• Paul G Richardson, David Siegel, Rachid Baz, Susan L Kelley, Nikhil C Munshi, Jacob Laubach, Daniel Sullivan, Melissa Alsina, Robert Schlossman, Irene M Ghobrial, Deborah Doss, Nora Loughney, Laura McBride, Elizabeth Bilotti, Palka Anand, Lisa Nardelli, Sandra Wear, Gail Larkins, Min Chen, Mohamad H Zaki, Christian Jacques, and Kenneth C Anderson.
• Division of Hematologic Malignancies, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, USA. Paul_Richardson@dfci.harvard.edu
• Blood. 2013 Mar 14;121(11):1961-7.

AbstractThis phase 1 dose-escalation study determined the maximum tolerated dose (MTD) of oral pomalidomide (4 dose levels) administered on days 1 to 21 of each 28-day cycle in patients with relapsed and refractory multiple myeloma (RRMM). After four cycles, patients who progressed or had not achieved minimal response (serum and urine M-protein reduction of ≥ 25% and ≥ 50%) could receive dexamethasone 40 mg per week. Safety and efficacy were evaluated. Thirty-eight patients who had received both bortezomib and lenalidomide (median 6 prior therapies) were enrolled; 63% were refractory to both lenalidomide and bortezomib. There were four dose-limiting toxicities (grade 4 neutropenia) at 5 mg per day and so the MTD was 4 mg per day. Rates of peripheral neuropathy and venous thromboembolism were low (≤ 5%). Among the 38 patients enrolled (including 22 with added dexamethasone), 42% achieved minimal response or better, 21% achieved partial response or better, and 3% achieved complete response. Median duration of response, progression-free survival, and overall survival were 4.6, 4.6, and 18.3 months, respectively. Pomalidomide 4 mg per day on days 1 to 21 of each 28-day cycle, with or without dexamethasone (40 mg/week), has encouraging activity with manageable toxicity in RRMM, including those refractory to both lenalidomide and bortezomib. This study is registered at http://www.clinicaltrials.gov as #NCT00833833.

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