Health, economic, and quality-of-life effects of erythropoietin and granulocyte colony-stimulating factor for the treatment of myelodysplastic syndromes: a randomized, controlled trial.

Abstract

In myelodysplastic syndromes (MDS), anemia responds to recombinant human erythropoietin (rHuEPO) alone and in combination with recombinant human granulocyte-colony-stimulating factor (rHuGCSF) in 10% to 20% and in 35% to 40% of patients, respectively. We randomly divided 60 patients with low-grade anemic MDS and serum EPO levels lower than 500 IU/L (500 mU/mL) into 2 groups: rHuEPO + rHuG-CSF (arm A) and supportive care (arm B). After 12 weeks, those who had erythroid responses were given rHuEPO alone for 40 additional weeks. They were also given rHuG-CSF if they had relapses. A response was considered major if the hemoglobin (Hb) level was 115 g/L (11.5 g/dL) or higher and minor Hb increase was 15 g/L (1.5 g/dL) or more or if it remained stable without transfusion. Ten of 24 patients responded in arm A, and 0 of 26 responded in arm B (P =.01). Eight patients in arm A continued rHuEPO therapy alone, and 6 had relapses. Responses were always restored when rHuG-CSF was reintroduced. Mean direct costs per patient were 26,723 euros (arm A) and 8,746 euros (arm B). Quality of life was assessed with a Functional Assessment of Cancer Therapy-Anemia (FACT-An) scale. Similar percentages of patients from both arms showed significant clinical improvement. rHuEPO plus rHuG-CSF led to responses in 41.7% of MDS patients. This treatment was expensive. No effect on quality of life was demonstrated.

• Authors: Nicole Casadevall, Pierre Durieux, Stéphanie Dubois, François Hemery, Eric Lepage, Marie-Catherine Quarré, Gandhi Damaj, Stéphane Giraudier, Agnès Guerci, Guy Laurent, Hervé Dombret, Christine Chomienne, Vincent Ribrag, Aspasia Stamatoullas, Jean-Pierre Marie, Anne Vekhoff, Frédéric Maloisel, Robert Navarro, François Dreyfus, Pierre Fenaux
• Journal: Blood (Blood) Vol. 104 Issue 2 Pg. 321-7 (Jul 15 2004) ISSN: 0006-4971 [Print] United States
• PMID: 15054036 (Publication Type: Clinical Trial, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Recombinant Proteins
• Erythropoietin
• Granulocyte Colony-Stimulating Factor

Topics

• Adult
• Aged
• Aged, 80 and over
• Anemia (drug therapy)
• Cost-Benefit Analysis
• Drug Costs
• Erythropoietin (administration & dosage, adverse effects, economics)
• Female
• Follow-Up Studies
• Granulocyte Colony-Stimulating Factor (administration & dosage, adverse effects, economics)
• Humans
• Male
• Middle Aged
• Myelodysplastic Syndromes (drug therapy)
• Quality of Life
• Recombinant Proteins
• Treatment Outcome