Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia

Exclusion Criteria

• •Patients who underwent bone marrow transplantation.
• •Patients with evidence of myelodysplasia, leukemia, or other concurrent malignancy.
• •Patients who have a history of allergic reactions to metformin or similar compounds.
• •Patients with a history of symptomatic hypoglycemia over the past year or hypoglycemia \< 50 mg/dL on screening and baseline laboratory assessments.
• •Patients with type 1 diabetes mellitus.
• •Patients with vitamin B12 deficiency.
• •Patients with Glucose-6-Phosphate Dehydrogenase deficiency.
• •Patients with abnormal Kidney function tests including serum creatinine, elevated liver function tests including live enzymes (ALT or AST \> 135 U/L, total bilirubin \> 1.5 x upper limit of normal for age, and/or patients with metabolic acidosis (bicarbonate \< 17 meq/L on venous blood gases).