Introduction: HSCT has been developed in few Wm cases and is nowadays challenged by other innovative approaches. However, high dose therapy followed by autologous HSCT (HD-auto) produces high response rate and some long term responses while allogeneic HSCT performed after either myeloablative (MA-allo) or reduced intensity conditioning (RIC-allo) regimens may be cure of Wm (Dreger 1998, Tournilhac 2003, Maloney 2006).

Methods: We updated and extended our retrospective experience on 32 HD-auto, 11 MA-allo and 11-RIC-allo performed from 1990 to 2006 in 51 patients from 18 institutions. A MA-allo and a RIC-allo were performed in 1 and 2 cases respectively following relapse after a 1st HD-auto.

Results: Data are presented in the table.

HD-autoMA-alloRIC-allo
Nb 32 11 11 
Median age at transplant 56 46 56 
Interval: diagnosis-transplant 38 50 74 
Chemoresistance at transplant 25% 36% 55% 
Conditioning regimen BEAM (13), TBI/melphalan (9), TBI/endoxan (7), other (3) TBI/endoxan (9), other (2) TBI/fluda (10), other (1) 
Donor  Sibling (9), unrelated (2) Sibling (8), unrelated (2), cord blood (1) 
Median follow up (m) 45 (3–121) 68 (3–132) 22(2–60) 
Relapse 18 (56%) 4 (36%) 0 
Transplant related mortality 12,5% (one 2th cancer) 36% 27% (one 2th cancer) 
overall survival (1;3;5y) 87%, 77%, 58% 64%, 54%, 54% 82%, 68%, 68% 
Event free survival (5y) 25% 48% 68% 
HD-autoMA-alloRIC-allo
Nb 32 11 11 
Median age at transplant 56 46 56 
Interval: diagnosis-transplant 38 50 74 
Chemoresistance at transplant 25% 36% 55% 
Conditioning regimen BEAM (13), TBI/melphalan (9), TBI/endoxan (7), other (3) TBI/endoxan (9), other (2) TBI/fluda (10), other (1) 
Donor  Sibling (9), unrelated (2) Sibling (8), unrelated (2), cord blood (1) 
Median follow up (m) 45 (3–121) 68 (3–132) 22(2–60) 
Relapse 18 (56%) 4 (36%) 0 
Transplant related mortality 12,5% (one 2th cancer) 36% 27% (one 2th cancer) 
overall survival (1;3;5y) 87%, 77%, 58% 64%, 54%, 54% 82%, 68%, 68% 
Event free survival (5y) 25% 48% 68% 
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Acute GVHD developed following 9 MA-allo [Grade III-IV (n=1)] and 8 RIC-allo [Grade III-IV (n=1)]. Chronic GVHD developed following 7 MA-allo [limited (n=5), extensive (n=2)] and 5 RIC-allo [limited (n=2), extensive (n=3)].

Conclusion: We confirm that autologous HSCT achieves some long term responses even in heavily pretreated patients. Allogeneic HSCT induces very long term disease control and may cure WM. Specially, the RIC-allo gives impressive results on disease control in a set of older patients, with refractory disease, mostly heavily pretreated.

Topics:
hematopoietic stem cell transplantation, waldenstrom macroglobulinemia, allopurinol, transplantation, allogeneic hematopoietic stem cell transplant, cancer, follow-up, graft-versus-host disease, acute, graft-versus-host disease, chronic, melphalan

Author notes

Disclosure: No relevant conflicts of interest to declare.

2007, The American Society of Hematology
2007
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