Autologous and allogeneic stem cell transplantation in Waldenstrom's macroglobulinemia: review of the literature and future directions.

Waldenstrom's macroglobulinemia (WM) is a chronic lymphoproliferative disorder characterized by lymphoplasmacytic infiltrate and a monoclonal IgM serum peak. Treatment includes cytotoxic chemotherapy with alkylators, or purine nucleoside analogues and monoclonal anti-CD20 antibody. The role of stem cell transplantation (SCT) in WM has not been established. We identified 24 published cases of WM treated with high-dose chemotherapy (HDC) followed by autologous SCT (ASCT). The median age was 50 years; half of the patients had refractory disease and received a variety of preparative regimens. Nine complete and 14 partial responses were observed, with one early death. Fifteen patients were alive and well at follow-ups ranging from 1 to 132 months. Six additional patients, with a median age of 45 years (range, 30 to 62), who received allogeneic SCT have been reported. All were heavily pretreated with refractory or relapsed disease. Median time from diagnosis to transplant was 3.1 years (range, 1.3 to 7). Two patients died of complications of the procedure while one died of disease progression. Three patients were alive and well between 5 and 112 months post-transplant. The small number of reported patients precludes any significant conclusion except that SCT is feasible in WM and long-term disease control can be achieved in selected patients with autologous or allogeneic SCT, even in those with refractory disease.