Long-term alpha interferon treatment is effective on anaemia and significantly reduces iron overload in congenital dyserythropoiesis type I.

Interferon has been shown to be an effective treatment of congenital dyserythropoiesis type I (CDA-I), but the optimal dose and the feasibility of this treatment remains to be determined. Here, in a 9-yr follow-up of a single patient, we show that interferon remains active during such a long period. The optimal dose of conventional alpha interferon could be evaluated at 2 million units twice a week. Pegylated interferon could be used as well at a dose of 30 microg/wk. During interferon treatment, serum and erythrocyte ferritin levels decreased progressively, and remained inversely correlated with haemoglobin levels. On repeated liver biopsies, iron overload could be normalized. Low dose interferon is a long-term treatment of CDA-I, and allows a significant decrease in iron overload, that could be interesting even in patients who are only moderately anaemic.