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

Background: This systematic review and meta-analysis evaluated the efficacy and safety of nilotinib in patients with steroid-refractory or steroid-dependent chronic graft-versus-host disease (SR/D cGVHD). Methods: Electronic databases and trial registries were searched from inception to June 2025. Study selection, data extraction, risk-of-bias assessment, and GRADE evaluation were performed independently. Proportions were pooled using random-effects meta-analysis. Results: Four prospective single-arm early-phase interventional studies (n = 112 patients) were included. The pooled overall response rate (ORR) was 36% (95% CI: 13% to 59%; I2 = 84.9%), but the estimate was strongly influenced by the sequential rituximab-nilotinib study by van der Wagen et al.; after omission of this study, ORR decreased to 24% (95% CI: 13% to 34%; I2 = 0%). Complete and partial response rates were 8% (95% CI: 2% to 14%) and 32% (95% CI: 10% to 54%), respectively. Treatment discontinuation due to adverse events occurred in 26% (95% CI: 9% to 43%; I2 = 82.5%). The certainty of evidence was very low for most outcomes. Conclusion: Nilotinib was associated with modest and heterogeneous efficacy in SR/D cGVHD. The available evidence does not support routine salvage use after imatinib failure or intolerance; any future role is likely to be restricted to carefully selected patients, preferably within biomarker-guided or dose-optimized protocols. Keywords: Chronic graft-versus-host disease; Meta-analysis; Nilotinib; Steroid-refractory; Tyrosine kinase inhibitors; cGVHD.

Citation

Bahbah et al (2026). Efficacy and safety of nilotinib in steroid-refractory chronic graft-versus-host disease: A systematic review and meta-analysis. Transplant immunology, 97, 102415. Advance online publication. https://doi.org/10.1016/j.trim.2026.102415

DOI

10.1016/j.trim.2026.102415

URI

https://hdl.handle.net/20.500.14709/1526