ABSTRACT
Non-transfusion dependent thalassemia (NTDT) refers to a group of thalassemic disorders who do not need regular transfusions for survival, however it may be needed in certain conditions. Metformin was reported as a potential fetal hemoglobin (HbF) inducing agent in vitro but its efficacy and safety in vivo was not fully studied. This is a prospective interventional study aimed at studying the effect of metformin on HbF change in NTDT. METHODS: Patients with established diagnosis of NTDT were enrolled. They were receiving a stable fixed dose of Hydroxyurea over the last 3 months. Patients were divided into two groups: a group that received Metformin for 6 months (Metformin group) and a control group. Complete blood picture, reticulocytic count, hemoglobin electrophoresis, liver enzymes, bilirubin, kidney functions, LDH and random blood sugar were performed at onset, 3 and 6 months of the study. All adverse events were recorded. RESULTS: Forty two patients aged 12-23 years were enrolled. Metformin intake over 6 months did not show any statistically significant difference in clinical or the laboratory variables of efficacy when compared to the control group apart from reticulocytic count which was higher in Metformin group throughout the study. CONCLUSION: Metformin intake, in addition to hydroxyurea, did not yield any extra benefit among patients with NTDT.
ABSTRACT
Patients with thalassemia major requiring regular blood transfusions accumulate iron that is toxic to the heart, liver, and endocrine systems. The following prospective, randomized trial was carried out to determine the effectiveness, in children and young adults, of combined deferiprone (DFP) and deferoxamine (DFO) in reducing transfusional iron overload compared to either drug alone and to assess the safety and tolerability of DFP. Sixty-six patients were randomized into three treatment arms: daily DFP combined with DFO twice weekly; daily DFP only; and DFO only 5 days/week. Fifty-six patients completed the 54 weeks and were assessed by different indices. A significant reduction of liver iron concentration and serum ferritin was observed in all three arms while significant reduction of liver iron score was observed in patients on combination therapy only. Cardiac function did not significantly change in any arm. Compliance improved in patients who received combined therapy. Toxicity of DFP was mild to moderate and acceptable; most commonly, transient arthropathy and nausea/vomiting were observed. Thus, combination therapy has shown to be effective in reducing iron overload in thalassemia patients.