded to belumosudil. Overall, 69 percent had a partial response, meaning some of their GVHD symptoms improved, while 6 percent had a complete response — their symptoms disappeared after treatment.

People taking belumosudil have reported developing infections, including serious ones. However, because many people taking this drug also use other medicines that weaken the immune system, it’s unclear if belumosudil alone increases infection risk. Other possible side effects include:

• Swelling
• High blood pressure
• Itchy skin
• Stomach problems — Stomach pain, appetite loss, diarrhea, trouble swallowing, and nausea
• Blood issues — Anemia (low red blood cell count), bleeding, and low white blood cell count
• Infections — Bacterial, viral, and general infections
• Nervous system and muscle issues — Headache, joint pain, weakness, muscle spasms, and body pain
• Breathing problems — Cough, trouble breathing, and stuffy nose

4. Ruxolitinib

The FDA sometimes approves existing medications for new uses. One example is ruxolitinib (Jakafi), which was originally approved in 2019 for treating steroid-refractory acute GVHD in adults and children aged 12 and older. In 2021, the FDA approved this medication for chronic GVHD as well. Adults and children 12 and up who tried one or two other chronic GVHD treatments without success can take ruxolitinib.

Ruxolitinib is a kinase inhibitor that blocks Janus kinase (JAK) proteins, which researchers believe play a role in GVHD inflammation. Clinical trials have shown that ruxolitinib is effective in treating both acute and chronic GVHD.

Potential side effects of ruxolitinib include:

• Swelling
• Infections
• Low blood cell counts — Reduced red blood cells, white blood cells, and platelets

Ruxolitinib is taken twice daily, with or without food. The starting dose for chronic GVHD is higher than the dose for acute disease.

5. Ibrutinib

The FDA has expanded the use of ibrutinib (Imbruvica) for GVHD over the years. Originally developed to treat certain blood cancers, ibrutinib became the first approved treatment for chronic GVHD in adults in 2017. Ibrutinib is now also approved for children ages 1 year and older with chronic GVHD who haven’t responded to at least one other treatment.

Ibrutinib is a kinase inhibitor that blocks Bruton’s kinase (BTK) and interleukin-2-inducible T-cell kinase (ITK) proteins. Studies show that these proteins activate certain immune cells in chronic GVHD. By stopping BTK and ITK from sending signals, ibrutinib can reduce inflammation. People who take this medication can lower their steroid dose and improve their overall quality of life.

The most common side effects of ibrutinib for chronic GVHD include:

• Bruising
• Fatigue
• Diarrhea
• Nausea
• Mouth sores
• Anemia
• Fever
• Pneumonia

For children, ibrutinib comes in tablets, capsules, and a liquid. Dosing depends on the child’s age, height and weight.

Talk to Your Healthcare Team About Your GVHD Treatment Options

If you’re interested in learning more about new treatment options for GVHD, talk to your GVHD specialist. They can determine which therapies may be best suited for you. You may also be eligible for clinical trials testing new drugs for GVHD. While not everyone qualifies, your GVHD specialist can discuss whether a trial may be an option for you.

Talk With Others Who Understand

On myGVHDteam, the site for people living 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 GVHD.

Have you tried a newly approved treatment for your graft-versus-host disease? Has it helped manage your symptoms or improved your quality of life? Share your experience in the comments below.