Clinical Course in 28 Unselected Patients with Aplastic Anaemia Treated with Anabolic Steroids

Abstract

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.

Prediction of Survival in Adults with Acquired Bi‐ or Pancytopenia

“…Furthermore, PI(Naj) and PI(Lyn) appeared rather robust to application to patients fulfilling more extensive criteria of selection, while our EP on the other hand seemed to retain its discriminative capacity in the more restricted groups of patients with A A retrospectively or initially. Thus, our study supports the predictive value of PI(Lyn) and DS(Cam) which has been questioned by te Velde & Haak (20) and van Hengstum et a1 (24), respectively. Conversely, EP(Roz) was not found significantly predictive, but PI(Lyn) also appeared less discriminative than originally reported (2).…”

In vitro steroid sensitivity testing: A possible means to predict response to therapy in primary hypoproliferative anemia

“…One group of hormones with known effects on in vitro erythropoiesis, the steroids, has been used extensively in the treatment of various anemia states, including aplastic anemia, myelofibrosis, refractory anemia, chronic lymphocytic leukemia, and the anemias of chronic renal failure and inflammatory disease [13,14]. Reports of steroid dependency in some patients with bone marrow failure states are well known [15][16][17], whereas other studies have demonstrated that the vast majority of patients treated with these agents show no hématologie improvement following therapy [18][19][20][21]. Clearly, an in vitro method to predict clinical response to steroids would be of great value in the selection of patients for this form of therapy.…”

Testosterone

“…The effects of androgen treatment on erythropoiesis have been well established (McCullagh and Jones 1942) and have been used in the treatment of a variety of anaemias (Hendler et al 1974;Hengstum et al 1979;Najean 1981). This results from a direct effect on the bone marrow (Mirand and Murphy 1971;) and from enhancement of erythropoietin production, even in anephric patients (Naets and Wittek 1968;Shahidi 1973).…”