Immunosuppressive therapy for severe aplastic anemia in children under the age of 3 years yields a high response rate: a North American collaborative study
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.
Authors
- Amy E. Geddis
- Michaela Cada
- Talya Wittmann Dayagi
- BE Glader
- Christopher Gamper (ORCID: https://orcid.org/0000-0003-0253-2177)
- Zora R. Rogers (ORCID: https://orcid.org/0000-0003-1478-2368)
- Taizo A. Nakano (ORCID: https://orcid.org/0000-0003-0389-2297)
- Staci D. Arnold (ORCID: https://orcid.org/0000-0001-6053-8521)
- Yigal Dror (ORCID: https://orcid.org/0000-0003-3953-1477)
- D A Williams
- Thomas D. Coates (ORCID: https://orcid.org/0000-0001-9878-6029)
- Gary M. Kupfer (ORCID: https://orcid.org/0000-0002-3988-2894)
- Jessica Boklan (ORCID: https://orcid.org/0000-0003-4251-8484)
- Catherine Corriveau-Bourque
- Timothy S. Olson
- Clifford Takemoto
- Akiko Shimamura
- Laura Willis
- Alexis A. Thompson
Institutions
- Seattle Children's Hospital (US)
- St. Jude Children's Research Hospital (US)
- Children's Hospital of Philadelphia (US)
- Children's Hospital of Los Angeles (US)
- Harvard University (US)
- University of Arizona (US)
- Johns Hopkins University (US)
- Palo Alto University (US)
- Georgetown University (US)
- University of Toronto (CA)
- Georgetown University Medical Center (US)
- Hospital for Sick Children (CA)
- Children's Hospital Colorado (US)
- Center for Cancer and Blood Disorders (US)
- Children's Healthcare of Atlanta (US)
- Stollery Children's Hospital (CA)
- Sidney Kimmel Comprehensive Cancer Center (US)
- Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US)
- The University of Texas Southwestern Medical Center (US)
- University of Colorado Denver (US)
- Stanford University (US)
Publication Details
- Journal
- Haematologica
- Published
- 2026-04-16
- DOI
- https://doi.org/10.3324/haematol.2025.300232
- Primary Topic
- Hematopoietic Stem Cell Transplantation
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Aplastic Anemia and Myelodysplasia Association of Canada