Guidelines for diagnosis and management of beta-thalassemia intermedia

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Informa Healthcare

Abstract

Beta-thalassemia intermedia (β-TI) is a genetic variant of beta-thalassemias with a clinical disorder whose severity falls between thalassemia minor and thalassemia major. Different genetic defects are involved in this disorder and, based on severity of disease, clinical complications like skeletal deformities and growth retardation, splenomegaly, extramedullary hematopoiesis, heart failure, and endocrine disorders may be present in untreated patients. Precise diagnosis and management are essential in these patients for prevention of later clinical complications. Diagnosis of TI is based on clinical and laboratory data. There are some treatment strategies like modulation of gamma-globulin chain production with hydroxyurea or other drugs, transfusion, splenectomy, and stem cell transplantation. Iron chelation therapy is also needed in many of these patients even if they are not transfused. The aim of this manuscript is to review the clinical manifestations, complications, genetic defects, and unmet treatments needs in TI. © 2014 Informa Healthcare USA, Inc.

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Beta-thalassemia intermedia, Clinical complications, Diagnosis, Management, Beta-thalassemia, Blood transfusion, Gamma-globulins, Humans, Iron chelating agents, Leg ulcer, Practice guidelines as topic, Quality of life, Anticoagulant agent, Antioxidant, Calcium, Carnitine, Ferritin, Folic acid, Hemoglobin a2, Hemoglobin beta chain, Hemoglobin f, Hemoglobin gamma chain, Hydroxyurea, Iron, Magnesium chloride, Recombinant erythropoietin, Sildenafil, Transferrin, Vitamin d, Immunoglobulin, Iron chelating agent, Alloimmunization, Beta globin gene, Beta thalassemia, Beta thalassemia intermedia, Brain ischemia, Cachexia, Cerebrovascular accident, Diet supplementation, Differential diagnosis, Dyserythropoiesis, Extramedullary hematopoiesis, Ferritin blood level, Gene, Gene locus, Gene mutation, Genetic disorder, Hemoglobin blood level, Hemolysis, Hemolytic anemia, Hepatitis c, Heterozygosity, Human, Hypercoagulability, Hyperuricemia, Ineffective erythropoiesis, Iron blood level, Iron chelation, Iron overload, Lung embolism, Nuclear magnetic resonance imaging, Phenotype, Portal vein thrombosis, Practice guideline, Pulmonary hypertension, Review, Splenectomy, Stem cell transplantation, Survival, Thalassemia intermedia, Thalassemia major, Thromboembolism, Biosynthesis, Complication, Genetics

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