Metformin effective for improving blood counts in children with Fanconi anemia: Study

Written By : Medha Baranwal |Medically Reviewed By : Dr. Kamal Kant Kohli Published On 2022-09-16T19:30:51+05:30 | Updated On 16 Sept 2022 7:30 PM IST

USA: Findings from a recent study in Blood Advances suggest that metformin is safe and well tolerated therapeutic approach in non-diabetic pediatric patients with fanconi anemia (FA).

Fanconi anemia is a genetic DNA repair disorder characterized by cancer susceptibility and failure of bone marrow. Metformin was shown to improve blood counts and delay tumor development in FA mice. Considering this, Jessica A. Pollard, Pediatric Hematology-Oncology, Boston Children's Hospital, Boston, MA, and colleagues conducted a single institution study of metformin in nondiabetic patients with FA to determine tolerability and feasibility of metformin treatment and to assess for improvement in blood counts.

Fourteen of 15 patients with at least 1 cytopenia (hemoglobin < 10 g/dL; platelet count < 100 000 cells/µL; or an absolute neutrophil count < 1000 cells/µL) met the eligivilty criteria to receive metformin for 6 months. Median age of patients was 9.4 years (range 6.0-26.5 ).

Based on the study, the researchers reported the following:

Thirteen of 14 subjects (93%) tolerated maximal dosing for age; 1 subject had dose reduction for grade 2 gastrointestinal symptoms.
No subjects developed hypoglycemia or metabolic acidosis.
No subjects had dose interruptions caused by toxicity, and no grade 3 or higher adverse events attributed to metformin were observed.
Hematologic response based on modified Myelodysplastic Syndrome International Working Group criteria was observed in 4 of 13 evaluable patients (30.8%).
Median time to response was 84.5 days (range 71-128 days).
Responses were noted in neutrophils (n = 3), platelets (n = 1), and red blood cells (n = 1).
No subjects met criteria for disease progression or relapse during treatment.
Correlative studies explored potential mechanisms of metformin activity in FA.
Plasma proteomics showed reduction in inflammatory pathways with metformin.

This led to the conclusion that metformin is safe and tolerable in nondiabetic patients with FA and may provide therapeutic benefit.

Reference:

Pollard JA, Furutani E, Liu S, Esrick E, Cohen LE, Bledsoe J, Liu CW, Lu K, de Haro MJR, Surrallés J, Malsch M, Kuniholm A, Galvin A, Armant M, Kim AS, Ballotti K, Moreau L, Zhou Y, Babushok D, Boulad F, Carroll C, Hartung H, Hont A, Nakano T, Olson T, Sze SG, Thompson AA, Wlodarski MW, Gu X, Libermann TA, D'Andrea A, Grompe M, Weller E, Shimamura A. Metformin for treatment of cytopenias in children and young adults with Fanconi anemia. Blood Adv. 2022 Jun 28;6(12):3803-3811. doi: 10.1182/bloodadvances.2021006490. PMID: 35500223.

Blood Advancesfanconi anemiametformin

Source : Blood Advances

Medha Baranwal

MSc. Biotechnology

Medha Baranwal joined Medical Dialogues as an Editor in 2018 for Speciality Medical Dialogues. She covers several medical specialties including Cardiac Sciences, Dentistry, Diabetes and Endo, Diagnostics, ENT, Gastroenterology, Neurosciences, and Radiology. She has completed her Bachelors in Biomedical Sciences from DU and then pursued Masters in Biotechnology from Amity University. She has a working experience of 5 years in the field of medical research writing, scientific writing, content writing, and content management. She can be contacted at editorial@medicaldialogues.in. Contact no. 011-43720751

Dr. Kamal Kant Kohli

Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as a Chief Editor of Medical News. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Email: drkohli@medicaldialogues.in. Contact no. 011-43720751