Pomalidomide for the treatment of relapsed and refractory multiple myeloma

Anna Guidetti, Claudia Paba Prada, Jacob P. Laubach, Cindy Varga, Michelle E. Maglio, Mary McKenney, Deborah Doss, Robert L. Schlossman, Constantine Mitsiades, Teru Hideshima, Güllü T. Görgün, Irene M. Ghobrial, Noopur Raje, Nikhil Munshi, Kenneth C. Anderson, Paul G. Richardson

Research output: Contribution to journalArticlepeer-review


Introduction: Multiple myeloma patients who are refractory to lenalidomide and bortezomib have a dismal prognosis. Pomalidomide is a new immunomodulatory agent approved for the treatment of relapsed and refractory multiple myeloma (RRMM) that is unique in that it demonstrates promising activity but appears to be associated with lower toxicity than thalidomide or lenalidomide. Areas covered: We review the mechanisms of action of pomalidomide, evaluate preclinical data, summarize the results of dose-finding Phase I studies and describe Phase II/III studies of this drug in combination with dexamethasone and other agents. Data presented were gathered from multiple sources, including articles from PubMed, published abstracts from the annual meetings of the American Society of Hematology and American Society of Clinical Oncology and websites such as http://clinicaltrials.gov/. Expert opinion: The regulatory approval of pomalidomide represents an important addition to a hematologist's armamentarium for the treatment of RRMM. Pomalidomide is well tolerated and demonstrates a high level of anti-myeloma activity. Pomalidomide combined with dexamethasone should be considered as standard-of-care therapy for advanced RRMM following progression on both lenalidomide and bortezomib. Ongoing and future studies will characterize the activity of different combinations intended to improve treatment responses, and the potential role of pomalidomide as maintenance therapy.

Original languageEnglish (US)
Pages (from-to)1089-1108
Number of pages20
JournalExpert Opinion on Orphan Drugs
Issue number10
StatePublished - Oct 1 2014
Externally publishedYes


  • Immunomodulatory drugs
  • Multiple myeloma
  • Pomalidomide
  • Relapsed and refractory

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


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