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Blood, 15 January 2008, Vol. 111, No. 2, pp. 574-582.
Prepublished online as a Blood First Edition Paper on October 16, 2007; DOI 10.1182/blood-2007-06-096370.


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CLINICAL TRIALS AND OBSERVATIONS

Predictive factors of response and survival in myelodysplastic syndrome treated with erythropoietin and G-CSF: the GFM experience

Sophie Park1, Sophie Grabar2, Charikleia Kelaidi3, Odile Beyne-Rauzy4, Françoise Picard5, Valérie Bardet5, Valérie Coiteux6, Geneviève Leroux7, Pascale Lepelley8, Marie-Thérèse Daniel9, Stéphane Cheze10, Béatrice Mahé11, Augustin Ferrant12, Christophe Ravoet13, Martine Escoffre-Barbe14, Lionel Adès3, Norbert Vey15, Lina Aljassem16, Aspasia Stamatoullas17, Lionel Mannone18, Hervé Dombret19, Keith Bourgeois20, Peter Greenberg21, Pierre Fenaux3, François Dreyfus1, for the GFM group (Groupe Francophone des Myélodysplasies)

1 Service d'hématologie, Hôpital Cochin, Assistance Publique des Hopitaux de Paris (APHP), Paris, France; 2 Département de Statistiques, Hôpital Cochin, APHP, Paris, France; 3 Service d'hématologie, Hôpital Avicenne, APHP, Bobigny, France; 4 Service de médecine interne, Hôpital Purpan, Toulouse, France; 5 Laboratoire d'hématologie, Hôpital Cochin, Paris, France; 6 Service des maladies du Sang, Hôpital Claude Huriez, Centre Hospitalier Régional Universitaire, (CHRU) Lille, Lille, France; 7 Laboratoire d'hématologie, Hôpital Avicenne, Bobigny, France; 8 Service d'hématologie, CHRU Lille, Lille, France; 9 Laboratoire d'hématologie, Hôpital Saint-Louis, Paris, France; 10 Service d'hématologie, Centre Hospitalier Universitaire (CHU) Caen, Caen, France; 11 Service d'hématologie, CHU Nantes, Nantes, France; 12 Département de Médecine Interne, Cliniques Universitaires Saint-Luc, Brussels, Belgium; 13 Institut Jules Bordet, Brussels, Belgium; 14 Service d'hématologie, CHU Pontchaillou, Rennes, France; 15 Département d'Onco-Hématologie, Centre Paoli-Calmettes, Marseille, France; 16 Service d'hématologie, Centre Hospitalier (CH) Chartres, Chartres, France; 17 Service d'hématologie, CHU Rouen, Rouen, France; 18 Service d'hématologie, CHU Nice, Nice, France; 19 Service d'hématologie, CHU Saint-Louis, Paris, France; 20 Department of Biostatistics and Computational Biology, University of Rochester, NY; and 21 Department of Medicine, Division Hematology, Stanford University Medical Center, Stanford, CA

We analyzed prognostic factors of response, response duration, and possible impact on survival of epoetin {alpha}, epoetin β, or darbepoetin {alpha} (DAR) with or without granulocyte colony-stimulating factor in 403 myelodysplastic syndrome (MDS) patients. Sixty-two percent (40% major and 22% minor) and 50% erythroid responses were seen, and median response duration was 20 and 24 months according to IWG 2000 and 2006 criteria, respectively. Significantly higher response rates were observed with less than 10% blasts, low and int-1 International Prognostic Scoring System (IPSS), red blood cell transfusion independence, serum EPO level less than 200 IU/L, and, with IWG 2006 criteria only, shorter interval between diagnosis and treatment. Significantly longer response duration was associated with major response (IWG 2000 criteria), IPSS low to INT-1, blasts less than 5%, and absence of multilineage dysplasia. Minor responses according to IWG 2000 were reclassified as "nonresponders" or "responders" according to IWG 2006 criteria. However, among those IWG 2000 minor responders, response duration did not differ between IWG 2006 responders and nonresponders. Multivariate adjusted comparisons of survival between our cohort and the untreated MDS cohort used to design IPSS showed similar rate of progression to acute myeloid leukemia in both cohorts, but significantly better overall survival in our cohort, suggesting that epoetin or DAR treatment may have a favorable survival impact in MDS.


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