Future therapeutic options for patients with Waldenstrom macroglobulinemia

被引：4

作者：

Castillo, Jorge J. ^{[1
]}

Hunter, Zachary R. ^{[1
]}

Yang, Guang ^{[1
]}

Argyropoulos, Kimon ^{[2
]}

Palomba, M. Lia ^{[2
]}

Treon, Steven P. ^{[1
]}

机构：

[1] Harvard Med Sch, Dana Farber Canc Inst, Bing Ctr Waldenstrom Macroglobulinemia, Boston, MA USA

[2] Mem Sloan Kettering Canc Ctr, Div Hematol & Oncol, 1275 York Ave, New York, NY 10021 USA

来源：

BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY | 2016年 / 29卷 / 02期

关键词：

BTK; BCL2; CD38; CXCR4; MYD88; Waldenstrom; CHRONIC LYMPHOCYTIC-LEUKEMIA; TYROSINE KINASE BTK; RELAPSED/REFRACTORY MULTIPLE-MYELOMA; ORAL PROTEASOME INHIBITOR; L265P SOMATIC MUTATION; PHASE-1; CLINICAL-TRIAL; END RESULTS DATABASE; MYD88; L265P; ANTITUMOR-ACTIVITY; IN-VIVO;

D O I：

10.1016/j.beha.2016.08.021

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Waldenstrom macroglobulinemia (WM) is a rare lymphoma characterized by the accumulation of IgM-producing lymphoplasmacytic cells. Although WM patients can experience prolonged remissions, the disease invariably recurs. Therefore, novel treatments associated with higher success rates and lower toxicity profiles are needed. The discovery of recurrent mutations in the MYD88 and CXCR4 genes has unraveled potential therapeutic targets in WM patients. As a result of these findings and based on the design and execution of a prospective clinical trial, the FDA granted approval to ibrutinib, an oral Bruton tyrosine kinase (BTK) inhibitor, to treat patients with symptomatic WM. The present review focuses on potential therapies that could change the landscape of treatment of patients with WM, specifically focusing on inhibitors or antagonists or the proteasome, BTK, CD38, BCL2 and the CXCR4 and MYD88 genes themselves. Novel agents with novel mechanisms of action should be evaluated in the context of carefully designed clinical trials. (C) 2016 Elsevier Ltd. All rights reserved.

引用

页码：206 / 215

页数：10

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