RESUMEN
Early death in Schimke immuno-osseous dysplasia often results from renal failure and/or cell-mediated immunodeficiency. Kidney transplants have improved renal function, but effective therapy for the immunodeficiency has not yet been reported. We describe markedly improved marrow function 2 years after bone marrow transplantation in a boy with Schimke immunoosseous dysplasia.
Asunto(s)
Trasplante de Médula Ósea , Osteocondrodisplasias/genética , Osteocondrodisplasias/terapia , Antígenos CD/sangre , Niño , Preescolar , Enfermedad Injerto contra Huésped/prevención & control , Humanos , Inmunosupresores/uso terapéutico , Recién Nacido , Trasplante de Riñón , Linfopenia/complicaciones , Linfopenia/diagnóstico , Masculino , Osteocondrodisplasias/complicaciones , Linaje , Insuficiencia Renal/complicaciones , Insuficiencia Renal/cirugía , Tacrolimus/uso terapéuticoAsunto(s)
Desamino Arginina Vasopresina/efectos adversos , Hemostasis Quirúrgica , Hiponatremia/inducido químicamente , Convulsiones/inducido químicamente , Niño , Desamino Arginina Vasopresina/administración & dosificación , Femenino , Humanos , Hiponatremia/sangre , Hiponatremia/orina , Lactante , Inyecciones Intravenosas , Masculino , Concentración Osmolar , Factores de TiempoRESUMEN
The effects of branched-chain amino acid metabolites on granulocyte-macrophage progenitor cell proliferation in marrow culture are reported. Isovalerate and propionate profoundly suppress granulopoiesis at both 3.2 and 6.4 mM concentrations, whereas methylmalonate and other metabolites suppress to a lesser degree. The parent branched-chain amino acids leucine, isoleucine, and valine do not suppress in vitro granulopoiesis at similar concentrations. Because the concentrations of the organic acids tested fall within the pathophysiologic ranges observed in patients with isovaleric, propionic, and methylmalonic acidemias, we suggest that elevated in vivo levels of isovalerate, propionate, and to a lesser degree methylmalonate are responsible for the neutropenia observed in these disorders.
Asunto(s)
Aminoácidos de Cadena Ramificada/farmacología , Granulocitos/fisiología , Células Madre Hematopoyéticas/efectos de los fármacos , Aminoácidos de Cadena Ramificada/metabolismo , Células Cultivadas , Ensayo de Unidades Formadoras de Colonias , Depresión Química , Células Madre Hematopoyéticas/fisiología , Humanos , Isoleucina/farmacología , Leucina/farmacología , Malonatos/farmacología , Propionatos/farmacología , Valina/farmacologíaRESUMEN
Thirty-seven children with acute lymphocytic leukemia in clinical remission for at least 6 months completed a 1-year trial in which they were randomly assigned in a double-blind fashion to receive co-trimoxazole twice daily for 6 months followed by placebo for 6 months (18 patients) or placebo followed by co-trimoxazole (19 patients). Total amounts of maintenance chemotherapy administered during both periods were similar. During administration of co-trimoxazole significant reductions were documented in the patients' average total white blood count (P less than 0.001), absolute neutrophil count (P less than 0.001), absolute lymphocyte count (P = 0.009), and platelet count (P = 0.002) compared with values obtained during the placebo period. Patients had on the average 1.6 infections during the co-trimoxazole period compared with 2.5 infections during placebo administration (P = 0.008). It is concluded that, although co-trimoxazole is an effective prophylactic antibiotic in children with acute lymphocytic leukemia, the resultant myelosuppression could potentially hamper the administration of maintenance cancer chemotherapy.