Translating Innovation Into Practice: Emerging Treatments and Real-World Data in GVHD

Panelists discuss how recent FDA-approved therapies for chronic graft-vs-host disease (GVHD; ibrutinib, ruxolitinib, belumosudil, and axatilimab) have transformed treatment approaches by enabling earlier intervention after steroid failure, targeting distinct immunological pathways, and improving patient outcomes through personalized treatment sequencing based on organ involvement, toxicity profiles, and practical considerations like oral vs intravenous administration.

EP. 1: Advances in Prevention and Treatment Approvals in Chronic GVHD

September 18th 2025

Panelists discuss how recent FDA approvals of 4 agents (ibrutinib, ruxolitinib, belumosudil, and axatilimab-csfr) have expanded treatment options for chronic graft-vs-host disease, with each drug targeting different mechanisms including BTK inhibition, JAK1/JAK2 inhibition, and ROCK2 pathway modulation, while also noting significant advances in disease prevention strategies.

EP. 2: Novel Mechanisms and Emerging Therapies in Chronic GVHD

September 18th 2025

Panelists discuss how belumosudil works through anti-inflammatory and antifibrotic mechanisms by preventing actin filament polymerization and sequestering transcription factors, while axatilimab-csfr represents a novel approach targeting monocyte and macrophage pathways through CSF-1R blockade to reduce tissue inflammation and fibrosis in patients with chronic GVHD who have failed multiple prior therapies.

EP. 3: Case 1: Multiorgan cGVHD With Pulmonary Involvement

September 25th 2025

Panelists discuss how a 52-year-old male patient developed multiorgan chronic GVHD 9 months post transplant with skin tightness, joint pain, and pulmonary involvement that showed minimal response to initial prednisone therapy and only moderate improvement after adding ruxolitinib, highlighting the importance of early detection through patient education and regular monitoring including spirometry to prevent irreversible fibrosis.

EP. 4: Assessment and Early Management After Steroid Failure in Chronic GVHD

September 25th 2025

Panelists discuss how the availability of novel agents has transformed chronic GVHD management by shortening steroid treatment duration to 2 to 3 weeks before adding second-line therapy, with response assessment focusing on dominant organ involvement rather than comprehensive National Institutes of Health criteria, and emphasizing patient-reported outcomes and quality of life measures as essential components of treatment evaluation in clinical practice.

EP. 5: Treatment Sequencing and Organ-Based Considerations in Chronic GVHD

October 2nd 2025

Panelists discuss how treatment selection for multiorgan chronic GVHD currently relies on trial data, toxicity profiles, and practical considerations like proximity to treatment centers and patient preference for oral vs intravenous therapy, while looking toward future developments in predictive biomarkers that could enable personalized treatment selection and early preemptive intervention before clinical symptoms develop.

EP. 6: Case 2: cGVHD With Ocular and Hepatic Involvement

October 2nd 2025

Panelists discuss how a 48-year-old female patient with chronic GVHD involving ocular, hepatic, and skin manifestations presents diagnostic challenges when elevated liver enzymes could indicate disease progression or drug toxicity from belumosudil and axicabtagene ciloleucel, leading to consideration of alternative treatments like mycophenolate or the preference for axicabtagene ciloleucel in patients with prior treatment failures or significant lung involvement despite the lack of head-to-head comparative data.

EP. 7: Insights Into Real-World Evidence in Chronic GVHD

October 9th 2025

Panelists discuss how real-world evidence shows lower response rates and shorter time to next treatment compared to clinical trials due to more advanced patient populations and different monitoring practices, while highlighting the potential value of combination therapies using multiple FDA-approved agents simultaneously despite the lack of formal trial data supporting such approaches.

EP. 8: Future Directions in Chronic GVHD Management

October 9th 2025

Panelists discuss how the future of chronic GVHD management looks promising with advances in graft manipulation strategies, expanded posttransplant cyclophosphamide use, dose optimization studies, oral CSF-1R inhibitors in development, and ongoing phase III trials studying current FDA-approved agents in earlier treatment lines and combination therapies to reduce patient morbidity and improve outcomes.