New Treatments & Clinical Trials for Graft Versus Host Disease

Graft-versus-host disease occurs when donor immune cells attack a transplant recipient's tissues, most often after stem cell or bone marrow transplant. It affects about half of transplant recipients and ranges from mild skin rashes to severe damage of skin, liver, gut, and other organs. Treatment focuses on dampening the immune attack without losing the transplant's cancer-fighting benefit.

What's actually going on in research

Trials are testing JAK inhibitors like ruxolitinib (FDA-approved for steroid-refractory acute GVHD in 2019), BTK inhibitors that calm B cells, drugs that preserve regulatory T cells, and treatments targeting specific inflammatory proteins like IL-6. Researchers are also studying ways to prevent GVHD before it starts, including removing certain immune cells from donor grafts and using low-dose medications early after transplant.

What to know before you search

Eligibility depends on whether GVHD is acute or chronic, which organs are affected, response to prior treatments like steroids, and time since transplant.

What types of trials are currently open

• Prevention trials — Testing medications or graft modifications given before or soon after transplant to reduce the chance GVHD will develop.
• Treatment trials — Testing new drugs for acute or chronic GVHD, often in people whose disease hasn't responded to steroids.
• JAK inhibitor trials — Studies of ruxolitinib and newer JAK inhibitors, testing whether they work for different GVHD types or stages.
• Cell therapy trials — Testing infusions of regulatory T cells or mesenchymal stromal cells to calm the immune system.
• Quality of life studies — Following transplant recipients to understand how GVHD affects daily function, symptoms, and long-term health.