Safety of deferasirox in sickle cell disease patients with co-existing liver impairment

摘要

We read with great interest the article by Vichinsky et al (2011) regarding the long-term safety and efficacy of defer-asirox (Exjade~R, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA) in transfusional iron-overloaded patients with sickle cell disease (SCD). In the initial study, deferasirox use had been associated with reversible increases in liver function tests (LFTs) in a relatively small percentage of patients (3.8%) (Vichinsky et al, 2007). Only one patient in this study was confirmed (with deferasirox re-challenge) to have LFT elevations caused by deferasirox. In the follow-up study three deaths were reported (Vichinsky et al, 2011), one of them in a patient with underlying liver disease secondary to hepatitis C virus (HCV) infection, in addition to haemosiderosis. This patient had presented with hepatitis C and abnormal LFTs at the time of enrolment and was diagnosed with hepatic failure following 40 months of treatment with deferasirox. According to the investigators' judgment, hepatic failure was attributed to the underlying liver disease and not to the study drug. In light of the above mentioned LFT elevations associated with the use of deferasirox in the initial report, we believe that Vichinsky et al (2011)should have provided more detailed information on the course of this patient to support their contention.