RESUMO
Since 1968, bone marrow transplantation became the first line therapy for selected metabolic and immunological hereditary disorders. Actually, advances in the supportive care in bone marrow transplantation and a better knowledge of the immunology of BMT complications has been associated with a better disease correction and an increase in long term survival. New approaches are under investigation and include: hematopoietic growth factors, enzymatic replacement and gene therapy. However at the present time BMT is still the only curative treatment for selected hereditary disorders.
Assuntos
Transplante de Medula Óssea , Doenças Genéticas Inatas/terapia , Síndrome de Chediak-Higashi/terapia , Doenças Genéticas Inatas/classificação , Substâncias de Crescimento/uso terapêutico , Humanos , Erros Inatos do Metabolismo/terapia , Síndrome de Wiskott-Aldrich/terapiaRESUMO
Curability in children suffering from malignant solid tumor is 50%. Thus, high dose chemotherapy with or without total body irradiation followed by autologous bone marrow transplantation (ABMT) has been proposed to patients suffering from cancer either at initial diagnosis (poor prognosis tumor) or at relapse. Thanks to these studies, drugs having dose effects properties have been selected. In some tumors, ABMT has significantly improved patients median survival. It remains to be determined if: 1. high dose chemotherapy protocols with ABMT are superior to new aggressive chemotherapeutic protocol without ABMT. 2. ABMT increases the curability of high risk patients.
Assuntos
Transplante de Medula Óssea , Neoplasias/terapia , Transplante Autólogo , Neoplasias Encefálicas/terapia , Criança , Terapia Combinada , Humanos , Neoplasias Renais/terapia , Neoplasias Embrionárias de Células Germinativas/terapia , Neuroblastoma/terapia , Sarcoma de Ewing/terapia , Neoplasias de Tecidos Moles/terapia , Tumor de Wilms/terapiaRESUMO
In a series of 51 children presenting with an accidental caustic burn, symptoms were analysed for their predictive value of significant i.e. necrotizing oesophageal lesions (grade II or III). For the whole group, the incidence of significant oesophageal lesions was 37%. Vomiting and/or respiratory distress were associated with high incidence of significant oesophageal burn (84% and 75% respectively). The particular location of each caustic burn was analysed for its association with caustic burns at other anatomical sites. Of the 18 patients with a laryngeal burn, 72% also had a grade II or III oesophageal burn. The 19 patients with a grade II or III oesophageal burn and the 18 patients with a laryngeal burn, all had lesions at other sites. In the group of 19 patients with a caustic lesion limited to one site, only 1 patient showed mild oesophagitis (grade I) without late sequelae.