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International Journal of Hematology
Published in: International Journal of Hematology 3/2024

03-03-2023 | Aplastic Anemia | Progress in Hematology

Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia

Author: Nao Yoshida

Published in: International Journal of Hematology | Issue 3/2024

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Abstract

Acquired aplastic anemia (AA) in children is a rare bone marrow failure that requires several special considerations for its diagnosis and treatment compared with that in adults. The most common issue is the differential diagnosis with refractory cytopenia of childhood and inherited bone marrow failure syndromes, which is crucial for making decisions on the appropriate treatment for pediatric AA. In addition to detailed morphological evaluation, a comprehensive diagnostic work-up that includes genetic analysis using next-generation sequencing will play an increasingly important role in identifying the underlying etiology of pediatric AA. When discussing treatment strategies for children with acquired AA, the long-term sequelae and level of hematopoietic recovery that affect daily or school life should also be considered, although the overall survival rate has reached 90% after immunosuppressive therapy or hematopoietic cell transplantation (HCT). Recent advances in HCT for pediatric patients with acquired AA have been remarkable, with the successful use of upfront bone marrow transplantation from a matched unrelated donor, unrelated cord blood transplantation or haploidentical HCT as salvage treatment, and fludarabine/melphalan-based conditioning regimens. This review discusses current clinical practices in the diagnosis and treatment of acquired AA in children based on the latest data.
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Metadata
Title
Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia
Author
Nao Yoshida
Publication date
03-03-2023
Publisher
Springer Nature Singapore
Published in
International Journal of Hematology / Issue 3/2024
Print ISSN: 0925-5710
Electronic ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-023-03564-4

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