A retrospective analysis of the clinical use and benefit of lenalidomide and thalidomide in myelofibrosis

Abstract Introduction Anemia in myelofibrosis (MF) occurs frequently, is poorly addressed by FDA-approved JAK inhibitors, and negatively impacts quality of life. Immunomodulatory imide agents (IMiDs), such as thalidomide and lenalidomide are among the limited treatment options that have demonstrated anemia benefit in single-arm studies. Methods To better understand the comparative impact of lenalidomide and thalidomide in MF patients, we analyzed 176 consecutive MF patients who received lenalidomide or thalidomide for at least four weeks. We sought to understand the variability in patient populations receiving lenalidomide versus thalidomide, assess the efficacy of these agents, and investigate clinical or genomic features that predict for response. Results Clinical benefit (CB) was assessable in 83 lenalidomide- and 67 thalidomide-treated patients. Thalidomide-treated patients were more likely to have thrombocytopenia (p Conclusion Overall, in this retrospective analysis, lenalidomide and thalidomide showed similar rates of clinical benefit in a cohort of MF patients that frequently harbored splicing mutations.