Efficacy and safety of axatilimab in chronic graft-versus-host disease: A systematic review and meta-analysis

Background

Chronic graft-versus-host disease (cGVHD) remains a significant challenge after allogeneic hematopoietic cell transplantation, with unmet needs for effective salvage therapies in refractory patients. Axatilimab, a CSF-1R inhibitor, targets macrophages central to cGVHD pathophysiology. We conducted a systematic review and meta-analysis to synthesize the available clinical evidence on axatilimab's efficacy and safety in cGVHD.

Methods

Following PRISMA guidelines, we searched major databases through May 2025 for studies evaluating axatilimab in cGVHD (clinical trials and cohorts). Two reviewers independently screened studies, extracted data, and assessed risk of bias using ROBINS-I, RoB 2.0, NOS, and certainty of evidence using the GRADE approach. Pooled proportions were calculated using random-effects models.

Results

Four studies comprising 304 heavily pre-treated patients were included. The pooled Overall Response Rate was 58 % (95 % CI: 45–72 %). Clinically meaningful symptom improvement occurred in 64 % (95 % CI: 53–75 %). Any-grade adverse events (AEs) occurred in 97 %, Grade ≥ 3 AEs in 58 % (95 % CI: 52–64 %), and treatment discontinuation due to AE in 16 % (95 % CI: 11–20 %). Common AEs included periorbital edema and laboratory abnormalities. Overall certainty of evidence was rated as Low by GRADE.

Conclusion

This meta-analysis suggests axatilimab is an effective and generally well-tolerated treatment option for refractory cGVHD, demonstrating clinically meaningful response rates, particularly in difficult-to-treat gastrointestinal involvement. These findings support its use in heavily pre-treated patients in the third-line or later setting. However, the overall certainty of evidence is Low due to limitations in current study designs, highlighting the critical need for future high-quality randomized comparative trials.