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Pomalidomide alone or in combination with low-dose dexamethasone in relapsed and refractory multiple myeloma: a randomized phase 2 study

Paul G. Richardson, David S. Siegel, Ravi Vij, Craig C. Hofmeister, Rachid Baz, Sundar Jagannath, Christine Chen, Sagar Lonial, Andrzej Jakubowiak, Nizar Bahlis, Kevin Song, Andrew Belch, Noopur Raje, Chaim Shustik, Suzanne Lentzsch, Martha Lacy, Joseph Mikhael, Jeffrey Matous, David Vesole, Min Chen, Mohamed H. Zaki, Christian Jacques, Zhinuan Yu, Kenneth Anderson
This article has an Erratum 123(20):3208

Key points

  • Pomalidomide plus low-dose dexamethasone significantly improved PFS versus pomalidomide alone in relapsed and refractory multiple myeloma

  • Pomalidomide plus low-dose dexamethasone is an important new treatment option for RRMM patients who had received multiple prior therapies

Abstract

This multicenter, open-label, randomized phase 2 study assessed the efficacy and safety of pomalidomide (POM) with/without low-dose dexamethasone (LoDEX) in patients with relapsed/refractory multiple myeloma (RRMM). Patients who had received ≥ 2 prior therapies (including lenalidomide [LEN] and bortezomib [BORT]) and had progressed within 60 days of their last therapy were randomized to POM (4 mg/day on days 1-21 of each 28-day cycle) with/without LoDEX (40 mg/week). The primary endpoint was progression-free survival (PFS). In total, 221 patients (median 5 prior therapies, range 1-13) received POM+LoDEX (n = 113) or POM (n = 108). With a median follow-up of 14.2 months, median PFS was 4.2 and 2.7 months (hazard ratio = 0.68, P = .003), ORRs were 33% and 18% (P = .013), median response duration was 8.3 and 10.7 months, and median OS was 16.5 and 13.6 months, respectively. Refractoriness to LEN, or resistance to both LEN and BORT, did not affect outcomes with POM+LoDEX (median PFS 3.8 months for both; ORRs 30% and 31%; and median OS 16 and 13.4 months). Grade 3-4 neutropenia occurred in 41% (POM+LoDEX) and 48% (POM); no grade 3-4 peripheral neuropathy was reported. POM+LoDEX was effective and generally well tolerated, and provides an important new treatment option for RRMM patients who have received multiple prior therapies (www.clinicaltrials.gov #NCT00833833).

  • Submitted November 15, 2013.
  • Accepted January 2, 2014.