This study compared the prognosis of patients treated for aplastic anaemia
at Great Ormond Street Hospital for Children from 1973-88 (group A; n = 38)
with a more recent cohort from 1989-96 (group B; n = 37). The two groups w
ere similar in terms of clinical history, age, and severity of aplasia. The
main treatment differences included the use of androgen treatment in group
A (21 of 38 patients) but not in group B, and the addition of cyclosporin
A to immunosuppressive treatment for 14 patients in group B. Actuarial surv
ival at eight years' follow up was significantly better for group B (84%; 9
5% CI, 68% to 93%) than for group A (45%; 95% CI, 30% to 60%), because of i
mproved outcome for both immunosuppressive treatment (86% nu 39%) and bone
marrow transplantation (93% nu 56%). There was no evidence for late clonal
disorders or secondary malignancies in survivors in either group. The progn
osis for aplastic anaemia has improved greatly in recent years so that over
80% of children are long term survivors.