Metformin as a Fetal Hemoglobin Inducer in Non-transfusion Dependent Thalassemia Patients

TL;DR

This study tested metformin as a treatment to increase fetal hemoglobin in thalassemia patients but found no significant benefits beyond existing therapy.

Contribution

The study is the first to evaluate metformin's in vivo efficacy as a fetal hemoglobin inducer in NTDT patients.

Findings

Metformin did not significantly change clinical or laboratory variables in NTDT patients.

Reticulocytic count was higher in the metformin group during the study.

No additional benefit of metformin was observed when combined with hydroxyurea.

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.