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Bone Marrow Transplant as Definitive Therapy for β-Thalassemia Major Patients: A literature review

Open Access

Journal Type:Review Article

Subject:Medicine, Health & Food

Subject Field:Internal Medicine

Volume:115, Issue: 1, December, 2022

Publish Date:December 26, 2022 7:00 pm

Pages:434-440

Download:613

Views:698

Abstract

Thalassemia is a group of hereditary hemoglobin disorders characterized by insufficient production of at least one globin chain, resulting in unbalanced production of globin chains. Homozygous mutations in the β-globin gene, resulting in the absence of the β-chain, are the main cause of β-thalassemia major. Because the β-chain in β-thalassemia major is not formed, there is an accumulation of free α-chains in red blood cells, which can trigger apoptosis and hemolysis resulting in ineffective erythropoiesis. Management for β-thalassemia major patients requires lifelong therapy with blood transfusions and medication. However, blood transfusions and routine administration of iron chelation drugs cannot cure β-thalassemia major. Thalassemia can be cured through definitive therapies including bone marrow or stem cell transplantation and genetic therapy. Bone marrow transplantation is a treatment option for children and adolescents suffering from certain types of cancer and other blood disorders  such as thalassemia.

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