title: A single-arm, phase II study of the anti-Blys monoclonal antibody belimumab in symptomatic Waldenstrom macroglobulinemia.
abstract: BACKGROUND
  The B-lymphocyte stimulator (BLYS) protein is known to regulate immunoglobulin in normal B cells, and be overexpressed in B-cell malignancies, including Waldenstrom macroglobulinemia (WM).
  
  
  METHODS
  This trial evaluated the safety and activity of belimumab, a monoclonal antibody targeting BLYS, in 12 patients with WM in a single-arm phase II study.
  
  
  RESULTS
  Ten patients had stable disease with therapy, although no objective responses were seen. Correlative studies showed patients to have low or undetectable baseline serum levels of BLYS, with the administration of belimumab having no effect on B-cell numbers.
  
  
  CONCLUSIONS
  Belimumab cannot be recommended as a single-agent therapy for the treatment of symptomatic WM, although further evaluation in combination with other agents would be justified.
authors.affiliation: Department of Haematology and Medical Oncology, Peter MacCallum Cancer Centre, Melbourne, Australia; Department of Haematology, Nottingham City Hospital, Hucknall Road, Nottingham, United Kingdom.
authors.forename: Mark
authors.initials: M
authors.lastname: Bishton
authors.forename: Andrew
authors.initials: A
authors.lastname: Spencer
authors.forename: Michael
authors.initials: M
authors.lastname: Dickinson
authors.forename: David
authors.initials: D
authors.lastname: Ritchie
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D061067:Antibodies, Monoclonal, Humanized / Q000494:pharmacology / Q000627:therapeutic use*; D053264:B-Cell Activating Factor / Q000037:antagonists & inhibitors* / Q000378:metabolism; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000494:pharmacology / Q000627:therapeutic use*; D016131:Lymphocyte Subsets / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D016896:Treatment Outcome; D008258:Waldenstrom Macroglobulinemia / Q000188:drug therapy* / Q000378:metabolism / Q000401:mortality
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D053264:B-Cell Activating Factor; D007166:Immunosuppressive Agents; C511911:belimumab
keywords: BLYS protein; Belimumab; Waldenstrom macroglobulinemia