Safety and efficacy of deferasirox in the management of transfusion-dependent patients with myelodysplastic syndrome and aplastic anaemia: a perspective review

摘要

Deferasirox is an orally administered, once-daily iron chelator with a generally good safety and efficacy profile. Reported adverse events in the older myelodysplastic population are somewhat different to the more intensively investigated and younger thalassaemic population. Renal impairment is the most concerning adverse event, but this is reversible if identified and the drug is withdrawn early. Gastrointestinal effects, particularly diarrhoea, can be troublesome for older patients, but can be minimized with tailored therapy. Negative iron balance can be achieved in most patients with a median dose of 20 mg/kg/day, and doses up to 40 mg/kg are possible in patients with severe iron overload, who are at risk of cardiac decompensation.Introduction Disorders characterized by chronic anaemia, such as myelodysplastic syndrome (MDS) and aplastic anaemia (AA), rely on supportive red blood cell (RBC) transfusions for the treatment of symptomatic anaemia. Several guidelines have been published, recommending the institution of iron chelation therapy to counteract the effects of transfusional iron overload [Bennett, 2008; Bird et al. 2012; Greenberg et al. 201 l].