Background Deferasirox is approved for the treatment of transfusional iron overload in thalasesmia disease. However, in real life, deferasirox is also used as an iron chelator for iron overload in other pathologies, such as myelodysplastic syndrome (MDS).
Purpose Our aim was to describe the effectiveness and safety of deferasirox in the treatment of transfusional iron overload in MDS in clinical practice.
Material and methods A longitudinal, retrospective, observational study was carried out in a university hospital. We included MDS patients who were treated with deferasirox for transfusion dependent iron overload during the period of study (from January 2011 to April 2015).
Treatment effectiveness was assessed by serum ferritin (SF) and liver iron concentration (LIC), measured by MRI. Adverse events and reasons for treatment discontinuation were collected from clinical records. The percentage of patients that had laboratory values for liver enzymes, bilirubin, glomerular filtration rate (GFR) and haemoglobin falling outside of the normal ranges during the treatment was also registered.
Results 35 patients were included (50.0% men). Median (p25, p75) SF at baseline was 1636 µg/L (1100, 1634), which fell to 1399 µg/L (824, 1772) during follow-up. Median LIC was 6.4 mg/g (5.2, 12.5) at baseline and 4.6 mg/g (3.1, 6.1) during follow-up.
Median treatment duration during the period of study was 11.0 months (3.0, 37.8). 57.1% of patients discontinued deferasirox therapy. Reasons for treatment discontinuation were: renal toxicity (35.0%), exitus (25.0%), maintained SF below 500 µg (15.0%), discontinuation of blood transfusions (10.0%), gastrointestinal intolerance (5.0%) and clinical worsening (5.0%). Treatment discontinuation data were missing in 5% of cases. Among those patients that had a baseline value of AST within the normal range when treatment was initiated, 13.6% had a serum AST level >38 U/L, 29.2% had ALT >42 U/L and 37.5% had bilirubin >1.1 mg/dL during follow-up. Renal function worsened in 40% of patients who had a GFR <60 ml/min/1.73m2 at some point during treatment.
Conclusion Deferasirox was effective in most of the patients with a reduction in SF and LIC. Renal toxicity was the most frequent adverse event and it was the first reason for treatment discontinuation.
No conflict of interest.
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