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KOS 2007: The role of Autologous Transplantation in Waldenstrom's Macroglobulinemia
By Athanasios Anagnostopoulos, MD
Athanasios Anagnostopoulos, MD
Department of Clinical Therapeutics
University of Athens School of Medicine
Athens, Greece
06.25.07
Department of Clinical Therapeutics
University of Athens School of Medicine
Athens, Greece

ABSTRACT: The published experience on high-dose treatment (HDT) supported by autologous stem cell transplantation (ASCT) for Waldenstrom’s Macroglobulinemia (WM) consists mostly of retrospective single Institution reports1-6 with small number of patients and 3 registry analysis from US, France, and EBMT; the last 2 are presented (or updated) in this Workshop. The limited experience with HDT in WM is mostly due to the advanced age at the time of disease diagnosis and the relatively indolent nature of the disease, which makes such an aggressive treatment option less attractive. However, all studies confirm that HDT with ASCT is feasible in WM patients even in heavily pretreated with a non relapse mortality rate at 6% in the largest series.10 Harvest of auto SC should be done prior to nucleoside analogue exposure – since these agents may impair SC collectability11 and before extensive bone marrow infiltration occurs. The occurrence of 2nd cancers in WM patients after autografting (at least 5 cases reported) should be taken into consideration.A small series of 7 patients received HDT as part of their induction regimen with promising results. Most patients have been transplanted in advanced phase of their disease, beyond 1st relapse. A variety of preparative regimens have been used such as BEAM, high dose melphalan or cyclophosphamide with or without total body irradiation. A high response rate and prolonged remissions have been observed in patients who were clearly resistant to conventional chemotherapy. With the available data we can argue that ASCT as upfront treatment for WM should be still considered only within the context of clinical trials, in younger patients with adverse prognostic factors. Moreover ASCT assalvage regimen should be considered for all fit patients with advanced disease where conventional treatment options have failed. The possibility of stem cell collection in patients pretreated with nucleoside analogues makes the option of SC harvest and storage early in the course of the disease reasonable for certain patients. Moreover, the use of novel agents like rituximab and bortezomib, with confirmed activity against WM, who spare patients’ stem cell pool warranties the planning of further studies with HDT based on those treatment options


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