Spanish registry of hemoglobinopathies and rare anemias (REHem-AR) : demographics, complications, and management of patients with β-thalassemia
Bardón-Cancho, Eduardo J. (Universidad Complutense de Madrid)
Marco-Sánchez, José Manuel (Universidad Complutense de Madrid)
Beneitez Pastor, David (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Payán-Pernía, Salvador (Universidad de Sevilla)
Llobet, Anna Ruiz (Institut de Recerca Pediàtrica Hospital San Joan de Déu de Barcelona)
Berrueco, Rubén (Institut de Recerca Pediàtrica Hospital San Joan de Déu de Barcelona)
García-Morin, Marina (Universidad Complutense de Madrid)
Beléndez, Cristina (Universidad Complutense de Madrid)
Senent, Leonor (Hospital Universitario y Politécnico La Fe)
Acosta, María José Ortega (Hospital Universitario Virgen de las Nieves (Granada))
Pleguezuelos, Irene Peláez (Hospital Universitario Virgen de las Nieves (Granada))
Velasco, Pablo (Hospital Universitari Vall d'Hebron)
Collado, Anna (Hospital Universitari Vall d'Hebron)
Moreno-Servet, Marta (Universidad Complutense de Madrid)
Argilés, Bienvenida (Hospital Universitario y Politécnico La Fe)
de Soto, Inmaculada Pérez (Universidad de Sevilla)
del Mar Bermúdez, María (Hospital Virgen de la Arrixaca)
Salido Fiérrez, Eduardo J. (Hospital Universitario Virgen de la Arrixaca)
Blanco, Adoración (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Navarro, Pablo González (Hospital General Universitario Gregorio)
Cela, Elena (Universidad Complutense de Madrid)
Universitat Autònoma de Barcelona

Date:	2024
Abstract:	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. We performed an observational, multicenter, and ambispective study, which included patients of any age with TDT and NTDT, registered up to December 31, 2021. 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). 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.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Anemia ; Complications ; Hemoglobinopathies ; Registry ; Spain ; Thalassemia
Published in:	Annals of Hematology, Vol. 103 (march 2024) , p. 1525-1539, ISSN 1432-0584

DOI: 10.1007/s00277-024-05694-z
PMID: 38519604

15 p, 2.4 MB

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