Lenalidomide in the Myelodysplastic Syndrome with Chromosome 5q Deletion

摘要: Background Severe, often refractory anemia is characteristic of the myelodysplastic syndrome associated with chromosome 5q31 deletion. We investigated whether lenalidomide (CC5013) could reduce transfusion requirement and suppress abnormal 5q31− clone in patients this disorder. Methods One hundred forty-eight received 10 mg for 21 days every 4 weeks or daily. Hematologic, bone marrow, cytogenetic changes were assessed after 24 treatment by an intention-to-treat analysis. Results Among 148 patients, 112 had a reduced need transfusions (76%; 95% confidence interval [CI], 68 to 82) 99 (67%; CI, 59 74) no longer required transfusions, regardless karyotype complexity. The response was rapid (median time response, 4.6 weeks; range, 1 49) sustained; median duration independence not been reached 104 follow-up. maximum hemoglobin concentration reached...