title: [Severe acquired aplastic anemia: historical outcome of patients treated by allogeneic bone marrow transplantation from matched sibling donors. A study by the Spanish Group for Bone Marrow Transplantation in Children (GETMON)].
abstract: BACKGROUND
  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.
  
  
  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.
authors.affiliation: Hospital Ramon y Cajal, Universidad de Alcalá, Madrid, Spain. amvillatv@yahoo.es
authors.forename: A
authors.initials: A
authors.lastname: Muñoz Villa
authors.forename: C
authors.initials: C
authors.lastname: Díaz de Heredia
authors.forename: M A
authors.initials: MA
authors.lastname: Díaz González
authors.forename: I
authors.initials: I
authors.lastname: Badell Serra
authors.forename: A
authors.initials: A
authors.lastname: Martínez Rubio
authors.forename: M A
authors.initials: MA
authors.lastname: González Valentín
authors.forename: M A
authors.initials: MA
authors.lastname: Dasí Carpio
authors.forename: P
authors.initials: P
authors.lastname: Gómez Pérez
authors.forename: E
authors.initials: E
authors.lastname: Bureo Dacal
authors.forename: T
authors.initials: T
authors.lastname: Olivé Oliveras
authors.forename: J M
authors.initials: JM
authors.lastname: Pérez Hurtado
authors.forename: M S
authors.initials: MS
authors.lastname: Maldonado Regalado
mesh_terms: D000741:Anemia, Aplastic / Q000175:diagnosis* / Q000188:drug therapy / Q000628:therapy*; D000970:Antineoplastic Agents / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D006084:Graft Rejection / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D012720:Severity of Illness Index; D035781:Siblings; D013030:Spain; D014019:Tissue Donors; D014184:Transplantation, Homologous
chemical_list: D000970:Antineoplastic Agents; D007166:Immunosuppressive Agents; D016572:Cyclosporine