[Acquired aplastic anemia in children. Therapy using androgens and antilymphocyte gamma globulin]

Authors describe initial characteristics, clinical course and response to treatment of 13 children, aged from eight months to nine years, with acquired aplastic anaemia. Six of the children had been exposed to products with potential bone-marrow toxicity: chloranphenicol (1), pirazolones (3) and insecticides (2). Pancytopenia was severe in twelve and moderate in one. Five patients with severe aplastic anaemia were given antilymphocytic (2) or antithymocytic (3) (ATG) gammaglobulins; two of those treated with ATG responded after two and five months respectively and there was no response in the other three. Twelve patients were given androgen treatment (oxymetholone or nandrolone decanoate): four, two of whom previously had received ATG, followed a favorable course. None of the severe initial pancytopenic patients responded to androgen single agent treatment. No marrow transplant was performed in any of the children. Eight of the thirteen patients died one to 37 months after onset (median, 6 months) and five (38%) are alive after 21 months to 10 years, corresponding to two patients with moderate and three with severe initial pancytopenia. Importance of an appropriate supportive treatment during initial stages of the disease and the probable efficacy or immunosupressive treatment in certain patients must be underlined.