P. Dreger et al., Myeloablative radiochemotherapy followed by reinfusion of purged autologous stem cells for Waldenstrom's macroglobulinaemia, BR J HAEM, 106(1), 1999, pp. 115-118
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.