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Erythropoiesis stimulating agents and other growth factors in low-risk MDS

Authors
  • Hellström-Lindberg, Eva
  • van de Loosdrecht, Arjan1, 2, 3, 4, 5, 6, 7
  • 1 Department of Medicine
  • 2 Division of Hematology
  • 3 Karolinska Institutet
  • 4 Karolinska University Hospital
  • 5 Department of Hematology
  • 6 Cancer Center Amsterdam
  • 7 VU University Medical Center
Type
Published Article
Journal
Best Practice & Research Clinical Haematology
Publisher
Elsevier
Publication Date
Jan 01, 2014
Volume
26
Issue
4
Pages
401–410
Identifiers
DOI: 10.1016/j.beha.2013.09.007
Source
Elsevier
Keywords
License
Unknown

Abstract

Anemia and transfusion need constitute major problems for patients with myelodysplastic syndromes (MDS) and are associated with reduced quality of life, poorer survival and an increased risk for transformation to AML. Treatment with erythropoiesis-stimulating agents (ESAs) is first-line treatment for the anemia of most patients with MDS. Erythropoietin acts synergistically with G-CSF to inhibit erythroid apoptosis and promote erythrocyte production. The median duration of response is 2–3 years, with patients responding for more than a decade. Onset of a permanent transfusion need is delayed if treatment is introduced early after the onset of symptomatic anemia. A positive effect on long-term outcome has been suggested by several large epidemiological studies, with no difference in the rate of leukemic transformation between treated and untreated patients. Moreover, responding patients show improvement of quality of life and exercise capacity. Response to treatment can be predicted by combining serum erythropoietin, transfusion rate, and flow cytometry profiling.

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