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Opinion|Videos|June 9, 2025

Patient Experiences and Considerations for Axatilimab Therapy in Steroid-Refractory cGVHD

Author(s)Catherine J. Lee, MD, MS, Michael Bishop, MD, Paul J. Shaughnessy, MD

Panelists discuss how axatilimab demonstrates particularly good responses for gastrointestinal graft-vs-host disease (GVHD) and may improve sclerotic skin changes despite lower rates of overall skin response.

Episodes in this series

3 experts are featured in this series.

EP. 1 An Overview to cGVHD: Disease Pathophysiology and Manifestations

3 experts are featured in this series.

EP. 2 Clinical Perspectives: Risk Factors and Patient Characteristics Associated with cGVHD

3 experts are featured in this series.

EP. 3 The Multidisciplinary Approach to cGVHD Diagnosis

3 experts are featured in this series.

EP. 4 Diagnosis of cGVHD: Challenges & Considerations

3 experts are featured in this series.

EP. 5 A 50-Year-Old-Woman With cGVHD

3 experts are featured in this series.

EP. 6 Steroid Therapy for cGVHD

3 experts are featured in this series.

EP. 7 Considerations for Steroid Therapy in cGVHD: Optimizing Patient Care

3 experts are featured in this series.

EP. 8 Clinical Perspectives for Systemic Therapy in Steroid-Refractory cGVHD

3 experts are featured in this series.

EP. 9 FDA Approvals of Systemic Therapy for Steroid-Refractory cGVHD

3 experts are featured in this series.

EP. 10 Frontline Ruxolitinib Therapy for Steroid-Refractory cGVHD

3 experts are featured in this series.

EP. 11 Considerations for Ruxolitinib Therapy in Steroid-Refractory cGVHD

3 experts are featured in this series.

EP. 12 Systemic Therapies for Steroid-Refractory cGVHD: Second Line and Beyond

3 experts are featured in this series.

EP. 13 Safety and Efficacy Results from AGAVE-201 (Axatilimab Therapy in cGVHD)

3 experts are featured in this series.

EP. 14 Patient Experiences and Considerations for Axatilimab Therapy in Steroid-Refractory cGVHD

3 experts are featured in this series.

EP. 15 Future Perspectives in cGVHD

Clinical Experience with Newer Agents

The final segment covers the panelists’ perspectives on the AGAVE-201 trial data and early clinical experience with axatilimab. They note:

Strong efficacy in gastrointestinal manifestations of chronic GVHD
Efficacy in heavily pretreated patients (85% had prior ruxolitinib/ibrutinib/belumosudil)
Different mechanism of action provides new options for refractory disease
Early clinical experience shows good tolerability
Potential adverse events to monitor: liver enzyme elevations, infusion reactions, periorbital edema

The panel discusses the practical aspects of intravenous administration (every 2 weeks) and their approaches to sequential vs combination therapy. Most prefer to add axatilimab while maintaining current therapy, then gradually tapering prior agents, though practices vary between centers.

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