title: Allogeneic hematopoietic stem cell transplantation allows long-term complete remission and curability in high-risk Waldenström’s macroglobulinemia. Results of a retrospective analysis of the Société Française de Greffe de Moelle et de Thérapie Cellulaire.
abstract: BACKGROUND
  Patients with poor-risk Waldenström's macroglobulinemia have suboptimal response and early post-treatment relapse with conventional therapies. Hence, new therapeutic approaches such as allogeneic stem cell transplantation should be evaluated in these patients.
  
  
  METHODS
  We examined the long-term outcome of allogeneic stem cell transplantation in Waldenström's macroglobulinemia by studying the records of 24 patients reported in the SFGM-TC database and one transplanted in the bone marrow unit in Hamburg.
  
  
  RESULTS
  Median age at the time of transplant was 48 years (range, 24-64). The patients had previously received a median of 3 lines of therapy (range, 1-6) and 44% of them had refractory disease at time of transplant. Allogeneic stem cell transplantation after myeloablative (n=12) or reduced-intensity (n=13) conditioning yielded an overall response rate of 92% and immunofixation-negative complete remission in 50% of evaluable patients. With a median follow-up of 64 months among survivors (range, 11-149 months), 5-year overall survival and progression-free survival rates were respectively, 67% (95% CI: 46-81) and 58% (95% CI: 38-75). The 5-year estimated risk of progression was 25% (95% CI: 10-36%), with only one relapse among the 12 patients who entered complete remission, versus 5 in the 12 patients who did not. Only one of the 6 relapses occurred more than three years post-transplant.
  
  
  CONCLUSIONS
  Allogeneic stem cell transplantation yields a high rate of complete remissions and is potentially curative in poor-risk Waldenström's macroglobulinemia.
authors.affiliation: Service d'Hématologie et de Thérapie Cellulaire, CHU Clermont-Ferrand, Hôpital Hotel Dieu, and Univ Clermont 1, EA3846 France Clermont-Ferrand, France.
authors.forename: Alice
authors.initials: A
authors.lastname: Garnier
authors.forename: Marie
authors.initials: M
authors.lastname: Robin
authors.forename: Fabrice
authors.initials: F
authors.lastname: Larosa
authors.forename: Jean-Louis
authors.initials: JL
authors.lastname: Golmard
authors.forename: Steven
authors.initials: S
authors.lastname: Le Gouill
authors.forename: Valérie
authors.initials: V
authors.lastname: Coiteux
authors.forename: Reza
authors.initials: R
authors.lastname: Tabrizi
authors.forename: Claude-Eric
authors.initials: CE
authors.lastname: Bulabois
authors.forename: Victoria
authors.initials: V
authors.lastname: Cacheux
authors.forename: Mathieu
authors.initials: M
authors.lastname: Kuentz
authors.forename: Brigitte
authors.initials: B
authors.lastname: Dreyfus
authors.forename: Peter
authors.initials: P
authors.lastname: Dreger
authors.forename: Bernard
authors.initials: B
authors.lastname: Rio
authors.forename: Marie-Pierre
authors.initials: MP
authors.lastname: Moles-Moreau
authors.forename: Karin
authors.initials: K
authors.lastname: Bilger
authors.forename: Jacques-Olivier
authors.initials: JO
authors.lastname: Bay
authors.forename: Véronique
authors.initials: V
authors.lastname: Leblond
authors.forename: Didier
authors.initials: D
authors.lastname: Blaise
authors.forename: Olivier
authors.initials: O
authors.lastname: Tournilhac
authors.forename: Nathalie
authors.initials: N
authors.lastname: Dhédin
mesh_terms: D000328:Adult; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D005602:France; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012074:Remission Induction; D012189:Retrospective Studies; D012307:Risk Factors; D012955:Societies, Medical; D013997:Time Factors; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D008258:Waldenstrom Macroglobulinemia / Q000401:mortality / Q000473:pathology / Q000601:surgery*; D055815:Young Adult