Cargando…

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

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 underlyin...

Descripción completa

Detalles Bibliográficos
Autores principales: Ojeda-Uribe, Mario, Rimelen, Valérie, Marzullo, Cathérine
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7025649/
https://www.ncbi.nlm.nih.gov/pubmed/32104129
http://dx.doi.org/10.2147/JBM.S233059
_version_ 1783498547355713536
author Ojeda-Uribe, Mario
Rimelen, Valérie
Marzullo, Cathérine
author_facet Ojeda-Uribe, Mario
Rimelen, Valérie
Marzullo, Cathérine
author_sort Ojeda-Uribe, Mario
collection PubMed
description 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.
format Online
Article
Text
id pubmed-7025649
institution National Center for Biotechnology Information
language English
publishDate 2020
publisher Dove
record_format MEDLINE/PubMed
spelling pubmed-70256492020-02-26 Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome Ojeda-Uribe, Mario Rimelen, Valérie Marzullo, Cathérine J Blood Med Case Series 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. Dove 2020-02-13 /pmc/articles/PMC7025649/ /pubmed/32104129 http://dx.doi.org/10.2147/JBM.S233059 Text en © 2020 Ojeda-Uribe et al. http://creativecommons.org/licenses/by-nc/3.0/ This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution – Non Commercial (unported, v3.0) License (http://creativecommons.org/licenses/by-nc/3.0/). By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms (https://www.dovepress.com/terms.php).
spellingShingle Case Series
Ojeda-Uribe, Mario
Rimelen, Valérie
Marzullo, Cathérine
Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome
title Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome
title_full Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome
title_fullStr Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome
title_full_unstemmed Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome
title_short Good Profile of Efficacy/Tolerance of Bortezomib or Idelalisib in Waldenström Macroglobulinemia Associated with Acquired Von Willebrand Syndrome
title_sort good profile of efficacy/tolerance of bortezomib or idelalisib in waldenström macroglobulinemia associated with acquired von willebrand syndrome
topic Case Series
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7025649/
https://www.ncbi.nlm.nih.gov/pubmed/32104129
http://dx.doi.org/10.2147/JBM.S233059
work_keys_str_mv AT ojedauribemario goodprofileofefficacytoleranceofbortezomiboridelalisibinwaldenstrommacroglobulinemiaassociatedwithacquiredvonwillebrandsyndrome
AT rimelenvalerie goodprofileofefficacytoleranceofbortezomiboridelalisibinwaldenstrommacroglobulinemiaassociatedwithacquiredvonwillebrandsyndrome
AT marzullocatherine goodprofileofefficacytoleranceofbortezomiboridelalisibinwaldenstrommacroglobulinemiaassociatedwithacquiredvonwillebrandsyndrome