The prospective 1-year Evaluation of Patients'
Iron Chelation with
Exjade (EPIC) study enrolled a large cohort of 116 patients with
aplastic anemia; the present analyses evaluated the efficacy and safety of
deferasirox in this patient population. After 1 year, median serum
ferritin decreased significantly from 3254 ng/mL at baseline to 1854 ng/mL (P < .001). Decreases occurred in chelation-naive (3229-1520 ng/mL; P < .001, last-observation-carried-forward analysis), and previously chelated (3263-2585 ng/mL; P = .21, last-observation-carried-forward analysis) patients and were reflective of dose adjustments and ongoing
iron intake. Baseline labile plasma
iron levels were within normal range despite high serum
ferritin levels. The most common
drug-related adverse events were
nausea (22%) and
diarrhea (16%). Serum
creatinine increases more than 33% above baseline and the upper limit of normal occurred in 29 patients (25%), but there were no progressive increases; concomitant use of
cyclosporine had a significant impact on serum
creatinine levels. The decrease in mean
alanine aminotransferase levels at 1 year correlated significantly with reduction in serum
ferritin (r = 0.40, P < .001). Mean absolute neutrophil and platelet counts remained stable during treatment, and there were no
drug-related
cytopenias. This prospective dataset confirms the efficacy and well characterizes the tolerability profile of
deferasirox in a large population of patients with
aplastic anemia. This study was registered at www.clinicaltrials.gov as #NCT00171821.