Prior presentation: Presented in abstract form at the 48th annual meeting of the American Society of Hematology, Orlando, Florida, 9–12 December 2006 and at the 43rd annual meeting of the American Society of Clinical Oncology, Chicago, Illinois, 1–5 June 2007.
Phase 2, single-arm trial to evaluate the effectiveness of darbepoetin alfa for correcting anaemia in patients with myelodysplastic syndromes
Version of Record online: 6 JUN 2008
© 2008 The Authors. Journal Compilation © 2008 Blackwell Publishing Ltd
British Journal of Haematology
Volume 142, Issue 3, pages 379–393, August 2008
How to Cite
Gabrilove, J., Paquette, R., Lyons, R. M., Mushtaq, C., Sekeres, M. A., Tomita, D. and Dreiling, L. (2008), Phase 2, single-arm trial to evaluate the effectiveness of darbepoetin alfa for correcting anaemia in patients with myelodysplastic syndromes. British Journal of Haematology, 142: 379–393. doi: 10.1111/j.1365-2141.2008.07181.x
Re-use of this article is permitted in accordance with the Creative Commons Deed, Attribution 2.5, which does not permit commercial exploitation.
- Issue online: 8 JUL 2008
- Version of Record online: 6 JUN 2008
- Received 30 September 2007; accepted for publication 14 February 2008
- myelodysplastic syndromes;
- erythropoiesis-stimulating agent;
- erythroid response;
Patients with myelodysplastic syndromes (MDS) often develop anaemia resulting in frequent transfusions and fatigue. Darbepoetin alfa is an erythropoiesis-stimulating agent (ESA) approved for treating chemotherapy-induced anaemia. This single-arm, phase 2 study examined the efficacy of darbepoetin alfa 500 μg every 3 weeks (Q3W) for treating anaemia in low-risk MDS patients (after 6 weeks, poor responders received darbepoetin alfa 500 μg every 2 weeks). The primary end-point was the incidence of erythroid responses (International Working Group criteria) after 13 weeks of therapy. Secondary end-points included the incidence of erythroid responses at weeks 28 and 55, [or weeks 27 and 53 for dose escalations to every two weeks (Q2W)], and safety parameters. Analyses were stratified by the patient’s previous ESA therapy status [ESA-naïve (n = 144) vs. prior ESA-treated (n = 62)]. After 13 weeks of therapy, 49% of ESA-naïve patients and 26% of prior ESA-treated patients achieved a major erythroid response. After 53/55 weeks, 59% of ESA-naïve patients and 34% of prior ESA-treated patients achieved a major erythroid response; 82% of ESA-naïve patients and 55% of prior ESA-treated patients achieved target haemoglobin of 110 g/l. Thromboembolic or related adverse events occurred in 2% of patients; no pulmonary embolisms were reported. In conclusion, darbepoetin alfa, 500 μg Q3W appeared well tolerated and increased haemoglobin levels in low-risk MDS patients.