Study of 22 Egyptian patients with Diamond-Blackfan anemia, corticosteroids, and cyclosporin therapy results.
OBJECTIVE
Diamond-Blackfan anemia is a rare congenital hypoproliferative anemia of infancy and early childhood. Treatment with corticosteroids is commonly used, but with limited success. Trials with cyclosporin-A (CSA) are not frequently reported. Therefore, in this study we analyzed our results in the management of this rare disease by different medical treatments.


METHODS
The results of 22 patients diagnosed at our Hematology Center in the New Cairo University Children's Hospital during the period 1991-2001 were retrospectively analyzed. Our patients first received prednisolone (2 mg/kg/d) for different courses according to their response. Since the year 2000, the steroid nonresponders received CSA (3-12 mg/kg/d) for 6 months unless treatment complications developed.


RESULTS
The age at the onset of the disease ranged from 1 to 24 months (median: 2.5 months). The mean values of the hemoglobin, the reticulocyte count, and the myeloid/erythroid ratio at the onset of the disease were 4.75 +/- 1.79 g/dL, 0.14 +/- 0.16, and 39.4 +/- 27.08, respectively. Patients received prednisolone from 0.25 to 10 years (median: 2 years). Ten patients were nonresponders (45.5%), and 5 patients (22.7%) responded to corticosteroid therapy. Two of 5 responders are off treatment with a hemoglobin level of >9 g/dL, and 3 of 5 are currently corticosteroid-dependent. Of 10 patients not responding to steroids, 8 received CSA for 6 months. Four patients (50%) responded to CSA therapy. A significant positive association was found between CSA dose and response.


CONCLUSIONS
CSA therapy should be tried in steroid-resistant Diamond-Blackfan anemia patients before blood transfusion or corticosteroid therapy complications are instituted.
D017668:Age of Onset; D029503:Anemia, Diamond-Blackfan / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D004534:Egypt; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use; D006454:Hemoglobins / Q000032:analysis; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D037102:Lectins; D008297:Male; D008775:Methylprednisolone / Q000627:therapeutic use; D011239:Prednisolone / Q000627:therapeutic use; D011241:Prednisone / Q000627:therapeutic use; D011247:Pregnancy; D011250:Pregnancy Complications, Hematologic; D017701:Reticulocyte Count; D012189:Retrospective Studies; D016896:Treatment Outcome
D005938:Glucocorticoids; D006454:Hemoglobins; D007166:Immunosuppressive Agents; D037102:Lectins; C071077:blood type B binding lectin, Charybdis japonica; D016572:Cyclosporine; D011239:Prednisolone; D011241:Prednisone; D008775:Methylprednisolone