As presented in a previous paper,1 prior to the discovery and use of the adrenal corticosteroids in congenital hypoplastic anemia, the only treatment was repeated blood transfusions. Although this permitted normal growth and development early in life, if continued, it resulted in extensive hemosiderosis and severe liver disease, with eventual liver failure. The chief hope for transfusion therapy was that it would enable the patient to survive until a spontaneous remission occurred.
In 1951, Gasser2 reported a corticosteroid-induced remission of this anemia and thereafter a number of additional patients elsewhere have been similarly treated. Although many of them have also had a steroid-induced remission, discouragingly, a significant number have not responded.
We have studied 22 children with congenital hypoplastic anemia given one or more trials of corticosteroids. In this paper we will show the results of such treatment, the conclusions we draw concerning the presence or failure of
ALLEN DM, DIAMOND LK. Congenital (Erythroid) Hypoplastic Anemia: Cortisone Treated. Am J Dis Child. 1961;102(3):416–423. doi:10.1001/archpedi.1961.02080010418021
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