Looking Ahead: Research Priorities and Final Insights

EP: 1.Early Challenges in the cGVHD Journey

EP: 2.Navigating the Path to Second-Line Therapy

EP: 3.Steroid-Refractory cGVHD: A Look at FDA-Approved Options

EP: 4.Emerging Data: Impacts of ROCKstar and AGAVE-201

EP: 5.Tailoring Therapy: Weighing Evidence, Organ Involvement, and Route of Administration

EP: 6.Sequencing Strategies and the Role of Combination Therapy

EP: 7.Pharmacist-Led Interventions for Adherence and Optimization

EP: 8.Overcoming Barriers to Access and Treatment

EP: 9.Multidisciplinary Collaboration in cGVHD Care

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EP: 10.Looking Ahead: Research Priorities and Final Insights

The discussion highlights critical areas for advancing chronic disease management, specifically in the context of chronic graft-vs-host disease (GVHD). A key point raised was the challenge of improving steroid response rates, which remain at around 50%. The possibility of adding drugs alongside steroids in the initial treatment regimen was discussed, though past attempts have been unsuccessful. There is hope that newer drugs might improve response rates when introduced early in the treatment process. Additionally, combination therapies, though mostly explored in retrospective studies, may hold promise. Larger, prospective trials are needed to better understand which drug combinations work best for managing chronic GVHD.

Another exciting area of future research is the identification and application of biomarkers to predict treatment responses. The ability to tailor therapies based on biomarkers could revolutionize treatment protocols and help personalize care for patients. Currently, treatments are largely guided by generalized algorithms, but the use of biomarkers would allow for more precise treatment choices, improving patient outcomes. This idea of “biomarker-guided” therapy could potentially change how clinicians approach chronic GVHD and other diseases.

Finally, the conversation emphasized the need for steroid-free first-line treatments. While steroids are effective in managing symptoms, their long-term adverse effects—such as cardiovascular complications, osteoporosis, and diabetes—pose significant challenges for patients. Researchers and clinicians alike are eager to find therapies that could reduce or eliminate the need for steroids in the initial treatment phase. Additionally, earlier intervention and better prophylaxis regimens could help prevent chronic GVHD altogether. These efforts align with ongoing research into real-world outcomes and the evolving role of prophylactic treatments in improving long-term patient health and quality of life.