Preclinical and clinical results with pomalidomide in the treatment of relapsed/refractory multiple myeloma

Abstract Despite the evolution of effective frontline treatment strategies, many patients with myeloma inevitably relapse. Treatment can be complicated by the interplay of disease-, treatment-, and patient-related factors. Unfortunately, many patients eventually develop disease that is refractory to lenalidomide and bortezomib and have few treatment options. Pomalidomide is a distinct IMiD® agent recently approved in the US and Europe. We review the pomalidomide mechanism of action, summarizing its direct antimyeloma, immunomodulatory, and stromal-support inhibitory activities. We also detail its clinical development, including establishment of the approved dose/schedule, phase 2 and 3 trials in relapsed and refractory patients, and novel pomalidomide-based combinations.