title: [Favorable current prognosis after HLA-identical bone marrow transplantation for children with required severe aplastic anemia; evaluation of 30 years of bone marrow transplantation at the Leiden University Medical Center].
abstract: OBJECTIVE
  To evaluate the results of 30 years of allogeneic HLA-identical bone marrow transplantation (BMT) as the treatment for children with acquired severe aplastic anaemia.
  
  
  METHODS
  Retrospective, descriptive.
  
  
  METHODS
  Of all patients who underwent an HLA-identical sibling-donor BMT for severe aplastic anaemia at the Department of Paediatrics, Leiden University Medical Center, in the period 1971-2000, and had a follow-up period of at least 1 year, the medical data were reviewed. The patients were split into 2 groups: patients transplanted before 1989 (n = 24), and patients who had their BMT from 1989 onwards (n = 20). This was due to a change in the treatment policy, namely a reduction in the period between diagnosis and BMT, resulting in fewer blood transfusions as well as changes in the prophylaxis against graft-versus-host disease (GvHD) from 1989 onwards (combination therapy using methotrexate and cyclosporin).
  
  
  RESULTS
  There was an increase in the 1-year actuarial survival rate from 67% in the period before 1989 to 90% thereafter. The incidence of GvHD has significantly decreased since the introduction, in 1989, of the combination therapy using methotrexate and cyclosporin, with only 1/20 patients suffering from acute GvHD versus 13/24 prior to 1989 (p = 0.002). No patients acquired chronic GvHD after 1989, whereas before 1989, 10 patients had acquired this (p = 0.001).
  
  
  CONCLUSIONS
  The prognosis of allogeneic HLA-identical sibling transplantation for paediatric patients with severe aplastic anaemia has considerably improved over the last 30 years due to improved supportive care, a significant decrease in GvHD and a shorter period between diagnosis and BMT, with the result that less blood transfusions have been required and less sensitisation has occurred. The long-term survival chance has increased to 90%.
authors.affiliation: Willem-Alexander Kinder- en Jeugdcentrum, sectie Immunologie, Hematologie, Oncologie, Beenmergtransplantatie en Auto-immuunziekten (IHOBA), Leids Universitair Medisch Centrum, Postbus 9600, 2300 RC Leiden.
authors.forename: M
authors.initials: M
authors.lastname: van Steekelenburg
authors.forename: M H
authors.initials: MH
authors.lastname: van Weel-Sipman
authors.forename: A H
authors.initials: AH
authors.lastname: Zwinderman
authors.forename: P M
authors.initials: PM
authors.lastname: Hoogerbrugge
authors.forename: J M J J
authors.initials: JM
authors.lastname: Vossen
authors.forename: R M
authors.initials: RM
authors.lastname: Egeler
mesh_terms: D000741:Anemia, Aplastic / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000453:epidemiology* / Q000517:prevention & control; D006680:HLA Antigens / Q000276:immunology; D006801:Humans; D007166:Immunosuppressive Agents; D007223:Infant; D008297:Male; D009679:Nuclear Family; D011379:Prognosis; D012189:Retrospective Studies; D016019:Survival Analysis; D015996:Survival Rate; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens; D007166:Immunosuppressive Agents