Targeting hedgehog signaling with glasdegib in patients with refractory sclerotic chronic GVHD: A report of two phase I/II trials

According to the results of two phase 1/2 trials (GETH-TC and FHD), the use of glasdegib among patients with active sclerotic chronic GVHD previously treated with more than 1-2 lines of therapy demonstrated promising responses, but tolerability was limited by muscle cramping. At 12 months, the skin/joint sclerotic GVHD overall response rate was 65% (all partial responses) in the GETH-TC trial and 47% (6 partial responses, 1 complete response) in the FHD cohort. Grade 3 muscle cramps were the most frequently reported adverse event (33%) in the FHD trial.