{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T04:10:59Z","timestamp":1777435859269,"version":"3.51.4"},"reference-count":63,"publisher":"Pensoft Publishers","issue":"2","license":[{"start":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T00:00:00Z","timestamp":1777507200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["FM"],"abstract":"<jats:p>Beta-thalassemia is a hereditary disease that affects the synthesis of \u03b2-globin, resulting in ineffective erythropoiesis, chronic anemia, and iron overload. Although the traditional treatment approach, which includes hypertransfusion and chelation therapy, has increased patient survival, it does not address the underlying disease mechanisms. Improving erythroid maturation, increasing fetal hemoglobin synthesis, lowering iron burden, and modifying the disease\u2019s genetic background are the goals that have resulted in the development of several therapeutic classes and treatment options. Erythroid maturation modifiers have been shown to significantly reduce transfusion requirements in patients with beta-thalassemia, and inducers of fetal hemoglobin and hepcidin agonists show promise in controlling ineffective erythropoiesis and secondary iron overload. Gene therapy using lentiviral vectors or CRISPR\/Cas9 genome editing has the potential to provide a significant proportion of patients with long-term transfusion independence. These therapeutic advances could significantly improve the quality of life and long-term survival of patients with beta-thalassemia. However, long-term studies are still needed to establish their safety profiles, refine patient selection criteria, and ensure greater access to these innovative treatments.<\/jats:p>","DOI":"10.3897\/folmed.68.e180627","type":"journal-article","created":{"date-parts":[[2026,4,28]],"date-time":"2026-04-28T11:57:16Z","timestamp":1777377436000},"source":"Crossref","is-referenced-by-count":0,"title":["Changing the treatment paradigm in beta thalassemia"],"prefix":"10.3897","volume":"68","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9892-7979","authenticated-orcid":true,"given":"Katya","family":"Sapunarova","sequence":"first","affiliation":[{"name":"First Department of Internal Medicine, Division of Hematology, Medical University of Plovdiv, Plovdiv, Bulgaria"},{"name":"Clinic of Clinical Hematology, St. George University Hospital, Plovdiv, Bulgaria"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"2258","published-online":{"date-parts":[[2026,4,30]]},"reference":[{"key":"180627_B1"},{"key":"180627_B2"},{"key":"180627_B3"},{"key":"180627_B4"},{"key":"180627_B5"},{"key":"180627_B6"},{"key":"180627_B7"},{"key":"180627_B8"},{"key":"180627_B9"},{"key":"180627_B10"},{"key":"180627_B11"},{"key":"180627_B12"},{"key":"180627_B13"},{"key":"180627_B14"},{"key":"180627_B15","doi-asserted-by":"crossref","unstructured":"Gerds AT, Vannucchi AM, Passamonti F, et al. Luspatercept in MF-associated anaemia. Blood 2019; 134(Suppl 1):557.","DOI":"10.1182\/blood-2019-122546"},{"key":"180627_B16"},{"key":"180627_B17"},{"key":"180627_B18"},{"key":"180627_B19"},{"key":"180627_B20"},{"key":"180627_B21"},{"key":"180627_B22"},{"key":"180627_B23"},{"key":"180627_B24"},{"key":"180627_B25"},{"key":"180627_B26"},{"key":"180627_B27"},{"key":"180627_B28","unstructured":"A study evaluating the efficacy and safety of mitapivat in participants with non-transfusion-dependent alpha- or beta-thalassemia (\u03b1- or \u03b2-NTDT). ClinicalTrials.gov. Identifier NCT04770753. 2021 [Accessed on 17.11.2025]. Available from: https:\/\/clinicaltrials.gov\/study\/NCT04770753"},{"key":"180627_B29","unstructured":"A study to determine the efficacy, safety, pharmacokinetics, and pharmacodynamics of AG-348 in adult participants with non-transfusion-dependent thalassemia. ClinicalTrials.gov ID NCT03692052. 2019 [Accsessed on 17.11.2025]. Available from: https:\/\/clinicaltrials.gov\/study\/NCT03692052"},{"key":"180627_B30"},{"key":"180627_B31"},{"key":"180627_B32"},{"key":"180627_B33"},{"key":"180627_B34"},{"key":"180627_B35"},{"key":"180627_B36"},{"key":"180627_B37"},{"key":"180627_B38"},{"key":"180627_B39","doi-asserted-by":"crossref","unstructured":"Goranov G, Nikolov P. [Periprocedural complications and risk factors after carotid stenting in patients with concomitant coronary artery disease]. Bulgarian Cardiology. 2021;27(4):72\u201379 [Bulgarian].","DOI":"10.3897\/bgcardio.27.e75361"},{"key":"180627_B40"},{"key":"180627_B41"},{"key":"180627_B42"},{"key":"180627_B43","doi-asserted-by":"crossref","unstructured":"Aghajan M, Casu C, Presti VL, et al. Developing a Galnac-Conjugated TMPRSS6 Antisense Therapy for the Treatment of \u03b2-Thalassemia. Blood 2016; 128 (22): 1013. Available from: https:\/\/ashpublications.org\/blood\/article\/128\/22\/1013\/96080\/.","DOI":"10.1182\/blood.V128.22.1013.1013"},{"key":"180627_B44"},{"key":"180627_B45"},{"key":"180627_B46"},{"key":"180627_B47"},{"key":"180627_B48"},{"key":"180627_B49"},{"key":"180627_B50"},{"key":"180627_B51"},{"key":"180627_B52","unstructured":"A trial testing SP-420 in subjects with transfusion-dependent \u03b2-thalassemia or low-risk myelodysplastic syndromes. ClinicalTrials.gov ID NCT05693909. Available from: https:\/\/clinicaltrials.gov\/study\/NCT05693909"},{"key":"180627_B53"},{"key":"180627_B54"},{"key":"180627_B55","doi-asserted-by":"crossref","unstructured":"Locatelli F, Thompson AA, Kwiatkowski JL, et al. Betibeglogene for non-\u03b20\/\u03b20. N Engl J Med 2022; 386(5):415\u201327.","DOI":"10.1056\/NEJMoa2113206"},{"key":"180627_B56"},{"key":"180627_B57","unstructured":"The Guardian. NHS to offer gene therapy for thalassemia. 2024. 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