title: Recombinant human G-CSF (Lenograstim) for infectious complications in glycogen storage disease type Ib. Report of 7 cases.
abstract: Seven patients with glycogen storage disease type Ib suffering from severe and/or recurrent bacterial infections were treated with glycosylated recombinant G-CSF (Lenograstim). Mean follow up was 20.8 months (range 9-30 months). In all cases a median dose of 5 micrograms/kg/day resulted in rapid clinical improvement, associated in 6/7 with an increase in absolute polymorphonuclear (PMN) count. In the remaining subject, a striking amelioration of infectious status contrasted with a persistently low PMN count. Liver transplantation in one patient resolved metabolic complications but did not improve PMN count or the infectious status, while neutropenia was corrected by G-CSF. Prevention of recurrent infections was achieved in all cases with intermittent therapy. Short term treatment was well tolerated, thrombocytopenia in two patients (WHO grade 0 and grade 3) recovering after decrease of G-CSF dosage.
authors.affiliation: Service de Pédiatrie Générale et Laboratoire d'Hématologie, Hôpital Bicêtre, Le Kremlin-Bicêtre, France.
authors.forename: J
authors.initials: J
authors.lastname: Donadieu
authors.forename: B
authors.initials: B
authors.lastname: Bader-Meunier
authors.forename: Y
authors.initials: Y
authors.lastname: Bertrand
authors.forename: A
authors.initials: A
authors.lastname: Lachaux
authors.forename: P
authors.initials: P
authors.lastname: Labrune
authors.forename: M A
authors.initials: MA
authors.lastname: Gougerot-Pocidalo
authors.forename: M
authors.initials: M
authors.lastname: Odièvre
authors.forename: P
authors.initials: P
authors.lastname: Gibeaud
authors.forename: A
authors.initials: A
authors.lastname: Yver
authors.forename: G
authors.initials: G
authors.lastname: Tchernia
mesh_terms: D000293:Adolescent; D001424:Bacterial Infections / Q000097:blood / Q000150:complications / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D005953:Glycogen Storage Disease Type I / Q000097:blood / Q000150:complications*; D016179:Granulocyte Colony-Stimulating Factor / Q000009:adverse effects / Q000627:therapeutic use*; D006403:Hematologic Tests; D006801:Humans; D000078224:Lenograstim; D008297:Male; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D012008:Recurrence
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; D000078224:Lenograstim