Analysis of REVLIMID in Patients with Non-Del-5Q MDS Demonstrated Improved Health Related Quality of Life Measures

Abstract #116 Celgene Corporation

today announced results from a pre-specified secondary endpoint analysis of MDS-005, a multicenter, randomized, placebo controlled phase III study of REVLMID® (lenalidomide) compared with placebo in patients with non-del-5q myelodysplastic syndromes (MDS). The analysis was presented during the 13th International Symposium on Myelodysplastic Syndromes. Results from MDS-005 were originally presented at the 2014 American Society of Hematology annual meeting and reported that significantly more patients treated with lenalidomide achieved red-blood-cell-transfusion independence (RBC-TI) of at least 56 days compared with placebo (26.9%, [43/160 patients] vs. 2.5%, [2/79 patients]; p<0.001), the primary endpoint of the study. In the current analysis, presented by Dr. Valeria Santini, HRQoL was assessed using the European Organization for Research and Treatment of Cancer QLQ-C30 questionnaire at baseline, every 12 weeks thereafter and at discontinuation. Patients who had a score change of at least 1 from baseline were assessed (lenalidomide n=122, placebo n=56). Clinically relevant domains of HRQoL were pre-selected as fatigue, dyspnea, physical functioning, emotional functioning and global quality of life. At week 12, mean changes in HRQoL scores from baseline were not significantly different between treatment arms for the pre-selected domains. At week 24, HRQoL score changes favored lenalidomide versus placebo for all pre-selected domains. After adjusting for baseline scores, improvement was statistically significant for emotional functioning (P=0.047) but not other domains. In lenalidomide patients, those who achieved RBC-TI and/or erythroid response at Week 24, an improving trend was observed with continued lenalidomide treatment. In a post hoc analysis, RBC-TI of at least 8 weeks was associated with significant improvement (P<0.01) across all preselected domains, with improvements also exceeding the pre-specified threshold for clinically meaningful change. "This analysis provides new insights into the clinical results of lenalidomide in non-del 5q patients," said Dr. Santini. "We now have a better understanding of how achievement of transfusion independence impacts quality of life measures." In the study, the most common adverse event associated with lenalidomide was related to myelosuppression including neutropenia (64.4% vs 11.4%) and thrombocytopenia (39.4% and 7.6%). Grade 3–4 neutropenia occurred in 61.9% versus 12.7% in the lenalidomide and placebo groups, respectively, and grade 3–4 thrombocytopenia occurred in 35.6% versus 3.8% in the lenalidomide and placebo groups, respectively. Based on the results of MDS-005, Celgene plans to submit a regulatory filing with the U.S. FDA in the second half of 2015. REVLIMID® is not approved for the treatment of patients with non-del-5q MDS.