RESUMO
INTRODUCTION: Allogeneic haematopoietic stem-cell transplantation is the treatment of choice for acquired aplastic anaemia in children. Experience with this approach from Spanish Working Party for Bone Marrow Transplantation in Children in two sequential time periods (1982-1990 and 1991-2004) is reported. PATIENTS AND METHODS: Sixty two consecutive patients with a median age of 10 years were transplanted; 18 in the 1982-1990 period and 44 in the 1991-2004 period. Conditioning regimen consisted mainly of irradiation and cyclophosphamide in the first period (72 % of patients) and cyclophosphamide +/- anti-thymocyte globulin (62 %) in the second. Graft versus host disease prophylaxis consisted of cyclosporine in most patients (57/62). RESULTS: Fifty one patients are alive and disease-free at a median follow-up of 127 months. Five years probability of event-free survival is 82 %. The survival increased from 61 % to 91 % during the two time periods. Eleven patients died from graft failure or rejection (3), acute or chronic graft versus host disease and infection (4) or multi-organ failure (4). Univariate analysis identified two significant prognostic factors: interval diagnostic/transplant and time period of transplant (for both p = 0.03). CONCLUSIONS: This experience corroborates that allogeneic haematopoietic stem-cell transplantation is the best treatment for severe acquired aplastic anaemia, with a current disease-free survival of 90 % of patients.
Assuntos
Anemia Aplástica/diagnóstico , Anemia Aplástica/terapia , Transplante de Medula Óssea/métodos , Irmãos , Anemia Aplástica/tratamento farmacológico , Antineoplásicos/uso terapêutico , Criança , Ciclosporina/uso terapêutico , Feminino , Rejeição de Enxerto/prevenção & controle , Humanos , Imunossupressores/uso terapêutico , Masculino , Índice de Gravidade de Doença , Espanha , Doadores de Tecidos , Transplante HomólogoRESUMO
There is considerable evidence supporting the disappearance of immunity against diseases for which there is active immune-prophylaxis available after allogeneic hematopoietic progenitor cell transplantation. These findings do not constitute merely an epidemiologic problem. From a strictly clinical point of view, these patients might benefit from the administration of different forms of vaccines against diseases to which they are particularly susceptible. There are, however, no unified criteria for the revaccination of these patients. Moreover, several surveys suggest that active immune-prophylaxis is frequently undervalued in these patients. In the present review, we provide up-to-date information on current trends as well as practical guidelines for the re-immunization of these patients in our environment.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Vacinação , Criança , Transplante de Células-Tronco Hematopoéticas/normas , Humanos , Esquemas de Imunização , Transplante Homólogo , Vacinação/normasRESUMO
OBJECTIVE: The objective of this study was to evaluate retrospectively the efficacy of allogeneic BMT in the treatment of childhood severe acquired aplastic anemia (SAAA). PATIENTS AND METHODS: Twenty-seven children aged 2 to 16 years (median 11 years) received a BMT from an HLA identical sibling. Conditioning consisted in irradiation (total, nodal or thoraco-abdominal) plus cyclophosphamide (120-200 mg/kg) in 15 patients and cyclophosphamide alone (200 mg/kg) in the rest. Prophylaxis for graft-versus-host disease (GVHD) was cyclosporine and methotrexate in most patients. RESULTS: Twenty-four children achieved the bone marrow graft at a median of 18 days (neutrophils) and 21 days (platelets). Two patients failed engraftment and 1 had a late graft rejection. Three patients developed acute GVHD grades 3-4 and six chronic GVHD, which was extensive in 4 of them. Twenty patients/71%) are alive and disease-free at a median follow-up of 110 months and the estimated disease free survival at 6 years is 67%. CONCLUSIONS: Our results confirm that allogeneic bone marrow transplantation from an HLA identical sibling is the best treatment modality for children with SAAA. Acute GVHD associated with infections and graft rejection were responsible for treatment failures.
Assuntos
Anemia Aplástica/terapia , Transplante de Medula Óssea , Doença Aguda , Adolescente , Anemia Aplástica/mortalidade , Transplante de Medula Óssea/métodos , Transplante de Medula Óssea/estatística & dados numéricos , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Seguimentos , Humanos , Masculino , Prognóstico , Modelos de Riscos Proporcionais , Estudos Retrospectivos , Transplante HomólogoRESUMO
No disponible
Assuntos
Masculino , Recém-Nascido , Humanos , Transtornos Mieloproliferativos/complicações , Dermatopatias Vesiculobolhosas/complicações , Síndrome de Down/complicações , Diagnóstico DiferencialRESUMO
Introducción: El trasplante de progenitores hematopoyéticos (TPH) de donante familiar compatible es el tratamiento de elección en la aplasia medular adquirida (AMA) grave en la infancia. Se presenta la experiencia de Grupo Español para el Trasplante de Médula Ósea en Niños en esta enfermedad a lo largo del período cronológico 1982-2004. Pacientes y métodos: Recibieron un trasplante 62 pacientes con una mediana de edad de 10 años. En el período 1982-1990 lo recibieron 18 pacientes y en el período 1991-2004, 44. El régimen de acondicionamiento varió según el período cronológico; en el primero se utilizó preferentemente la asociación de radioterapia y ciclofosfamida (72 % de los casos) y en el segundo ciclofosfamida con o sin globulina antitimocitaria (62 %). La profilaxis de enfermedad injerto contra huésped más utilizada fue la ciclosporina (57/62 pacientes). Resultados: Un total de 51 pacientes están vivos y en remisión completa de su aplasia con períodos de observación de entre 24 y 289 meses (mediana de 127 meses). La probabilidad de supervivencia actuarial libre de eventos a 5 años es del 82 %. Dicha supervivencia se incrementó del 61 al 90 % entre los dos períodos analizados. Un total de 11 pacientes fallecieron por fracaso o pérdida del injerto (3), enfermedad injerto contra huésped aguda o crónica asociada a infecciones (4) o fallo multiorgánico (4). El análisis univariante evidenció dos factores con valor predictivo para la supervivencia: el intervalo diagnóstico/trasplante y el período cronológico en que se efectuó (en ambos, p = 0,03). Conclusiones: Esta experiencia confirma que el trasplante de progenitores hematopoyéticos de donante familiar compatible es el tratamiento de elección para la aplasia medular grave adquirida, con un porcentaje de supervivencia libre de episodios del 90 % en la actualidad (AU)
Allogeneic haematopoietic stem-cell transplantation is the treatment of choice for acquired aplastic anaemia in children. Experience with this approach from Spanish Working Party for Bone Marrow Transplantation in Children in two sequential time periods (1982-1990 and 1991-2004) is reported. Patients and methods: Sixty two consecutive patients with a median age of 10 years were transplanted; 18 in the 1982-1990 period and 44 in the 1991-2004 period. Conditioning regimen consisted mainly of irradiation and cyclophosphamide in the first period (72 % of patients) and cyclophosphamide ± anti-thymocyte globulin (62 %) in the second. Graft versus host disease prophylaxis consisted of cyclosporine in most patients (57/62). Results: Fifty one patients are alive and disease-free at a median follow-up of 127 months. Five years probability of event-free survival is 82 %. The survival increased from 61 % to 91 % during the two time periods. Eleven patients died from graft failure or rejection (3), acute or chronic graft versus host disease and infection (4) or multi-organ failure (4). Univariate analysis identified two significant prognostic factors: interval diagnostic/transplant and time period of transplant (for both p = 0.03). Conclusions: This experience corroborates that allogeneic haematopoietic stem-cell transplantation is the best treatment for severe acquired aplastic anaemia, with a current disease - free survival of 90 % of patients (AU)
Assuntos
Humanos , Masculino , Feminino , Criança , Transplante de Medula Óssea/história , Transplante de Medula Óssea/métodos , Transplante de Medula Óssea/tendências , Doenças da Medula Óssea/congênito , Doenças da Medula Óssea/complicações , Ciclofosfamida/uso terapêutico , Terapia de Imunossupressão/métodos , Anemia Aplástica/congênito , Anemia Aplástica/complicações , Anemia Aplástica/tratamento farmacológico , Doenças da Medula Óssea/patologia , Terapia de Imunossupressão/tendências , Terapia de Imunossupressão , Consentimento Livre e Esclarecido/normas , Valor Preditivo dos Testes , Anemia Aplástica/patologia , Anemia Aplástica/radioterapiaRESUMO
Existen diferentes evidencias de la desaparición de inmunidad frente a las enfermedades para las que existe inmunoprofilaxis activa, tras un trasplante alogénico de medula ósea. Estos hallazgos no constituyen sólo un problema epidemiológico. Desde un punto de vista estrictamente clínico, estos pacientes podrían beneficiarse de la administración de determinadas formas de vacunas frente a enfermedades a las que son especialmente susceptibles. Sin embargo, no existe en la actualidad una pauta unificada para la revacunación de estos pacientes. Más aún, diferentes encuestas sugieren que la inmunoprofilaxis activa es con frecuencia infravalorada en estos pacientes. En la presente revisión, se pretende ofrecer tanto una información actualizada sobre las actuales tendencias, como una guía práctica para la reinmunización de estos pacientes de acuerdo con las necesidades de nuestro medio (AU)