Real-World Data on the Safety and Efficacy of Pomalidomide as a Single Agent and in Combination with Corticosteroids in Relapsed and Refractory Multiple Myeloma

FAQ Library published on November 14, 2019
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Beth Faiman, PhD, RN, MSN, APRN-BC, AOCN
Department of Hematology and Medical Oncology
Cleveland Clinic Taussig Cancer Institute
Cleveland, Ohio

Welcome to Managing Myeloma. My name is Beth Faiman and today I’d like to discuss real-world data on the safety and efficacy of pomalidomide as a single agent and in combination with corticosteroids in relapsed/refractory multiple myeloma. This was a paper that was presented at the International Myeloma Workshop in Boston in 2019. As you very well know, pomalidomide is an immunomodulatory agent that is FDA-approved in combination with low-dose corticosteroids, among other agents, and in patients that are refractory to bortezomib and lenalidomide. That was the first indication, so at our institution we wanted to evaluate the safety and efficacy of pomalidomide in the real-world setting. Patients at the Cleveland Clinic from 2013 until 2018 were evaluated in a retrospective cohort analysis. All these patients had relapsed and refractory multiple myeloma. The primary objective was efficacy because safety was already assessed in the context of well-designed randomized clinical trials. The response rate or overall response rate was defined as a partial response or better, and progression-free survival and overall survival among the primary endpoints were assessed. So, 74 patients met criteria for inclusion in this study because they only received pomalidomide and dexamethasone. The dose of pomalidomide we included was just the 4 mg dose, days 1-21 of a 28-day schedule, and dexamethasone 20-40 mg weekly on that same schedule. The median age at diagnosis was 63 years and the median number of prior lines of therapy was three. A number of patients had undergone prior transplant and a number of patients were refractory to lenalidomide and bortezomib, which constituted 64% and 41% respectively. What is interesting about our analysis is that after a median follow-up of 39.6 months, the median progression-free survival was 14.4 months, which was longer than what was seen in clinical trials as well as an overall survival of 76.8 months. Adverse effects were very similar, hematologic toxicities in terms of neutropenia in 11% of patients, anemia and thrombocytopenia consistent with prior clinical trials. So in conclusion to the best of our knowledge, this is first real-word experience in pomalidomide in combination with dexamethasone in relapsed/refractory multiple myeloma and actually in this cohort we observed a very similar overall response rate but a much longer overall survival at 76.8 months versus 12.7 months, respectively. We did have a longer median follow-up and the safety was consistent with prior clinical trials, but these results underscore the importance considering pomalidomide and dexamethasone as a possible future therapy in patients with more than three lines of therapy. Of course, our study was limited by a small sample, single institution design, however, is something you can consider in your patients as well. I’d like to thank you at this point for viewing this activity.

Reference: Atieh T, et al. Real World Data on the Safety and Efficacy of Pomalidomide as a Single Agent and in Combination with Corticosteroids in Relapsed and Refractory Multiple Myeloma. IMW 2019. SP-073.

Last modified: November 13, 2019