title: [Long-term treatment with recombinant human granulocyte colony stimulating factor in patients with severe congenital neutropenia].
abstract: Severe congenital neutropenia is a disorder of myelopoiesis characterized by severe neutropenia secondary to either a maturational arrest of myelopoiesis at the level of promyelocytes (Kostmann-Syndrome; SCN) or regular cyclic fluctuations in the number of blood neutrophils with a median ANC below 500/microliter (cyclic neutropenia). We have treated 32 patients with SCN and 4 patients with cyclic neutropenia. Thirty of 32 patients with SCN and all 4 patients with SCN responded to r-met HuG-CSF treatment with an increase of the median ANC to above 1000/microliter. The doses needed to achieve and maintain the response varied between 0.8 and 120 micrograms/kg/d. Long-term treatment did not exhaust the myelopoiesis: The mean ANC remained stable up to 5 years of treatment. The increase in ANC was associated with dramatic clinical responses: significant reduction of severe bacterial infections, reduction of intravenous antibiotic treatment episodes, and reduction of hospitalizations. No severe bacterial infections occurred in any of the r-met HuG-CSF responders during long-term treatment. Severe adverse event, most likely associated with the underlying disease, included the development of MDS/Leukemia in two patients, and osteopenia/osteoporosis in 12 patients. These results demonstrate the beneficial effects of r-met HuG-CSF treatment in severe congenital neutropenia patients.
authors.affiliation: Abteilung für Pädiatrische Hämatologie und Onkologie, Medizinische Hochschule Hannover.
authors.forename: C
authors.initials: C
authors.lastname: Zeidler
authors.forename: A
authors.initials: A
authors.lastname: Reiter
authors.forename: E
authors.initials: E
authors.lastname: Yakisan
authors.forename: B
authors.initials: B
authors.lastname: Koci
authors.forename: H
authors.initials: H
authors.lastname: Riehm
authors.forename: K
authors.initials: K
authors.lastname: Welte
mesh_terms: D000293:Adolescent; D015519:Bone Density / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D005260:Female; D000069585:Filgrastim; D005500:Follow-Up Studies; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage*; D006410:Hematopoiesis / Q000187:drug effects; D006801:Humans; D007223:Infant; D007958:Leukocyte Count / Q000187:drug effects; D008134:Long-Term Care; D008297:Male; D009503:Neutropenia / Q000151:congenital* / Q000276:immunology / Q000628:therapy; D009504:Neutrophils / Q000187:drug effects; D011994:Recombinant Proteins / Q000008:administration & dosage
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; D000069585:Filgrastim