Real-world data show that axatilimab (Niktimvo) is highly effective for patients with severe, treatment-resistant chronic graft-versus-host disease (cGVHD).
The analysis included 104 patients, most of whom had severe disease affecting multiple organs and had tried a median of four prior therapies, including ruxolitinib (Jakafi) and belumosudil (Rezurock). Pediatric patients under 17 made up 10.6% of the group.
Axatilimab produced strong results: 86.5% of patients experienced a clinical benefit, and the overall response rate among those on multiple cycles reached 70.8%. At the program’s conclusion, 67.3% of patients transitioned to the commercial version of the drug, indicating lasting benefit.
The treatment was well-tolerated, with common side effects including elevated liver enzymes and fatigue. Only 4.8% discontinued therapy due to side effects.
Researchers concluded that axatilimab provides a critical treatment option for patients who have failed multiple prior therapies, offering effective, real-world management of refractory cGVHD.