title: Acquired aplastic anaemia: still a serious disease.
abstract: Over 15 years, 42 children aged 2-14 years were diagnosed as having acquired aplastic anaemia. Adequate clinical details were available for 38 children who were categorised as very severe (n = 13), severe (n = 16), or nonsevere (n = 9) by the modified Camitta criteria. Treatment varied over the study period. Seven children received a bone marrow allograft from a full match family donor and three a matched unrelated donor transplant after failed treatment with antilymphocyte globulin. The remainder were treated with antilymphocyte globulin (n = 11), antilymphocyte globulin and oxymetholone (n = 4), oxymetholone with or without prednisolone (n = 12), or supportive treatment alone (n = 1). With a minimum follow up of one year since treatment, the five year survival was 70% for bone marrow transplantation with a family donor, 30% for antilymphocyte globulin, and 25% for oxymetholone. All three children with a matched unrelated donor transplant died. The prognosis of acquired aplastic anaemia remains poor for most children and new approaches to treatment are urgently required.
authors.affiliation: Department of Haematology and Oncology, Hospital for Sick Children, London.
authors.forename: D K
authors.initials: DK
authors.lastname: Webb
authors.forename: I M
authors.initials: IM
authors.lastname: Hann
authors.forename: J M
authors.initials: JM
authors.lastname: Chessells
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality / Q000601:surgery / Q000628:therapy*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007165:Immunosuppression Therapy; D008297:Male; D010110:Oxymetholone / Q000627:therapeutic use; D011379:Prognosis
chemical_list: D010110:Oxymetholone