Hematopoietic stem cell transplantation in childhood inherited bone marrow failure syndrome.
Aplastic anemia is a rare disease in children that is most commonly idiopathic and less often a hereditary disorder. Hereditary bone marrow failure (BMF) syndromes, however, should be considered both in children and in adults before any attempt at treatment. Precise diagnosis is important because it will modify prognostic treatment options and the results of bone marrow transplantation. In this review, we will report recent results of treatment of Fanconi anemia and other hereditary BMF syndromes.
Gluckman
E
E
Hematology Department, Eurocord Hôpital, Saint Louis, Paris, France. eliane.gluckman@sls.aphp.fr
Wagner
J E
JE
D029502:Anemia, Hypoplastic, Congenital / Q000235:genetics / Q000628:therapy*; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D053208:Kaplan-Meier Estimate; D019653:Myeloablative Agonists / Q000627:therapeutic use; D012801:Sibling Relations; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous / Q000379:methods
D019653:Myeloablative Agonists