Disorders of neutrophil number and function

UMMS Affiliation

Department of Pediatrics

Publication Date


Document Type



Gene Therapy; Granulocyte Colony-Stimulating Factor; Humans; Leukemia, Myeloid; Myelodysplastic Syndromes; *Neutropenia; Sepsis


Life Sciences | Medicine and Health Sciences


This review of disorders of neutrophil number and function will discuss important research advances in the field and then provide a clinical diagnostic approach. The focus will be on two recent clinical developments in the field of phagocyte disorders. First, an important natural history study from the Severe Chronic Neutropenia International Registry has recently quantitated the incidence and risk factors for death from sepsis and for progression to myelodysplastic syndrome and acute myeloid leukemia in a large cohort of severe chronic neutropenia patients, many of whom were followed 10 or more years on treatment with granulocyte colony-stimulating factor. Second, in the past year, a multinational group has announced successful gene therapy of two adults with chronic granulomatous disease, the most common disorder of neutrophil function. However, monitoring of retroviral insertion sites revealed expansion of the multiclonal population of gene-modified cells, raising concerns about eventual leukemogenesis. The review also provides a pragmatic approach to the evaluation of a patient with a suspected disorder of neutrophil number or function.

DOI of Published Version



Hematology Am Soc Hematol Educ Program. 2006;:104-10. Link to article on publisher's site

Journal/Book/Conference Title

Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program

Related Resources

Link to Article in PubMed

PubMed ID