title: High-dose therapy and autologous stem-cell transplantation in Waldenstrom macroglobulinemia: the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation.
abstract: OBJECTIVE
  The role of autologous stem-cell transplantation (ASCT) in Waldenström macroglobulinemia (WM) is not defined. The aim of this study was to analyze the results of ASCT in patients with WM and to determine the prognostic factors that have a significant impact on outcome.
  
  
  METHODS
  We analyzed 158 adult patients with WM reported to the European Group for Blood and Marrow Transplantation (EBMT) between January 1991 and December 2005. Median time from diagnosis to ASCT was 1.7 years (range, 0.3 to 20.3 years), 32% of the patients experienced treatment failure with at least three lines of therapy, and 93% had sensitive disease at the time of ASCT. Conditioning regimen was total-body irradiation-based in 45 patients. Median follow-up for surviving patients was 4.2 years (range, 0.5 to 14.8 years).
  
  
  RESULTS
  Nonrelapse mortality was 3.8% at 1 year. Ten patients developed a secondary malignancy, with a cumulative incidence of 8.4% at 5 years. Relapse rate was 52.1% at 5 years. Progression-free survival (PFS) and overall survival were 39.7% and 68.5%, respectively, at 5 years and were significantly influenced by number of lines of therapy and chemorefractoriness at ASCT. The achievement of a negative immunofixation after ASCT had a positive impact on PFS after ASCT. When used as consolidation at first response, ASCT provided a PFS of 44% at 5 years.
  
  
  CONCLUSIONS
  ASCT is a feasible procedure in young patients with advanced WM. ASCT should not be offered to patients with chemoresistant disease and to those who received more than three lines of therapy.
authors.affiliation: North West London, NHS Trust, Watford Rd, Harrow, Middlessex, HA1 3UJ, United Kingdom. c.kyriakou@btinternet.com
authors.forename: Charalampia
authors.initials: C
authors.lastname: Kyriakou
authors.forename: Carmen
authors.initials: C
authors.lastname: Canals
authors.forename: David
authors.initials: D
authors.lastname: Sibon
authors.forename: Jean Yves
authors.initials: JY
authors.lastname: Cahn
authors.forename: Majid
authors.initials: M
authors.lastname: Kazmi
authors.forename: William
authors.initials: W
authors.lastname: Arcese
authors.forename: Karin
authors.initials: K
authors.lastname: Kolbe
authors.forename: Norbert Claude
authors.initials: NC
authors.lastname: Gorin
authors.forename: Kristy
authors.initials: K
authors.lastname: Thomson
authors.forename: Noel
authors.initials: N
authors.lastname: Milpied
authors.forename: Dietger
authors.initials: D
authors.lastname: Niederwieser
authors.forename: Karel
authors.initials: K
authors.lastname: Indrák
authors.forename: Paolo
authors.initials: P
authors.lastname: Corradini
authors.forename: Anna
authors.initials: A
authors.lastname: Sureda
authors.forename: Norbert
authors.initials: N
authors.lastname: Schmitz
mesh_terms: D000328:Adult; D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D018572:Disease-Free Survival; D005060:Europe; D005240:Feasibility Studies; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage* / Q000009:adverse effects; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D018579:Patient Selection; D016016:Proportional Hazards Models; D012008:Recurrence; D012042:Registries; D012189:Retrospective Studies; D018570:Risk Assessment; D012307:Risk Factors; D033581:Stem Cell Transplantation / Q000009:adverse effects; D013997:Time Factors; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods*; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D008258:Waldenstrom Macroglobulinemia / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery / Q000628:therapy*; D014916:Whole-Body Irradiation / Q000009:adverse effects; D055815:Young Adult
chemical_list: D007155:Immunologic Factors