Response to iron chelation therapy may predict disease improvement in patients with myelofibrosis receiving treatment with deferasirox for transfusion-dependent anemia, according to results from a retrospective analysis published in the British Journal of Haematology.

A high prevalence of transfusion-dependent anemia characterizes myelofibrosis, a clonal myeloproliferative neoplasm. This can result in iron overload, which can be treated by iron chelation therapy with deferasirox.

Researchers assessed efficacy of deferasirox with regard to iron chelation response, hematologic improvement, and influence of deferasirox on survival and leukemic evolution in 45 consecutive patients with myelofibrosis at centers in Italy.

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At baseline, 40% of patients were cytoreduction-naive and 60% were on cytoreductive therapy. Of those patients on cytoreductive therapy, 12 received hydroxycarbamide, 14 received ruxolitinib, and 1 was on other therapy.

After a median myelofibrosis duration of 26.8 months (interquartile range [IQR], 2.6-208.7) and transfusion-dependent latency of 12.5 months (IQR, 1.2-146.1), patients received deferasirox. The majority of patients (91.1%) were evaluable for response to deferasirox.

Patients who experienced iron chelation response had significantly lower transfusion burden of red blood cell units prior to iron chelation therapy (16 vs 31 units transfused; P =.032), lower ferritin values (1390 mg/L vs 2000 mg/L; P =.0018), and decreased exposure to transfusion dependence before iron chelation therapy (8.7 vs 16.7 months; P =.04).

Patients who experienced iron chelation response had a progressive and substantial decrease in ferritin levels at 6, 12, and 18 months compared with baseline values.

Seven patients experienced a complete response (CR; 17%), 11 experienced a partial response (PR; 26.8%), and 23 experienced no response (56.1%). Achieving an iron chelation response predicted improvement in myelofibrosis: 91.7% of patients who experienced iron chelation response had a CR or PR compared with just 24.1% of patients with no iron chelation response (P <.001).

“Further controlled prospective trials are required to confirm a positive effect of iron chelation therapy with deferasirox on outcome of disease,” concluded the authors.

Reference

1.     Elli EM, Iurlo A, Aroldi A, et al. Deferasirox in the management of iron-overload in patients with myelofibrosis: a multicentre study from the Rete Ematologica Lombarda (IRON-M study) [published online May 20, 2019]. Br J Haematol. doi:10.1111/bjh.15964