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
Moxibustion for Steroid-Refractory Acute Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
This study is a prospective, multicenter, open-label, phase II clinical trial designed to evaluate the safety and efficacy of moxibustion in patients with steroid-refractory acute graft-versus-host disease (SR-aGVHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT). A total of 42 patients with SR-aGVHD, primarily involving the gastrointestinal tract and presenting with abdominal pain and diarrhea, will be enrolled. All participants will receive standard second-line therapy based on best available treatment (BAT), including ruxolitinib, basiliximab, or methotrexate, according to clinical judgment. In addition, patients will receive moxibustion at specific acupoints (Tianshu \[ST25\], Shenque \[CV8\], and Qihai \[CV6\]) for 30 minutes once or twice daily for 28 days. The primary endpoint is the overall response rate (ORR) at Day 28. Secondary endpoints include durable ORR at Day 56, incidence and severity of chronic GVHD (cGVHD), non-relapse mortality (NRM), overall survival (OS), and changes in traditional Chinese medicine (TCM) syndrome scores. Safety will be assessed by monitoring adverse events throughout the study period. This study aims to explore whether moxibustion, as an adjunctive therapy, can improve clinical outcomes and provide a safe and effective treatment strategy for patients with SR-aGVHD after allo-HSCT.
Letermovir Prophylaxis Duration Guided by CMV-Specific T-cell Monitoring After Allo-HSCT.
The purpose of this study is to evaluate the efficacy and safety of a personalized strategy for discontinuing Letermovir (a drug used to prevent Cytomegalovirus \[CMV\] infection) based on the recovery of the patient's own immune system. Cytomegalovirus (CMV) is a common and serious complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Currently, Letermovir is typically given as a standard prevention for about 100 days post-transplant. However, some patients may recover their CMV-specific immunity earlier, while others may need longer protection. In this study, researchers will use a dynamic monitoring technology (QuantiFERON-CMV) to detect the level of CMV-specific T-cells in patients. Participants will be randomly assigned to either the experimental group or the control group: Experimental Group: Letermovir discontinuation will be guided by T-cell recovery. If the test shows that the patient's CMV-specific T-cells have recovered, Letermovir may be stopped earlier than the standard 100 days. Control Group: Patients will receive the standard Letermovir prophylaxis for approximately 100 days, regardless of T-cell status. The study aims to determine if this immune-guided strategy can effectively prevent CMV infection while potentially reducing the duration of medication and associated costs, without increasing the risk of CMV disease.
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