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Patients with chronic graft-versus-host disease (cGvHD) often have platelet-derived growth factor receptor (PDGF-R) antibodies. Imatinib is a tyrosine kinase inhibitor with multiple targets including PDGF-R.
To assess whether imatinib might be active in cGvHD, Italian investigators conducted a prospective, multicenter, phase II trial involving 39 cGvHD patients unresponsive to steroid therapy (mean age, 48; median duration of multiorgan cGvHD, 26 months). Imatinib was started at 100 mg per day and gradually increased to 400 mg per day; 34 patients received the drug for ≥3 months.
Toxicities were generally mild and were mainly fatigue, muscle pain, and weakness, but 12 patients developed infections and 2 died of Aspergillus pneumonia. No patie…