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.
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.
Garnier
Alice
A
Service d'Hématologie et de Thérapie Cellulaire, CHU Clermont-Ferrand, Hôpital Hotel Dieu, and Univ Clermont 1, EA3846 France Clermont-Ferrand, France.
Robin
Marie
M
Larosa
Fabrice
F
Golmard
Jean-Louis
JL
Le Gouill
Steven
S
Coiteux
Valérie
V
Tabrizi
Reza
R
Bulabois
Claude-Eric
CE
Cacheux
Victoria
V
Kuentz
Mathieu
M
Dreyfus
Brigitte
B
Dreger
Peter
P
Rio
Bernard
B
Moles-Moreau
Marie-Pierre
MP
Bilger
Karin
K
Bay
Jacques-Olivier
JO
Leblond
Véronique
V
Blaise
Didier
D
Tournilhac
Olivier
O
Dhédin
Nathalie
N
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