Immunosuppressive therapy for severe aplastic anemia in children under the age of 3 years yields a high response rate: a North American collaborative study
Year: 2026
Type: article
Abstract: Distinguishing acquired (idiopathic/immune) severe aplastic anemia (SAA) from inherited bone marrow failure syndromes (IBMFS) is critical since only acquired cases respond to immunosuppressive therapy (IST), the standard treatment in cases without a matched sibling donor. Differentiating SAA from IBMFS is particularly challenging in children younger than 3 years, in whom inherited disorders are more prevalent. No age threshold exists to guide IST use in this population. Thus, we evaluated IST outcomes in children aged.
Source: Haematologica
Authors: Talya Wittmann Dayagi, Laura Willis, Staci D. Arnold, Jessica Boklan, Michaela Cada, Thomas D. Coates, Catherine Corriveau-Bourque, Christopher J. Gamper, Amy E. Geddis, Bertil Glader, Gary M. Kupfer, Taizo A. Nakano, Timothy S. Olson, Zora R. Rogers, Akiko Shimamura, Clifford Takemoto, Alexis A. Thompson, David A. Williams, Yigal Dror
Institutions: University of Toronto, Hospital for Sick Children, Emory University, Children's Healthcare of Atlanta, University of Arizona College of Medicine- Phoenix, University of Southern California, Children's Hospital of Los Angeles, University of Alberta, Stollery Children's Hospital, Johns Hopkins University, Johns Hopkins Medicine, Sidney Kimmel Comprehensive Cancer Center, Seattle Children's Hospital, Stanford Medicine, Stanford University, Georgetown Lombardi Comprehensive Cancer Center, Children's Hospital Colorado, University of Colorado Anschutz, Children's Hospital of Philadelphia, University of Pennsylvania, The University of Texas Southwestern Medical Center, Harvard University, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, St. Jude Children's Research Hospital
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $2,157
OpenAlex ID: W7154563752
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