European Society for Paediatric Research Abstract

IMPROVED SURVIVAL FROM GRANULOCYTE-COLONY STIMULATING FACTOR THERAPY FOR CONGENITAL NEUTROPENIA UNMASKS PREDISPOSITION TO MYELODYSPLASIA AND ACUTE MYELOID LEUKEMIA

Abstract 19 First part

First part

Background. Over the last decade, recombinant human granulocyte-colony stimulating factor (G-CSF) has had a major impact on management of "severe chronic neutropenia", a term used collectively that refers to congenital idiopathic, or cyclic neutropenia. More than 90% of patients respond to G-CSF with an increase in neutrophils to > 1.5 × 109/L, and with a marked reduction in infections and a vastly improved survival. Several reports indicate that responders with congenital neutropenia have developed myelodysplastic syndromes and acute myeloblastic leukemia (MDS/AML) raising the question on the role of G-CSF in the pathogenesis of the malignant transformation.

Methods. The Severe Chronic Neutropenia International Registry (SCNIR) has collected data on 531 neutropenic patients from 13 countries treated with G-CSF from 1987 to 1997, including 302 patients with congenital neutropenia. To clarify the risk of MDS/AML in congenital neutropenia and the potential role of G-CSF in pathogenesis, these data were analyzed with respect to treatment and patient demographics.

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