Refractory anaemia with excess of myeloblasts in the bone marrow: a clinical trial of androgens in 90 patients.

In a cooperative trial, 90 patients with refractory anaemia with an excess of blast cells in the marrow were evaluated and treated with androgens. The clinical presentation was very similar to previously published observations: features of medullary insufficiency were less marked than in primary aplastic anaemia; bone marrow blastic infiltration varied from case to case, and remained stable until death or until an acute leukaemic change. All the patients were treated with high doses of androgens as for aplastic anaemia. The efficacy of this therapy was poor. The average life expectancy was 13 months, 64% of deaths being associated with a change to acute myeloid leukaemia. A severe bone marrow deficiency foreshadowed early death, but myeloblastic transformation was observed whatever the initial degree of blastic infiltration of the bone marrow. A comparison with the literature suggests that androgen therapy may accelerate the change to acute leukaemia.