Oncotarget

Research Papers:

Safety and tolerability of deferasirox in pediatric hematopoietic stem cell transplant recipients: one facility’s five years’ experience of chelation treatment

Natalia Maximova _, Massimo Gregori, Roberto Simeone, Aurelio Sonzogni, Giulia Boz, Carmen Fucile, Valeria Marini, Antonietta Martelli and Francesca Mattioli

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Oncotarget. 2017; 8:63177-63186. https://doi.org/10.18632/oncotarget.18725

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Abstract

Natalia Maximova1, Massimo Gregori2, Roberto Simeone3, Aurelio Sonzogni4, Giulia Boz5, Carmen Fucile6, Valeria Marini6, Antonietta Martelli6 and Francesca Mattioli6

1Bone Marrow Transplant Unit, Institute for Maternal and Child Health - IRCCS Burlo Garofolo, Trieste, Italy

2Department of Pediatric Radiology, Institute for Maternal and Child Health - IRCCS Burlo Garofolo, Trieste, Italy

3University of Trieste, Trieste, Italy

4Department of Pathology, Ospedale Beato Papa Giovanni XIII, Bergamo, Italy

5University of Cagliari, Faculty of Medicine, Cagliari, Italy

6Pharmacology and Toxicology Unit, University of Genoa, Genoa, Italy

Correspondence to:

Maximova Natalia, email: natalia.maximova@burlo.trieste.it

Keywords: deferasirox, allogeneic hematopoietic stem cell transplantation (allo-HSCT), pediatric, ductopenia, therapeutic drug monitoring

Received: April 05, 2017     Accepted: May 23, 2017     Published: June 28, 2017

ABSTRACT

42 pediatric patients with iron overload, who underwent liver biopsy and DFX treatment after hematopoietic stem cell transplantation were included in the study group. The patients were divided into two groups diversified according to deferasirox trough plasma concentrations (DFX Ctrough) with cut-off equal to10 mcg/mL. The average dose of DFX was 25.9 mg/kg in the DFX Ctrough < 10 mcg/mL group versus 19.2 mg/kg in the DFX Ctrough > 10 mcg/mL group (p=0,0003). The mean duration of DFX treatment was 135.7 days in the DFX Ctrough < 10 mcg/mL group versus 41.8 days in the DFX Ctrough > 10 mcg/mL group (p<0.0001). The mean tissue iron concentration in the DFX Ctrough < 10 mcg/mL group was 261.9 μmol/g versus 133.4 μmol/g in the DFX Ctrough > 10 mcg/mL group (p < 0.0001). 21 patients (100%) in the DFX Ctrough > 10 mcg/mL group had ductopenia which was complete in 47.6% of them and severe in 52.4%. All patients with particularly high Ctrough (> 25 mcg/mL) were found to have total ductopenia. 90.5% of all deferasirox-related adverse events and 100% of major adverse events occurred in the DFX Ctrough > 10 mcg/mL group. In the DFX Ctrough < 10 mcg/mL group only one patient interrupted chelation therapy versus 16 (84.2%) patients in the DFX Ctrough > 10 mcg/mL group. We would recommend a close monitoring in pediatric hematopoietic transplant recipients subjected to deferasirox-based therapy because we have observed a high incidence of adverse events and discontinuation of chelation treatment.


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