Graft versus host disease (GVHD) occurs when donor immune cells from a stem cell transplant attack the recipient's tissues — most often the skin, gut, and liver. It remains a major cause of illness and death after allogeneic transplant, and chronic GVHD can persist and impair quality of life for years.
What's actually going on in research
Ruxolitinib, a JAK inhibitor, has become the standard treatment for steroid-refractory acute GVHD after showing superior responses over best available therapy in a large trial. Belumosudil, a ROCK2 inhibitor, is now a standard option for chronic GVHD after multiple prior therapies. Earlier interventions targeting the donor T-cell response and new prophylaxis regimens are being tested to prevent GVHD from developing at all.
JAK inhibitors
Ruxolitinib blocks inflammatory signaling in both acute and chronic GVHD and is being tested in earlier lines, in combination, and as prophylaxis to prevent GVHD after transplant.
ROCK2 inhibitor (belumosudil)
Belumosudil shifts immune cell activity from inflammatory to regulatory, reducing chronic GVHD activity in multiple organs. Trials are moving it into earlier treatment positions.
GVHD prevention
Post-transplant cyclophosphamide given as GVHD prophylaxis dramatically reduces severe GVHD and is being combined with other approaches to further lower GVHD incidence without impairing the anti-cancer effect.
What to know before you search
Eligibility requires allogeneic stem cell transplant history, confirmed GVHD diagnosis, organ involvement, steroid refractoriness for many trials, and adequate organ function.
What types of trials are currently open
- Acute GVHD trials — Testing new drugs and combinations for steroid-refractory acute GVHD.
- Chronic GVHD trials — Evaluating treatments for chronic GVHD affecting skin, mouth, lung, joints, and other organs.
- Prevention trials — Testing prophylaxis regimens including post-transplant cyclophosphamide and regulatory T-cell infusions.
- Microbiome trials — Evaluating gut microbiome interventions and fecal transplant to prevent or treat gastrointestinal GVHD.
- Quality of life trials — Testing physical and psychological rehabilitation for chronic GVHD survivors.
Recently added Graft Versus Host Disease trials
A Prospective Study of Madecassoside Tablets in Sclerotic Chronic Graft-versus-Host Disease After Allo-HSCT
Sclerotic chronic graft-versus-host disease (scl-cGVHD) is a severe subtype of chronic GVHD after allogeneic hematopoietic stem cell transplantation (allo-HSCT), characterized by skin sclerosis, fascial involvement, and restricted joint mobility, leading to substantial functional impairment and reduced quality of life. Current standard therapies, including corticosteroids and other immunosuppressive agents, have limited efficacy in patients with refractory or steroid-dependent disease. Madecassoside tablets, a standardized extract of Centella asiatica, have demonstrated anti-inflammatory and anti-fibrotic properties in previous studies and may provide therapeutic benefit in fibrotic diseases. This study is a prospective, multicenter, single-arm phase 2 clinical trial designed to evaluate the efficacy and safety of madecassoside tablets combined with standard therapy in patients with scl-cGVHD after allo-HSCT. Eligible participants will receive oral madecassoside tablets (0.2 g three times daily) in addition to standard treatment for 6 months. Clinical assessments will be performed at baseline and at weeks 4, 8, 12, and 24. The primary endpoint is the improvement rate of skin and/or joint/fascia NIH cGVHD scores at 6 months according to the 2014 NIH consensus criteria. Secondary endpoints include overall cGVHD response rate, failure-free survival, non-relapse mortality, corticosteroid dose reduction, patient-reported outcomes, and safety assessments. Peripheral blood samples will also be collected to explore changes in inflammatory cytokines and lymphocyte subsets during treatment.
A Study of Ruxolitinib in Patients With Graft-Versus Host Disease
This study aims to evaluate the safety and effectiveness of Jakavi® (ruxolitinib) in patients with acute graft-versus host disease (GVHD) and chronic GVHD in clinical practice.
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