Tolerated, Low-Dose Deferasirox: Successful Three Cases of Aplastic Anemia and 34 Cases of MDS

Abstract 1187

It has been shown that serum ferritin (SF) levels are associated with mortality in transfusion-dependent patients with MDS. Optimal dosing with deferasirox has been shown to decrease SF level even in transfusion-dependent patients and to be able to decrease the adverse effects of repeated transfusion. Previous studies have however shown that about 50% of patients withdraw from treatment because of side effects in the early phase of treatment.

Low-dose deferasirox (mean 6.3 mg/kg, range 1.6∼25.6) was administered to 53 patients (MDS 34, AA 13, ALL 2, MF 2, AML 1,ML 1) with dose escalation up to the maximum dose tolerated (mean 9.8 mg/kg, range 1.6∼31.0). In 47 eligible cases, a mean SF of 1579 ng/ml (range 320∼4477) decreased to 1101 ng/ml (range 233∼1680) at 18 months with 71% of continuous rate. Just six cases had treatment stopped, solely because of renal dysfunction. In three cases with transfusion-dependent aplastic anemia, deferasirox (6.25∼10.8 mg/kg) was given with G-CSF, cyclosporin A, and metenolone with the successful abreaction from transfusion dependency and even with elevation of the platelet count (from 10 to 30∼50×10⁹/L).

Continuous administration of low-dose deferasirox, as tolerated, was thought to be sufficient to prevent a rise in SF in transfusion-dependent patients. A prospective study that will examine OS in such MDS patients has been started.