RESUMO
OBJECTIVES: To prospectively define the lowest possible doses of recombinant human granulocyte colony-stimulating factor (rhG-CSF) that would benefit selected children with chronic idiopathic neutropenia whose disease was severe enough to interfere appreciably with quality of life. STUDY DESIGN: The efficacy of low-dose rhG-CSF therapy was investigated in six children with symptomatic chronic idiopathic neutropenia. All patients received rhG-CSF, 5 micrograms/kg subcutaneously, as a single daily dose until an absolute neutrophil count (ANC) above 1.5 x 10(9)/L was observed. The rhG-CSF dosage interval and amount were then increased and decreased, respectively, in an alternating fashion until the lowest rhG-CSF dose that would maintain the ANC above 1.0 x 10(9)/L (1000/mm3) was reached. RESULTS: Although the minimal dose requirements varied, all patients were able to maintain a mean ANC > 1.0 x 10(9)/L during a mean follow-up period of 14 months at doses ranging from 1.0 microgram/kg once weekly to 5.0 micrograms/kg every other day. Administration of rhG-CSF resulted in resolution of all preexisting chronic infections, reduction in the frequency of new infectious episodes, and discontinuation of prophylactic antibiotics. In all patients the ANC decreased to pretreatment values when further reduction or discontinuation of rhG-CSF therapy was attempted. By identifying the minimal effective dose in each patient, we were able to reduce the treatment cost by a mean of 81% compared with daily dosage at 5 micrograms/kg. CONCLUSIONS: Recombinant human granulocyte colony-stimulating factor therapy at low doses (< or = 5 micrograms/kg) every 2 to 7 days to symptomatic children with chronic idiopathic neutropenia is effective and no more costly than supportive treatment with antibiotics.
Assuntos
Fator Estimulador de Colônias de Granulócitos/administração & dosagem , Neutropenia/tratamento farmacológico , Criança , Pré-Escolar , Doença Crônica , Feminino , Custos de Cuidados de Saúde , Humanos , Lactente , Contagem de Leucócitos , Masculino , Neutropenia/economia , Neutropenia/etiologia , Estudos Prospectivos , Qualidade de Vida , Proteínas RecombinantesRESUMO
OBJECTIVES: To assess the efficacy and toxicity of very high doses of glucocorticoids in patients with congenital pure red cell aplasia (Diamond-Blackfan anemia) who did not respond to standard doses of prednisone. STUDY DESIGNS: We prospectively treated eight patients with transfusion-dependent Diamond-Blackfan anemia with high intravenous doses of methylprednisolone. All patients had previously not responded to one or more oral courses of prednisone in standard doses and were dependent on erythrocyte transfusions. Every patient initially received methylprednisolone at a dose of 30 mg/kg per day, followed by slow tapering for 4 weeks, but none responded. All patients then received a second treatment course starting at 100 mg of methylprednisolone per kilogram per day, again followed by slow tapering of the dosage. RESULTS: Three patients had a complete response that has been sustained for 21+, 31+, and 41+ months, respectively. One patient had a partial response. Toxic effects included a rise in serum alanine aminotransferase activity in all patients, transient diabetes mellitus in one child, and three episodes of bacteremia in two patients with intravenous access devices. CONCLUSIONS: We conclude that very high doses of methylprednisolone may induce sustained remission in some patients with transfusion-dependent Diamond-Blackfan anemia refractory to standard-dose prednisone therapy.
Assuntos
Anemia Refratária/tratamento farmacológico , Relação Dose-Resposta a Droga , Anemia de Fanconi/tratamento farmacológico , Glucocorticoides/administração & dosagem , Glucocorticoides/uso terapêutico , Metilprednisolona/administração & dosagem , Metilprednisolona/uso terapêutico , Adolescente , Idade de Início , Anti-Infecciosos/administração & dosagem , Anti-Infecciosos/uso terapêutico , Pré-Escolar , Protocolos Clínicos , Glucocorticoides/efeitos adversos , Humanos , Lactente , Metilprednisolona/efeitos adversos , Pneumocystis , Infecções por Pneumocystis/tratamento farmacológico , Infecções por Pneumocystis/prevenção & controle , Prednisona/administração & dosagem , Prednisona/uso terapêutico , Estudos Prospectivos , Combinação Trimetoprima e Sulfametoxazol/administração & dosagem , Combinação Trimetoprima e Sulfametoxazol/uso terapêuticoRESUMO
A 2-year-old boy with sickle cell anemia had a massive, fatal hemolytic reaction after administration of an intravenous dose of ceftriaxone. Laboratory studies demonstrated the presence of an IgM antibody against ceftriaxone, binding to and destroying the patient's erythrocytes by an immune complex mechanism. This rare complication should be considered in the differential diagnosis when hemoglobinuria develops in a child after administration of ceftriaxone or a similar agent.
Assuntos
Anemia Hemolítica Autoimune/induzido quimicamente , Anemia Falciforme/tratamento farmacológico , Ceftriaxona/efeitos adversos , Hemoglobinúria/induzido quimicamente , Anemia Hemolítica Autoimune/sangue , Anemia Hemolítica Autoimune/imunologia , Ceftriaxona/uso terapêutico , Pré-Escolar , Evolução Fatal , Hemoglobinúria/sangue , Hemólise , Humanos , Imunoglobulina M/sangue , MasculinoRESUMO
We recently encountered a previously healthy 3-year-old girl who had severe bleeding resulting from a severe deficiency of prothrombin. A lupus anticoagulant was identified by several different methods. The patient was successfully treated with glucocorticoids. This rare complication of a lupus anticoagulant should be considered in the differential diagnosis of a previously well child who suddenly has hemorrhage.