Between 1990 and 1999, 36 children with refractory anaemia with excess blasts (
RAEB) and
RAEB in transformation (RAEBt), not associated with
Down's syndrome, were diagnosed in Britain. A total of 31 children received intensive
chemotherapy, six of whom proceeded to a bone marrow allograft in first remission, whereas two received an autograft. Of the 23 given
chemotherapy only, four died of toxicity, 10 relapsed and nine are alive in first remission. Out of the 10 who relapsed, four are alive and disease-free following an allograft. Out of the 6 children given an allograft in first remission, two died of disease and four are alive in first remission. Both children given an autograft died of disease. Two children received an allograft without prior
chemotherapy but died of toxicity. Three children received supportive care only, and one child survived. The overall survival was 51% at 5 years, and was superior in children with RAEBt (63%) compared with
RAEB (28%, P = 0.03). Cytogenetics were available in 35 cases.
Monosomy 7 was the most common abnormality (33% of cases). Survival in children with
monosomy 7 was 22% at 5 years compared with 66% for the other patients (P = 0.05). Allowing for cytogenetics, outcomes of
therapy appear similar to those for de novo acute myeloid leukaemia (AML), and it is appropriate for children with
RAEB/RAEBt to be registered in AML trials.