Phase II clinical trials for Waldenstrom's macroglobulinemia

Research output: Contribution to journalReview articlepeer-review


Introduction: Waldenstrom's macroglobulinemia (WM) is an indolent lymphoma, characterized by infiltration of bone marrow by lymphoplasmacytic cells producing monoclonal immunoglobulin M protein. Many patients with WM may be observed, with chemotherapy reserved for symptomatic disease. Five-year survival rates vary from 36% in high-risk to 87% in low-risk patients. Studies in WM have shown improving survival over time. Recent insights into pathogenesis have revealed new targets for therapy, including mammalian target of rapamycin (mTOR) pathway and PI3K/Akt/PKC pathway among others. Whole genome sequencing has identified novel activating somatic mutations in WM, including MYD88 and CXCR4, which could serve as potential targets for management.Areas covered: This paper summarizes Phase II trials incorporating novel therapeutic agents, including proteasome inhibitors, immunomodulators, inhibitors of mTOR and PI3K/Akt/PKC pathways, Bruton tyrosine kinase inhibitors, bendamustine and HDAC inhibitors. A comprehensive literature search was undertaken in PubMed, Ovid Medline, Ovid Embase and Web of Science databases and all relevant studies were included.Expert opinion: Rituximab is considered as a first-line agent in the management of WM, with rituximab-cyclophosphamide-dexamethasone and rituximab-bendamustine being preferred regimens. Second-line regimens include proteasome inhibitors and purine analogs.

Original languageEnglish (US)
Pages (from-to)537-547
Number of pages11
JournalExpert Opinion on Orphan Drugs
Issue number5
StatePublished - May 1 2015


  • Bendamustine
  • Bruton tyrosine kinase inhibitors
  • HDAC inhibitors
  • Immunomodulators
  • MYD88 (L265P)
  • Mammalian target of rapamycin inhibitors
  • PI3K/AKT/PKC inhibitors
  • Proteasome inhibitors
  • Waldenstrom's macroglobulinemia

ASJC Scopus subject areas

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


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