Metformin effective against nondiabetic patients of Fanconi anemia: Study

2. There were no cases of hypoglycemia or metabolic acidosis.

3. There were no dosage interruptions owing to toxicity, and there were no grade 3 or higher adverse events associated with metformin.

4. Four of the thirteen evaluable patients had a hematologic response (HR) based on modified MDS IWG criteria.

5. The average time to respond was 84.5 days (range 71-128). Neutrophils (n=3), platelets (n=1), and red blood cells (n=1) all showed responses.

6. During therapy, no patients satisfied the criteria for illness progression or relapse.

7. Correlative research looked at the various mechanisms of metformin activity in FA.

8. Metformin reduced inflammatory pathways in plasma proteomics.

In conclusion, Metformin may be a safe and acceptable treatment option in non-diabetic patients with FA, according to this study. Additional research is required to strengthen the current evidence.

Reference:

Pollard, J., Furutani, E., Liu, S., Esrick, E. B., Cohen, L. E., Bledsoe, J. R., Liu, C.-W., Lu, K., Ramírez, M. J., Surrallés, J., Malsch, M. M., Kuniholm, A., Galvin, A., Armant, M., Kim, A. S., Ballotti, K., Moreau, L. A., Zhou, Y., Babushok, D. V., … Shimamura, A. (2022). Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia. In Blood Advances. American Society of Hematology. https://doi.org/10.1182/bloodadvances.2021006490