RECOMBINANT HUMAN G-CSF (LENOGRASTIM) FOR INFECTIOUS COMPLICATIONS INGLYCOGEN-STORAGE-DISEASE TYPE-IB - REPORT OF 7 CASES

Seven patients with glycogen storage disease type Ib suffering from se vere and/or recurrent bacterial infections were treated with glycosyla ted recombinant G-CSF (Lenograstim). Mean follow up was 20.8 months (r ange 9-30 months). In all cases a medium dose of 5 mu g/kg/day resulte d in rapid clinical improvement, associated in 6/7 with an increase in absolute polymorphonuclear (PMN) count. In the remaining subject, a s triking amelioration of infectious status contrasted with a persistent ly low PMN count. Liver transplantation in one patient resolved metabo lic complications but did not improve PMN count or the infectious stat us, 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 (W HO grade 0 and grade 3) recovering after decrease of G-CSF dosage.