[Aplastic anemia in children: recent advances in diagnosis and treatment].

Abstract

Pediatric aplastic anemia (AA) is a rare bone marrow failure that requires several special considerations for its diagnosis and treatment compared with adult AA. Accurate differential diagnosis is crucial, and distinguishing acquired AA from inherited bone marrow failure syndromes is particularly important. In addition to detailed morphological evaluation, comprehensive diagnostic workups incorporating genetic analysis using next-generation sequencing have become increasingly important for guiding treatment decisions. When discussing treatment strategies for pediatric AA, the long-term sequelae and level of hematopoietic recovery should also be considered, although the survival rates now exceed 90% following immunosuppressive therapy or hematopoietic cell transplantation (HCT). Recent advances in HCT include improved conditioning regimens, successful upfront HCT from matched unrelated donors, and expanded salvage treatment options such as unrelated cord blood transplantation or haploidentical HCT. This review outlines these recent advances in the diagnosis and treatment of AA in children based on the latest data.