title: Multicenter clinical trial of bortezomib in relapsed/refractory Waldenstrom's macroglobulinemia: results of WMCTG Trial 03-248.
abstract: OBJECTIVE
  Waldenstrom's macroglobulinemia (WM) is a B-cell disorder. Despite advances in the therapy, WM remains incurable. As such, novel therapeutic agents are needed for the treatment of WM.
  
  
  METHODS
  In this multicenter study, 27 patients with WM received up to eight cycles of bortezomib at 1.3 mg/m(2) on days 1, 4, 8, and 11. All but one patient had relapsed/or refractory disease.
  
  
  RESULTS
  Following therapy, median serum IgM levels declined from 4,660 to 2,092 mg/dL (P < 0.0001). The overall response rate was 85%, with 10 and 13 patients achieving minor and major responses, respectively. Responses were prompt and occurred at median of 1.4 months. The median time to progression for all responding patients was 7.9 (range, 3-21.4+) months. The most common grade III/IV toxicities occurring in > or =5% of patients were sensory neuropathies (22.2%), leukopenia (18.5%), neutropenia (14.8%), dizziness (11.1%), and thrombocytopenia (7.4%). Sensory neuropathies resolved or improved in nearly all patients following cessation of therapy.
  
  
  CONCLUSIONS
  The results of these studies show that bortezomib is an active agent in relapsed and refractory WM.
authors.affiliation: Bing Center for Waldenstrom's Macroglobulinemia, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02115, USA. steven_treon@dfci.harvard.edu
authors.forename: Steven P
authors.initials: SP
authors.lastname: Treon
authors.forename: Zachary R
authors.initials: ZR
authors.lastname: Hunter
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Matous
authors.forename: Robin M
authors.initials: RM
authors.lastname: Joyce
authors.forename: Brian
authors.initials: B
authors.lastname: Mannion
authors.forename: Ranjana
authors.initials: R
authors.lastname: Advani
authors.forename: David
authors.initials: D
authors.lastname: Cook
authors.forename: Joseph
authors.initials: J
authors.lastname: Songer
authors.forename: John
authors.initials: J
authors.lastname: Hill
authors.forename: Bruce R
authors.initials: BR
authors.lastname: Kaden
authors.forename: David
authors.initials: D
authors.lastname: Sharon
authors.forename: Ronald
authors.initials: R
authors.lastname: Steiss
authors.forename: Xavier
authors.initials: X
authors.lastname: Leleu
authors.forename: Andrew R
authors.initials: AR
authors.lastname: Branagan
authors.forename: Ashraf
authors.initials: A
authors.lastname: Badros
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001853:Bone Marrow / Q000187:drug effects; D001897:Boronic Acids / Q000627:therapeutic use*; D000069286:Bortezomib; D002986:Clinical Trials as Topic; D005260:Female; D006801:Humans; D007075:Immunoglobulin M / Q000097:blood; D008297:Male; D008875:Middle Aged; D011719:Pyrazines / Q000627:therapeutic use*; D012008:Recurrence; D013997:Time Factors; D016896:Treatment Outcome; D008258:Waldenstrom Macroglobulinemia / Q000188:drug therapy*
chemical_list: D000970:Antineoplastic Agents; D001897:Boronic Acids; D007075:Immunoglobulin M; D011719:Pyrazines; D000069286:Bortezomib