Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia

Fanconi anemia (FA), a genetic disorder affecting DNA repair, is characterized by bone marrow failure and cancer susceptibility. In FA mouse models, metformin (N,N-dimethylguanide) a biguanide metabolic agent, improves blood counts and delays tumor development. Given these findings, we conducted a s...

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Bibliographic Details
Published in:Blood 2021-11, Vol.138 (Supplement 1), p.1102-1102
Main Authors: Pollard, Jessica A., Furutani, Elissa M., Liu, Shanshan, Esrick, Erica B., Cohen, Laurie, Bledsoe, Jacob, Lu, Kun, Ramirez, Maria Jose, Surrallés, Jordi, Malsch, Maggie, Kuniholm, Ashley, Galvin, Ashley, Armant, Myriam, Kim, Annette S., Ballotti, Kaitlyn, Moreau, Lisa, Zhou, Yu, Babushok, Daria V., Boulad, Farid, Carroll, Clinton, Hartung, Helge, Hont, Amy, Nakano, Taizo A., Olson, Timothy S., Sze, Sei-Gyung K., Thompson, Alexis A, Wlodarski, Marcin W., Libermann, Towia A, D'Andrea, Alan D., Grompe, Markus, Weller, Edie A., Shimamura, Akiko
Format: Article
Language:English
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Summary:Fanconi anemia (FA), a genetic disorder affecting DNA repair, is characterized by bone marrow failure and cancer susceptibility. In FA mouse models, metformin (N,N-dimethylguanide) a biguanide metabolic agent, improves blood counts and delays tumor development. Given these findings, we conducted a single institution pilot study of metformin in non-diabetic patients with FA to assess feasibility, tolerability, and impact of metformin on hematologic response as defined by modified MDS International Working Group (IWG) criteria (Table 1). Fourteen of 15 patients with at least 1 cytopenia (hemoglobin
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2021-153598