Emerging treatment options for myelofibrosis: focus on pacritinib

被引:4
|
作者
Chow, Vivian [1 ]
Weissman, Ashley [2 ]
O'Connell, Casey Lee [3 ,4 ]
Mehrvar, Azim [5 ]
Akhtari, Mojtaba [3 ,4 ]
机构
[1] Univ So Calif, Dept Clin Pharm & Pharmaceut Econ & Policy, Los Angeles, CA 90033 USA
[2] Univ So Calif, Kenneth Norris Jr Comprehens Canc Ctr, Dept Pharm, Los Angeles, CA 90033 USA
[3] Univ So Calif, Kenneth Norris Jr Comprehens Canc Ctr, Jane Anne Nohl Div Hematol, 1441 Eastlake Ave,Norris Topping Tower 3463, Los Angeles, CA 90033 USA
[4] Univ So Calif, Kenneth Norris Jr Comprehens Canc Ctr, Ctr Study Blood Dis, 1441 Eastlake Ave,Norris Topping Tower 3463, Los Angeles, CA 90033 USA
[5] Mahak Childrens Canc Treatment & Res Ctr, Tehran, Iran
来源
ONCOTARGETS AND THERAPY | 2016年 / 9卷
关键词
JAK2; FLT3; myeloproliferative neoplasms; SB1518; INTERNATIONAL WORKING GROUP; MYELOPROLIFERATIVE NEOPLASMS; JAK INHIBITION; JAK2/FLT3; INHIBITOR; MYELOID METAPLASIA; AVAILABLE THERAPY; NATURAL-HISTORY; CELL-FUNCTION; RUXOLITINIB; SURVIVAL;
D O I
10.2147/OTT.S93875
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Myelofibrosis (MF) is a myeloid malignancy associated with a heavy symptomatic burden that decreases quality of life and presents a risk for leukemic transformation. While there are limited curative treatments, the recent discovery of the Janus kinase/signal transducer and activator of transcription (JAK/STAT) pathway dysregulation has led to many clinical investigations for new treatment approaches. This review provides practical knowledge on the disease state, an overview of treatment options, and specifically focuses on the efficacy and safety of pacritinib in the management of MF. Pacritinib is a novel selective inhibitor of JAK2 and FMS-related tyrosine kinase 3 (FLT3) currently in Phase III trials for the treatment of MF. Thus far, studies have demonstrated clinical efficacy in reducing splenomegaly and constitutional symptoms. Common adverse events were gastrointestinal in nature, while hematologic toxicity was limited. However, it was announced that all ongoing clinical trials on pacritinib have been placed on hold by the US Food and Drug Administration in February 2016, due to concerns for increased intracranial hemorrhage and cardiac events. With comprehensive risk-benefit analysis of clinical trial data, the utility of pacritinib in the management of MF may be more clearly defined.
引用
收藏
页码:2655 / 2665
页数:11
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