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Imatinib mesylate and nilotinib are small molecule tyrosine kinase inhibitors (TKIs) that target BCR-ABL, platelet-derived growth factor receptor (PDGF-R), and c-KIT. In the context of chronic graft-versus-host disease (cGVHD), these agents are investigated for their ability to inhibit the PDGF-R and transforming growth factor-β (TGF-β) pathways, which are key drivers of the fibrotic processes characteristic of the disease. This specific entry refers to a clinical strategy evaluated by the University Hospital, Lille (CHU de Lille), where nilotinib was studied as a salvage therapy for patients with steroid-refractory cGVHD who had previously failed or were intolerant to imatinib mesylate. A prospective, multicenter Phase 2 trial (NCT02891395) conducted by the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) investigated this switch; however, the trial failed to demonstrate sufficient efficacy for nilotinib as a salvage treatment in this specific patient population.
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