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Autologous stem cell transplantation in Waldenström macroglobulinemia


Authors: M. Kaščák 1;  J. Ďuraš 1;  M. Navrátil 1;  R. Hájek 1,2,3
Authors‘ workplace: Klinika hematoonkologie, Fakultní nemocnice Ostrava 1;  Lékařská fakulta, Ostravská univerzita, Ostrava 2;  Babákova myelomová skupina, Ústav patologické fyziologie, Masarykova univerzita, Brno 3
Published in: Transfuze Hematol. dnes,22, 2016, No. 1, p. 28-38.
Category: Comprehensive Reports, Original Papers, Case Reports

Overview

Waldenström macroglobulinemia is a rare incurable monoclonal B lymphoproliferative disease, characterized by bone marrow infiltration and production of IgM paraprotein. Given its clinical course, it is indolent in nature, with a median overall survival of 6 years. Primary treatment using rituximab based regimens has overall response rates of 80% to 90% with median remission duration of 3 to 4 years. High dose chemotherapy with rescue autologous stem cell transplantation is an effective modality for treating patients with Waldenström macroglobulinemia. However, the issue of correct timing and selection of a suitable candidate for autologous transplantation remains unclear. There is no consensus regarding the most appropriate conditioning myeloablative regimen. High dose melphalan, carmustine or thiotepa based chemotherapy regimens and total-body irradiation based regimens are most commonly used. Autologous stem cell transplantation is a safe procedure with a low 1 year post-transplant mortality, usually not exceeding 5% even in an elderly patient population. In relapsed Waldenström macroglobulinemia, autologous transplantation has overall response rates of 80% to 90% with 20% f complete remissions. The expected median duration of remission is 3 to 4 years with median overall survival exceeding 60% after 5 years. Autologous transplantation should always be considered for the treatment of younger patients in first chemosensitive relapse and with a short duration of remission (less than 1–2 years) as well as in patients with a high risk prognostic score. In the future, it is expected that autologous transplantation will be used to treat early stage disease, at the time of best achieved response, similar to multiple myeloma treatment. This paper summarizes current knowledge regarding the use of autologous stem cell transplantation in Waldenström macroglobulinemia.

Key words:
Waldenström macroglobulinemia – autologous stem cell transplantation – high dose therapy


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Haematology Internal medicine Clinical oncology

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