Incidence and Response to Treatment of Adenovirus (ADV) infection With cidofavir (C) in Pediatric Patients After Allogeneic HCT

initiated pre-transplant in one patient, and 21 days to greater than 2 years in six patients (median of 61days).We analyzed 7 patients with CMV viremia who were treated under emergency IND because of lack of other reasonable therapeutic options.All 7 patients had failed conventional antiviral therapy: all patients had received ganciclovir and/or valganciclovir, 6 had received foscarnet, and 4 had received CMV IG.The doses of CMX001 in these patients ranged from 80 mg to 300 mg (approximately 2 to 4 mg/kg); follow-up data was available for at least 4 weeks in all patients.Virologic response was defined as more than a 90% reduction (1 log 10) in viral load (VL) and complete response was defined as an undetectable viral load.The 4 males and 3 females treated had a median age of 55 years (range 11 to 69 years); they were treated with CMX001 for a median of 88 days (range 29-131 days).The median reduction in VL was greater than 1.2 log 10 at 4 weeks.A complete response was observed in 3/5 (60%) patients who did not have mutations in the CMV polymerase UL54 gene; 2/5 had an average reduction in CMV by PCR of 1.2 log 10.Neither of two patients with a relevant mutation in UL54 (L501F and A987G) had a 1 log reduction in viremia at the last time point.Two patients had pre-existing renal insufficiency; no dose adjustment was needed based on kidney function.Four of 7 patients had GVHD during treatment with CMX001.One patient experienced C. difficile colitis, one experienced pancytopenia, one with a seizure disorder experienced seizure, and one experienced severe GVHD.No trends were observed in SAEs.This case series demonstrates that CMX001 is a promising therapeutic option for the treatment of CMV disease in stem cell transplant patients.