RESUMO
Two children with glycogen storage disease type Ib associated with numerous recurrent bacterial infections as a result of neutropenia and neutrophil dysfunction were treated with recombinant human granulocyte colony-stimulating factor (G-CSF). One of the two patients was previously treated with recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF); therapy had to be discontinued because of severe local side effects. Both colony-stimulating factors at dosages of 3 and 8 micrograms/kg/per day, respectively, increased the average neutrophil counts from less than 300 cells/microliters to more than 1200 cells/microliters. Two subpopulations of neutrophils could be identified by their capacity to produce H2O2: one subpopulation generated H2O2 normally and a second was defective in H2O2 production. The doses of G-CSF effectively enhanced and maintained that subpopulation of neutrophils which produced normal amounts of H2O2. Moreover, these colony-stimulating factor-induced neutrophils demonstrated effective phagocytosis of zymosan particles and killing of staphylococci. Chemotaxis was decreased and could not be normalized by treatment with G-CSF. We conclude that maintenance treatment with G-CSF improved the quality of life in both patients: The number and severity of bacterial infections decreased markedly during treatment. Long-term treatment with G-CSF (12 and 10 months, respectively) was well tolerated, and no adverse clinical events were observed.
Assuntos
Doença de Depósito de Glicogênio Tipo I/terapia , Fator Estimulador de Colônias de Granulócitos/uso terapêutico , Fator Estimulador de Colônias de Granulócitos e Macrófagos/uso terapêutico , Neutropenia/terapia , Adolescente , Bacteriólise , Quimiotaxia de Leucócito/fisiologia , Feminino , Citometria de Fluxo , Doença de Depósito de Glicogênio Tipo I/fisiopatologia , Fator Estimulador de Colônias de Granulócitos/administração & dosagem , Fator Estimulador de Colônias de Granulócitos e Macrófagos/administração & dosagem , Humanos , Peróxido de Hidrogênio/metabolismo , Lactente , Injeções Subcutâneas , Contagem de Leucócitos , Masculino , Neutropenia/fisiopatologia , Neutrófilos/metabolismo , Neutrófilos/patologia , Neutrófilos/fisiologia , Proteínas Recombinantes , Staphylococcus aureus/fisiologia , Superóxidos/metabolismoRESUMO
A prospective randomized study comparing 7S immunoglobulin G to a 5S IgG preparation for therapy of acute idiopathic thrombocytopenic purpura was conducted. The 5S preparation differed from the 7S preparation in that it lacked part of the Fc portion of the IgG molecule. Both groups were given 400 mg IgG/kg body weight over 5 days. All patients had platelet counts less than or equal to 30 X 10(9)/L before IgG infusion. Nine of the 10 patients in the 7S treatment group, compared with three of 10 patients in the 5S treatment group, responded to therapy with an increment in platelet counts greater than 100 X 10(9)/L within 4 days of completing the infusion treatment. Furthermore, the rate of increase of the platelets was more rapid in the 7S group. The results emphasize the efficacy of partial Fc for management of acute idiopathic thrombocytopenic purpura.