title: Human granulocyte colony-stimulating factor (rHuG-CSF) for treatment of neutropenia in Shwachman syndrome.
abstract: We report a boy affected by Shwachman syndrome (SS) who presented severe neutropenia and frequent suppurative infections, which we treated successfully with granulocyte colony-stimulating factor (rHuG-CSF). Daily injections of 7.5 micrograms/kg/day significantly increased the absolute neutrophil count and he was free from infections. In order to avoid the risk of side effects and to improve the child's quality of life, we intermittently administered lower doses of rHuG-CSF. A weekly dose of 2 micrograms/kg/day was able to maintain the absolute neutrophil count high enough (0.58-1.2 x 10(9)/L) to prevent suppurative infections. During the follow-up period (2.5 years) we tried suspending rHuG-CSF twice, but the absolute neutrophil count (0.18-0.31 x 10(9)/L) fell significantly and suppurative infections reoccurred (otitis, perianal abscess). rHuG-CSF may be a useful therapeutic agent in patients with symptomatic neutropenia in SS.
authors.affiliation: Istituto di Clinica Pediatrica, Policlinico Santa Chiara, Università di Pisa, Italy.
authors.forename: A
authors.initials: A
authors.lastname: Ventura
authors.forename: D
authors.initials: D
authors.lastname: Dragovich
authors.forename: P
authors.initials: P
authors.lastname: Luxardo
authors.forename: G
authors.initials: G
authors.lastname: Zanazzo
mesh_terms: D001855:Bone Marrow Diseases / Q000097:blood*; D002675:Child, Preschool; D010188:Exocrine Pancreatic Insufficiency / Q000097:blood*; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D009503:Neutropenia / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000627:therapeutic use; D013577:Syndrome
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor