Deferasirox Decreases Serum Ferritin, Iron in RBC Transfusion-Dependent MDS
The 3-year, prospective, multicenter trial administered deferasirox at a starting dose of 20mg/kg (with dose escalation up to 40mg/kg/day permitted) to 173 patients with serum ferritin levels of ≥1,000µg/L who had received ≥20 units of RBCs with ongoing transfusion requirements.
“Median serum ferritin decreased 23% in the 53% of patients who completed 12 months of treatment (n=91), 36.7% in patients who completed 2 years (n=49), and 36.5% in patients who completed 3 years (n=33) despite continued transfusion requirement,” the investigators found.
Reduced serum ferritin levels correlated with ALT improvement (P<0.001). At baseline, 68 patients (39.3%) had elevated labile plasma iron, which was subsequently measured quarterly during the first year. By week 13, labile plasma iron levels had normalized in all patients with an abnormal baseline level. By International Working Group 2006 criteria, 51 patients (28%) experienced hematologic improvement; of these, only 7 received growth factors or treatment for MDS.
Over 3 years, 138 (79.8%) patients discontinued therapy, 43 (24.8%) due to adverse events or disease progression and 23 (13.2%) due to abnormal laboratory values. The most common drug-related adverse events were gastrointestinal disturbances and increased serum creatinine. A total of 28 patients died; none were considered related to treatment with deferasirox.
“Overall, this study demonstrated improvements in iron parameters with prolonged deferasirox chelation in a cohort of heavily transfused lower risk patients with MDS accompanied by improvements in hepatic transaminases and hematologic improvement,” the investigators wrote. “A randomized controlled trial is warranted to better ascertain the clinical impact of deferasirox therapy in lower risk patients with MDS.”