Prior to novel targeted agents for chronic lymphocytic leukemia (CLL), the best chemoimmunotherapy regimen in patients with non-del(11q) disease was unclear. The role of lenalidomide was also not defined. This phase 2 study randomized 342 untreated patients with non-del(11q) CLL requiring therapy to fludarabine plus rituximab (FR; n 5 123), FR plus lenalidomide consolidation (FR1L; n 5 109), or FR plus cyclophosphamide (FCR; n 5 110) and compared 2-year progression-free survival (PFS) rates of each to the historical control rate with FC (60%). Patients with del(11q) in at least 20% of pretreatment cells continued with FCR (n 5 27) or were reassigned to FCR1L (n 5 31) and excluded from the primary analysis. Among non-del(11q) patients, 2-year PFS rates were 64% (90% confidence interval [CI], 57-71; FR), 72% (90% CI, 65-79; FR1L), and 74% (90% CI, 66-80; FCR); FR1L and FCR had rates significantly greater than historical control. Median PFS was significantly shorter with FR compared with FR1L (P 5 .04) and FCR (P, .001): 43 (95% CI, 33-50), 61 (95% CI, 45-71), and 97 (95% CI, 61 to not reached) months, respectively. Median follow-up was 73 months and median overall survival (OS) was only reached with FCR (101 months; 95% CI, 96 to not reached). With FR1L, the risk of death decreased over time and was lower than with FR at later time points (P 5 .01), but not significantly different from FCR (P 5 .21). Future studies incorporating short courses of lenalidomide into other novel treatment regimens are justified.
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