Myeloablative radiochemotherapy followed by reinfusion of purged autologous stem cells for Waldenstrom's macroglobulinaemia

Waldenstrom's macroglobulinaemia (WM) is an incurable lymphoproliferative d isorder. The purpose of this study was to investigate the role of autologou s peripheral blood stem cell transplantation (ASCT) for the treatment of WM . Seven patients (untreated or after first-line therapy) with symptomatic W M underwent two or three cycles of Dexra-BEAM chemotherapy + G-CSF with ste m cell harvesting and proceeded to total body irradiation and high-dose cyc lophosphamide followed by reinfusion of ex-vivo, B-cell-depleted stem cells , Engraftment was prompt, and procedure-related deaths did not occur. A str ong reduction or normalization of BM infiltration and serum IgM levels occu rred in all evaluable patients, but immunofixation electrophoresis revealed persistent paraproteinaemia in five of them. With 3-30 months of follow-up , all patients are alive without clinical or serological signs of disease p rogression, This pilot trial shows for the first time that high-dose radioc hemotherapy with purged stem cells is effective and may improve the course of patients with WM. In the majority of cases, however, complete eradicatio n of the disease does not appear to be possible with ASCT alone.