Disorders of neutrophil number and function
Department of Pediatrics
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
Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
Newburger, Peter E., "Disorders of neutrophil number and function" (2006). Open Access Articles. 621.