Combining Axatilimab and Ruxolitinib to Minimize Steroid Use in cGVHD

Amandeep Salhotra, MD, of City of Hope National Medical Center in Duarte, California, explores the safety and tolerability of combining axatilimab and ruxolitinib for the treatment of chronic graft-versus-host disease (cGVHD). Although both agents are individually approved for this condition, the combination arm of the study sought to determine if they could work synergistically without introducing additive toxicity. The results were highly encouraging, particularly regarding treatment adherence. Only 17% of patients in the combination arm discontinued treatment, and only one patient required an additional systemic therapy, demonstrating high stability within the cohort over a median follow-up of 4.3 months.

Regarding the safety profile, the adverse events (AEs) observed were consistent with those typically seen with these individual agents. Approximately 75% of patients in the combination arm experienced treatment-related AEs, primarily cytopenias—a known AE of ruxolitinib—along with elevations in transaminases, amylase, and lipase. Despite these findings, the overall conclusion was that the combination is well-tolerated. There was no evidence of unusual or unexpected additive toxicities that would preclude the use of both drugs simultaneously.

The ultimate goal of this clinical trial is to evaluate the 6-month overall response rate. With accrual now complete, the medical community awaits efficacy data to confirm if this dual-agent approach can provide a superior response. Salhotra expresses hope that this combination will eventually allow clinicians to “break free” from the standard use of corticosteroids in the upfront setting for cGVHD, thereby sparing patients from the significant long-term AEs associated with chronic steroid use.