Deferasirox In The Treatment Of Iron Overload During Myeloproliferative Neoplasms (MPN)

Deferasirox is an oral iron chelator widely employed in the treatment of iron overload during thalassemic syndromes and myelodysplastic syndromes.

At present, very few data are available on the efficacy and safety of a treatment with deferasirox in patients with Ph- Myeloproliferative Neoplasms (MPN) and transfusional requirement.

To address this issue, we report here on 23 patients (M/F 17/6) with MPN and iron overload secondary to transfusional requirement enrolled in the database of our regional cooperative group who received a treatment with deferasirox. Of them, 20 had a primary Myelofibrosis, 2 a post Essential Thrombocythemia myelofibrotic phase and 1 a post Polycythemia Vera myelofibrotic phase.

The main features of the patients at diagnosis and at baseline of deferasirox treatment are reported in the Table: 

Treatment with deferasirox was started after a median interval from diagnosis of 9.8 months (IR 6.7 – 21.7) and from transfusion requirement of 7.3 months (IR 5.7 – 15.3). Starting deferasirox dose was 1,500 mg/day in 11 patients, 1,250 mg/day in 4 patients, 1,000 mg/day in 7 patients and <1,000 mg/day in 1 patient. All patients were evaluable for toxicity: extra-hematological toxicity was reported in 12/23 patients and consisted of gastro-intestinal symptoms in 6 patients, renal impairment in 4 patients and skin reactions in 2 patients. A dose reduction/temporary discontinuation was needed in 11 cases, but only 1 patient went off treatment due to recurrent renal toxicity. Eighteen patients were considered evaluable for response (≥ 6 months of treatment) and 5 were too early. As to the iron overload decrement, after a median treatment period of 14.4 months (IR 7.1 – 21.3) 2 patients achieved ferritin levels < 500 ng/ml, 7 patients ferritin levels < 1,000 ng/ml and 9 patients did not have any ferritin reduction. As to hematological improvement, 5/18 patients showed an unexpected and persistent rise of Hb levels > 1.5 g/dl, with disappearance of transfusional requirement in 3 cases.

Treatment with deferasirox is feasible and effective in MPN with iron overload. Moreover, also in this setting an hematological improvement can occur in a sizeable rate of patients.

No relevant conflicts of interest to declare.