Assessment and Management of Iron Overload in β-Thalassemia Major Patients in Italy: A Real-Life Experience From the Webthal® Project
Authors: Piga Antonio , Longo Filomena , Musallam Khaled M , Cappellini Maria Domenica , Forni Gian Luca , Quarta Giovanni , Chiavilli Francesco , Commendatore Francesca Valeria , Mulas Sergio , Caruso Vincenzo , Galanello Renzo 
Published: October 2019

Abstract 1030

Background:

As the ideal assessment and management strategies of iron overload in patients with β-thalassemia major (TM) remain unclear, evaluation of real-life experiences is warranted.

Methods:

This was a cross-sectional study of 924 TM patients actively treated at nine Italian centers. Data were retrieved on 15-09-2009 through a common software (Webthal®) used by participating centers, and included: demographics; splenectomy and transfusion history; detailed iron overload assessment history; type of iron chelator used; existence of hepatitis C virus, heart disease, or diabetes mellitus; alanine aminotransferase and serum creatinine levels.

Results:

The mean age of patients was 30.1 ± 9.1 years (48.8% males, 42.8% splenectomized). Among the 924 patients, 83.9% had at least one previous liver iron concentration (LIC) measurement and 68.7% had it within two years prior. On step-wise multivariate logistic regression, a serum ferritin level <2500 ng/ml was a risk factor for absence of LIC measurement, while absence of heart disease and a history of normal cardiac magnetic resonance (CMR) T2* value were risk factors for a delay in LIC measurement >2 years. Moreover, 69.6% had at least one previous CMR T2* measurement and 73.4% patients had it within two years prior. Patients who never had a CMR T2* were <18 years, had iron intake ≤0.4 mg/kg/day, or a serum ferritin level <2500 ng/ml; while a history of a normal CMR T2* value was the main risk factor for a delay in CMR T2* assessment for >2 years. Using similar multivariate modeling, desferrioxamine (23.3%) was more commonly used in patients with hepatitis C virus infection or high serum creatinine level. Deferiprone (20.6%) was less commonly prescribed in patients with elevated alanine aminotransferase; while a desferrioxamine+deferiprone combination (17.9%) was more commonly used in patients with serum ferritin levels >2500 ng/ml or CMR T2* <20 ms. Deferasirox (38.3%) was more commonly prescribed in patients <18 years, but less commonly used in patients who had heart disease or those with a high iron intake.

Conclusion:

Assessment and management of iron overload in TM patients in Italy largely reflects findings from clinical research and recommendations from available guidelines, although some practices are expected to change in light of evolving evidence.

Disclosures:

Piga: Apopharma: Research Funding; Novartis Pharmaceuticals: Research Funding; Ferrokin: Research Funding. Musallam:Novartis Pharmaceuticals: Honoraria. Cappellini:Novartis Pharmaceuticals: Honoraria, Research Funding. Forni:Novartis Pharmaceuticals: Research Funding; Ferrokin: Research Funding. Quarta:Novartis Pharmaceuticals: Research Funding. Galanello:Apopharma: Honoraria, Research Funding; Novartis Pharmaceuticals: Honoraria, Research Funding; Ferrokin: Research Funding.