@article {117554, title = {Study of 22 Egyptian patients with Diamond-Blackfan anemia, corticosteroids, and cyclosporin therapy results.}, journal = {Pediatrics}, volume = {110}, year = {2002}, month = {2002 Oct}, pages = {e44}, abstract = {

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.

DESIGN: The results of 22 patients diagnosed at our Hematology Center in the New Cairo University Children{\textquoteright}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.

CONCLUSION: CSA therapy should be tried in steroid-resistant Diamond-Blackfan anemia patients before blood transfusion or corticosteroid therapy complications are instituted.

}, keywords = {Age of Onset, Anemia, Diamond-Blackfan, Child, Child, Preschool, Cyclosporine, Drug Therapy, Combination, Egypt, Female, Glucocorticoids, Hemoglobins, Humans, Immunosuppressive Agents, Infant, Lectins, Male, Methylprednisolone, Prednisolone, Prednisone, Pregnancy, Pregnancy Complications, Hematologic, Reticulocyte Count, Retrospective Studies, Treatment Outcome}, issn = {1098-4275}, author = {Amal El-Beshlawy and Ibrahim, Ilham Youssry and samia rizk and Eid, Khalid} }