RESUMO
Over 400 cases of pediatric SAA occur annually in the United States. A growing number of children with SAA may have had their stem cells harvested through cord blood collection. We describe a nine-yr-old male with SAA treated successfully with an autologous cord blood transplant following immunoablative chemotherapy. With the increasing number of people cryopreserving autologous cord blood, the use of autologous cord blood in the treatment of SAA might be considered as initial therapy. This case serves to discuss approaches to preparative therapy as well as the potential complications in this growing cohort of patients.
Assuntos
Anemia Aplástica/terapia , Transplante de Células-Tronco de Sangue do Cordão Umbilical/métodos , Bancos de Sangue , Criança , Criopreservação , Sangue Fetal/citologia , Humanos , Imunossupressores/efeitos adversos , Masculino , Condicionamento Pré-Transplante/efeitos adversos , Transplante Autólogo , Resultado do TratamentoRESUMO
CDA is a heterogeneous group of disorders that result in morphologically abnormal erythroid maturation and ineffective erythropoiesis. Curative therapy for CDA focuses on the use of HSCT using fully matched sibling donors. This is the first report of a Type II CDA patient with severe iron overload who was successfully treated with HSCT using a HLA-matched unrelated donor after aggressive chelation therapy. Given the challenges of HSCT in any patient with CDA and severe iron overload, the role of novel approaches to iron chelation and HSCT is discussed.
Assuntos
Anemia Diseritropoética Congênita/complicações , Transplante de Células-Tronco Hematopoéticas/métodos , Sobrecarga de Ferro/complicações , Benzoatos/farmacologia , Transplante de Medula Óssea/métodos , Pré-Escolar , Deferasirox , Desferroxamina/farmacologia , Feminino , Humanos , Lactente , Ferro/química , Quelantes de Ferro/farmacologia , Masculino , Triazóis/farmacologia , Doadores não RelacionadosRESUMO
Gastrointestinal complications following HSCT are numerous and include a variety of issues resulting in hepatic, biliary, pancreatic, and intestinal compromise. In the context of an underlying state of immune dysregulation, novel complications may arise including autoimmunity. To our knowledge, this is the first report of a patient with XLP who was successfully treated with HSCT using an HLA-matched unrelated cord blood unit that was complicated by the development of inflammatory polyps of the colon. Given the underlying diagnosis of XLP and its associated immune dysregulation, the challenge of understanding unique gastrointestinal manifestations of autoimmunity following HSCT is discussed.