A safety profile of medications used to treat Waldenström's macroglobulinemia

Expert Opin Drug Saf. 2018 Jun;17(6):609-621. doi: 10.1080/14740338.2018.1477936. Epub 2018 Jun 6.


Introduction: Waldenström's macroglobulinemia (WM) is a B-cell lymphoproliferative disease with serum IgM monoclonal component and bone marrow infiltration by lymphoplasmacytic lymphoma. Traditional therapy was based on that regimens used for closely related entities, such as chronic lymphocytic leukemia or multiple myeloma. This resulted in a lack of drugs specifically approved for WM, until the discovery of the Bruton Tyrosine Kinase (BTK) inhibitors.

Areas covered: Two main therapeutic attitudes are possible: (1) conventional therapies based on combinations with alkylating agents or proteasome inhibitors with steroids and anti-CD20 monoclonal antibodies or (2) new approaches with BTK inhibitors, usually alone. Other possibilities such as BCL2 inhibitors, PI3K/AKT inhibitors, and others are currently under evaluation, but we will focus the review on the most consolidated approaches that are available for patients with WM at different stages of the disease. PubMed, Web of Science, and clinicaltrials.gov were queried for the keywords 'Waldenstrom macroglobulinemia' and the different drugs here evaluated through 1 February 2018.

Expert opinion: Although WM has no many specific drugs, there are many possible therapies, including Ibrutinib, the first formally approved drug for this disorder.

Keywords: BTK inhibitors; IMiDs; Waldenström macroglobulinemia; anti-cd20; bendamustine; cyclophosphamide; dexamethasone; proteasome inhibitor.

Publication types

  • Review

MeSH terms

  • Agammaglobulinaemia Tyrosine Kinase
  • Animals
  • Drug Approval
  • Drug Design
  • Humans
  • Protein Kinase Inhibitors / adverse effects
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use*
  • Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Waldenstrom Macroglobulinemia / drug therapy*
  • Waldenstrom Macroglobulinemia / physiopathology


  • Protein Kinase Inhibitors
  • Protein-Tyrosine Kinases
  • Agammaglobulinaemia Tyrosine Kinase
  • BTK protein, human