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 the biguanide metabolic agent metformin improves blood counts and delays tumor development. We conducted a single institution pilot study of metformin i...

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Veröffentlicht in:	Blood advances 2022-06, Vol.6 (12), p.3803-3811
Hauptverfasser:	Pollard, Jessica A., Furutani, Elissa, Liu, Shanshan, Esrick, Erica, Cohen, Laurie E., Bledsoe, Jacob, Liu, Chih-Wei, Lu, Kun, de Haro, Maria Jose Ramirez, Surrallés, Jordi, Malsch, Maggie, Kuniholm, Ashley, Galvin, Ashley, Armant, Myriam, Kim, Annette S., Ballotti, Kaitlyn, Moreau, Lisa, Zhou, Yu, Babushok, Daria, Boulad, Farid, Carroll, Clint, Hartung, Helge, Hont, Amy, Nakano, Taizo, Olson, Tim, Sze, Sei-Gyung, Thompson, Alexis A., Wlodarski, Marcin W., Gu, Xuesong, Libermann, Towia A, D’Andrea, Alan, Grompe, Markus, Weller, Edie, Shimamura, Akiko
Format:	Artikel
Sprache:	eng
Schlagworte:	anemia bone marrow failure Child Fanconi anemia Fanconi Anemia - drug therapy Fanconi Anemia - genetics hematopoiesis Humans metformin Metformin - therapeutic use neutropenia thrombocytopenia Young Adult
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Zusammenfassung:	Fanconi anemia (FA), a genetic disorder affecting DNA repair, is characterized by bone marrow failure and cancer susceptibility. In FA mouse models the biguanide metabolic agent metformin improves blood counts and delays tumor development. We conducted a single institution pilot study of metformin in non-diabetic patients with FA to assess feasibility and tolerability of metformin treatment and to determine whether metformin could improve blood counts. Fourteen of 15 patients with at least 1 cytopenia (hemoglobin
ISSN:	2473-9529 2473-9537 2473-9537
DOI:	10.1182/bloodadvances.2021006490