Blood Journal
Leading the way in experimental and clinical research in hematology

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

  1. Paul G. Richardson1,*,
  2. David S. Siegel2,
  3. Ravi Vij3,
  4. Craig C. Hofmeister4,
  5. Rachid Baz5,
  6. Sundar Jagannath6,
  7. Christine Chen7,
  8. Sagar Lonial8,
  9. Andrzej Jakubowiak9,
  10. Nizar Bahlis10,
  11. Kevin Song11,
  12. Andrew Belch12,
  13. Noopur Raje13,
  14. Chaim Shustik14,
  15. Suzanne Lentzsch15,
  16. Martha Lacy16,
  17. Joseph Mikhael17,
  18. Jeffrey Matous18,
  19. David Vesole2,
  20. Min Chen19,
  21. Mohamed H. Zaki19,
  22. Christian Jacques19,
  23. Zhinuan Yu19, and
  24. Kenneth Anderson1
  1. 1 Dana-Farber Cancer Institute, Boston, MA, United States;
  2. 2 John Theurer Cancer Center, Hackensack, NJ, United States;
  3. 3 Washington University School of Medicine, St. Louis, MO, United States;
  4. 4 Ohio State University, Columbus, OH, United States;
  5. 5 H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL, United States;
  6. 6 Mount Sinai Medical Center, New York, NY, United States;
  7. 7 Princess Margaret Hospital, Toronto, ON, Canada;
  8. 8 Emory University School of Medicine, Atlanta, GA, United States;
  9. 9 University of Chicago Medical Center, Chicago, IL, United States;
  10. 10 University of Calgary, Calgary, AB, Canada;
  11. 11 Vancouver General Hospital, Vancouver, BC, Canada;
  12. 12 University of Alberta, Edmonton, AL, Canada;
  13. 13 Massachusetts General Hospital, Boston, MA, United States;
  14. 14 Royal Victoria Hospital, Montreal, QC, Canada;
  15. 15 College of Physicians and Surgeons of Columbia University, New York, NY, United States;
  16. 16 Mayo Clinic, Rochester, MN, United States;
  17. 17 Mayo Clinic, Scottsdale, AZ, United States;
  18. 18 Colorado Blood Cancer Institute, Denver, CO, United States;
  19. 19 Celgene Corporation, Summit, NJ, United States
  1. * Corresponding author; email: paul_richardson{at}
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


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 ( #NCT00833833).

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