Author:

Keywords:

Adolescent, Adult, Anemia, Aplastic, Benzoic Acids, Chelation Therapy, Creatinine, Female, Ferritins, Humans, Iron Chelating Agents, Male, Middle Aged, Prospective Studies, Triazoles, Young Adult, Science & Technology, Life Sciences & Biomedicine, Hematology, TRANSFUSION-DEPENDENT ANEMIAS, LABILE PLASMA IRON, MYELODYSPLASTIC SYNDROMES, BETA-THALASSEMIA, OVERLOADED PATIENTS, OXIDATIVE STRESS, SERUM FERRITIN, COMPLICATIONS, PATHOPHYSIOLOGY, EPIDEMIOLOGY, Benzoates, Deferasirox, EPIC study investigators, 1102 Cardiorespiratory Medicine and Haematology, 1103 Clinical Sciences, 1114 Paediatrics and Reproductive Medicine, Immunology, 3101 Biochemistry and cell biology, 3201 Cardiovascular medicine and haematology, 3213 Paediatrics

Abstract:

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.