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Successful reduced-intensity conditioning hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia in two children.
Andolina, Jeffrey R; Reinish, Ariel L; Akhtar, Razia; Noronha, Suzie; Shand, Jessica C; Girvin, Angela; Korones, David N; Bruckner, Lauren B; Mullen, Craig A; Curran, Kevin J; Boulad, Farid.
Afiliação
  • Andolina JR; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Reinish AL; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Akhtar R; Department of Pediatrics, Mouth Sinai, New York City, New York.
  • Noronha S; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Shand JC; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Girvin A; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Korones DN; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Bruckner LB; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Mullen CA; Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York.
  • Curran KJ; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, New York.
  • Boulad F; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, New York.
Pediatr Blood Cancer ; 65(8): e27218, 2018 08.
Article em En | MEDLINE | ID: mdl-29722478
ABSTRACT
Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare cause of bone marrow failure in children. We report two children who presented with pancytopenia, and were diagnosed with PNH with severe aplastic anemia. Both children underwent upfront, successful hematopoietic stem cell transplantation with reduced-intensity conditioning. One patient had a syngeneic donor, and one patient had a 10/10 matched unrelated donor. Neither patient developed graft versus host disease, infections, or recurrent PNH. Reduced-intensity conditioning hematopoietic stem cell transplantation is a reasonable therapy for PNH with marrow failure in children.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Condicionamento Pré-Transplante / Hemoglobinúria Paroxística / Anemia Aplástica Limite: Adolescent / Humans / Male Idioma: En Revista: Pediatr Blood Cancer Assunto da revista: HEMATOLOGIA / NEOPLASIAS / PEDIATRIA Ano de publicação: 2018 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Condicionamento Pré-Transplante / Hemoglobinúria Paroxística / Anemia Aplástica Limite: Adolescent / Humans / Male Idioma: En Revista: Pediatr Blood Cancer Assunto da revista: HEMATOLOGIA / NEOPLASIAS / PEDIATRIA Ano de publicação: 2018 Tipo de documento: Article