Axatilimab Plus Ruxolitinib Shows Promising Safety as First-Line cGVHD Treatment

Interim results show that combining axatilimab with ruxolitinib appears safe and well tolerated as a first-line treatment for moderate-to-severe chronic graft-versus-host disease.

The study tested this combination because the two drugs work in different ways. Axatilimab targets immune cells that drive fibrosis, while ruxolitinib reduces inflammation by blocking cytokine signaling. Patients were assigned to receive the combination, ruxolitinib alone, or standard steroid treatment.

The safety results were encouraging, with no clear increase in severe side effects from using both drugs together. Serious adverse events occurred in 25% of patients on the combination, compared with 15.8% for ruxolitinib alone and 18.8% for steroids. The most common side effect was anemia, along with fatigue, diarrhea, and mild liver enzyme increases. Only one patient stopped the combination due to side effects.

More patients stayed on the combination therapy compared with the other treatments, suggesting good tolerability and durability. Nearly 74% of patients remained on treatment, compared with about 58% on ruxolitinib alone and 32% on steroids.

The trial is still ongoing, and researchers are now waiting for results on how effective the combination is, especially the response rate after six months of treatment.