Congenital hypoplastic anaemia refractory to corticosteroids but responding to cyclophosphamide and antilymphocytic globulin. Report of a case having responded with a transitory wave of dyserythropoiesis.

A 7-year-old male child with congenital hypoplastic anaemia (Diamond-Blackfan), having originally responded to corticosteroids, but having subsequently become refractory and erythroblastopenic since 4 years, was treated with a combination of cyclophosphamide (CY; 2.1 g) and antilymphocytic globulin (ALG; 8 g), both by the intravenous route. Erythroblastic repopulation of the bone marrow, albeit dyserythropoietic in character, reticulocytosis and erythrocytic increment took place, but only for a short period. The significance of these findings is discussed in the light of recent progress in the understanding of the disease and of its treatment.