Overview and new treatments in Beta-thalassemia
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Angulo Ureña G, Koss Hernández R, Monge Ortiz JM. Overview and new treatments in Beta-thalassemia. Rev.méd.sinerg. [Internet]. 2020Dec.1 [cited 2024Nov.24];5(12):e549. Available from: https://revistamedicasinergia.com/index.php/rms/article/view/549

Abstract

Beta-thalassemia is a congenital disorder generally caused by point mutations in the gene that encodes for the synthesis of globin beta chains, which produces an ineffective erythropoiesis phenomenon, and reduces the half-life of erythrocytes in the most severe forms. However, the classification includes forms of thalassemia of various clinical behaviors; from severe forms to asymptomatic forms. Complications are usually associated with the chronic treatment utilized in this disorder, which includes repeated blood transfusions, amongst others. New therapies seek to reduce the adverse effects associated with transfusions, as well as targeting genetic components of the disease.

https://doi.org/10.31434/rms.v5i12.549

Keywords

thalassemia. hemoglobins. genetic therapy. blood transfusion. iron chelating agents.
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References

Origa R. β-Thalassemia.Genetics in Medicine [Internet]. 2016 [Citado 27/06/2020];19(6):609-619. https://doi.org/10.1038/gim.2016.173

Shah F, Sayani F, Trompeter S, Drasar E, Piga A. Challenges of blood transfusions in β-thalassemia, Blood Reviews [Internet]. 2019 [Citado 27/06/2020]; 37:100588. https://doi.org/10.1097/gim.0b013e3181cd68ed.

Cappellini M, Porter J, Viprakasit V, Taher A. A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies? Blood Reviews [Internet]. 2018 [Citado 27/06/2020];32(4):300-311. https://doi.org/10.1016/j.bj.2015.10.001

Taher A, Karakas Z, Cassinerio E, Siritanaratkul N, Kattamis A, Maggio A et al. Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study. Blood [Internet]. 2018 [Citado 29/06/2020];131(2):263-265.https://doi.org/10.1182/blood-2017-06-790121

Hoffman R, Benz E, Silberstein L, Heslop H, Weitz J, Anastasi J et al. Hematology. Philadelphia, PA: Elsevier; 2018.

Khandros E, Kwiatkowski J. Beta Thalassemia. Hematology/Oncology Clinics of North America [Internet] 2019 [Citado 28/06/2020];33(3):339-353.https://doi.org/10.1016/j.hoc.2019.01.003

de Dreuzy E, Bhukhai K, Leboulch P, Payen E. Current and future alternative therapies for beta-thalassemia major. Biomedical Journal [Internet]. 2016 [Citado 25/06/2020];39(1):24-38. https://dx.doi.org/10.1016%2Fj.bj.2015.10.001

Salah, Naouel Ben, et al. Revisiting Beta Thalassemia Intermedia: Past, Present, and Future Prospects. Hematology [Internet]. 2017 [Citado 1/08/2020];22(20):607-616. https://doi.org/10.1080/10245332.2017.1333246

Hoffbrand A, Steensma, P. Essential Hematology. Philadelpia, PA: Wiley Blackwell, 2019.

Cappellini M, Viprakasit V, Taher A, Georgiev P, Kuo K, Coates T et al. A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia. New England Journal of Medicine. [Internet] 2020 [Citado 29/06/2020] ;382(13):1219-1231. https://doi.org/10.1056/nejmoa1910182

Betts, Marissa, et al. Systematic Literature Review of the Burden of Disease and Treatment for Transfusion-Dependent β-Thalassemia. Clinical Therapeutics [Internet]. 2020 [Citado 29/06/2020]; 42(2):322-337.https://doi.org/10.1016/j.clinthera.2019.12.003

Soni S. Novel and innovative approaches for treatment of β-thalassemia. Pediatric Hematology Oncology Journal [Internet]. 2017 [Citado 29/06/2020];2(4):121-126.https://doi.org/10.1016/j.phoj.2017.11.153

El-Beshlawy, Amal, and Mona El-Ghamrawy. Recent Trends in Treatment of Thalassemia. Blood Cells, Molecules, and Diseases [Internet]. 2019 [Citado 29/06/2020]; 79:53-59. https://doi.org/10.1016/j.bcmd.2019.01.006

Thein S. Molecular basis of β thalassemia and potential therapeutic targets. Blood Cells, Molecules, and Diseases [Internet]. 2018 [Citado 29/06/2020]; 70:54-65.https://dx.doi.org/10.1016%2Fj.bcmd.2017.06.001

Richard F, Lier J, Roubert B, Haboubi T, Göhring U, Dürrenberger F. Oral ferroportin inhibitor VIT‐2763: First‐in‐human, phase 1 study in healthy volunteers. American Journal of Hematology [Internet]. 2019 [Citado 29/06/2020];95(1):68-77.https://dx.doi.org/10.1002%2Fajh.25670

Karponi G, Zogas N. Gene Therapy for Beta-Thalassemia: Updated Perspectives. The Application of Clinical Genetics [Internet]. 2019 [Citado 29/06/2020]; 12:167-180. https://dx.doi.org/10.2147%2FTACG.S178546

Boulad, Farid, et al. Gene Therapy and Genome Editing.” Hematology/Oncology Clinics of North America [Internet]. 2018 [Citado 29/06/2020]; 32(2):329-342. https://doi.org/10.1016/j.hoc.2017.11.

Wienert, Beeke, et al. Wake-up Sleepy Gene: Reactivating Fetal Globin for β-Hemoglobinopathies. Trends in Genetics [Internet]. 2018 [Citado 29/06/2020]; 34(12): 927-940. https://doi.org/10.1016/j.tig.2018.09.004

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