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Blood, 15 July 2004, Vol. 104, No. 2, pp. 321-327.
Prepublished online as a Blood First Edition Paper on March 30, 2004; DOI 10.1182/blood-2003-07-2252.
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CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Health, economic, and quality-of-life effects of erythropoietin and granulocyte colony-stimulating factor for the treatment of myelodysplastic syndromes: a randomized, controlled trial
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, and
Pierre Fenaux, for the Groupe Français des Myélodysplasies
From the Service d'Hématologie Biologique et Clinique, Hôtel Dieu, Assistance Publique Hôpitaux de Paris (AP-HP), Paris; Santé Publique et Informatique Médicale, Hôpital Européen Georges Pompidou (HEGP), Paris; Unité d'Information Médicale, Hôpital Henri Mondor, Créteil; Services d'Hématologie, Hôpital Cochin, Paris; Hôpital Necker, Paris; Hôpital Henri Mondor, Créteil; Centre Hospitalier Universitaire (CHU) Nancy-Brabois; Hôpital Purpan, Toulouse; Hôpital Saint-Louis, Paris; Institut Gustave Roussy, Villejuif; Centre Henri Becquerel, Rouen; Hôpital de Hautepierre, Strasbourg; Hôpital Lapeyronie, Montpellier; and CHU de Lille, France.
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 ( 26 723) (arm A) and 8746 (arm B). Quality of life was aszsessed 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. (Blood. 2004;104: 321-327)

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