Ontology highlight
ABSTRACT:
SUBMITTER: Donadieu J
PROVIDER: S-EPMC9821599 | biostudies-literature | 2022 Dec
REPOSITORIES: biostudies-literature
Donadieu Jean J Bellanné-Chantelot Christine C
Hematology. American Society of Hematology. Education Program 20221201 1
Severe congenital neutropenias (SCNs) are rare diseases, and to date about 30 subtypes have been described according to their genetic causes. Standard care aims to prevent infections and limit the risk of leukemic transformation; however, several subtypes may have additional organ dysfunction(s), requiring specialized care. Granulocyte colony-stimulating factor and hematopoietic stem cell transplantation are now the bedrock of standard care. Better understanding of SCN mechanisms now offers the ...[more]