Health, economic, and quality-of-life effects of erythropoietin and granulocyte colony-stimulating factor for the treatment of myelodysplastic syndromes: a randomized, controlled trial.
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.
Casadevall
Nicole
N
Hôtel Dieu, Service d'Hématologie Biologique, 1 place du parvis Notre Dame, 75004 Paris, France. nicole.casadevall@htd.ap-hop-paris.fr
Durieux
Pierre
P
Dubois
Stéphanie
S
Hemery
François
F
Lepage
Eric
E
Quarré
Marie-Catherine
MC
Damaj
Gandhi
G
Giraudier
Stéphane
S
Guerci
Agnès
A
Laurent
Guy
G
Dombret
Hervé
H
Chomienne
Christine
C
Ribrag
Vincent
V
Stamatoullas
Aspasia
A
Marie
Jean-Pierre
JP
Vekhoff
Anne
A
Maloisel
Frédéric
F
Navarro
Robert
R
Dreyfus
François
F
Fenaux
Pierre
P
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000188:drug therapy; D003362:Cost-Benefit Analysis; D016527:Drug Costs; D004921:Erythropoietin / Q000008:administration & dosage* / Q000009:adverse effects / Q000191:economics; D005260:Female; D005500:Follow-Up Studies; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage* / Q000009:adverse effects / Q000191:economics; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D011788:Quality of Life; D011994:Recombinant Proteins; D016896:Treatment Outcome
D011994:Recombinant Proteins; D004921:Erythropoietin; D016179:Granulocyte Colony-Stimulating Factor