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Multicenter Study
. 2022 Apr;197(2):190-200.
doi: 10.1111/bjh.18057. Epub 2022 Feb 8.

Deferasirox in the management of iron overload in patients with myelofibrosis treated with ruxolitinib: The multicentre retrospective RUX-IOL study

Elena Maria Elli  1 , Ambra Di Veroli  2 , Daniela Bartoletti  3   4 , Alessandra Iurlo  5 , Ida Carmosino  6 , Giulia Benevolo  7 , Elisabetta Abruzzese  8 , Massimiliano Bonifacio  9 , Micaela Bergamaschi  10 , Nicola Polverelli  11 , Marianna Caramella  12 , Daniela Cilloni  13 , Mario Tiribelli  14 , Novella Pugliese  15 , Giovanni Caocci  16 , Elena Crisà  17 , Raffaele Porrini  18 , Uros Markovic  19 , Rossella Renso  1 , Giuseppe Auteri  3   4 , Daniele Cattaneo  5 , Malgorzata Monika Trawinska  8 , Luigi Scaffidi  9 , Lucia Biale  20 , Cristina Bucelli  5 , Massimo Breccia  6 , Carlo Gambacorti-Passerini  1 , Giuseppe Alberto Palumbo  21 , Roberto Latagliata  2 , Francesca Palandri  3
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Free article
Multicenter Study

Deferasirox in the management of iron overload in patients with myelofibrosis treated with ruxolitinib: The multicentre retrospective RUX-IOL study

Elena Maria Elli et al. Br J Haematol. 2022 Apr.
Free article

Abstract

Deferasirox (DFX) is used for the management of iron overload (IOL) in many haematological malignancies including myelofibrosis (MF). The 'RUX-IOL' study retrospectively collected 69 MF patients treated with ruxolitinib (RUX) and DFX for IOL to assess: safety, efficacy in term of iron chelation response (ICR) and erythroid response (ER), and impact on overall survival of the combination therapy. The RUX-DFX therapy was administered for a median time of 12.4 months (interquartile range 3.1-71.2). During treatment, 36 (52.2%) and 34 (49.3%) patients required RUX and DFX dose reductions, while eight (11.6%) and nine (13.1%) patients discontinued due to RUX- or DFX-related adverse events; no unexpected toxicity was reported. ICR and ER were achieved by 33 (47.8%) and 32 patients (46.4%) respectively. Thirteen (18.9%) patients became transfusion-independent. Median time to ICR and ER was 6.2 and 2 months respectively. Patients achieving an ER were more likely to obtain an ICR also (p = 0.04). In multivariable analysis, the absence of leukocytosis at baseline (p = 0.02) and achievement of an ICR at any time (p = 0.02) predicted improved survival. In many MF patients, the RUX-DFX combination provided ICR and ER responses that correlated with improved outcome in the absence of unexpected toxicities. This strategy deserves further clinical investigation.

Keywords: cancer; deferasirox; iron overload; myelofibrosis; ruxolitinib.

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