Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis.

ABSTRACT:

Purpose

Myelofibrosis is a myeloid malignancy associated with anemia, splenomegaly, and constitutional symptoms. Patients frequently harbor JAK-STAT activating mutations that are sensitive to TG101348, a selective small-molecule Janus kinase 2 (JAK2) inhibitor.

Patients and methods

In a multicenter phase I trial, oral TG101348 was administered once a day to patients with high- or intermediate-risk primary or post-polycythemia vera/essential thrombocythemia myelofibrosis.

Results

Fifty-nine patients were treated, including 28 in the dose-escalation phase. The maximum-tolerated dose was 680 mg/d, and dose-limiting toxicity was a reversible and asymptomatic increase in the serum amylase level. Forty-three patients (73%) continued treatment beyond six cycles; the median c

SUBMITTER: Pardanani A 

PROVIDER: S-EPMC4979099 | biostudies-literature | 2011 Mar

REPOSITORIES: biostudies-literature