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World J Clin Cases. Apr 6, 2025; 13(10): 100223
Published online Apr 6, 2025. doi: 10.12998/wjcc.v13.i10.100223
Beta thalassemia syndromes: New insights
Ana Dordevic, Ines Mrakovcic-Sutic, Sonja Pavlovic, Milena Ugrin, Jelena Roganovic
Ana Dordevic, Department of Business Development, Jadran Galenski Laboratorij, Rijeka 51000, Croatia
Ines Mrakovcic-Sutic, Faculty of Medicine, University of Rijeka, Rijeka 51000, Croatia
Sonja Pavlovic, Milena Ugrin, Institute of Molecular Genetics and Genetic Engineering, University of Belgrade, Belgrade 11000, Serbia
Jelena Roganovic, Department of Pediatric Hematology and Oncology, Children’s Hospital Zagreb, Zagreb 10000, Croatia
Jelena Roganovic, Faculty of Biotechnology and Drug Development, University of Rijeka, Rijeka 51000, Croatia
Author contributions: Dordevic A performed the research and wrote the original draft; Mrakovcic-Sutic I contributed to the design of the manuscript, and supervision; Pavlovic S was involved in conceptualization, writing, and supervision; Ugrin M contributed to the writing and literature review; Roganovic J designed the overall concept, and is responsible for the editing and submission of the current version of the manuscript.
Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Jelena Roganovic, MD, PhD, Department of Pediatric Hematology and Oncology, Children’s Hospital Zagreb, Klaiceva 16, Zagreb 10000, Croatia. jelena.roganovic@kdb.hr
Received: August 10, 2024
Revised: November 6, 2024
Accepted: December 2, 2024
Published online: April 6, 2025
Processing time: 130 Days and 18.4 Hours
Core Tip

Core Tip: Beta thalassemia syndromes are among the most common monogenic disorders worldwide, characterized by impaired hemoglobin synthesis that leads to ineffective erythropoiesis, chronic hemolysis, iron overload, and subsequent complications. The clinical manifestations are diverse, as a result of the underlying beta-globin gene variants and the coinheritance of modifying factors. Recent advances in the understanding of the underlying molecular and cellular mechanisms have facilitated the development of novel therapeutic strategies. The purpose of this article is to briefly describe new insights in the pathophysiology of this old disease, and discuss revolutionary changes in the treatment landscape for severe forms of beta thalassemia.