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.
D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D000741:Anemia, Aplastic / Q000097:blood / Q000151:congenital* / Q000628:therapy; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D001853:Bone Marrow / Q000473:pathology; D002648:Child; D003520:Cyclophosphamide / Q000627:therapeutic use*; D004900:Erythroblasts; D004920:Erythropoiesis; D006801:Humans; D007166:Immunosuppressive Agents; D008297:Male; D012075:Remission, Spontaneous; D013577:Syndrome
D000305:Adrenal Cortex Hormones; D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide