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Metformin for treatment of cytopenias in children and young adults with Fanconi anemia
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Pollard, Jessica A. (Department of Pediatrics, Harvard Medical School, Boston, MA) ;
Furutani, Elissa (Department of Pediatrics, Harvard Medical School, Boston, MA) ;
Liu, Shanshan (Biostatistics and Research Design Center, Institutional Centers for Clinical and Translational Research, Harvard Medical School, Boston, MA) ;
Esrick, Erica (Department of Pediatrics, Harvard Medical School, Boston, MA) ;
Cohen, Laurie E. (Department of Endocrinology, and) ;
Bledsoe, Jacob (Department of Pathology, Boston Children's Hospital, Boston, MA) ;
Liu, Chih-Wei (Department of Environmental Sciences and Engineering, University of North Carolina at Chapel Hill, Chapel Hill, NC) ;
Lu, Kun (Department of Environmental Sciences and Engineering, University of North Carolina at Chapel Hill, Chapel Hill, NC) ;
Ramírez de Haro, Ma. José (Institut d'Investigació Biomèdica Sant Pau) ;
Surrallés i Calonge, Jordi (Institut d'Investigació Biomèdica Sant Pau) ;
Malsch, Maggie (Clinical Research Operations Center, Institutional Centers for Clinical and Translational Research, Boston Children's Hospital, Boston, MA) ;
Kuniholm, Ashley (Clinical Research Operations Center, Institutional Centers for Clinical and Translational Research, Boston Children's Hospital, Boston, MA) ;
Galvin, Ashley (Clinical Research Operations Center, Institutional Centers for Clinical and Translational Research, Boston Children's Hospital, Boston, MA) ;
Armant, Myriam (Trans Laboratory, Boston Children's Hospital, Boston, MA) ;
Kim, Annette S. (Department of Pathology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA) ;
Ballotti, Kaitlyn (Pediatric Hematology-Oncology, Boston Children's Hospital, Boston, MA) ;
Moreau, Lisa (Comprehensive Center for Fanconi Anemia, Dana-Farber Cancer Institute, Boston, MA) ;
Zhou, Yu (Pediatric Hematology-Oncology, Boston Children's Hospital, Boston, MA) ;
Babushok, Daria (Division of Hematology-Oncology, University of Pennsylvania, Philadelphia, PA) ;
Boulad, Farid (Pediatric Hematology-Oncology, Memorial Sloan-Kettering Cancer Center, New York, NY) ;
Carroll, Clint (Pediatric Hematology-Oncology, The Children's Hospital at TriStar Centennial, Nashville, TN) ;
Hartung, Helge (Pediatric Hematology-Oncology, Children's Hospital of Philadelphia, Philadelphia, PA) ;
Hont, Amy (Pediatric Hematology-Oncology, Children's National Medical Center, Washington, DC) ;
Nakano, Taizo (Pediatric Hematology-Oncology, Children's Hospital Colorado, Denver, CO) ;
Olson, Tim (Pediatric Hematology-Oncology, Children's Hospital of Philadelphia, Philadelphia, PA) ;
Sze, Sei-Gyung (Department of Pediatrics, Maine Medical Center, Tufts University School of Medicine, Portland, ME) ;
Thompson, Alexis A. (Pediatric Hematology-Oncology, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL) ;
Wlodarski, Marcin W. (Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN) ;
Gu, Xuesong (Beth Israel Deaconess Medical Center Genomics, Proteomics, Bioinformatics and Systems Biology Center, Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA) ;
Libermann, Towia A. (Beth Israel Deaconess Medical Center Genomics, Proteomics, Bioinformatics and Systems Biology Center, Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA) ;
D'Andrea, Alan (Radiation Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA) ;
Grompe, Markus (Oregon Stem Cell Center, Department of Pediatrics, Papé Family Institute, Oregon Health and Science University, Portland, OR; and) ;
Weller, Edie (Biostatistics and Research Design Center, Institutional Centers for Clinical and Translational Research, Harvard Medical School, Boston, MA) ;
Shimamura, Akiko (Department of Pediatrics, Harvard Medical School, Boston, MA)
Fanconi anemia (FA), a genetic DNA repair disorder characterized by marrow failure and cancer susceptibility. In FA mice, metformin improves blood counts and delays tumor development. We conducted a single institution study of metformin in nondiabetic patients with FA to determine feasibility and tolerability of metformin treatment and to assess for improvement in blood counts. [...]
2022 -  10.1182/bloodadvances.2021006490
Blood advances, Vol. 6 (june 2022) , p. 3803-3811
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