title: Bone marrow transplantation for severe aplastic anaemia using cyclosporin: long-term follow-up.
abstract: Between 1980 and 1986 we transplanted 49 patients from genotypically identical sibling donors for severe aplastic anaemia (SAA). Patients were predominantly adults, median age 22 years (3-47). Forty-six were multiply transfused prior to referral. The median pre-transplant disease duration was 4 months (1-72). Pre-transplant conditioning was with cyclophosphamide (CY) 200 mg/kg. Cyclosporin A (CSA) was given from 1 day before the transplant and continued for 9-12 months. Eight of 48 evaluable patients did not achieve initial engraftment (early graft failure). Six of these episodes occurred in the eight cases transplanted more than 1 year after diagnosis, four of whom died. Thirteen of 44 (30%) evaluable patients (with stable engraftment after one or two transplants) had grade III-IV acute graft-versus-host disease (GVHD). Only three of 35 patients surviving more than 100 days with sustained engraftment developed generalized chronic GVHD; two died. An additional 10 patients developed localized chronic GVHD, which was very mild, transient and related to CSA withdrawal. Four long-term survivors are known to have autologous marrow function. All survivors have Karnofsky scores of greater than 90%. We conclude that the use of CSA after bone marrow transplantation for SAA is associated with good long-term survival and minimal on-going chronic GVHD. Early graft failure was frequent when transplantation was delayed beyond 1 year from diagnosis.
authors.affiliation: Department of Haematology, Royal Postgraduate Medical School, London, UK.
authors.forename: J M
authors.initials: JM
authors.lastname: Hows
authors.forename: J C
authors.initials: JC
authors.lastname: Marsh
authors.forename: J L
authors.initials: JL
authors.lastname: Yin
authors.forename: S
authors.initials: S
authors.lastname: Durrant
authors.forename: D
authors.initials: D
authors.lastname: Swirsky
authors.forename: A
authors.initials: A
authors.lastname: Worsley
authors.forename: S M
authors.initials: SM
authors.lastname: Fairhead
authors.forename: P M
authors.initials: PM
authors.lastname: Chipping
authors.forename: S
authors.initials: S
authors.lastname: Palmer
authors.forename: E C
authors.initials: EC
authors.lastname: Gordon Smith
mesh_terms: D000741:Anemia, Aplastic / Q000601:surgery*; D016026:Bone Marrow Transplantation; D003524:Cyclosporins / Q000627:therapeutic use*; D005500:Follow-Up Studies; D006084:Graft Rejection / Q000187:drug effects; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D008212:Lymphocyte Depletion
chemical_list: D003524:Cyclosporins