IRON CHELATION THERAPY WITH DEFERASIROX IN THE MANAGEMENT OF IRON OVERLOAD IN PRIMARY MYELOFIBROSIS

Elena Maria Elli, Angelo Belotti, Andrea Aroldi, Matteo Parma, Pietro Pioltelli, Enrico Maria Pogliani
  • Elena Maria Elli
    Division of Hematology, San Gerardo Hospital, Monza, Italy | elena.elli@libero.it
  • Angelo Belotti
    Division of Hematology, San Gerardo Hospital, Monza, Italy
  • Andrea Aroldi
    Division of Hematology, San Gerardo Hospital, Monza, Italy
  • Matteo Parma
    Division of Hematology, San Gerardo Hospital, Monza, Italy
  • Pietro Pioltelli
    Division of Hematology, San Gerardo Hospital, Monza, Italy
  • Enrico Maria Pogliani
    Division of Hematology, San Gerardo Hospital, Monza, Italy

Abstract

Deferasirox (DSX) is the principal option currently available for iron-chelation-therapy (ICT), principally in the management of myelodysplastic syndromes (MDS), while in primary myelofibrosis (PMF) the expertise is limited. We analyzed our experience in 10 PMF with transfusion-dependent anemia, treated with DSX from September 2010 to December 2013. The median dose tolerated of DSX was 750 mg/day (10 mg/kg/day), with 3 transient interruption of treatment for drug-related adverse events (AEs) and 3 definitive discontinuation for grade 3/4 AEs. According to IWG 2006 criteria, erythroid responses with DSX were observed in 4/10 patients (40%), 2 of them (20%) obtaining transfusion independence. Absolute changes in median serum ferritin levels (Delta ferritin) were greater in hematologic responder (HR) compared with non-responder (NR)  patients, already at 6 months of ICT respect to baseline. Our preliminary data open new insights regarding the benefit of ICT not only in MDS, but also in PMF with the possibility to obtain an erythroid response, overall in 40 % of patients. HR patients receiving DSX seem to have a better survival and a lower incidence of leukemic transformation (PMF-BP). Delta ferritin evaluation at 6 months could represent a significant predictor for a different survival and PMF-BP.  However, the tolerability of the drug seems to be lower compared to MDS, both in terms of lower median tolerated dose and for higher frequency of discontinuation for AEs. The biological mechanism of action of DSX in chronic myeloproliferative setting through an independent NF-κB inhibition could be involved, but further investigations are required.

Keywords

Iron overload; Primary Myelofibrosis; Deferasirox

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Submitted: 2014-07-21 14:09:07
Published: 2014-05-30 00:00:00
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