Congenital Neutropenia and Rare Functional Phagocyte Disorders in Children

Hematol Oncol Clin North Am. 2019 Jun;33(3):533-551. doi: 10.1016/j.hoc.2019.01.004. Epub 2019 Apr 1.

Abstract

Both profound neutropenia and functional phagocyte disorders render patients susceptible to recurrent, unusual, and/or life-threatening infections. Many disorders also have nonhematologic manifestations and a substantial risk of leukemogenesis. Diagnosis relies on clinical suspicion and interrogation of the complete blood count with differential/bone marrow examination coupled with immunologic and genetic analyses. Treatment of the quantitative neutrophil disorders depends on granulocyte colony-stimulating factor, whereas management of functional phagocyte disease is reliant on antimicrobials and/or targeted therapies. Hematopoietic stem cell transplant remains the only curative option for most disorders but is not used on a routine basis.

Keywords: ELANE; Granulocyte colony-stimulating factor (G-CSF); Neutropenia; Oxidative burst; Phagocytes.

Publication types

  • Review

MeSH terms

  • Child
  • Congenital Bone Marrow Failure Syndromes / genetics
  • Congenital Bone Marrow Failure Syndromes / immunology
  • Congenital Bone Marrow Failure Syndromes / therapy*
  • Granulocyte Colony-Stimulating Factor / therapeutic use*
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Leukemia / genetics
  • Leukemia / immunology
  • Leukemia / therapy
  • Myelodysplastic Syndromes / genetics
  • Myelodysplastic Syndromes / immunology
  • Myelodysplastic Syndromes / therapy
  • Neutropenia / congenital*
  • Neutropenia / genetics
  • Neutropenia / immunology
  • Neutropenia / therapy
  • Neutrophils / immunology
  • Neutrophils / metabolism
  • Phagocytes / immunology
  • Phagocytes / metabolism*
  • Rare Diseases / genetics
  • Rare Diseases / immunology
  • Rare Diseases / therapy*

Substances

  • Granulocyte Colony-Stimulating Factor

Supplementary concepts

  • Neutropenia, Severe Congenital, Autosomal Recessive 3