Pomalidomide – An Appraisal of Its Clinical Development and Role in the Treatment of Relapsed/Refractory Multiple Myeloma
Authors: Richardson Paul G , Palumbo Antonio , Schey Stephen A , Dimopoulos Meletios A , Facon Thierry , Weisel Katja C , O’Gorman Peter , Leleu Xavier , Lacy Martha Q , Streetly Matthew J , Mikhael Joseph R , Siegel David S , San Miguel Jesus F , Anderson Kenneth C ,   Clinical Director of the Myeloma Program, Dana-Farber Cancer Institute, Boston, MA, US,   Chief of the Myeloma Unit of the Department of Oncology, University of Torino, Torino, Italy,   Clinical Lead and Consultant Haematologist, Department of Haematology, King’s College Hospital, London, UK,   Professor and Chairman, Department of Clinical Therapeutics, University of Athens School of Medicine, Athens, Greece,   Professor of Hematology, Lille University Hospital, Service des Maladies du Sang, Hôpital Claude Huriez, Lille, France,   Senior Physician, Department of Hematology, Oncology, Immunology, Rheumatology and Pulmonology, University Hospital of Tuebingen, Tuebingen, Germany,   Clinical Director of Pathology/Consultant Haematologist, Mater Misericordiae University Hospital, Dublin, Ireland,   Associate Professor, Department of Haematology, Lille University Hospital, Service des Maladies du Sang, Hôpital Claude Huriez, Lille, France,   Professor of Medicine, Chair of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN, US,   Consultant Haematologist and Clinical Lead for Myeloma, Department of Haematology, Guy’s Hospital, Guy’s and Thomas’ NHS Foundation Trust, London, UK,   Associate Professor, Mayo Clinic, Scottsdale, AZ, US,   Chief, Multiple Myeloma Division, John Theurer Cancer Center, Hackensack, NJ, US,   Director of Clinical & Translational Medicine, Clínica Universidad de Navarra, Centro de Investigación Médica Aplicada, IDISNA, Pamplona, Spain,   Program Director, Jerome Lipper Multiple Myeloma Center and LeBow Institute for Myeloma Therapeutics, Dana-Farber Cancer Institute, Boston, MA, US
Published: December 2015

Pomalidomide is a distinct immunomodulatory agent with significant activity in relapsed/refractory multiple myeloma (RRMM). The optimal treatment schedule in patients with RRMM who have received multiple lines of treatment, including bortezomib and lenalidomide, is 4 mg/day on days 1–21 of a 28-day cycle in combination with weekly low-dose dexamethasone. Improved responses and outcomes relative to traditional therapies continue to be confirmed in recently completed and ongoing trials. Pomalidomide exhibits direct tumoricidal, immunomodulatory, anti-angiogenic and anti-inflammatory activities, which facilitate combination therapy with agents with complementary mechanisms of action, resulting in greater anti-myeloma effects than single-agent therapy or previous combination therapies. For example, in combination with proteasome inhibitors and traditional chemotherapeutic agents in doublet or triplet regimens, pomalidomide provides high rates of durable response, and represents an important new treatment option for patients with RRMM requiring effective new therapies. Additionally, pomalidomide maintains its efficacy and tolerability profile in difficult-to-treat patients, including the elderly, patients with poor cytogenetics and those with renal impairment. This review summarises the clinical development of pomalidomide and discusses this effective agent for the treatment of patients with RRMM in the context of current myeloma treatment options, as well as potential future directions to further improve patient outcomes.