G-Csf Vs Haematopoietic Stem Cell Transplantation In Severe Congenital Neutropenia With Elane Mutation: Role Of G-Csf Dose. A Retrospective Controlled Study On Behalf Of Saawp (Severe Aplastic Anemia Working Party) Of The Ebmt, Of The Stem Cell Transplant For Immunodeficiency Group In Europe (Scetide), Of The Severe Chronic Neutropenia French Registry (Scnfr) And Italian Neutropenia Registry (Inr)

BLOOD(2017)

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摘要
Background: Established indications for Hematopietic Stem Cell Transplantation (HSCT) in SCN include transformation to acute leukemia (AL) and/or myelodisplasia (MDS) and G-CSF resistance (need for >20mcg/kg/d). Indication for HSCT also appears appropriate in patients with poor response to G-CSF (10-20 mcg/kg/d) and, as a consequence, a high incidence of infectious events. Indication is less clear in cases requiring a G-CSF doses up to 10 mcg/kg but with good control of infections.
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