Thesis (M.Sc.) - Cairo University - Faculty of Medicine - Department of Pediatrics

Background: Metformin was reported as a potential fetal hemoglobin (HbF) inducing agent but its efficacy and safety in Non-Transfusion-Dependent Thalassemia (NTDT) patients were not fully studied. Objectives: A prospective interventional study aimed to study the effect of metformin on HbF change in NTDT and its role in ameliorating disease severity. Methods: Forty-two patients with established diagnosis of NTDT aged 12-23 years old were enrolled. They were receiving a stable dose of Hydroxyurea (HU) over the last 3 months. Inclusion criteria:- Confirmed diagnosis of NTDT based on hemoglobin Electrophoresis and/or high performance liquid Chromatography .- Age greater than or equal to 12 years of age.- Stable dose of HU over the last 3 months.- Serum creatinine less than or equals to 1.4 mg/dl . - Liver enzymes (ALT and AST) less than or equal to 3 times upper limits of normal .- Patients/ parents giving willingly informed consents Exclusion Criteria: - Simple or chronic red blood cell transfusion therapy in the last 3 months.- Patients with diabetes mellitus who are receiving other anti-diabetic medications.- Patients with chronic hypoxemic conditions associated with lactic acidosis, such as cardiovascular, renal, hepatic and pulmonary diseases

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