NO RESPONSE TO RECOMBINANT HUMAN ERYTHROPOIETIN THERAPY IN PATIENTS WITH CONGENITAL DYSERYTHROPOIETIC ANEMIA TYPE I

Congenital dyserythropoietic anemia (CDA) type I is a rare inherited bone marrow disorder characterized by moderate to severe macrocytic anemia with pathognomonic cytopathology of nucleated red blood cells. Previous studies have suggested that serum erythropoietin levels in affected patients are low...

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Bibliographic Details
Published in:Pediatric hematology and oncology 1999, Vol.16 (2), p.165-168
Main Author: Hannah Tamary, Petah Tiqva, Hanna Shalev, Vered Pinsk, Beer Sheva, Meira Zoldan, Rina Zaizov, Petah Tiqva
Format: Article
Language:English
Subjects:
Adolescent
Anemia, Congenital Dyserythropoietic Anemia Type I, Recombinant Human Erythropoietin, Treatment
Anemia, Dyserythropoietic, Congenital - drug therapy
Anemias. Hemoglobinopathies
Biological and medical sciences
Child
Child, Preschool
Diseases of red blood cells
Erythropoietin - therapeutic use
Female
Hematologic and hematopoietic diseases
Humans
Male
Medical sciences
Recombinant Proteins
Treatment Failure
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Summary:Congenital dyserythropoietic anemia (CDA) type I is a rare inherited bone marrow disorder characterized by moderate to severe macrocytic anemia with pathognomonic cytopathology of nucleated red blood cells. Previous studies have suggested that serum erythropoietin levels in affected patients are lower than expected for the degree of anemia. An earlier study demonstrated a substantial increase in the number of CFU-E in CDA type I pattern on addition of exogenous erythropoietin. The present study reports on the response to recombinant human erythropoietin in 8 patients with CDA type I. Eighteen weeks of treatment, starting at 300 IU/kg twice a week and gradually increasing to 500 IU/kg three times a week, did not have a substantial effect on the mean hemoglobin value. These results indicate that recombinant human erythropoietin (rHuEpo) is not beneficial to patients with CDA type I and that the relatively low levels of serum erythropoietin probably play no major role in the pathogenesis of the disease.
ISSN:0888-0018
1521-0669
DOI:10.1080/088800199277498