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

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 -globin gene, resulting in the absence of the -chain, are the main cause of -thalassemia major. Because the -chain in -thalassemia major is not formed, there is an accumulation of free -chains in red blood cells, which can trigger apoptosis and hemolysis resulting in ineffective erythropoiesis. Management for -thalassemia major patients requires lifelong therapy with blood transfusions and medication. However, blood transfusions and routine administration of iron chelation drugs cannot cure -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.