Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
نویسندگان
چکیده
منابع مشابه
Prevalence of Diabetes Mellitus in Patients with Transfusion Dependent β Thalassemia
Introduction: Thalassemia is common in Iran. Appropriate therapy for this disease includes a regular blood transfusion and chelation therapy. However, patients will inevitably confront with side effects, particularly iron overloads in critical organ including heart, ductless glands and liver. This study tries to determine the prevalence of diabetes mellitus in transfusion dependent β thalassemi...
متن کاملHydroxyurea Treatment in Transfusion-Dependent β-Thalassemia Patients
BACKGROUND β-Thalassemia is an inherited hemoglobin disorder caused by defective synthesis of ß-globin chains. Hemoglobin (Hb) F induction is a possible therapeutic approach which can partially compensate for α and non-α globin chains imbalance. OBJECTIVES We aimed to investigate the efficacy and safety of Hydroxyurea (HU) in diminishing transfusion requirements of patients with β-thalassemia...
متن کاملhydroxyurea treatment in transfusion-dependent β-thalassemia patients
conclusions: hydroxyurea can be safely used in some transfusion-dependent β-thalassemia patients to decrease their transfusion needs. background: β-thalassemia is an inherited hemoglobin disorder caused by defective synthesis of ß-globin chains. hemoglobin (hb) f induction is a possible therapeutic approach which can partially compensate for α and non-α globin chains imbalance. objectives: we a...
متن کاملBone density in transfusion dependent thalassemia patients in Urmia, Iran
Background Patients with thalassemia major and intermedia are susceptible to osteopenia and osteoporosis. The mechanism of osteoporosis in these patients is multifactorial. Transfusion related iron overload in endocrine organs leads to impaired growth hormone secretion, diabetes mellitus, hypothyroidism, hypoparathyroidism, lack of sex steroids and vitamin D deficiency that contribute t...
متن کاملPrevalence of Alloantibodies and Autoantibodies in Transfusion Dependent Thalassemia Patients
Background: The development of anti-red blood cell alloantibodies remains a major problem in transfusion of blood in thalassemia major patients. Also, Autoantibodies can result in clinical hemolysis and difficulty in cross-matching blood. We studied the frequency of red blood cell alloimmunization and autoimmunization among thalassemia patients who received regular transfusions in Ilam province...
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ژورنال
عنوان ژورنال: New England Journal of Medicine
سال: 2018
ISSN: 0028-4793,1533-4406
DOI: 10.1056/nejmoa1705342