GvHD Hub

The GvHD Hub held a virtual symposium on October 21, 2025, titled, Guidance vs practice: How can we improve treatment of SR-GvHD? Here, we share a presentation by Daniel Wolff, University Hospital Regensburg, DE, discussing the early treatment strategies for SR-GvHD. Wolff provided an overview of the treatment options for second-line treatment after steroids and subsequent advanced-line options, emphasizing that early detection of disease progression and sufficient treatment intervention are crucial, as advanced disease is often nonreversible. Wolff discussed response assessments to first-line steroid treatment and second-line treatments, and outlined the treatment options for patients with SR-cGvHD, including ruxolitinib, belumosudil, axatilimab, ibrutinib, and extracorporeal photopheresis. This educational resource is independently supported by Sanofi.​ All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

The GvHD Hub held a virtual symposium on October 21, 2025, titled, Guidance vs practice: How can we improve treatment of SR-GvHD. Here, we share a presentation by Steven Pavletic, Bethesda, US, discussing quality of life and symptoms in cGvHD. Pavletic discussed the importance of the assessment of quality of life using patient-reported outcome measures (PROMs) in both clinical trials and clinical practice in cGvHD, outlining the frequently used PROMs in GvHD, including cGvHD-specific and dimension-specific measures. Pavletic highlighted that incorporation of PROMs into standard of care practice may ultimately lead to more tailored and effective clinical assessments. This educational resource is independently supported by Sanofi. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

The GvHD Hub was pleased to speak with Mohamad Mohty. We asked, What are the treatment options for ruxolitinib-refractory acute GvHD?In this interview, Mohamad Mohty discusses the evolving management of steroid-refractory acute GvHD, highlighting ruxolitinib as the standard of care and the importance of robust supportive measures. Mohty explores treatment options for ruxolitinib-refractory disease – including extracorporeal photopheresis, mesenchymal stromal cells, and emerging targeted approaches. Mohty highlights the value of referral to specialized centers and clinical trial enrolment as treatment shifts toward precision-guided, biology-driven strategies.This educational resource is independently supported by Medac. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

The GvHD Hub was pleased to speak with Robert Zeiser. We asked, What are the current treatment options for steroid-refractory chronic GvHD (SR-cGvHD)? In this interview, Zeiser explores the current treatment options for SR-cGvHD, such as ruxolitinib, ibrutinib, belumosudil, and axatilimab, and discusses the different mechanisms of action involved in targeting distinct pathways involved in cGvHD pathogenesis. Zeiser notes that future directions may focus on identifying optimal combination therapies for patients with SR-cGvHD. This educational resource is independently supported by Sanofi. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

Despite advances in preventing chronic graft-versus-host disease (GvHD), transplant recipients continue to be affected, with steroid-refractoriness associated with significantly increased morbidity and mortality.1The GvHD Hub spoke with Corey Cutler, Dana-Farber Cancer Institute, Boston, US. We asked, What are the major challenges in the treatment of steroid-refractory GvHD (SR-GvHD), and how can clinicians overcome these?This educational resource is independently supported by Sanofi. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

During the GvHD Hub Steering Committee Meeting on May 12, 2025, key opinion leaders met to discuss how to improve measurement of quality of life (QoL) and use of patient-reported outcomes (PROs) in cGvHD. The discussion was preceded by a presentation by Mohamad Mohty, and the discussion featured Nico Gagelmann, Bipin Savani, Ali Bazarbachi, Corey Cutler, and Andrew Harris. Mohty began by explaining the necessity of measuring QoL in patients with graft-versus-host disease (GvHD) and the current use of PROs in trials and clinical practice. During the discussion, the steering committee members provided insight into the difficulties implementing QoL assessment in clinical practice, and potential solutions.This independent educational activity is supported by Sanofi. All content was developed independently. The funder was allowed no influence on the content of this activity. 

During the GvHD Hub Steering Committee Meeting on May 12, 2025, key opinion leaders met to discuss the use of extracorporeal photopheresis + ruxolitinib to prevent cGvHD in patients with SRaGvHD. The discussion was preceded by a presentation by Nico Gagelmann, chaired by Mohamad Mohty, and featured Robert Zeiser, Bipin Savani, Daniel Wolff, Corey Cutler, and Andrew Harris.  Gagelmann began by presenting the design and rationale of a real-world retrospective study in Germany, response and survival outcomes, and key takeaways. During the discussion, the steering committee members provided their thoughts on the use of combination therapies, specifically ECP + ruxolitinib, in SR-GvHD, and potential directions for future studies.  This independent educational activity was supported by Therakos. All content was developed independently. The funder was allowed no influence on the content of this activity. 

Real-world evidence (RWE) and randomized controlled trial (RCT) data each have both advantages and disadvantages, and they can be complementary. In general, outcomes of real-world studies are given lower credibility compared with RCT research, which is characterized as having the highest reliability. However, real-world studies can be particularly advantageous in specific patient populations, for instance in chronic graft-versus-host disease (cGvHD), which may have a lack of RCT data. The GvHD Hub spoke to Steven Pavletic, National Cancer Institute, Bethesda, US, about his perspectives on how RWE can complement RCTs, and why it may be important for small patient populations. RCT vs RWE Data from RCTs are considered the ‘gold-standard’ for investigating the efficacy and safety of treatments, owing to the well-controlled, observational environment, with strict inclusion and exclusion criteria. A major challenge with RCT data is that they generally apply to a specific population of patients but may not be relevant for all individuals with the disease.  Ideally, RCTs and real-world studies should be developed to be complementary. For example, if an RCT investigates the efficacy of a drug, the real-world studies can focus on the epidemiology, effectiveness, safety, or costs of treatment related to that drug.  Real-world data in cGvHD cGvHD is heterogeneous in presentation, with variability in severity, organ involvement, and manifestations. It is important to identify less common adverse effects that may result from treatment in patients with varied clinical manifestations of cGvHD, which is not always possible in clinical trials. Therefore, further RWE and observational studies are needed to provide additional data on the safety and efficacy of treatments, particularly in cGvHD which can have distinct patient populations. However, these studies lack the consistency of RCTs, and caution should be used when interpreting results. Several recent prospective phase II/III studies have led to the approval of new agents for cGvHD, including ibrutinib, ruxolitinib, and belumosudil. In the case of belumosudil, real-world studies have consistently shown safety and effectiveness similar to the RCTs, which is encouraging for the broader cGvHD patient population. Both RWE and RCTs can be helpful tools for advancing clinical research and informing patient care, especially when considered together.