RESUMO
Malignant extracranial germ cell tumors (GCTs) are rare in pediatric patients and are usually extremely sensitive to chemotherapy. Relapsed or refractory tumors, although rare, established the need for second-line therapies, including high-dose chemotherapy with autologous stem cell transplantation (HDCT/ASCT). However, there are few data on its use in children with GCTs. We present a retrospective analysis of all patients diagnosed with extracranial GCTs who received HDCT/ASCT at two Brazilian pediatric cancer centers from May 1999 to December 2019. We identified a total of 34 patients with a median age at diagnosis of 2.8 years (range, 0 to 18.8), who received HDCT/ASCT. Most patients (73%) received carboplatin, etoposide and melphalan (CEM) as a HDCT regimen. Fourteen patients received a second-line conventional dose chemotherapy (CDCT), 14 received a third-line CDCT and five received even a fourth-line CDCT prior to HDCT/ASCT. After a median follow-up of 22.7 months (range, 0.3 to 198.1), 16 patients had died after tumor relapse/progression and 2 patients died from HDCT/ASCT toxicity. We observed a 5-year OS of 47.1% and 5-year EFS of 44.1%. The 5-year OS for patients referred for HDCT/ASCT with progressive disease was 10% compared to 62.5% for those who achieved disease control before HDCT/ASCT (p = 0.001). In our experience, heavily pretreated children and adolescents with extracranial GCTs achieved considerable survival rates with HDCT/ASCT since, at least, partial control of their disease was possible before starting HDCT/ASCT. The role of HDCT/ASCT in pediatric patients with GCTs should be investigated in prospective trials.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Neoplasias Embrionárias de Células Germinativas , Adolescente , Humanos , Criança , Recém-Nascido , Lactente , Pré-Escolar , Estudos Retrospectivos , Estudos Prospectivos , Brasil , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Recidiva Local de Neoplasia , Transplante Autólogo , Neoplasias Embrionárias de Células Germinativas/tratamento farmacológico , Etoposídeo/uso terapêutico , Terapia de Salvação , Transplante de Células-TroncoRESUMO
The present review discusses the use of autologous hematopoietic stem cell transplantation (HSCT) for the treatment of diabetes mellitus type 1 (DM 1). It has been observed that high dose immunosuppression followed by HSCT shows better results among other immunotherapeutic treatments for the disease as the patients with adequate beta cell reserve achieve insulin independence. However, this response is not maintained and reoccurrence of the disease is major a major challenge to use HSCT in future to prevent or control relapse of DM 1.
Assuntos
Diabetes Mellitus Tipo 1/terapia , Transplante de Células-Tronco , Animais , Transplante de Células-Tronco de Sangue do Cordão Umbilical , Diabetes Mellitus Tipo 1/complicações , Diabetes Mellitus Tipo 1/imunologia , Células-Tronco Embrionárias/transplante , Doenças Hematológicas/complicações , Doenças Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas , Humanos , Terapia de Imunossupressão , Células Secretoras de Insulina/transplante , Transplante de Células-Tronco Mesenquimais , Camundongos , Transplante AutólogoRESUMO
Nesta revisão são discutidas várias alternativas de regeneração do conjunto de células produtoras de insulina do pâncreas, usando células-tronco embrionárias do cordão umbilical e adultas, e o trabalho que está sendo realizado em nosso grupo de pesquisas utilizando imunossupressão em altas doses combinada com a infusão de células-tronco hematopoéticas autólogas em diabete do tipo 1 recém-diagnosticado.
In this review, we discuss several alternatives for the regeneration of the pool of insulin-producing cells by the pancreas using embryonic, cord blood or adult stem cells and the work being carried out by our research group using high dose immunosuppression with autologous hematopoietic stem cells in newly diagnosed type 1 diabetes mellitus.
Assuntos
Humanos , Medula Óssea , Terapia Baseada em Transplante de Células e Tecidos , Diabetes Mellitus , Transplante de Células-Tronco Hematopoéticas , Células-Tronco , Cordão UmbilicalRESUMO
In this review, we present 1) scientific basis for the use of high dose immunosuppression followed by autologous peripheral blood hematopoietic stem cell transplantation for newly diagnosed type 1 diabetes mellitus, 2) an update of clinical and laboratory outcomes in 21 patients transplanted at the University Hospital of the Ribeirão Preto Medical School, University of São Paulo, Brazil, including 6 relapses in patients without previous ketoacidosis and 3) a discussion of future prospectives of cellular therapy for type 1 diabetes mellitus.
Nesta revisão, são apresentadas: 1) as bases científicas para o uso de imunossupressão em alta dose seguida de transplante autólogo de células-tronco hematopoéticas do sangue periférico no diabete melito do tipo 1 recém-diagnosticado; 2) uma atualização da evolução clínica e laboratorial de 21 pacientes transplantados no Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto, da Universidade de São Paulo, Brasil, incluindo recaídas em seis pacientes transplantados sem cetoacidose prévia; e 3) uma discussão das perspectivas futuras de terapia celular no diabete melito do tipo 1.
