Celgene Offers Results from Phase III Study of REVLIMID, Showed Improved Transfusion Independence

Celgene Corporation CELG today announced results from an international phase III study of REVLIMID® (lenalidomide) compared with placebo in patients with red-blood cell (RBC) transfusion dependent low-risk myelodysplastic syndromes (MDS) who were unresponsive or refractory to erythropoietin stimulating agents (ESA) and did not have a deletion 5q cytogenic abnormality. The results were presented during the 56th American Society of Hematology (ASH) annual meeting. The study, presented by Valeria Santini, M.D., found that significantly more patients treated with lenalidomide achieved RBC-transfusion independence 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. The majority of patients (90%) with transfusion independence responded within 16 weeks of treatment. For patients who became transfusion independent, the median duration of transfusion independence was 8.2 months (range 5.2-17.8). Additionally, transfusion independence of at least 168 days was reached in 17.5% (28/160) of patients receiving lenalidomide compared with no patients receiving placebo. The incidence of AML progression (per 100 person-years) was 1.91 (95% CI 0.80-4.59) and 2.46 (95% CI 0.79-7.64) for lenalidomide and placebo patients, respectively. The follow-up period was not long enough to permit an overall survival comparison. Myelosuppression was the main adverse event (AE). 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. “This confirmation of the Phase II data is extremely encouraging. Based on this study, REVLIMID may offer this refractory patient population an additional option beyond their current limited choices,” said Guillermo Garcia-Manero, M.D. of the M.D. Anderson Cancer Center at the University of Texas. REVLIMID is not indicated for the treatment of non-del5q MDS in any country.