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Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome

Authors Ojeda-Uribe M, Rimelen V, Marzullo C

Received 1 October 2019

Accepted for publication 23 January 2020

Published 13 February 2020 Volume 2020:11 Pages 67—72


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 2

Editor who approved publication: Dr Martin H. Bluth

Mario Ojeda-Uribe,1 Valérie Rimelen,2 Cathérine Marzullo3

1Department of Hematology and Cellular Therapy Unit, Groupe Hospitalier Region Mulhouse-Sud-Alsace (GHRMSA), Mulhouse 68070, France; 2Laboratory of Molecular Biology CHRU Strasbourg, Strasbourg 67200, France; 3Laboratory of Immunology, Groupe Hospitalier Region Mulhouse-Sud-Alsace (GHRMSA), Mulhouse 68070, France

Correspondence: Mario Ojeda-Uribe
Department of Hematology and Cellular Therapy Unit, Groupe Hospitalier Region Mulhouse-Sud-Alsace (GHRMSA), 20 Avenue du Dr Laennec, Mulhouse Cedex 68070, France
Tel +33389647755
Fax +33389647747

Abstract: Acquired von Willebrand syndrome (AVWS) in the setting of Waldenström macroglobulinemia (WM) is a challenging condition. No real standard of care is recommended for these patients, although the therapeutic strategy should include a rapid approach to the emergency bleeding events and to the underlying malignant lymphoid disorder. We report here our experience treating three elderly patients with these concomitant hematologic entities. The use of a bortezomib-based chemotherapy regimen showed a good profile of tolerance and efficacy even in a long-term follow-up period. These patients were treated for several years before switching their therapy to idelalisib, a targeted oral therapy that inhibits phosphatidylinositol 3-kinase isoform-delta (PI3KD), which is part of the signaling pathway downstream B-cell receptor. This approach was well tolerated and efficacious, although some adverse effects were observed, particularly at hepatic levels, but were all reversible. The same profile of tolerance/efficacy was observed in one very old patient who received idelalisib as a first-line therapy. We think that bortezomib-based therapy could be considered in refractory patients with AVWS associated with WM.

Keywords: waldenström macroglobulinemia, acquired von Willebrand syndrome, bortezomib, idelalisib, proteasome inhibitors, von Willebrand factor

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