TY - JOUR
T1 - Waldenström macroglobulinemia
AU - Kasi, Pashtoon Murtaza
AU - Ansell, Stephen M.
AU - Gertz, Morie A.
N1 - Publisher Copyright:
© 2015 Millennium Medical Publishing, Inc. All Rights reserved.
PY - 2015
Y1 - 2015
N2 - Waldenström macroglobulinemia (WM) is an indolent low-grade lymphoma characterized by bone marrow infltration with lymphoplasmacytic cells associated with a monoclonal immu-noglobulin M protein. It is considered incurable. The 5-year survival rate for patients with symptomatic WM is 87% for those with low-risk disease, 68% for those with intermediate-risk disease, and 36% for those with high-risk disease. Owing to recent advances in therapy with new targeted treatment options, relative survival has improved. Insights into mutations in MYD88 L265P and the WHIM-like CXCR4 have been shown to be signifcant not just in terms of their diagnostic and prognostic value, but also as potential targets for therapy. For patients with symptomatic WM, the different classes of agents used to treat WM include alkylating agents (eg, cyclophosphamide and chlorambucil), nucleoside analogues (eg, cladribine and fudarabine) and monoclonal antibodies (eg, rituximab and alemtuzumab). With an increasing number of novel treatment options available including everolimus, bendamustine, bortezomib, ibrutinib, carflzomib, lenalidomide, and panobinostat, the optimal timing and introduction of these options in the absence of phase 3 trials remains controversial. A treatment algorithm based on Mayo Stratifcation for Macroglobu-linemia and Risk-Adapted Therapy (mSMART) and a comparison of important clinical trials in WM is provided.
AB - Waldenström macroglobulinemia (WM) is an indolent low-grade lymphoma characterized by bone marrow infltration with lymphoplasmacytic cells associated with a monoclonal immu-noglobulin M protein. It is considered incurable. The 5-year survival rate for patients with symptomatic WM is 87% for those with low-risk disease, 68% for those with intermediate-risk disease, and 36% for those with high-risk disease. Owing to recent advances in therapy with new targeted treatment options, relative survival has improved. Insights into mutations in MYD88 L265P and the WHIM-like CXCR4 have been shown to be signifcant not just in terms of their diagnostic and prognostic value, but also as potential targets for therapy. For patients with symptomatic WM, the different classes of agents used to treat WM include alkylating agents (eg, cyclophosphamide and chlorambucil), nucleoside analogues (eg, cladribine and fudarabine) and monoclonal antibodies (eg, rituximab and alemtuzumab). With an increasing number of novel treatment options available including everolimus, bendamustine, bortezomib, ibrutinib, carflzomib, lenalidomide, and panobinostat, the optimal timing and introduction of these options in the absence of phase 3 trials remains controversial. A treatment algorithm based on Mayo Stratifcation for Macroglobu-linemia and Risk-Adapted Therapy (mSMART) and a comparison of important clinical trials in WM is provided.
KW - Bortezomib
KW - Everolimus
KW - International prognostic scoring system for WM
KW - MYD88
KW - Rituximab
KW - WHIM-like CXCR4
KW - Waldenström macroglobulinemia
UR - http://www.scopus.com/inward/record.url?scp=84921642461&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84921642461&partnerID=8YFLogxK
M3 - Article
C2 - 25679974
AN - SCOPUS:84921642461
SN - 1543-0790
VL - 13
SP - 56
EP - 66
JO - Clinical Advances in Hematology and Oncology
JF - Clinical Advances in Hematology and Oncology
IS - 1
ER -