RESUMEN
OBJECTIVE: To compare 2 active agents, vinblastine and etoposide, in the treatment of multisystem Langerhans' cell histiocytosis (LCH) in an international randomized study. STUDY DESIGN: One hundred forty-three untreated patients were randomly assigned to receive 24 weeks of vinblastine (6 mg/m(2), given intravenously every week) or etoposide (150 mg/m(2)/d, given intravenously for 3 days every 3 weeks), and a single initial dose of corticosteroids. RESULTS: Vinblastine and etoposide were equivalent (P > or = .2) in all respects: response at week 6 (57% and 49%); response at the last evaluation (58% and 69%); toxicity (47% and 58%); and probability of survival (76% and 83%) [corrected], of disease reactivation (61% and 55%), and of developing permanent consequences (39% and 51%) including diabetes insipidus (22% and 23%). LCH reactivations were usually mild, as was toxicity. All children > or = 2 years old without risk organ involvement (liver, lungs, hematopoietic system, or spleen) survived. With such involvement, lack of rapid (within 6 weeks) response was identified as a new prognostic indicator, predicting a high (66%) mortality rate. CONCLUSIONS: Vinblastine and etoposide, with one dose of corticosteroids, are equally effective treatments for multisystem LCH, but patients who do not respond within 6 weeks are at increased risk for treatment failure and may require different therapy.
Asunto(s)
Etopósido/uso terapéutico , Histiocitosis de Células de Langerhans/tratamiento farmacológico , Metilprednisolona/uso terapéutico , Vinblastina/uso terapéutico , Adolescente , Niño , Quimioterapia Combinada , Etopósido/efectos adversos , Humanos , Medición de Riesgo , Análisis de Supervivencia , Resultado del Tratamiento , Vinblastina/efectos adversosAsunto(s)
Histiocitosis de Células de Langerhans/inmunología , Hipergammaglobulinemia/etiología , Adolescente , Factores de Edad , Niño , Preescolar , Hemaglutininas/análisis , Histiocitosis de Células de Langerhans/complicaciones , Humanos , Inmunoglobulina M/análisis , Inmunoglobulinas/análisis , Lactante , Recién Nacido , Activación de LinfocitosRESUMEN
Fifty-seven patients younger than 25 years with severe aplastic anemia underwent either bone marrow transplantation or antithymocyte globulin therapy (ATG) to ascertain which approach should be used in young patients. Thirty-five patients who had an HLA-identical sibling donor underwent bone marrow transplantation after conditioning with cyclophosphamide and low-dose total-body radiation. Twenty-two patients who did not have an HLA-identical donor received ATG. The 2-year actuarial survival of patients after transplant is 72% (95%, CI 64% to 80%), versus 45% (95%, CI 29% to 61%) in those given ATG therapy (P = 0.18). In those patients surviving 6 months after treatment, return of peripheral blood counts to normal values was more common in patients who received marrow transplant compared with those given ATG therapy (P less than 0.001). Furthermore, 24 of 26 transplant survivors had Karnofsky performance scores greater than 90%, compared with only five of 13 ATG survivors. These data suggest that bone marrow transplantation is the preferred therapy for severe aplastic anemia in young patients who have an HLA-identical sibling donor. ATG should be reversed for those young patients with severe aplastic anemia who do not have a histocompatible marrow donor.
Asunto(s)
Anemia Aplásica/terapia , Suero Antilinfocítico/uso terapéutico , Trasplante de Médula Ósea , Linfocitos T/inmunología , Adolescente , Adulto , Anemia Aplásica/mortalidad , Suero Antilinfocítico/efectos adversos , California , Niño , Preescolar , Femenino , Enfermedad Injerto contra Huésped/etiología , Humanos , Lactante , Masculino , Fibrosis Pulmonar/etiología , Calidad de VidaRESUMEN
Three children developed extensive extrapulmonary disease in the course of fatal adenovirus type 7 pneumonia. Several clinical features, including the unexpected onset of coma, suggested the development of Reye syndrome, but biochemical and histopathologic findings were inconsistent with this diagnosis. Virologic and pathologic studies did not reveal evidence of extrapulmonary adenovirus infection, despite clinical involvement of the liver, skeletal muscle, and central nervous system. The detection in premortem sera from all three patients of adenovirus penton antigen, known to be cytotoxic in vitro, suggests a possible mechanism for the production of extrapulmonary pathology in the absence of extrapulmonary virus infection.