No response to recombinant human erythropoietin therapy in patients with congenital dyserythropoietic anemia type I

Hannah Tamary, Hanna Shalev, Vered Pinsk, Meira Zoldan, Rina Zaizov

Research output: Contribution to journalArticlepeer-review

13 Scopus citations

Abstract

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.

Original languageEnglish
Pages (from-to)165-168
Number of pages4
JournalPediatric Hematology and Oncology
Volume16
Issue number2
DOIs
StatePublished - 1 Jan 1999
Externally publishedYes

Keywords

  • Anemia
  • Congenital dyserythropoietic anemia type I
  • Recombinant human erythropoietin
  • Treatment

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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