Achievement of red blood cell transfusion independence in red blood cell transfusion-dependent patients with lower-risk non-del(5q) myelodysplastic syndromes correlates with serum erythropoietin levels

Abstract

In the randomized, phase 3, MDS-005 study (NCT01029262), lenalidomide-induced red blood cell transfusion independence (RBC-TI) in 27% of transfusion-dependent patients with lower-risk non-del(5q) myelodysplastic syndromes (MDS) ineligible for or refractory to erythropoiesis-stimulating agents. To determine the influence of erythropoietin (EPO) level on response, 155 patients treated with lenalidomide in MDS-005 were categorized into four groups by baseline EPO level. The EPO >500 mU/mL group had higher RBC transfusion burden and the lowest proportion of patients with ring sideroblasts ≥15% versus lower EPO groups. Achievement of RBC-TI ≥8 weeks inversely correlated with EPO level, ranging from 42.5 to 15.5%. EPO level did not affect erythroid hematologic improvement response (36.2–44.4%). This analysis suggests patients with lower EPO levels experience the strongest benefit from lenalidomide. Although meaningful improvements were observed in some patients with EPO level >500 mU/mL, new treatments are needed for this population.

Keywords:

• Erythropoietin
• lenalidomide
• myelodysplastic syndromes

Acknowledgments

This study was sponsored by Celgene Corporation, Summit, NJ, USA. The authors received editorial and writing support provided by Christian Geest, PhD, formerly of Excerpta Medica, funded by Celgene Corporation. The authors are fully responsible for all content and editorial decisions.

Disclosure statement

VS: Amgen, Astex, Celgene, Janssen, Novartis, Takeda – honoraria. AA: Alexion, Bristol-Myers Squibb, Shire - speakers bureau; Celgene Corporation – consultancy, research funding, speakers bureau; Novartis – consultancy, speakers bureau. AG: Celgene Corporation – consultancy. BS, CLB, and JW: Celgene Corporation – employment, equity ownership. NT: Celgene Corporation – formerly employment, equity ownership. PF: Astex, Janssen – research funding; Celgene Corporation, Novartis, Teva Pharmaceutical Industries - research funding, honoraria.