Longitudinal Prospective Comparison of Pancreatic Iron by Magnetic Resonance in Thalassemia Patients Transfusion-dependent Since Early Childhood Treated with Combination Deferiprone-desferrioxamine Vs Deferiprone or Deferasirox Monotherapy

Overview

Journal Blood Transfus

Specialty Hematology

Date 2023 May 5

PMID 37146300

Authors

Paolo Ricchi

Antonella Meloni

Laura Pistoia

Maria R Gamberini

Liana Cuccia

Massimo Allo

Maria C Putti

Anna Spasiano

Rosamaria Rosso

Valerio Cecinati

Riccardo Righi

Stefania Renne

Giuseppe Peritore

Antonino Vallone

Vincenzo Positano

Emilio Quaia

Filippo Cademartiri

Alessia Pepe

Affiliations

Soon will be listed here.

Abstract

Background: In transfusion-dependent thalassemia patients who started regular transfusions in early childhood, we prospectively and longitudinally evaluated the efficacy on pancreatic iron of a combined deferiprone (DFP) + desferrioxamine (DFO) regimen versus either oral iron chelator as monotherapy over a follow-up of 18 months.

Materials And Methods: We selected patients consecutively enrolled in the Extension-Myocardial Iron Overload in Thalassemia network who received a combined regimen of DFO+DFP (No.=28) or DFP (No.=61) or deferasirox (DFX) (No.=159) monotherapy between the two magnetic resonance imaging scans. Pancreatic iron overload was quantified by the T2* technique.

Results: At baseline no patient in the combined treatment group had a normal global pancreas T2* (≥26 ms). At follow-up the percentage of patients who maintained a normal pancreas T2* was comparable between the DFP and DFX groups (57.1 vs 70%; p=0.517).Among the patients with pancreatic iron overload at baseline, global pancreatic T2* values were significantly lower in the combined DFO+DFP group than in the DFP or DFX groups. Since changes in global pancreas T2* values were negatively correlated with baseline pancreas T2* values, the percent changes in global pancreas T2* values, normalized for the baseline values, were considered. The percent changes in global pancreas T2* values were significantly higher in the combined DFO+DFP group than in either the DFP (p=0.036) or DFX (p=0.030) groups.

Discussion: In transfusion-dependent patients who started regular transfusions in early childhood, combined DFP+DFO was significantly more effective in reducing pancreatic iron than was either DFP or DFX.

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