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Front Pediatr. 2019 Oct 24;7:436. doi: 10.3389/fped.2019.00436. eCollection 2019.

The Evidence for Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Neutrophil Disorders: A Comprehensive Review by the Inborn Errors Working Party Group of the EBMT.

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Division for Pediatric Stem Cell Transplantation and Immunology, University Hospital Frankfurt, Frankfurt, Germany.
Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah Medical Center, Jerusalem, Israel.
Department of Immunology, Garvan Institute of Medical Research, Darlinghurst, NSW, Australia.
Graduate Research School, University of New South Wales, Kensington, NSW, Australia.


Congenital disorders of the immune system affecting maturation and/or function of phagocytic leucocytes can result in severe infectious and inflammatory complications with high mortality and morbidity. Further complications include progression to MDS/AML in some cases. Allogeneic stem cell transplantation is the only curative treatment for most patients with these diseases. In this review, we provide a detailed update on indications and outcomes of alloHSCT for congenital neutrophil disorders, based on data from the available literature.


granulocyte colony-stimulating factor; hematopoietic stem cell transplantation; leukemia; neutropenia; neutrophils

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