Failure of immunosuppressive therapy and high-dose intravenous immunoglobulins in four transfusion-dependent, steroid-unresponsive Blackfan-Diamond anemia patients.

Blackfan-Diamond anemia (BDA) is a rare hypoproliferative anemia occurring in infancy or in early childhood. Patients who fail on usual doses of steroids did not achieve remission with other pharmacological agents. Claims that other molecules such as cyclosporin A (CSA) or antithymocyte globulin (ATG) are effective require substantiation. We treated four transfusion-dependent, steroid-unresponsive BDA patients with ATG and methylprednisolone (MP). Only a transient response was obtained in one case. None of these patients responded to high-dose intravenous immunoglobulins (HDIg) or CSA.