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Deferasirox in the management of iron overload in patients with myelofibrosis treated with ruxolitinib: The

Elena Maria Elli1, Ambra Di Veroli2, Daniela Bartoletti3,4

  • 1Hematology Division and Bone Marrow Unit, Ospedale San Gerardo, Monza, Italy.

British Journal of Haematology
|February 9, 2022
PubMed

Insights

The combination of ruxolitinib (RUX) and deferasirox (DFX) effectively manages iron overload (IOL) in myelofibrosis (MF) patients, showing good safety and improving survival outcomes. This RUX-DFX therapy achieved significant iron chelation and erythroid responses.

Area of Science:

  • Hematology
  • Oncology
  • Pharmacology

Background:

  • Iron overload (IOL) is a common complication in hematological malignancies like myelofibrosis (MF).
  • Deferasirox (DFX) is a standard treatment for IOL, while ruxolitinib (RUX) targets myelofibrosis symptoms.

Purpose of the Study:

  • To retrospectively evaluate the safety and efficacy of combined ruxolitinib (RUX) and deferasirox (DFX) therapy in myelofibrosis (MF) patients with iron overload (IOL).
  • To assess iron chelation response (ICR), erythroid response (ER), and impact on overall survival.

Main Methods:

  • Retrospective analysis of 69 MF patients treated with RUX and DFX for IOL.
  • Data collected on RUX-DFX therapy duration, dose adjustments, adverse events, ICR, ER, and survival.

Main Results:

  • RUX-DFX therapy was administered for a median of 12.4 months.
  • Dose reductions or discontinuations due to adverse events were reported in 52.2% and 49.3% for RUX, and 11.6% and 13.1% for DFX, respectively; no unexpected toxicity.
  • Iron chelation response (ICR) and erythroid response (ER) were achieved in 47.8% and 46.4% of patients, respectively. Thirteen patients (18.9%) became transfusion-independent.
  • Achieving ER correlated with ICR (p=0.04). Absence of leukocytosis at baseline and achieving ICR predicted improved survival.

Conclusions:

  • The combination of RUX and DFX is a safe and effective strategy for managing IOL in MF patients.
  • The therapy demonstrated significant ICR and ER, correlating with improved survival outcomes.
  • Further clinical investigation of this RUX-DFX combination strategy is warranted.
Keywords:
cancerdeferasiroxiron overloadmyelofibrosisruxolitinib

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