High‐dose darbepoetin alpha in the treatment of anaemia of lower risk myelodysplastic syndrome results of a phase II study

• Published in: British journal of haematology Vol. 133; no. 5; pp. 513 - 519
• Main Authors: Mannone, L., Gardin, C., Quarre, M. C., Bernard, J. F., Vassilieff, D., Ades, L., Park, S., Vaultier, S., Hamza, F., Beyne‐rauzy, M. O., Cheze, S., Giraudier, S., Agape, P., Legros, L., Voillat, L., Dreyfus, F., Fenaux, P.
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.06.2006; Blackwell
• Subjects: Aged; Aged, 80 and over; anaemia; Anemia - complications; Anemia - drug therapy; Anemias. Hemoglobinopathies; Biological and medical sciences; darbepoetin; Darbepoetin alfa; Diseases of red blood cells; Drug Administration Schedule; Drug Therapy, Combination; Erythropoiesis - drug effects; erythropoietin; Erythropoietin - administration & dosage; Erythropoietin - adverse effects; Erythropoietin - analogs & derivatives; Erythropoietin - therapeutic use; Female; Granulocyte Colony-Stimulating Factor - administration & dosage; Hematologic and hematopoietic diseases; Hematology; Humans; Injections, Subcutaneous; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical sciences; Middle Aged; myelodysplastic syndromes; Myelodysplastic Syndromes - complications; Myelodysplastic Syndromes - drug therapy; Risk Factors; Treatment Outcome; anaemia; Hematology; Anemia; Malignant hemopathy; Epidemiology; Myelodysplastic syndrome; Treatment; Darbepoetin alpha; Phase II trial; Risk factor; myelodysplastic syndromes; High dose; darbepoetin; erythropoietin
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/j.1365-2141.2006.06070.x

Summary An open‐label, phase II non‐randomised trial was conducted with darbepoetin (DAR), an erythropoiesis‐stimulating factor with prolonged half‐life, at a weekly dose of 300 μg subcutaneously in 62 anaemic patients with myelodysplastic syndrome (MDS) with an endogenous erythropoietin (EPO) level <500 mU/ml. Most of the patients were classified as low or intermediate 1 according to the International Prognostic Scoring System. After 12 weeks, 44 (71%) patients had an erythroid response (34 major and 10 minor), including eight of 13 patients who were previous non‐responders to conventional EPO. Two additional responses (one minor and one major) occurred, in 10 non‐responders, after the addition of granulocyte colony‐stimulating factor (G‐CSF). Thirty‐six of the 46 total responders (31/35 major and 5/11 minor) continued to respond on maintenance DAR after a median of 40 weeks (range 4–84). Median dose of DAR required to maintain response was 300 μg every 14 d. The only prognostic factors of favourable response were low endogenous EPO level and low or absent red blood cell transfusion requirement. Those results suggest that high‐dose DAR alone yields high erythroid response rates in anaemia of lower risk MDS, possibly equivalent to those obtained with conventional EPO + G‐CSF, although this will need to be confirmed in larger and randomised trials.