Published in:
Open Access
23-03-2024 | Thalassemia | Original Article
Spanish registry of hemoglobinopathies and rare anemias (REHem-AR): demographics, complications, and management of patients with β-thalassemia
Authors:
Eduardo J. Bardón-Cancho, José Manuel Marco-Sánchez, David Benéitez-Pastor, Salvador Payán-Pernía, Anna Ruiz Llobet, Rubén Berrueco, Marina García-Morin, Cristina Beléndez, Leonor Senent, María José Ortega Acosta, Irene Peláez Pleguezuelos, Pablo Velasco, Anna Collado, Marta Moreno-Servet, Bienvenida Argilés, Inmaculada Pérez de Soto, María del Mar Bermúdez, Eduardo J. Salido Fiérrez, Adoración Blanco-Álvarez, Pablo González Navarro, Elena Cela
Published in:
Annals of Hematology
|
Issue 5/2024
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Abstract
Introduction
The increase in the number of patients with hemoglobinopathies in Europe in recent decades highlights the need for more detailed epidemiological information in Spain. To fulfil this need, the Spanish Society of Pediatric Hematology and Oncology (SEHOP) sponsored the creation of a national registry of hemoglobinopathies known as REHem-AR (Spanish Registry of Hemoglobinopathies and Rare Anemias). Data from the transfusion-dependent (TDT) and non–transfusion-dependent (NTDT) β-thalassemia cohorts are described and analyzed.
Methods
We performed an observational, multicenter, and ambispective study, which included patients of any age with TDT and NTDT, registered up to December 31, 2021.
Results
Among the 1741 patients included, 168 cases of thalassemia were identified (103 TDT and 65 NTDT-patients). Survival at 18 years was 93% for TDT and 100% for NTDT. Regarding management, 80 patients with TDT (77.7%) and 23 patients with NTDT (35.4%) started chelation treatment during follow-up, with deferasirox being the most widely used. A total of 76 patients within the TDT cohort presented at least 1 complication (73.8%), the most frequent being hemosiderosis and osteopenia-osteoporosis. Comparison of both cohorts revealed significant differences in the diagnosis of hepatic hemosiderosis (p = 0.00024), although these were not observed in the case of cardiac iron overload (p = 0.27).
Discussion
Our registry enabled us to describe the management of β thalassemia in Spain and to analyze the morbidity and mortality of the cohorts of patients with TDT and NTDT. Complications related to iron overload in TDT and NTDT account for most of the morbidity and mortality of the disease, which is associated with a considerable social, psychological, and economic impact, although cardiac, osteopathy and endocrinological complications requiring more attention. The convenience and simplicity of online registries make it possible to homogenize variables and periodically update data, thus providing valuable information on these diseases.