Clinical course in 28 unselected patients with aplastic anaemia treated with anabolic steroids

Br J Haematol. 1979 Mar;41(3):323-33.

Clinical course in 28 unselected patients with aplastic anaemia treated with
anabolic steroids.

Van Hengstum M, Steenbergen J, Haanen C.

Twenty-eight unselected patients with histologically proven aplastic anaemia were
electively treated with anabolic steroids (75-150 mg orally q.d.) Additional
supportive treatment with blood cell components and antibiotics was given if
indicated. Response to therapy was defined as favourable if after 3 months of
anabolic therapy overt bleeding tendency had disappeared, there was no need for
transfusion therapy, a spontaneous increase of haemoglobin had occurred of
greater than 3 g/dl above the initial level, and a platelet rise of twofold the
initial count (up to at least greater than 30 x 10(9) /L) had occurred. Of 22
patients evaluable for the results of long-term (greater than 3 months) anabolic
treatment, six showed a partial response and eleven responded favourably. These
11 are all alive at the end of the study. Five of these patients proved to be
anabolic steroid-dependent. The 50% actuarial survival is approximately 4 years
after diagnosis, which compares favourably with the best published results from
bone marrow transplantation for aplastic anaemia. It is concluded that anabolic
therapy in aplastic anaemia should be tried for 2-3 months before the bone marrow
transplantation or immunosuppressive therapy is taken into consideration.