Ponatinib in the leukemia world: why a reevaluation is necessary for Philadelphia chromosome-positive patients with T315I mutation

被引：8

作者：

Goodrich, Angelina Daisy ^{[1
]}

机构：

[1] Univ Nevada, Reno, NV 89557 USA

来源：

EXPERT REVIEW OF HEMATOLOGY | 2014年 / 7卷 / 05期

关键词：

leukemia; Philadelphia chromosome; ponatinib; T315I mutation; tyrosine kinase inhibitor; CHRONIC MYELOID-LEUKEMIA; BCR-ABL INHIBITOR; RESISTANCE; IMATINIB; AP24534;

D O I：

10.1586/17474086.2014.958465

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Strategic drug design is used to meet the needs of numerous diseases for which there is no other recourse. Take the T315I mutation, for example, which occurs in Philadelphia chromosome-positive leukemias and renders all currently available tissue kinase inhibitors useless. The US FDA therefore saw it fit to avail ponatinib, the therapeutic result of careful drug design, to patients based on early data. However, its sales and marketing were later suspended due to emerging safety concerns. This drug has now returned to market albeit with tighter labeling. While the lesson for early approvals may be to restrict the drug to as narrow a patient population as possible, the potential benefits of this drug for the target population must not be lost amidst the controversy.

引用

页码：513 / 515

页数：3

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